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The purpose of this study is to evaluate the safety and efficacy of the CCR5 antagonist GW873140 or placebo in combination with an optimized background regimen in treatment-experienced HIV-infected subjects with R5-tropic virus
null
Infection, Human Immunodeficiency Virus I
HIV-1 GW873140 CCR5 antagonist experienced
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intervention 1: 400 mg twice daily
intervention 1: GW873140
55
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NCT00197145
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The objective of this clinical study was to evaluate whether CXCL8 (CXC ligand 8 \[formerly interleukin (IL)-8\]) inhibition with repertaxin leads to reduced severity of primary graft dysfunction, as the result of improved functional and clinical outcomes in lung transplantation patients. The safety of repertaxin in the specific clinical setting was also evaluated. The ability of repertaxin to reduce target cells (polymorphonuclear leukocyte \[PMN\]) infiltration into the graft was evaluated to confirm its mechanism of action.
This was a phase 2, multi-center, randomized, double-blind, placebo-controlled, parallel-group (two arms) study. A total of 100 patients accepted and listed for lung transplantation, who met all of the study inclusion and none of the exclusion criteria described in Sections 9.3.1 and 9.3.2 of this report, were planned to be enrolled in the study. These patients were randomly assigned in a 1:1 ratio to receive either repertaxin or placebo, by continuous intravenous infusion for a period of 48 hours to start approximately 2 hours before reperfusion of the (first) transplanted lung occurred. The experimental treatment was additional to the standard treatment of lung transplant recipients. An initial 'loading dose' of repertaxin of 4.488 mg/kg body weight/hour was to be administered over 30 minutes followed by a maintenance dose of 2.772 mg/kg body weight/hour lasting 47.5 hours. Placebo was to be volume matched saline. Total infusion volume was not to exceed 500 mL/24 hours. Study medication was to be provided as clear glass class I ampoules, each containing 10 mL of the following products: repertaxin (33 mg/mL aqueous injectable solution) and placebo (9 mg/mL aqueous injectable solution of NaCl). The double-blind was to be maintained for the main part of the study only, i.e. up to the Month 1 (at least 30 days post-transplant) follow-up visit of the last patient in. After database lock of Month 1 data the study proceeded in an open fashion.
Ischemia-Reperfusion Injury Lung Transplantation
Lung transplantation Reperfusion Injury Survival
Prot_000.pdf: Study REPO 104 Repertaxin in Lung Transplantation Page 1 of73 PPD CLINICAL STUDY PROTOCOL • CONFIDENTIAL Study Number: Investigational Product: Title: Protocol Version Date of protocol: REP0104 Repertaxin A phase 2, multi-center, randomized, double-blind, placebo- controlled, parallel-group study of repertaxin in the prevention of primary grafi dysfunction after lung transplantation. Final Revision No. 3 20 March 2006 This protocol results from revision of "Final Revised Protocol No. 2 (30 August 2005)" according to Amendment No. 2 dated 20 March 1006. STATEMENT OF CONFIDENTIALITY Information in this protocol and accompanying documents contains privileged or confidential information that is the property of Dompé pha.r.ma s.p.a. lt is understood that the information will not be used, divulged, or published without prior written consent of Dompé, except to the extent such disclosure is requìred by applicable laws and regulations. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 2 of73 CONTACTINFORMATION SPONSOR Dompé pha.r.ma s.p.a. [Dompé] Via Campo di Pile; 67100 L'Aquila, Italy Milan Offices: Via S. Martino 12-12/ A, 20122 Milan, Italy Phone: +39 02 583831; Fax: +39 02 58383324 Medicai Director, Coordinator Clinical Trial Manager CLINICAL RESEARCH ORGANIZATION Project Leader Pharmacovigilance Contact PD PD PRIMARY INVESTIGATOR PPD Final Revision No. 3, 20-March-2006 Study REP0l04 Repertaxin in Lung Transplantation Page 3 of73 INVESTI GATOR A GREEMENT SIGNATURES: CLINICAL CENTER: PRlNCIPAL lNVESTIGATOR I have read study protocol REP0104 "A phase 2, multi-center, randomized, double-blind, placebo-controlled, para11el-group study of repertaxin in the prevention of primary graft dysfunction after lung transplantation" and agree to conduct the study as outlined in the protocol, and in accordance with ICH-GCP and any local regulations. Name of Principal Investigator: Signa ture: Date: PROTOCOL APPROVAL SIGNATURES: DOtvfPÉ PPD Signing of this page of the protocol by the Sponsor and CRO indicates that the protocolformat and content have been agreed arul approved by both partìes; and that it ìs agreed that no further changes to the protocol are required at the time of sìgning. A plzotocopy of thi!t fully signed page will be provided to all participatitig fovestigational Sites for filing. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in lung Transplantation Page 4 of73 TABLE 014"' CONTENTS 1. STUDY SYNOPSIS AND OVERALL DESIGN ................................................ 9 2. BACKGROUND INFOR.l\1A TION ................................................................... 12 2.1. RELEV ANT PRE-CLINICAL RESULTS ........................................................... 12 2.2. A SUMMARY OFTOXICOLOGY DATA ......................................................... 13 2.3. A SUMMA.RY OFPHASE 1 DATA .................................................................... 14 2.4. DISEASE REVIEW AND STUDY RATIONALE .............................................. 15 2.4.1. Selectìon of dose and treatment schedule in the study .......................................... 16 2.4.2. Alternative treatments ............................................................................................ 17 2.4.3. Risk - benefit evaluation ........................................................................................ 17 3. OVERALL STUDY DESIGN AN'D PLAN DESCRIPTION .......................... 19 3.1. STUDYDESIGN .................................................................................................. 19 3.2. STUDYTIME TABIB ......................................................................................... 19 4. OB.JECTIVES AND ENDPOINTS .................................................................... 20 4.1. STlJDY OBJECTIVES ......................................................................................... 20 4.2. STUDYENDPOINTS ........................................................................................... 20 4.2.1. Efficacy endpoints ................................................................................................. 20 4.2.2. Mechanism of action-targeted endpoints ............................................................... 21 4.2.3. Pharmaco.kinetic endpoints .................................................................................... 21 4.2.4. Safety endpoints ..................................................................................................... 21 5. STUDY PO PULA TI ON ....................................................................................................... 22 5.1. lNCLUSION CRIIBRIA ...................................................................................... 22 5.2. EXCLUSION CRITERIA ..................................................................................... 22 5.3. ASSIGNMENT OFPATIENT NUMBER ............................................................ 23 6. Srf"UDY MEDICA1"'ION ....................................................................................................... 24 6.1. PRESENTATION, STO RAGE, PAC.K.A.GING AND LABELING ..................... 24 6.1.1. Presentation of Investigational Products ............................................................... 24 6.1.2. Receipt of drug at the center/Stability during shipment ........................................ 24 6.1.3. Preparatìon of the Dosing So!ution ........................................................................ 25 6.1.4. Storage/Stability of Investìgational Products ........................................................ 25 6.1.5. Packaging/Labeling of Investigatìonal Products ................................................... 25 6.1.6. Blinding ................................................................................................................. 26 6.2. DOSE, ROUTE AND SCHEDULE OF INVESTIGATIONAL PRODUCT AD:tv1INJSTRA.TION ............................................................................................. 26 6.3. CRITERIA FOR SCHEDULE ADJUSTMENT/DOSE-MODIFICATION/ DISCONTINUATION OF INVESTIGATIONAL PRODUCT ........................... 27 6.3.l. Criteria for schedule adjustment or dose modification .......................................... 27 6.3.2. Criteria for discontinuation of Investigational Product ......................................... 27 Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 5 of73 6.4. ACCOUNT ABILITY ............................................................................................ 28 6.4.1. Assessment of compliance ..................................................................................... 29 6.5. CONCOMITANTTIIBRAPY .............................................................................. 29 6.5.1. Immunosuppressive therapy .................................................................................. 29 6.5.2. Prophylaxis of opportunistic ìnfections ................................................................. 30 6.5.3. Other treatrnents ..................................................................................................... 30 7. STUDY PROCEDURE ANO ASSESSMENTS ................................................ 31 7 .1. SCREENING AND RANDOMIZATION ............................................................ 31 7.2. 7.3. 7.4. 7.5. 7.5.1. 7.5.2. 7.5.3. 7.6. 7.7. 7.8. 7.9. 7.10. 7.10.1. 7.10.2. 8. 8.1. 8.1.1. 8.1.2. 8.2. 8.3. 8.3.1. 8.3.2. 8.3.3. 8.3.4. 8.4. 8.4.1. 8.4.2. 8.4.3. 8.5. 9. 9.1. ORGAN PROCUREMENT .................................................................................. 32 OPERATIVE COlJRSE ........................................................................................ 32 ADMli'l"ISTRATION OF INVESTIGATIONAL PRODUCT IN THE OPERATIN'G ROOM ............................................................................................ 33 ADMISSION 1'0 rc·u ........................................................................................... 33 Arri val to the ICU .................................................................................................. 33 Assessment in the ICU ........................................................................................... 33 \Veaning mechanical ventilation ............................................................................ 34 ICU DISCHARGE - HOSPIT AL STA Y .............................................................. 35 DRUG ADMINISTRATION Ll\f THE ICU/HOSPITAL AND BLOOD SAIVIPLING FOR PHARNIACOKINETIC ASSA Y ............................................ 35 MONTH 1 POST-TRANSPLANT ....................................................................... 35 MONTHS 6 AND 12 POST-TRANSPLANT ....................................................... 36 EARLY PATIENT WITHDRA W AL ................................................................... 36 Withdrawal cri te.ria ................................................................................................ 36 Replacement policy ............................................................................................... 36 AD'1"ERSE EVENTS ........................................................................................... 38 DEFINITIONS ...................................................................................................... 38 Definiti on of an Ad verse Event ............................................................................. 38 Definiti on of a Serious Adverse Event .................................................................. 38 E:tvfERGENCY PROCEDURES ........................................................................... 38 .RECORDlNG ........................................................................................................ 39 AE reporting peri od ............................................................................................... 39 Relatìonship of AEs to the InvestigationaJ Product. .............................................. 39 Seve1ity of AEs ...................................................................................................... 40 Frequency of AEs .................................................................................................. 40 SERIOUS AD VERSE EVENT REPORTll~G ...................................................... 40 Reporting Procedure for Investigators to PPD ................................................. 40 Reporting Procedure for Investigators to IRB/REB ............................................. .41 Reporting Procedure for Regulatory Authorities .................................................. .41 DATA MONITORINO COMMlTTEE ................................................................. 41 STA T.ISTICAL ISSUES ...................................................................................... 43 SAl\.1PLE SIZE ...................................................................................................... 43 Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in lwig Transplantation Page 6 of73 9.2. 9.3. 9.4. 9.4.1. 9.4.2. 9.4.3. 9.4.4. 9.4.5. 9.4.6. 10. 10.1. 10.2. 10.3. 10.4. 11.. 11.1. 11.2. 11.3. 11.4. 11.5. 12. 12.1. 12.2. 12.3. 12.3.1. 12.4. 12.5. 12.6. 12.7. 13. 14. 14.1. 14.1.1. 14.1.2. 14.1.3. 14.2. RANDOMIZATION ............................................................................................. 43 ANAL YSIS POPULATION ................................................................................. 43 STATISTICAL METHODOLOGY ...................................................................... 44 Demographic and baseline characteristics ............................................................. 44 Primary efficacy analysis ....................................................................................... 44 Secondary efficacy analysis ................................................................................... 45 Analysis of the endpoint targeted to the mechanism of action .............................. 45 Analysis of pharmacok.inetic endpoints ................................................................. 45 Safety analysi.s ....................................................................................................... 46 ETHICAL CONSIDERATIONS ....................................................................... 47 L~STITUTIONAL REVIEW BOARD/RESEARCH ETHICS BOARD ............. 47 INFORMED CONSENT ....................................................................................... 48 CONFIDENTIALITY ........................................................................................... 48 COMPENSATION FOR MEDICINE-INDUCED INJURY AND INDElVIN'IFICATION ........................................................................................... 48 DATA HANDLJNG AND RECORD KEEPING ............................................. 50 CASE REPORT FOR.MS COìvlPLETION ........................ , .................................. 50 DATA 1/fANAGEMENT ...................................................................................... 50 DOCUME~1T ATI ON REQUIRED PRIOR TO INITIATION ............................. 51 DOCUMENTATION REQUIRED DURINO THE STUDY ............................... 51 ESSENTIAL DOClJMENT RETENTION ........................................................... 52 STlJDY ~IANAGEl\lIBNT .................................................................................. 53 REGULATORY BODY APPROVAL. ................................................................. 53 STAFF Il\1FORMATION & RESPONSIBILITIES .............................................. 53 MO~TIORlNG ...................................................................................................... 53 Access to records ................................................................................................... 54 AUDIT AND IN"SPECTION ................................................................................. 54 PROTOCOL DEVIATIONS/AMENDMENTS .................................................... 54 DISCONTINUATION OF THE STUDY ............................................................. 54 PlJBLICATIONS ................................................................................... , .............. 55 REFEREN CES .................................................................................................... 56 APPENDICES ...................................................................................................... 59 APPEl'{DIX 1 - 11:ETHODOLOGICAL DET AILS .............................................. 59 PGD score - ISHL T system for grading of severity of primary graft dysfunction [ Christie et al, 2004] .......................................................................... 59 Bronchoalveolar la vage methods ........................................................................... 59 Handling of samples for pharmacokinetic assay ................................................... 60 APPENDL'X 2-PACKAGING AL'ID LABELJ.i'.TG DETAILS ............................. 62 Final Revision No.3, 20-March-2006 Study REP0104 Repe,taxin in Lun.g Transplantation Page 7 of73 14.3. APPENDIX 3 - STUDY DRUG INFUSION RATE BY BODY WEIGHT ......... 66 14.4. APPENDIX 4 - STUDY FLOW CHART ............................................................. 69 14.5. APPENDIX 5 - WORLD MEDICAL ASSOCIA TION DECLARATION OF lIBLSlL~KI. ...................................................................................................... 70 14.6. APPENDIX 6 - ACCEPTABLE TIME WINDOWS POR ASSESSMENTS/ PROCEDURES ..................................................................................................... 73 Fina! Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ALT AE ADR A.i--iOVA AST AUC BAL BOS BP oc CLcr Cmax CO CRA CRF Css CVP CXCL8 DGF DMC EMEA ESRD FDA FEV1 Fi02 FVC g GCP HR ICH ICU IND IRB/REB ISHLT Ì.V. kg mg mL mml-Ig ng NSAID PA Pa02 PCWP PEEP PGD PMN p.o. PVR SAE s.c. SOP Sp02 SVR t½ ~tg UNOS Alanine Aminotransferase Ad verse Event Adverse Drug Reaction Analysis ofVariance Asparlate Aminotransferase Area Under the Curve Bronchoalveolar Lavage Bronchioli.tis Obliterans Syndrome Blood Pressure Degrees Celsius Calculated Creatinine Clearance (Cockcroft - Gault formula) Maximum Plasma Concentration Cardiac Output Clinica! Research Associate Case Report Form Steady State Concentration Right Atrial Pressure CXC ligand 8 [formerly interleukin (IL)-8] Delayed Graft Function Data Monitoring Committee European Medicines Evaluation Agency End Stage Rena! Disease Food and Drug Adrninistration Forced Expiratory Volume in One Second Fraction of Inspired Oxygen Forced Vital Capacity Grams Good Clinica! Practice Heart Rate Internatìonal Conference on Hannonization Intensive Care Unit Investìgational New Drug Instìtutìonal Review Board/Research Ethics Board International Society of Heart and Lung Transplantation Intravenous Kilogram Milligram Millilitre Millimeters mercury Nanogram Non Steroidal Anti-Inì1ammatory Drug Pulmonary Artery Partial Pressure of Arteria! Oxygen Pulrnonary Capillary Wedge Pressure Positive End Expiratory Pressure Primary Graft Dysfunction Polymorphonuclear leukocyte per os (taken by mouth) Pulmonary Vascu!ar Resistance Serious Adverse Event Subcutaneous Standard Operating Procedure Peripheral Oxygen Saturation Systemic Vascular Resistance Elìrnination half life Microgram United Network for Organ Sharing Final Revision No. 3, 20-March-2006 Page 8 of 73 Study REP0104 Repertaxin in Lung Transplantation Page 9 of73 1. STUDY SYNOPSIS AND OVERALL DESIGN Study title A phase 2, multi-center, randornized, double-blind, placebo-controlled, parallel-group study of repertaxin in the prevention of primary graft dysfunction after lung transp]antation. Study Number REP0104 Study period Study design Actua1 starting date (first-patient-in): 1 May 2005 Projected completi on of patient accrual (last-patient-in): September 2006 Projected study end date (last-patient-last-visit): September 2007 The study will be a phase 2, multi-center, randomìzed, double-blind, placebo-controlled, parallel-group (two arms) study. It will involve 100 lung transplant recipients. Objectives/endpoints The objectìve of this clinìcal trial is to evaluate whether CXCL8 inhibition with repertaxin leads to reduced severity of PGD. The safety of repertaxin in the specìfic clìnical setting will be also evaluated. Ptimary efficacy endpoint will be: PaO2/FiO2 ratio measured on ICU admission and at 24 hours after ICU admission. , Secondary efficacy endpoints will be: the time profile of Pa0 2/FiO2 ratio (ICU adrnission, then q24 hours up to extubation or up to 72 hours; addìtional data obtained as per center standard of care will be collected in patients wìth clinica) diagnosis of PGD up to the clinica I. recovery of the condition); PGD score (ICU admission, 24 hours, 48 hours, and 72 hours after ICU admission; in patients with clinical diagnosis of PGD additional scoring will be calculated up to the clinica) recovery of the condition from available data); time to freedom from mechanical ventilation; duration of ICU stay; ICU mortality; mortality in the first 30 days post-transplant; FEV 1 and FVC at month 1, 6, and 12 post-transplant; BOS score at month 6 and 12 post-transplant; cumulative acute rejection episodes at month 6 and 12 post- transplant; patient survival rate evaluated at month 6 and 12 post-transplant. Any additional information from BAUbiopsies performed according to center standard of care in patients with clinica! diagnosis of PGD will be collected up to the clinical recovery of the condition. PMN count in BAL specimen obtained during mechanical ventìlation, in the inter-val between 12 and 24 hours after reperfusion will be evaluated as a "mechanism of action-targeted" endpoint. Also. plasma levels of repe1iaxin and its major metabolite, DF2243Y, will be evaluated at steady state conditions (24±6 hours and 48 hours from the start of infusion) and then in the interval 2-6 hours after the end of drug infusjon. Safety endpoints will be; AE recordìng; standard laboratory tests performed at screening and at hospital discharge; vita} signs (BP & HR) evaluated at screening, at ICU admission and then q12 hours up to ICU discharge or up to 5 days after ICU admission; cardiopulmonary physiologic measurements (CO, PA pressure, SVR, PVR, PCWP, CVP), when PA catheter is Final Revision No.3, 20-March-2006 Study REP0l04 Repertaxin in lung Transplantation Page 10 of73 present, at ICU admission and then q12 hours up to ICU dìscharge or up to 5 days after ICU admìssion. Additiona1 cardiopulmonary data obtained as per standard of care will be collected in patients with clinical dìagnosis of PGD up to the clinica} recovery of the conditi on. Number of patients One-hundred patients undergoing single or bilatera} lung transplant. Inclusion/exclusion criteria Patients accepted and listed for transplantation (planned isolated lung transplant from a non- livìng donar with brain death), ages 18-65 years, body weight 30-95 kg, given written informed consent will be included. Patient must have normai renai function (as per calcu.lated creatinìne clearance 60 mUmin). Patients will be excluded if they are: recipients of an intended multiple organ transp1ant, including heart-lung and liver-lung transplantation; recipients of a lung from a living lobar donor or a Iung from a non-heart beatìng donor; recipìents of a re-do lung transplantatìon. Patients requiring mechanical ventilation at the time of transplant, or with extra-respiratory tract site of infection, or with hepatic dysfunction at the time of transpla.nt, or with hypersensitivity to ibuprofen orto more than one NSAID, orto medìcations belonging to the class of sulfonamides wìll be excluded as well. Also, patients planned to receive Orthoclone OKT3 or Campath induction immunosuppression or planned to receive sirolìrnus in the first three months after transplantation, or simultaneously participating in any other studies ìnvolving a study drug to be administered concomitantly with the Investìgational Product and/or a study dmg intended to prevent ischemia/reperfusìon injury will be excluded. Pregnant or breast feeding women will be exc]uded. Investigational drug Repertaxin: 33 mg/mL aqueous injectable solution of repertaxin (per single 10 m.L ampoule). Placebo: 9 rng/rnL aqueous injectable solution of sodium chloride (NaCI) (per single 10 mLampoule) For each 24 hour administration period, a dosing soJution will be prepared from the contents of 23 ampoules (230 mL), cliluted with 370 mL of 0.9 % sterile saline. The study drugs wìll be administered as a contìnuous i.v. infusion into a (high flow) central vein, by an infusion pump, starting approximately 2 hours before reperfusion of the (first) transplanted lung occurs. An initial 'loading dose' of repertaxin of 4.488 mg/kg body weight/hour will be administered over 30 min followed by a maintenance dose of 2.772 mg/kg body weight/hour lasting 47 .5 hours. Placebo will be volume matched saline. Total infusion volume wìll not exceed 500 mU24 hours. Statistics Data for the double-blind (maìn) phase of the study (up to 30 days post-treatment) will be presented in the clinica] study report. Data collected during the open phase of the study (from l month up to 12 months) will be presented in an addendum to the cHnica1 study report. Appropriate descriptive statistics will be produced, accordìng te the variable. Final Revision No. 3, 20-March-2006 Study REPO 104 Repertaxin in Lung Transplantation Page 11 of73 The ptimary endpoint will be analyzed by ANOV A and separate comparisons at two time points (O hours on ICU admission and 24 hours after ICU admission). Appropriate descriptive statìstics wilJ be produced for all secondary endpoints. Inferential tests will be applied only to PaOi/FiO2 ratio, PGD Score, time to freedom from mechanical ventilati on and durati on of ICU stay. Safety data will be presented with the appropriate desc1iptive statistics. Final Revision No. 3, 20-March-2006 Study REP0104 Repettaxin in Lung Transplantation Page 12 of73 2. BACKGROUNDINFORMATION Repertaxin (DF1681 Y) is a specific inhìbitor of CXC lìgand 8 [CXCL8; fo1merly interleukin (IL)-8) bio]ogìcal activity, stemming from a program of drug design of molecules intended to modulate chemokine action. Repertaxin is the first low molecula:r weight blocker of CXCL8 biologica! activity jn clinical development. Dompé is currently committed to evaluating the clinical use of repertaxin in the prevention of Delayed Graft Function (DGF) after solid organ transplantations, a clinical condition that can be consìdered a paradìgrn of ischernìa/reperfusion injury. Repertaxin received the orphan drug designation by the European Committee of Orphan Medicinal Products in September 2001 and by the Food and Drug Administrntion (FDA) in January 2003 for such clinica! condition. In the 4th quarter of 2003, Dompé has obtained protocol assistance by the European Medicines EvaJuation Agency (EN.lEA) for the development of repertaxin, and a pre-Investìgation New Drug (IND) meeting was held with the Food and Drng Administration (FDA) to discuss IND submission. Relevant pre-clinical, toxicological and phase 1 clini e al data are summarized below. Please also referto the Investigator's Brochure for more detailed information. As mentioned in the Investigator's Brochure, all previous non clinical and clinica! (phase 1) studies were performed with the lysine salt of repertaxin. Repertaxin (free acid) formulated in a lysine buffer will be used for this phase 2 Clinical Trial. In order to allow a readi1y- accessed infonnation and easy understanding of the study protocol, any doses of the experimental drug mentioned in this protocol are reported as repertaxin. 2.1. RELEV ANT PRE-CLINICAL RESULTS Repertaxin is in vitro a potent and specific inhibitor of CXCL8 biologica} activity. In vitro chemotaxis experiments have shown that repertaxin inhibits CXCL8-induced chemotaxis of human polymorphonuclear leukocytes (PMN) in the low nanomolar range. Studies to elucidate the mechanism of action have shown that repertaxin is a non-competitive allosteric inhibitor of the CXCL8 receptors CXCRl and CXCR2. Interaction of repertaxìn with CXCL8 receptors inhibits the intracellular signal transduction events activated by binding of CXCL8 to CXCRI and CXCR2 [Bertini, 2004; Souz,a, 2004]. In vivo, treatment of rats with repertaxin lysine sa1t prevented PJ\,1N infìltration ìnto the reperfused transplanted kìdney and reduced kidney damage, as assessed by an increase in serum creatinine Levels. Moreover, repertaxìn prevented PMN infiltration and tissue damage in animai models of ischemia/reperfusion injury of lìver, brain, intestine, heart and spinal cord. In these models, repertaxin inhibition of PMN recruitment ranged from 40 to 90%, and inhìbìtion of functional damage ranged from 50 to 80%. Efficacy was seen in all rnodels at repertaxin dose of 9 .90 mg/kg. More recently, repertaxin 1ysine salt has been evaluated in a rat model of lung transplantation. Doses of repertaxin of 9.90 and 19.80 mg/kg given i.v. 15 nùn before and s.c. 2 and 4 hours Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 13 of73 after the reperfusion, reduced PMN infiltration, along with organ damage (]ung edema) and functional impainnent (decreased PaO2) induced by ischemia/reperfusion. The phannacokinetics of repertaxin was evaluated after i.v. or p.o. single dose in rats, after i.v. single dose in dogs and after i.v. repeated bolus or i.v. continuous infusìon as part of toxicity studìes in both species. In rats, repertaxin is rapidly eliminated with a half-lìfe (tiri) of between 0.5 and 3 hours. lt is elìminated mainly by rnetabolism with negligible amounts of parent compound excreted in the urine. Phaimacokinetìcs were linear over a wide range of doses in rats. In dogs, a different pharmacokinetic profile was observed, i.e. repertaxin was less rapidly eliminated (t 112 12-28 hours) both after single bolus i.v. administration and i.v. continuous infusion. Metabolic studies inclicated the presence of 8 and 10 metabolites in rats and dogs, respectively. Ibuprofen is an expected mìnor metabolite. Repertaxin inhibited in vitro the isoenzymes CYP3A4 and, to a minor extent, CYP2Cl9. A phase I metabolism study of [14C]-repe1taxin indicated that repertaxin is catalysed by CYP2C9 and to a lesser extent by CYP2Cl 9 with the production of two major metabolites. In vitro protein bincling of [14C]-repertaxìn showed that repe1taxin is highly bound (approxirnately 99%) to plasma proteins in rats, dogs, rabbits, cynomolgus monkeys and humans. Albumìn ìs likely to be the mirjor binding protein in plasma in ali species. 2.2. A SlTh'lMARY OF TOXICOLOGY DATA Repeitaxin was tested for toxicity in rodent and non-rodent anima! species after single and repeated ì.v. doses. The repeated dose administration studies were conducted by i.v. continuous infusion, according to the foreseen human admìnistratìon route. The genera] toxicological profile of i.v. repertaxin, in the studies conducted to date, is characterized by a low toxicity after sìngle or repeated dose administrations in rats (L.Dso = 229.68 mg/kg i.v.; 660.00 mg/kg/day as No Observed Adverse Effect Level from 4 weeks studies) and mice (401.94 mg/kg i.v.). Continuous i.v. administration to dogs for 2 weeks resulted in a safe dose of 39.60 mg/kg/day. Continuous i.v. infusion of repettaxin to the male and fema1e rat at dose levels of up to 660.00 mg/kg/day did not have any sìgnificant adverse effects on mating performance and fertilìty. Repertaxìn poses no genotoxìc hazard for humans. Repertaxin lysine salt, at doses in excess of those intended to be used in humans, has a safe pharmacology profile in the renal, cardiovascular and respiratory systerns of rats and dogs. The locai tolerabìlity of repertaxin lysine salt was assayed in the rabbit ear lateral vein. The compound was well tolerated in concentrations up to 4.95 mg/mL (1 mlJkg) infused over a minute. In order to provide evidence of the safety of DF2243Y, the main metabolite of repertaxin excreted in urine in hurnans, safety pharmaco1ogy and toxicity studies have been ìmplemented Final Revision No. 3, 20-March-2006 Study REP0I04 Repertaxin in Lung Transplantation Page 14 of73 at doses 2 to 3 tirnes higher that those reached in man, as may occur during the treatment of patients receiving kidney transplantation. 2.3. A SUJ\t1MARY OFPHASE 1 DATA Four phase 1 pharmacokinetics/safety studies have been implemented: three healthy volunteers studies have been completed, and one study is ongoing in patients with chronic renal disease. In the first study _, single doses of repertaxin of 0.66 to 10.56 mg/kg were administered by 30-min i.v. infusion. Each dose group consisted of 4 subjects treated wìth repertaxin and 2 with placebo. The compound was well tolerated at all doses, with minor and unspecific adverse events that were not dose-related. Repertaxin peak plasma concentrations were achieved at approximately 30 min post-dose. Linear kinetics were observed over the dose range of 0.66-5.28 mg/kg. Repertaxin was eliminated from plasma with a terminal half- life ranging from 0.94 to 1.28 hours. Total body clearance and volume of distribution appeared to be ìndependent of dose. Unchanged repertaxin was unmeasurable in urine and a very small amount was excreted in the f eces over 72 hours. In a second study - repertaxin was adminìstered as 48-hour i.v. infusions of doses targeted to achieve repertaxin plasma steady state concentrations (Css) of 10, 20 and 30 µ,g/mL. Three dose levels were administered: 2.05 mg/kg/h x 30 min fo1lowed by 0.66 mg/kg/h x 47.5 h; 2.51 mgfk.g/h x 30 min followed by 1.32 mg/kg/h x 47.5 h; and 4.49 mg/kg/h x 30 min fotlowed by 2.77 mg/kg/h x 47.5 h. Twelve subjects (9 active, 3 placebo) were dosed in each dose level. These treatment schedules were chosen in order to closely mimi e the possible dose regimen to be used in efficacy trials. Plasma levels of repe11axin, and its metabolites ibuprofen, DF2243Y and DF2188Y were determined. Repettaxin free fraction appeared to increase with total concentration. Elimination from plasma was rapid with terminal half-Iìfe of approximately 1.2-1.5 hours. The pham1acokinetics of repertaxin did not appear to be Jinear, with total clearance and volume of distribution increasing with dose, while maximum plasma concentration (Cmax) and area under the curve (AUC) appeared to be less than dose proportional. Repertaxin was metabolized and excreted into urine as ibuprofen (about 1 % of the dose), DF2243Y (30-40% of the dose), DF 2188Y (18-25% of the dose) and methanesulfonamide (10-12% of the dose). Renal clearance for repertaxin and ibuprofen was dose-dependent, but not for DF2243Y and DF2188Y. The total recovery of rnetabolites in urine accounted far 60-75% of the admìnistered dose. Repertaxin and ibuprofen were detectable in the feces over 60 hours at very low arnounts and only in few subjects. The metabolite DF2188Y had a terminal half-life similar to repertaxin, while tenninal half-lives for ibuprofen and DF2243Y were longer than that for repertaxin, suggesting elimination-rate limited pharmacokinetics for these two metabolìtes. Overall, the pharmacok.inetic profiles of repertaxin and its major metabolites are sirrrilar in rats and humans. Repertaxìn was well tolerated again, resulting in adverse events that were minor and not dose related. The local reactions observed have been reduced by administering a more dilute solution at a higher infusion rate. The study showed that, in healthy volunteers, the proposed dose regimen that gives rise to target concentrations expected to be clinically effective is feasible, reproducible and safe. A third study - was performed to verify whether repertaxin, at plasma concentrations egual to or exceedìng that thought to be required for therapeutic efficacy, Fìnal Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplcmtation Page 15 of73 could affect drug metabolism through CYP3A4 and CYP2C9 ìn a clinically significant manner. No clinically significant pharrn.acokinetic interaction was observed between repertaxin and rnidazolam or tolbutamide, probe substrates for CYP3A4 and CYP2C9 respectively. Repertaxin free fraction appeared to increase by at least 50% over the infusion period, after mìdazolam/tolbutarnide co-adminìstration, while tota! repertaxin concentrations remained stable. The fourth study design - was implemented to obtain initial information on the safety and phannacokinetics of repertaxìn in subjects with renal impairment by exposing a very few subject.s with the most severe degree of renai impairment, i.e. patients with end stage renal disease (ESRD) undergoing hemodìalysis (stage A) to a limited dose of the compound. After determìning appropriate dosing for renal failure, a more complete evaluation of the safety and phannacokinetics of repertaxin was obtained by administering the appropriate dose to a representative number of subjects with various degrees of renal impairment and to male and female heaJthy volunteers (stage B). A dose of 1.32 mg/kg/h for 6 hours by i.v. continuous infusion is being adminìstered in both ESRD patients and healthy volunteers. Results obtained indicate that the pharmacokinetics profile of repertaxin is not influenced, as expected, by the degree of renai function. On the other hand, rena] function had a profound effect on plasma concentrations of the two major metabolites, which were found to increase over time along with the increase of the e]imination half-life. Based upon the .limited number of subjects there appears to be no apparent gender differences in the phannacokinetics profile of repertaxin and its metabolites. Repertaxin was well tolerated also in ESRD patients. Very few adverse events were reported, the majority of which was mìld in intensity and unlikely due to repe1taxìn. 2.4. DISEASE REVIE'\iV AND STUDY RA TIONALE Lung transplantation has become a standard therapy for patients with end-stage lw1g disease. Within last decades, donor management, organ preservation, immunosuppressive regimens and controJ of infectious complications have been substantially improved. In addition, the operative techniques of transplantation procedures have been developed to an intemational standard of high quality [Lau & Patterson, 2003]. However, despite these refinernents, significant reperfusion injury occurs in up to 10-20% of Iung transplant recipients as the consequence of unavoìdable processes of procurement, preservation and restoring bJood flow. This clinica! condition, recently termed primary graft dysfunction (PGD), remains an important problem after lung transplantation [Christie, 1998; King, 2000], and still represents the single bìggest cause of early morbidity and mortality for lung recipients [Fischer, 2001; Thabut. 2002]. In additi on, there is some evidence to suggest a relationship between repe1fusìon ìnjury, acute rejection, and the subsequent development of chronic graft dysfunction [Waddell, 1996; Fìser, 2002]. Prediction of PGD is made difficuJt by the complexity of the interactions between the donor lung and the recipient [Sommers, 1996; McRae, 2000]. In post-ischemia reperfusion, restoration of the blood supply (reperfusion) after prolonged tissue ischemia is associated with an inflammatory reaction characterized by massive polymorphonuclear neutrophil infiltration into the reperfused tissue [de Perrot 2003]. The infiltrating inflammatory cells can perpetuate the initial inflarnmatory reactìon and induce further injuries. Final Revision No.3, 20-March-2006 Study REP0104 Repenaxin in lung Transplantation Page 16 of 73 CXCL8 is a member of a class of chemokines involved in leukocyte recruitment and activatjon in tissues [Baggiolini, 1994]; this chemokine is believed to play a key role in the recruitment and activation of polymorphonuclear neutrophils in post-ischemia repe.rfusion injury [Welbourn, 1991; Lefer, 1991; Matsumoto, 1997]. The importance of CXCL8 in lung tissue during the ischemie time and after reperfusion has been clearly demonstrated. Reperfusion of ischemie lung in a rabbit model of lung reperfusion injury caused neutrophil infiltration and tissue damage, as well as a local production of CXCL8. The admìnistration of a neutralizing monoclonal antibody against CXCL8 prevented neutrophil infiltration and tissue injury, providing a causal role of locally-produced CXCL8 in this model [Sekido, 1993]. The release of CXCL8 early after reperfusion could initiate neutrophil recruìtment and thus induce secondary lung injury [de Perrot, 2002]. As a resu1t of these findings, the modulati on or inhibition of CXCL8 acti vity ìs considered a valid target for the development of innovative treatments for a vaiiety of severe clinical conditions, including ischemia/reperfusion injury occurring after solid organ transplantation. No cmTently avai1able pharmacological treatments are intended to cure or to prevent the occurrence of ischemia/reperfusion injury after lung transplantation. The current standard of care in preventing this clinica} condition focuses on prevention by way of surgical techniques in the procurement, storage and implantation of grafi lungs. The efficacy of repertaxin in preventing polymorphonuclear neutrophil infiltration and tissue damage in rat models of kidney transplantation and lung transplantation, as well as the safety shown in human phase 1 studìes, provide the rationale for a clinical study aimed at evaluating the effect of repertaxìn in preventing PGD after lung transplantation. 2.4.1. Selection of dose and treatment schedule in the study "In vitro", repertaxin inhibits CXCL8-induced chemotaxis of human PMN wìth an ICso of 1 nM, corresponding to about 0.3 ng/mL. CXCL8 ìs a key mediator of P:tvll'-i infiltration (and hence of tissue damage) in post- ischemia/reperfusion injury. In anima! models of ischemia/reperfusion, CXCL8 (and corresponding proteins in animals) is upregulated in the reperfused tissue within minutes from reperfusion, peaks after 4-6 hours and drops to undetectable levels within 24-48 hours from reperfusion [Sekido, 1993; Yamaguchi, 2000; Miura, 2001; Morita, 2001; DeVries, 2003]. Repe1taxin lysine sa.lt was tested in several anirnal models of ischemia/reperfusion. Repertaxin was admìnistered either by repeated bolus treatments (s.c. and/or i.v.) or by continuous i.v. infusion and was paired with observations of PMN infiltration and assessment of tissue damage (routineJy 24 hours after reperfusion). In both circumstances, repertaxin treatment was started before reperfusion occurred. For convenience and ease of treatment of animals, most expetiments were carried out by repeated bolus treatments. In repeated bolus treatments, repertaxìn was most efficacious in inhibiting PMN infiltratìon and reducing tissue damage in the reperfused tissue at doses ranging from 9.90 to 19.80 mg/kg. Furthennore, there was no overt increase in efficacy at doses of 19.80 vs. 9.90 mg/kg. At the dose of 9.90 mg/kg, Cmax of total plasma repertaxin was 34 ~tg/rnL, corresponding to 68 ng/mL of protein unbound (free) repertaxin. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lzmg Transplantation Page 17 of73 In continuous i.v. infusion animai experiments, repertaxin was found to be efficacìous at total plasma concentrations of 15 and 3.6 µg/mL, corresponding to 140 and 10 ng/mL of protein unbound (free) repertaxin, respectìvely. Repertaxìn was less efficacious at 0.5 ~tg/mL tota! repertaxin, corresponding to approx. 1-2 ng/mL of protein unbound drug. Although repertaxin was efficacious in animals when given either by repeated boluses or by continuous i.v. infusion, the proposed treatment in humans will be by continuous infusion in order to expose target cells (polymorphonuclear neutrophils) to steady state concentrations in the pharmacological range, thus maximizing the pharmacological activity. It should also be emphasized that repertaxin ìs a reversible inhibitor of CXCRI/2 activation induced by CXCL8 and that repertaxin has a short plasma ha]f Jife (0.95 to 1.28 hours in humans). The proposed dose in this clinical study was found safe in -previous phase l studies and should produce total plasma concentrations of 30 µg/mL, coITesponding to 30 ng/mL of protein unbound (free) repertaxin. Thus, the target plasma concentration of repertaxin obtainable in patients with the proposed dose ìs in the range found efficacious in both in vitro studies of CXCL8 inhibition and in animai rnodels of ischemia/reperfusion. The proposed schedule of administration (48 hours) is intended to expose the patient to repertaxin for the lag time when CXCL8 is believed to be upregulated in ischemia/reperfusion [Sekido, 1993; Yamaguchi, 2000; Miura, 2001; Monta, 2001; DeVries, 2003]. 2.4.2. Alternative treatments Because there is no other standard of care pharmacologic agent to prevent or treat PGD, patients not willing to participate in the study will not be offered any specific alternative treatment for the prevention of ischemia/reperfusion injury. Ali patients, regardless of study participation, will receive the standard of optimal care for the transplanted organ (procurement, storage, reperfusion) and the recipìent. Treatment of lung dysfunction, jncJucling the use of supporti ve measures, as well as overall management of lung transplant recipients will be the same, regardless of partìcipation in the study, 2.4.3. Risk - benefit evaluation 2.4.3.1. Treatment-related risks Results from preclinical studies support the level of drug exposure planned in this study. Furthennore, the sarne dose regimen that is proposed for this study has been tested in healthy volunteers without observing any significant, systemic adverse events. The tisk of toxicity at the infusion site, reported in a cohort of volunteers adrninistered by i.v. infusion via a peripheral vein, will be minimized by administe1ing the experimental drug via a high flow centrai vein. 2.4.3.2. Centrai line The study dmg will be adrninistered as a continuous i.v. infusion aver 48 hours with a catheter inserted in a (high flow) centra! vein. The risks of a central line are not considered Final Revision No.3, 20-March-2006 Stud y REPO 104 Repertaxin in Lung Transplantation Page 18 of73 study-related risks since preliminary survey reveals that centrai line will be routinely placed in all patients undergoing lung transplantation for their clirucal care. This clinically required access line will be maintained for a period of time that is longer than that required for the administration of the Investigational Product. Centrai line care as routinely practiced by the participating sites will mìnimize the risk of thrombosis and infections. 2.4.3.3. Bronchoalveolar Lavage Broncoalveolar lavage (BAL) is an invasive procedure used to monitor emerging problems in lung transplant recipients. A BAL specimen wiII be obtaìned from all study patients on mechanical ventilation, in the interval between 12 and 24 hours after reperfusion, to explore the repe11axin effect in reducing target cells polymorphonuclear neutrophil trafficking. To nùnimize the additional risk of the stucly specific BAL, the procedure will not be performed in patients with any clinical conditions which would be worsened by BAL procedures, accorcling to the Investigator's judgment. 2.4.3.4. Blood sampling In addition to the routine blood sa:mpling, participation in the study will require three pharmacological blood samplings (18 mL total), to detennine plasma concentrations of repertaxin (total and unbound) and its major metabolite, DF2243Y, in the specific clinical setting. Blood sampling will be performed at steady state conditions (24±6 hours and 48 hours from the start of infusion) and then in the interval 2-6 hours after the end of infusion. To maintain the study blind, blood samples will be obtained from all patients recruited in the study. Moreover, the study will require additional blood samplings at 12, 24, and 36 hours after the beginning of Investigational Product administration, to ensure that rena] and hepatic function are compatible with the ongoing jnvestigational treatment. 2.4.3.5. Other study relateci procedures No other procedures specifically related to the study are required. The specific timing of some measurements such as blood gas measurements will be guided by the protocol, but such measurements are firmly entrenched as pare of standard patient care and would undoubtedly be perforrned regardless of study partici patì on. 2.4.3.6. Potential benefit To the patients: It is qui te likely that there will be no direct benefit to individual patients participating in the trial. Half the patients will be receiving placebo and will therefore obtain no benefit other than the potential benefit of increased scrutiny and monito1ing that comes with being part of a clinical ttial The patients receiving the Investigational Product may possibly benefit with improved early lung function, but that is not certain. To society: This study may identify a useful medication that will make lung transplantation safer and more effecti ve for future recipients. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 19 of73 3. OVERALL STUDY DESIGN AND PLAN DESCRIPTION 3.1. STUDY DESIGN The study will be a phase 2, multi-center, randomized, double-blind, placebo-controlled, parallel-group (two arms) study. It wil1 involve 100 lung transplant recipients, randomly assigned in a 1:1 ratio to receive repertaxin or placebo, by contìnuous i.v. infusion for a per:iod of 48 hours starting approximately 2 hours before reperfusion of the (first) transplanted Iung occurs. The experimental treatment will be additional to the standard treatment of lung transplant recipients. Recruitment will be competitive among the study sjtes, unti I the planned number of patients is enrolled. Competitive recruitment has been chosen to increase the speed of recruitment and to account for any difference in t:ransplant rate among study sites. Each patient will be involved in the study for 48 hour treatment, for measurements up to hospital discharge, and then for three visits (@ 1, 6, and 12 months post-transp1ant). The double-blind will be maintained for the main part of the study only, i.e. up t:o the 1 rnonth (at least 30 days post-transplant) follow-up visit of the last patient in. After database lock the randonnzation code will be broken and the results of the blìnd phase will be analyzed and the study will proceed in an open fashion. 3.2. STUDY TIME TABLE • Actual starting date (first-patient-in): • Projected completion of patient accrual (last-patient-in): • Projected study end date (last-patient-last-visit): Final Revision No. 3, 20-March-2006 1 May 2005 September 2006 September 2007 Study REP0104 Repertaxin in Lun.g Transplantation Page 20 of73 4. OBJECTIVES AND ENDPOINTS 4.1. STUDY OBJECTIVES The objective of this clinical trial is to evaluate whether CXCL8 inhibition with repertaxin leads to reduced seve1ity of PGD, as the result of improved functional and clinical outcomes in lung transplantatìon patìents. The safety of repertaxin in the specific clinical setting will be also evaluated. The ability of repertaxin to reduce target cells (PMN) infiltration into the graft will be evaluated to confinn its mechanism of action. Plasma levels of repertaxin and its major metabolite will be measured at steady state conditions to provide population pharmacokinetic profile in this specìfic clinical setting. 4.2. STUDY ENDPOINTS 4.2.1. Efficacy endpoints 4.2.1.1. Prirnary efficacy endpoint • Pa02/FiO 2 ratio measured at FiO2 = 1 during mechanical ventìlation on ICU admission (time O) and at 24 hours after ICU admission. Patients on FiO2 < 1 will be tumed back to FiO2 = l for 5 minutes before gas measurement. 4.2.1.2. Secondary efficacy (exp1oratory) endpoints • Time profile of PaO2/FìO2 ratio measured at FiO2 = 1 during mechanical ventilation at four potential time points: ICU admission (time O) and then q24 hours, up to extubation or up to 72 hours after ICU admission, whichever occurs earlier. Additional data obtained as per center standard of care will be collected in patients with clinical diagnosis of PGD up to the clinica! recovery of the conditi on. • PGD score (stratified by single or bilateral transplant), evaluated accordìng to the scming system described by Christie et al. of the working group on primary lung graft dysfunction in the Scientific Council on Pulmonary Transplantation, Intemational Society for Hea1t and Lung transplantation (2005; see Appendix 1 for methodological details). Specific planned comparison of PGD scores will be made at four time points: ICU admission (time O), 24 hours, 48 hours, and 72 hours after ICU admissìon. In patients with clinical diagnosis of PGD additional scoring will be calculated up to the clinica! recovery of the condition from available data as per center standard of care. • Time to freedom from mechanical ventilation, defined as time between admissjon to the ICU and the initial time of first extubation (breathing off mechanical venti lati on without a tube) which is maintained far more than 24 hours. • Duration ofICU stay. • ICU mortality, defined as vita] status at the ICU dìscharge. • Mortality, defined as any death occurring in the first 30 days post-transplant, regardless of hospital discharge. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page21 of73 • FEV1 and FVC (stratìfied by single or bilatera] transplant) measured at month 1, 6, and 12 post-transplant. • BOS score (stratified by single or bilateral transplant) evaluated according to Estenne et al. (2002). BOS score will be measured at month 6, and 12 post-transplant. • Cumulative acute rejection episodes, biopsy proven, evaluated accordìng to Yousem et al. (1996) at month 6 and 12 post-transplant. • Patient survival rate evaluated at month 6 and 12 post-transplant. • Any additional infonnation from BAUbiopsies pe1formed according to standard of care in patients with clinica} diagnosis of PGD will be collected up to the clinica! recovery of the condition. 4.2.2. Mechanism of action-targeted endpoints • PMN count in BAL specimen obtained during mechanical ventilation, in the interval between 12 and 24 hours after reperfusion. 4.2.3. Pharmacokinetic endpoints • Plasma Jevels of repertax.in (tota! and unbound) and its major metabolite, DF2243Y, at steady state conditions (24±6hours and 48 hours from the start of infusion) and then in the interval 2-6 hours after the end of study drug infusi on. 4.2.4. Safety endpoints • AE recording. • Standard laboratory tests including hematology (hematocrit, hemoglobin, red blood cells, platelets, white blood cells, differential white blood cells count), and clinical chenùstry (sodium, chloride, potassium, bicarbonate, serum creatinine, blood urea nitrogen, total bilirubin, alanine arn.inotransferase (ALT), aspartate aminotransferase (AST), prothrombin time or intematìonal normalized ratio, partial thromboplastin time). Laboratory tests will be performed at screening and at hospital discharge. • Vita] signs, i.e. blood pressure (BP) and heart rate (HR), assessed at screening, on ICU admission, and then q 12 hours up to ICU discharge or up to 5 days after ICU ad.missi on, whichever occurs earlier. • Cardiopulmonary physiologic measurements, ìncluding cardiac output (CO), pulmonary artery pressure (PA pressure), systemic vascular resìstance (SVR), pulmonary vascular resjstance (PVR), pulmonary capillary wedge pressure (PCWP), right atrial pressure (CVP). Assessment will be perfonned on ICU admission, and then ql2 hours when PA catheter is present, up to ICU discharge or up to 5 days after ICU admission, whichever occurs earlier. Additional cardiopulmonary data obtained as per center standard of care will be collected in patients with clinical diagnosis of PGD up to the cHnical recovery of the condition. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in lung Transplantation Page22 of73 S. STUDYPOPULATION One hundred patients, receiving lung transplantation, will be included in the study, each one being randomized to receive either repertaxin or placebo. Patients will be selected from those on the lung transplant waiting list. Each prospective patient will be enrolled provided that (s)he fully meets ali of the study Inclusion and none of the Exclusion Criteria described in Sections 5.1. and 5.2. 5.1. INCLUSION CRITERIA To be eligible for inclusion into thìs study, each patient must fulfill the following inclusion criteri a: 1. Patients accepted and listecl for transplantatìon due to irreversìble, progressive disabling, end-stage pulmonary disease. 2. Ages 18-65 years 3. Body weight 30 - 95 kg, inclusive (i.e. up to 95.99 kg). 4. Planned isolated (single and bi-lateral) lung transplant from a non-living donor with brain death. This includes lobar lung transplant involving excisjon and sizing of a cadaver donor lobe to meet the thoracic dimension of the recipient before being transplanted. 5. Normal renal function at the time of transplant as per calculated creatinine clearance ,2:. 60 mUmin. Creatinine clearance will be calculated (CLcr) accordìng to the Cockcroft-Gault formula (1976; see section 7.1). 6. Patìent willing and able to comply with the protocol procedures for the duration of the study, including scheduled follow-up visits and examinations. 7. Patient given wrìtten infom1ed consent, prior to any study-related procedure not part of norma! medical care, with the understanding that consent may be withdrawn by the patient at any tjme without prejudice to their future medica! care. 5.2. EXCLUSION CRITERIA To be eligible for inclusion into this study, each patient must not fulfill any of the following: J. Recipients of an intended multiple organ transplant, including heart-lung and liver-lung transplantatìon. 2. Recipients of a lung from a living lobar donor. 3. Recipients of a lung from a non-heart beating donor. 4. Re-do lung transplantation. 5. Recipients requiring mechanical ventilation at the time of transplant. 6. Recipients with extra-respiratory tract site of infection (positive blood culture(s) ancl/or fever, associated with other signs of systemic sepsis syndrome). The criterion is not meant to exclude bacteremic cystic fibrosis patients with or without fever, unless they present with other signs of sepsis. 7. Recìpients with hepatic dysfunction (bilirubin exceeding 3 mg/dL and/or transaminases >3X upper limit of nonna I) at the time of transplant. Final Revìsion No. 3, 20-March-2006 Study REP0104 Repenaxin in Lung Transplantation Page 23 of73 8. Hypersensitivìty to: a) ibuprofen orto more than one non steroidal anti-inflammatory drug (NSAID). b) medications belonging to the class of sulfonamides, such as sulfamethazine, sulfamethoxazole, sulfasalazine, nimesulide or celecoxib. 9. Patients simultaneously partìcipating in any other studies involving a study drug to be administered concomitantly with the Investigational Product and/or a study drug intended to prevent ischernia/reperfusìon injury. 10. P1anned use of anti-CD3 monoclonal antibody (Orthoclone OKT3) or alemtuzumab (Campath) induction immunosuppression. 11. Planned use of sirolimus in the first three months after transplantation. 12. Pregnant or breast-feeding women. (NB: pregnancy should be avoided in patients or partners during the first month of participation in the study; no other specific wamings are desc1ibed, considering even stiicter general recommendations concerning pregnancy in transplanted patients, the treatment course of the Investigational Product, its phrumacokinetic profile, and the lack of significant adverse effects on mating performance and fertility in animal studies). 5.3. ASSIGNMENT OF PATIENT NUMBER The randomization number will be assigned in a sequen6al manner as patients are found to be ehgible for entry into the study and are enrolled. It will consìst of the site number, and the patient number e.g. 0210, where first 2 digìts represent site number, last 2 digits patient number. Ifa patìent is dropped from the study for any reason, the patient's number will not be reassigned. From the sìgning of the Patient Informed Consent Document until t.he allocation of a randomizatìon number, patients will be identified by their initials and date of bìrth. Final Revision No. 3, 20-March-2006 Stuòy REP0104 Repertaxìn in Lung Transplantation Page24 of73 6. STUDY MEDICATI ON Repertaxin is a new chemical entity with chernical narne R(-)-4-lsobutyl-alpha- rneth ylphen ylacety 1-methanesulfonamìde. 6.1. PRESENTATION, STORAGE, PACKAGING AND LABELING 6.1.1. Presentation of Investigational Products In this study the Investigational Products will be repertaxin and placebo. They will be provided as clear glass class I ampoules, each containing 10 mL of the following products: Repertaxin: 33 mg/mL aqueous injectable solution of repertaxin wìth the following composition (per single 10 mL unit): NAME0F INGREDIENT Repertaxin (DF 1681 Y) . . .. PER-IJNrr: • •• FORMULA. 330mg F\JNCTI0NOF INGREDIENT Active substance REFERENCE T0 QUALITY STANDARDS Manufacturer monograph and s ecification Placebo: 9 mg/mL aqueous injectable solution of sodiurn chloride (NaCl) with the following compositi on (per single 10 mL unit): NAMES0F PER·UNIT FuNCTION 0F • .REFERENCE T0 QUALffY · INGREDIENT · · FORMULA INGREDIENT STANDARDS. Sodium Chloride 90mg Isotonizing agent European Phannacopoeia - currenl edition Water for injections qstolOmL Solvent European Pharmacopoeia - current eclition Certificates of Analysis will be provided together with the Investigational products. 6.1.2. Receipt of drug at the center/Stability during shipment No Investigational Product wìll be sent to the Investigator before receiving Institutional Review Board (IRB)fResearch Ethics Board (REB) approvai. A temperature probe and data legger will accompany the drug on shipment. It is essentia] that centers read the graphical record on receipt in arder to verify the temperature range reached Final Revision No. 3, 20-March-2006 ' -- --- -- -- • - Study REP0104 Repertaxin in Lung Transplantation Page 25 of73 duting shipment, so that potential stability concerns during shipment can be investigated and appropriate action taken. 6.1.3. Preparation of the Dosing Solution The dosing solution for ìnfosìon wìll be prepared aseptically at the designated Pharmacy within each center. For each 24 hour administration period, the contents of 23 ampoules (230 mL) will be cliluted with 370 mL of 0.9 % sterile saline. This volume includes that needed for dead-space priming. Thus, repe1taxin will be dispensed as a 12.65 mg/mL solution. The dosing solution will be placed i11 a 1000 mL sterile empty infusion bag. Bags will be identified as Infusion Bag No. l (0-24 hour infusion) and Infusion Bag No. 2 (24 - 48 hour infusion). Detailed instrnction for the preparation of the dosing solution will be reported in the 'Instrnctions to the Phrumacy' provided with each 'Patient Box' of medicati on (see below ). Dosing solutions will be prepared and used within 72 hours from preparation. 6.1.4. Storage/Stability of Investigational Products Investigational Products must be stored not above 30°C (86°F). All Investigational Products must be stored in a secure locatìon, preferably in a temperature controlled locked room, and may be dispensed only by the Pharmacist or by a mernber of staff specifically authorized by the Investigator, as appropriate. Any deviations from the recommended storage conditions should be immediately rep01ted to PPD and the use of the drug suspended until authmization for its continued use has been given by Dompé. 6.1.S. Packaging/Labeling of Investigational Products Investigational Products have been manufactured by Current Repertaxin/Placebo ampoules have been packaged by . Any ~s will be packaged by , (formerly known as ----- All study medication is provided as 'Patient Boxes'. Each Patie11t Box wìll contain 2 'Treatment Boxes' (Treatment Box No. l = 0-24 hour infusion; Treatment Box No. 2 = 24-48 hour infusion) of 25 ampoules each, 23 to be used for the preparation of the dosing solution, 2 to be kept as reserve. Two sterile empty bags (lnfusion Bag No. 1 and Infusion Bag No. 2), plus an additional reserve bag, will be included in each Patient Box, along with the 'Instrnctions to the Pharmacy', a booklet detailing instrnctions for Investigational Product handlìng and preparati on of the dosing solution. If unused, the two ampoules in excess must remain in the Treatment Box. If they are used, the reasons for use must be documented. Dosing solutions will be prepared at the center Pharmacy. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lwrg Trcmsplanuuion Page26 ofi3 Current labeling of all study materials has been provided by Any future labeling will be provided by -· Labeling is prepared to meet locai regulatory requirements. Details of packaging and labeling are reported in Appendix 2. 6.1.6. Blinding In vesti gati onal Products Repertaxin and placebo will be packaged in identica! containers (boxes. ampoules. bags) displaying the same information on the labels so as to preserve the study blind. Moreover, solutions of repertaxin and placebo, either in the ampoules or in the bag (dosing solutìons), will be similar as per appearance and smell. Nevertheless, study Pharrnacists might not be fully blinded, since a transient foamy Jayer occasionally results from the preparation of the repertaxin dosing solution. Once the foamy layer, if any, has subsided, the dosing solution of repertaxin will be indistinguishable from that of placebo. Study blind After database lock of data recorded in the main part of the study, corresponding to the 1 month follow-up visit of the last patient in, the b1ind code will be broken and the study will proceed in an open fashion. 6.2. DOSE, ROUTE AND SCHEDULE OF INVESTIGATIONAL PRODUCT ADMINISTRA TION According to the parallel-group design, patients will receive either repertaxin or placebo as determined by a previously generated randomization schedule. An ìnitial 'loading dose' of repe1taxin of 4.488 mg/kg body weight/hour will be administered over 30 minutes followed by a maìntenance dose of 2.772 mg/kg body weight/hour lasting 47.5 hours. Placebo will be volume matched saline. The study drugs will be administered as a continuous ì.v. infusion into a (high flow) centrai vein, by an infusion pump adequate to provide reliable infusion rates (see below), as per treatment schedule. Total infusion volume will not exceed 500 mU24 hours. The infusion will start approxìmately 2 hours before the anticipateci time of reperfusion. For the purpose of this trial, reperfusion is defined as "the time of resumption of blood flow after vascular de-clamping of the first allograft". If cardiopulmonary bypass is used, the time of reperfusion is when the patient comes off the bypass, unless partial de-clamping occurs prior to complete stop of cardiopulmonary bypass. The surgeon will identify the time to start study drug infusion. In most instances, this time will be close to the time of the incision that begins the operation. The loading and maintenance doses will be gi ven using the same dosing soluti on (repertaxin 12.65 mg/mL), but t11e purnp rate will be altered to provide an infusion rate of approximately Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantatio11 Page27 of73 0.35 mLJkg/hour and 0.22 mIJkg/hour, respectively. Actual infusion rate (mUhour), adjusted to body weight, is tabulated in Appendix 3. Figures in the appendix represent mathematical rounding of origina! infusion rates derived from the following formula: Infusion rate (mL/hour) = dose (mg/kg/hour) x body weight (kg) 12.65 mg/mL Such a rounding affects actua] administered dose/kg/hour by less than 1 %. Technical characteristics of the infusion pump should include ìnfusion rate covering the range from 6.6 to 31.9 mUhour, in 0.1 rnUhourincrements, and capacity ìn excess of 600 mL'day. 6.3. 6.3.1. CRITERIA FOR SCHEDULE ADJUSTMENT/DOSE-MODIFICATION/ DISCONTINUATION OF INv'ESTIGATIONAL PRODUCT Criteria for schedule adjustment or dose modification No schedule adjustment and/or dose modificatìon is foreseen, except for discontinuation of drug as detailed below. 6.3.2. Criteria for discontinuation of Investigational Product Administration of the Investigatìonal Product should be immediately discontinued in case the patient develops renai or hepatic dysfunction defined as: renai dysftmction: the occurrence of both CLcr < 60 mL/min and urine output < 0.5 mIJkg/hour on two consecutive sampling time points (12, 24, 36 hours after the begjnning of Investigational Product infusion). hepatic dys.function: bilirubin exceeding 3 mg/d.L and/or transarrùnases >3X upper limit of norma!). Phase 1 studies in patients with ESRD have shown that renal function has a profound effect on plasma concentrations of a major, marginally active metabolite, carboxy-repertaxin (DF2243Y), whìch was found to accumulate over time along with the increased elimination half-life. Even if toxicological results to date suggest that DF2243Y does not raise any safety concem, limited experience in humans recommends to discontinue the drug in case of renal impairment as it would not be possible to predi.et the risk assocìatcd with elevated plasma levels. However, transient alterations of serurn creatinine or urine output, as defined above, do not indicate a renai dysfunction that would cause impaired excretion of DF2243Y. Because repertaxin undergoes extensive hepatic metabolism, it is also recommended that the infusi on is discontinued in case of hepatic dysfunction to avoid an increase of plasma level of repertaxin, exceeding the target and safe concentration. CLcr, urine output, bilirubìn and transaminases will be evaluated at 12, 24 and 36 hours after the beginning of Investigational Product infusion. Samples will be processed imrnediately and results made available as soon as possible to the Investigator. Urine output will be calculated for each of the 12 hour periods: from start of infusion to 12 hours, 12-24 hours, and 24-36 hours. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lzmg Transplantation Page28 of73 Investigational Product will also be immediately discontinued in the event of any other possibly drug related occun-ences that the Investigator believes might compromise patient safety. If the Investigational Product therapy is prematurely discontinued the primary reason for discontinuation must be recorded in the CRF. Patients who discontinue the treatment with the Investìgational Product will not be withdrawn from the study by default, but will complete observations as per the protocol, unless otherwise withdrawn at the Investigator's decision. 6.4. ACCOUNTABILITY Ali supplies will be maintained under adequate security by the responsible member of the Pharmacy staff. The Investìgator wìll ensure that study treatment is only admin:istered by designated staff wìthin the center. In particula:r, for U.S. sites, the Investigator will ensure that study treatrnent is only administered by those named as sub-investigators on the FDA form 1572 and designated staff. When Investjgational Product is received by the Phannacist (or designee), he/she will check for accurate delìvery and acknowledge receìpt by signing and dating the documentation provided by or on behalf of PPD and retuming it to PPD . A copy will be retained for the Investigator/Pharmacy file. The dispensing of the Investigational Product will be carefully recorded on appropriate drug accountability fo1ms (these will be provided by PPD ) and an accurate accounting will be available for verification by the CRA at each monitoring visit. Drug accountability records will include: • confinnation of delivery of the Investigati on al Product to the trial site, • the inventory at the site (ampoules/bags), • the use by each patient, • the retum to Dompé or alternative disposition of unused product(s), • accounts of any Investigational Product accidentally or deliberately destroyed, • accounts of an y reserve ampoules used. They should ìnclude dates, quantities, batch numbers, expiration dates (ff applicable), and any unique code numbers assigned to the Investigational Product(s) ancl/or patients. Investigators should maintain records which document adequately that: • the patients were provìded the doses specified by the protocoJ/amendment(s), • ali Investigational Product provided was fully reconciled. The PPD will review the drug accountability fonns and check ali Investigational Product ampoules (both unused and used) prior to making arrangements for their retum to Dompé, or authorizing their destruction by the study site. Investìgational Product which has been dispensed to a patient and retumed unused will not be re-dispensed to a dìfferent patient. Pina) Revision No. 3, 20-March-2006 Study REP0 l 04 Repertaxin in Lung Transplantation Page 29 of73 Unused Investìgational Product must not be discarded or used for any purpose other than the present study. Any remaining test material at the end of the trial will be returned to Dompé or disposed of, as detennined by the Sponsor. 6.4.1. Assessment of compliance Compliance will be assured by the person(s) within the center in charge of Investigational Product administration. Imn1ediately before the start of each 24-hours treatment period, the removable label on the Infusion Bag will be detached and attached to the relevant page of the CRF. Actual date and time of infusion start, switch of infusion rate (first 24 hours period only) and end of infusion will be recorded in the CRF, as well as infusion rates in each period. Compliance with the study product dosing schedule will be verified by a CRA during on-site monitoring visìts, as per records in the CRF, versus accountability records. 6.5. CONCOMITANT THERAPY Any medications required for the patient's welfare are permitted and will be gi ven at the discretion of the Investigator, except for drugs described in paragraphs below. Administration of all prior and concomitant medications, apart frorn the agents lìsted below, from the time of enrolment until 10 days after the end of study drng admìnistratìon or until hospital discharge (whichever occurs earlier) will be reported in the appropliate section of the CRF. For the intervals between 10 days or hospìtal dìscharge up to 12 month vìsit, only medications used far immunosuppression, prophylaxis of inf ections, and AE that the Investigator assesses as at least possibly related to the Investigational Produci will be reported. Ali the details as per the CRF fi.elds (sequential number, drng name, indication, startìng dose, doses at month 1, 6, and 12, route of administratìon, start/stop dates) will be recorded for the medications used for immunosuppression and prophylaxis of infections. Only the sequential number, the drng name, the ìndication and the start date wi11 be recorded for ali the other concomitant medications. The following agents do not need to be recorded: Saline and other hydration solutìons (including additional electrolytes) TPN and enteral f eeds ' Homeopatic medications Elective vitamins and minerals Topica! agents (apart from inhaled) with no or negligible systemic absorption Osmotic laxatì ves and locally acting antacids 6.5.1. Imrnunosuppressive therapy Neither guidelines nor fonnal consensus on "best" or "standard" immunosuppressive strategies after human lung transplantation are currently available. Therefore, Final Revision No. 3, 20-March-2006 Study REPO 104 Repertaxin in lung Transplantation Page 30 of73 irrummosuppression after lung transplantation is derived from the wider experience gained in renai and heart transplant recipients integrated by the results of specific lung trials. 6.5.1.1. Induction Data from the Intemational Socìety for Heart and Lung Transplantation (ISHLT) registry indicate that about 45% of lung transplant recipients receive some type of induction therapy, mainly polyclonal antilymphocyte preparations or anti-ìnterleukin (IL)-2-receptor antibodies. The use of Orthoclone OKT3 has been frequently associated to a cytokine release syndrome, which may induce cardiopulmonary instability. Also, experience with anti-CD52 (Campath) in the specific clinica} setting is very limited. Therefore, induction ,vith Orthoclone OKT3 or Campath will not be allowed in the trial. Induction therapy will not be mandatory in this trial and the decision, including the choice of the immunosuppressive agent, with the exclusion of Orthoclone OKT3 and Campath, will be based on center' s practice. 6.5.1.2. Maintenance At the present time, maintenance with a triple-drug regimen can be considered the norm. Therefore, this study will follow the common practice currently in use, recommending a ttiple-drug regimen of either cyclosporin A or tacrolimus, plus steroids (at a moderate dose in the first seven days), plus either azathioprine or mycophenolate mofetil/mycophenolic acid. The use of sirolimus will be discouraged, at least in the first three rnonths after lung transplantation, as cases of branchia] anastomotic dehiscence, rnost fatai, bave been repo1ted in de novo lung transplant patients when the dmg has been used as part of an immunosuppressive regimen. Programs intendìng to use sirolìmus in the first three months should refrain from enrollìng such patients in the t:rial. Monitoring strategies for dose adjustment as well as strategies for weanìng steroids will be as per center' s practice. 6.5.1.3. Treatment of acute and chronic rejection Treatment of acute rejections will be handled as per center practice. Treatment of established chronic rejection is currently net supported by evidence obtained in adequately designed clinical studies. Any strategy deemed useful by the Investigator will be allowed. 6.5.2. Prophylaxis of opportunistic infections Prophylaxis of oppo1tunistic ìnfections will be handled as per center practice. 6.5.3. Other treatments Nitric Oxide, Surfactant, Prostaglandin El will be administered as per center practice. Final Revìsion No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 31 of73 7. STUDY PROCEDURE AND ASSESS1"1ENTS A schedule for the tests and evaluations to be conducted in this study is found in the flow chart in Appendix 4. A list of acceptable assessment/procedure time windows is detailed in Appendix 6. 7.1. SCREENING AND RANDOMIZATION Potential study patients will be identified whilst on the center transplant waiting list. On anival at the center for transplantatìon, consented patients will undergo confirmatory screening for the study. Compliance with inclusion/exclusion criteria will be verified vs demographic and clinica! inforrnation available as per Organ Procurement Agency - e.g. United Network for Organ Sharing (UNOS) - form and standard clinica! practice. Laboratory tests (hematocrit, hemoglobìn, red blood cells, plateJets, white blood cells, differential white blood cells count, sodium, chloride, potassium, bicarbonate, serum creatinine, blood urea nitrogen, total bilirnbin, ALT, AST, prothrombin time or intemational normalized ratio, partial thromboplastin time) will be performed by local laboratories. Screening also includes measurement of BP, HR, body weight (kg), and testing for renai and hepatic function, as well as for extra-respiratory tract site of infection. Renal function will be evaluated by CLcr, calculated according to the following formula ( Cockcroft-Gault; 1976): [ 140 - age (years)J • Weight (kg) Male: CLcr = Female: CLcr = Serum Creatinine (mmol / L) • 815 [140 - age (years)] • Weight (kg) ------------- • 0.85 Serum Creatinine (mmol / L) • 815 Hepatic function will be evaluated by bilirubin (mg/dL) and transaminases (ALT and AST). Extra-respiratory tract sites of infection will be diagnosed by blood culture(s) ancl/or fever associated with other signs of systemic sepsis syndrome. Previous results of blood cultures can be used to verify inc1usion criteria, provided that the sample has not been obtained earlier than 72 hours prior to randomization. Eligible patients will be randomized (randomìzation number assigned) by an Investigator or designee before proceeding to the operating room. The Investigator will forward to the Pharmacy an order to start preparati on of Investigational Product dosìng solution. A screening form will be completed for all patients who signed the Patient Informed Consent Document, regardless of sequent entry into the study. Patients will be identified by their Final Revision No.3, 20-March-2006 Study REP0!04 Repertaxin in Lung Tra11splantatio11 Page 32 of73 initials and date of birth; in addition their race, sex and reasons for exclusion from the study wiH be recorded. Jf entered into the study, the folJowing recipient data wi11 be recorded in the CRF: - demographic, i.e. age, sex, race, weight, height, - immediate pretransplant and transplant clinica! information as per Organ Procurement Agency - e.g. UNOS - form, - results of laboratory tests. 7.2. ORGAN PROCUREMENT Standard protocols for procuring and preserving donor lungs will be used. Lungs will be resected en bloc following systemic hepalinization. Lungs will be washed by anterograde plus retrograde perfusion via the pulmonary artery with Low Potassium Dextran (Pe1iadex®) - based solution (with or without additions as per center practice) at 4°C. The lungs will be transported in the partially inflated state immersed in Pe1fadex® solution at 4°C. Donor lung data will be recorded in the CRF as per Organ Procurement Agency - e.g. UNOS - form. 7.3. OPERATIVE COURSE In principle, standard surgery procedures, excluding possible graft reflush with leukocyte depleted blood, will be applied as per center practice. However, it is suggested to adopt whenever possible, the following strategks: - Application of topìcal hypothermìa during implantation. - After implantation, blood flow will be progressively, in a controJled way, restored over a period of up to 10 rrùnutes (minimum 2 uùn). - Protective venti1ation strategy will be adopted during the initial period of repetfusion. It is suggested that the new]y implanted lung allograft is gently reinflated with a sustained airway pressure of 20 cmH20 before reperfusion and then ventilateci with Fi02 of 50%, PEEP =5 cmH2O, and pressure contro] ventilation limitìng peak airway pressures to 20 to 25 cmH20. After reperfusion has occurred, changes of ventilation will be directed by the anesthesìologist according to his/her judgment of the functional status of the implanted lung. Relevant operative details wìll be recorded in the CRF, including gross abnonnalities of the donor lung(s), duration of cold and warm ischemia, time of reperfusion, use and duration of cardiopulmonary bypass, Jung reduction procedures, anastomotic difficulties, hypoxemia, and pulmonary edema, use of nitroglycerin/nitric oxide/inhaled prostacyc1in/suifactant. Final Revision No.3, 20-March-2006 e ---- -- ·-- . - Study REP0104 Repertaxin ìn lung Transpla11tation Page 33 of73 7.4. ADMINISTR.,i TION OF IlWESTIGATIONAL PRODUCT IN THE OPERA TING ROOM The infusion Bag No. I, containing the dosing solution matching the patient randomization number, should be available in the operating room prior to anesthesia induction. Infusion of the Investigationa1 Product will start approximately 2 hours before the anticipated time of reperlusion of first allograft. The surgeon will identify the time to start study drug infusi on. The infusion pump will be set to provide the infusion rate corresponding to the loading dose. The loading dose will be infused for 30 min. Thereafter, the infusion rate will be reduced to provide the maintenance dose. Infusion rate (mL/hour) corresponding to each unit increment (kg) of body weight, is reported in Appendix 3. Immediately before the start of infusion, the removable 1abel on the lnfusion Bag will be detached and attached to the relevant page of the CRF. Starting time of drug administratìon, time of switch to maintenance dose and infusion rates will be recorded in the CRF. 7.5. ADMISSION TO ICU 7.5.1. Anival to the ICU Date and time of ICU admission will be recorded in the CRF. All patients will be mechanically ventilated through a ora! endotracheal tube. The ventìlator mode and rate will be set as per center practice, to ensure best standard of patient care. Upon arriva! to the ICU, the patient wilJ be hemodynamically stabilized and p]aced on FiO2 =1.0 and PEEP =5 cmH2O (or greater, if clinically necessary, to achieve SpO2 > 92% and PaO2 75mmHg). The following will be assessed as per center practice (unless otherwise specified) on affival to the ICU and recorded in the CRF, apart from SVR and PVR which wìll be calculated during data-management: • PaO2/FiO2 ratio (FiO2 =1; PEEP =5). • Chest X-rays (see Appendix 1 for methodological details). • BP,HR. • CO, PA pressure, SVR, PVR, PCWP, CVP. 7.5.2. Assessment in the ICU The following will be assessed during ICU stay and recorded in the CRF, apart from SVR and PVR which will be calculated during data-management. Measurement will be performed as per center practice, unless otherwise specified. For the purpose of this study, clinically diagnosed PGD is defined as: Final Revision No.3, 20-March-2006 Study REP0104 Repert<.L~in in Lung Transplantation Page 34 of73 - An episode of decreased PaOf_FiO2 ratio and chest X-ray results consistent with a PGD score of grade 3, which is not the result of some other secondary cause of graft dysfunction [Christie, 2005]. When diagnosed, the condition is considered clinically recovered for a PGD score of 1. • PaO2/FiO2 ratio. PaOiFiO2 ratio will be measured at FiO2 = 1 q24 hours, up to extubation or up to 72 hours after ICU admission, wbichever occurs earlier. Patients on FiO2 < 1 will be iurned back to FiO2 = 1 for 5 minutes before gas measurement. Addìtional PaO2/FiO2 ratio values obtained as per center standard of care in patients with clinical diagnosis of PGD will be recorded in the CRF up to the clinica] recovery of the condition. • Chest X-rays. A chest X-rays will be obtained 24 hours, 48 hours, and 72 hours after ICU admission (see Appendix 1 for details). • BP, HR. Vita! signs wìll be measured q12 hours up to ICU dìscharge or up to 5 days after ICU admission, whichever occurs earlier. • CO, PA pressure, SVR, PVR, PCWP, CVP. Cardiopulmonary physiologic measurements will be assessed q12 hours when a PA catheter is present, up to ICU discharge or up to 5 days after ICU admission, whichever occurs earlier. Additional measurements obtaìned as per standard of care in patients with clinica] diagnosis of PGD will be recorded in the CRF up to the clinica! recovery of the condition. For all measurements above, the actual date and time of assessment will be recorded in the CRF. • A BAL wm be perforrned during mechanical ventilation, in the interval between 12 and 24 hours after reperfusion, unless the patient is suffering from any clinica] conditions which would be worsened by BAL procedures, in the opìnion of the Investigator (see Appendix 1 for methodological details). Date and tìme of samp1ing will be recorded in the CRF along wìth location of BAL (lung, site) number of aliquots injected, amount recovered and summary of the most recent chest X-rays report. Specific reasons (hemodynamìc ìnstability, severe hypoxemia, etc.) for the decision to omit the BAL should be recorded in the CRF. Rena] (CLcr, urine output) and hepatic (bilirubin, transaminases) function wi11 be tested at 12, 24, and 36 hours after the begjnning of Investigation Product infusion. Other clìnical findings, including bronchoscopic and/or BAL findings, repo1ts for all routine chest X-rays and thoracìc CT scans, and results of routine biopsy will be copied ìnto the CRF from the medical charts. AEs will be also recorded. 7.5.3. Weaning mechanical ventilation \.Veaning procedures will be as per center practice. Every moming (@08:00) that a patient has not started weaning procedures, reasons for not weaning are recorded in the CRF. Date and time of first extubation which is maintained for more than 24 hours are recorded in the CRF. Final Revision No. 3, 20-March-2006 StudyREP0104 Repertaxìn in Lung Transplantation Page 35 of73 7.6. ICUDISCHARGE • HOSPITAL STAY Date and time of ICU discharge will be recorded in the CRF, as well as vital status at ICU discharge. Every moming (@08:00) that a patient has not been discharged from the ICU, reasons for not discharging are recorded in the CRF. Rena! (CLcr, urine output) and hepatic (bilirubin, transaminases) function wìll be tested at 12, 24, and 36 hours after the beginning of Investigati on Product infusi on. Before hospital discharge, laboratory tests will be pe1formed. Resu1ts will be recorded in the CRF. Length of hospital stay, vital status at hospitaJ discharge and AEs will be recorded. 7.7. DRUG ADMINISTRATION IN THE ICU/HOSPITAL AND BLOOD SAMPLING FOR PHARMACOKINETIC ASSA Y Drug ad:mìnistration (maintenance dose) will continue during ICU/hospital stay. Twenty-four hours after the start of the infusion, Infusion Bag No. 1 will be replaced by Infusion Bag No. 2. The removable label on Bag No. 2 will be detached and attached to the relevant page of the CRF. A blood sample will be obtained for pharmacokìnetic assay @ 24±6 hours. Infusion of Investigational Product will continue up to 48 hours. Immediately before the infusion is terminated, a blood sample will be obtained for pharmacokinetic assay. An additional blood sample will be obtained in the interval 2-6 hours after the end of infusi on. Blood sampling for phaimacokinetic assay will be vja an indwelling intravenous (or intra- arterial, if already in place) cannula or by direct venipuncture. On each time poìnt, a 6 mL sarnple will be taken into lithium heparin monovettes. Details of sample handling are reported in Appendix 1. Time of bag replacement, end of ìnfusìon, and blood sampling will be recorded in the CRF. 7.8. MONTH 1 POST•TRANSPLANT Patient will attend the center for study assessment at month 1 (at least 30 days post transplant). The following measurements will be performed as per center practice and recorded in the CRF: • FEV 1, FVC. Actua] date of assessment will be recorded in the CRF. Any AEs that occutTed du1ing the interval i.e. sìnce 10 days after the end of study drug administration or since hospital discharge (if applicable), and that the Investigator assesses as at least possibly related to the Investigational Product, will be recorded at this visit, as well as patient vita] status. Final Revision No. 3, 20-March-2006 Study REP0 104 Repertaxin in Lung Transplantation Page 36 of73 7.9. MONTHS 6 AND 12 POST~TRANSPLANT Patient will attend the center at months 6 and 12 +/- 2 weeks post-transplant. The fo11owing measurements will be perlonned as per center practice (unless otherwise specified) and recorded in the CRF: • FEV1. FVC. BOS score will be assessed at each visit. BOS score will be evaluated according to Estenne et al. (2002). • Acute, biops v proven rejection episodes diagnosed according to Y ousem et al. (1996). Cumulative assessment at each visit. Por all measurements above, actual date of assessment will be recorded in the CRF. Diagnosis and treatment of acute rejection, if any, will be also recorded in the CRF. Patient vital status, as well as any i\Es occuning during the intervals between study visits that the Investigator assesses as at least possibly related to the lnvestigational Product wiIJ be recorded at each visit. 7.10. EARL Y PATIENT 'WITHDRA ,v AL 7.10.1. vVithdrawal criteria Patients will be informed that they have the 1ight to withdraw from the study at any time, without prejudice to their medicai care, and are not obliged to state their reasons. Any withdrawals must be fu11y documented in the CRF and should be followed up by the Investigator. Additionally, the Investigator may withdraw a patient at any time if (s)he considers this to be in the patient's best interest. Also, a patient might be withdrawn from the study, at the Investigator' s judgment, in case of protocol violations, including (but not limited to) non-compliance with study procedures, patient lost to follow-up or administrative reasons. Patients must be annulled from the study for the following reasons: • Patient was randomized, but did not receive the Investigational Product, • Patient was randornized, but did not proceed to lung transplantation. If a patient fai 1s to return to the center for a scheduled visit, attempts should be made to contact the patient to ensure that the reason for not retuming is not an AE that may at least possibly be related to Investigatìonal Product. Likewise ifa patient declares his/her wish to discontinue from the study e.g. far persona! reasons, an attempt should be made to establish that the trne reason is not an AE (bearing in mind the patient is not obliged to state his/her reasons). 7.10.2. Replacement policy Only patients withdrawn due to one of the following reasons will be replaced: • Patient was randomized, but did not receive any Investigatìonal Product, Final Revision No.3, 20-March-2006 \ -- -- -- --- . - Study REP0104 Repertaxin in lung Transplantation Page 37 of73 • Patient was randomized, but did not proceed to lung transplantation. Should a patient be replaced, his/her number will not be real1ocated. A new patient will be enrolled and assigned the next number available. Final Revision No. 3, 20-March-2006 Study REPO 104 Repertaxin in Lung Transplantation Page 38 of73 8. ADVERSE EVENTS 8.1. DEFINITIONS 8.1.1. Definition of an Adverse Event An adverse event (AE) is defined as any untoward medicai occurrence in a patient or clinica! investigation subject administered a pharmaceutical product, which does not necessarily have a causai relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnorrnal laboratory finding), symptom, or disease temporally associated with the use of a medicina! (investigational) product, whether or not related to the medicina! (investigational) product [ Clinica/ safety data management: Definitions and Standardsfor Expedited Reporting]. 8.1.2. Definition of a Serious Ad verse Event A Serious Adverse Event (SAE) is defined [ Clinica/ safety data management: Definitions and Standards far Expedited Reporting] as any untoward medicai occurrence that at any dose: • • • • • • 8.2. results in death, is life-threatening (i.e. the patient was at risk of death at the time of the event. It does not referto an event which hypothetically might have caused death if it were more severe), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenita! anomaly/birth defect, is an important medicai event that based upon appropriate medicai judgment, may jeopardize the patient and may require medicai or surgical intervention to prevent one of the outcomes listed above. EMERGENCYPROCEDURES Ali AEs should be followed-up to determine outcome of the reaction. The Investigator should follow up the event unti! resolution or stabilization of the condition. It is the Investigator's responsibility to assure that the subjects experiencing AEs receive definite treatment for any AE, if required. The treatment allocation for each patient will be provided in individuai sealed envelopes. The locati on of these envelopes must be communicated to relevant study site staff and documented in the Investigator file. An envelope should only be opened in case of emergency where knowledge of the double-blind treatment may influence the further care of the patient. Ali such envelopes (both opened and unopened) will be collected by as each patient completes the study. If an envelope is opened for any reason, the Investigator will notify immediately and a record will be kept of when it was opened, by whom and why. Final Revision No. 3, 20-March-2006 CONFIDENTIAL PPD PPD Study REP0104 Repertaxin in Luns Transplantation Page 39 of73 8.3. RECORDING AE data should be obtained through observation of the patient, from any information volunteered by the patìent, or through patient questioning. All AEs encountered during the clinica! study, including SAEs wìll be reported in the appropriate section of the CRF. It is important that this ìncludes the duration of the AE (onset/resolution dates), the relationship to the drng, the severity, and relevant concomitant treatments dispensed (or other action taken) (see Sections 8.3.2., 8.3.3., and 8.3.4. below). 8.3.1. AE reporting period All AEs which occur during the treatment period (i.e. the first to last day of Investigational Product administration) and up to 10 days after the end of Investigational Product adnùnistration or up to hospìtal discharge, whìchever occurs earlìer, will be recorded in the CRF. In addition, any known untoward event that occurs subsequent to the AE reporting period that the Investigator assesses as at least possìbly related to the Investigational Product shou1d also be reported as an AE. 8.3.2. Relationship of AEs to the Investigational Product The Investigator wi11 assess the possible relationship between the AE and the investigational medication, according to the crite1ia in Table 1 below: Table 1: Relationship of the Adverse Event to the Investigational Product None (lntercurrent An event that is not and cannot be related to the Investigational Product, Event) e.g. patient js a passenger in a road traffic accident Relationship is not likely e.g. a clìnical event including laboratory test Unlikely (remote) abnormality with temporal relationship to drug administration which makes a causa! relationship improbable and in which ol11er drugs, chemicals or underlying disease provìde plausible explanatìons Possible ReJationship may exist, but could have been produced by the patient's conditi on or treatment or other cause Probable Relationship is likely, the AE ~bates upon discontinuati on of Investigational Product and cannot be due to the patient's conclition Strong relationship, the event abates upon cliscontinuation of H.ighly Probable Investigational Product and, ìf appJicable, re-appears upon repeat exposure An Ad verse Drug Reaction (ADR) is defined as an adverse experience which is a reasonably likely to have been caused by the drng. Any AE reported in the study having a possible, probable or highly probable relationship to study drug wil1 be considered as an ADR. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page40 of73 8.3.3. Severity of AEs The Investigator will grade the severity of any AE using the definitions in Table 2. For each epìsode, the highest seve1ity grade attained should be reported. Table 2: Severity of the Ad verse Event Mild Grade 1 - Does not interfere with patient's usual function (awareness of svmotoms or sjgns, but easilv tolerated [acceptable]). Moderate Grade 2 - Intetferes to some extent with patient's usual functi.on (enough discomfort to jnterfere with usnal activity [disturbing]). Severe C1Tade 3 - Interferes significantly with patient's usual function (ìncapacity to work orto do usual activities [unacceptable]) Life Threatening Grade 4 - Results .in risk of death, organ damage, or pennanent disabj]ìty ( unacceptable] 8.3.4. Frequency of AEs The frequency of AEs should be described as per Table 3. Table 3: Frequency of the Adverse Event Once The AE occurred only once and resolved in < 24 hours Occasionally The AE occmTed sporadìcally or episodically between the onset ancl resolutìon dates (and times) Continuously The AE was present for the entire time between onset and resolution dates (and times) and was > 24 hours duration 8.4. SERIOUS ADVERSE EVENT REPORTING 8.4.1. Reporting Procedure for Investigators to PPD The Investigator must report all SAEs, regardless of presumed causai relationship, to the PD Pharmacovigilance Depattment, by fax. within 24 hours of leaming of the event. Details of the relevant fax number for SAE repo1ting are provided in the section ''Contact Information". Infonnacion on SAEs wrn be recorded on a specific Non-Carbon Repeat SAE fonn. Blank copìes will be included in the Investigator's Site File. Follow-up reports (as many as required) should be completed and faxed following the sarne procedure above. A final report is required ìn any case once the condition is resolved or stabilized and no more information about the event ls expected. The final report should be completed and faxed following the same procedure above. PPD will report to Dompé alJ information received by the Investigators, by fax within 24 hours of knowledge. Final Revision No. 3, 20-March-2006 Study REP0104 Reperta.>:in in Lung Transplantation Page41 of73 8.4.2. Reporting Procedure for Investigators to IRB/REB In addition to reportìng the SAE to PPD , the Investigator rnust also comply with the requirements related to the reporting of SAEs to the IRB/REB which approved the study. The requirements of IRB/REB vaiies from one state and indeed one country to another, however as a minimum requirement a11 Investigators must promptly report all serious unexpected* ADRs, life-threatening problems or deaths to their IRB/REB. PPD will ìnforrn Investigators of all serious unexpected ADRs, which are reported to PPD from other Investigators. These SAEs should also be reported promptly to the IRB/REB in compliance with the locai regu1ations. Copies of all co1Tespondence relating to reporting of any SAEs to the IRB/REB should be maintained in the Investigator's Files and provided to PPD • * An unexpected ADR is defined as any adverse drug experience, the nature or seve1ity of which is not consìstent with the current Investigator's Brochure. 8.4.3. Reporting Procedure for Regulatory Authorities This study is jntended to take place at centers in the USA and Canada and therefore should satisf y both FDA and Health Canada SAE reporting requirements. During the course of the clinica} tiial, Dompé (or designee) shall inform FDA/Health Canada of any serious unexpected ADR as soon as possible and in no event later than: (a) seven calendar days after becoming aware of the info11nation if it ìs fata] or !ife threatening; and (b) fifteen ca1endar days after becoming aware of the information ìf it is neither fatal nor life threatening. Dompé (or designee) shall, within eight days after having infonned Health Canada under paragraph (a), submit a complete report in respect of that information that includes an assessment of the impo:rtance and imphcation of any findings made. Furthermore Dompé shall promptly investigate all safety information received by it and follow up information to a safety report shall be submitted to the appropriate authorities as soon as the relevant information is available. If the results of an investìgation show that an adverse drug reaction not initially determined to be reportable is reclassified as reportable, Dompé (or designee) shall repo1t such reaction in a written safety report as soon as possible, but in no event later than 15 calendar days after the deterrnination is made. 8.5. DATA MONITORINGCOMMITTEE An independent Data Monitoring Committee (DMC) wilJ be established to ensure patient safety, to consider patient risks against the potential for meeting tria! objectives, and to • previde recommendations to Dompé with respect to the conduct and analysis of the tiial. The DMC will operate independently of Dompé, and its members will not have connections to the Sponsor with the exception of the compensation to DMC members related to DMC activities. The DMC will comprise three voting members. They will be a multidisciplinary group that will include: Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page42 of73 • A surgeon with extensive experience in lung transplantation, • A physician with relevant experience in pulmonary and criticai care medicine (e.g. a. Medical Director of a Lung Transplant Program), • A biostatistician. The DMC will perfonn the following major functions: • The DMC will be responsible for the ongoing review of safety data throughout the tria!. Primary among the safety data that will be reviewed are serious AEs, ADRs and relevant available clinica] laboratory results. • The DMC may review acute and additional safety data, on a patient-by-patìent basis, at their discretion. This review would be warranted if significant unexpected ADRs are observed. • The DMC may request additional safety data if, in the view of the Committee, these adclitional data are needed in order to make an informed, responsible decision about the conduct of the study. • The DMC will advìse Dompé regarding safety of patients and continuing validity and scientific merit of the trial. The DMC will also monitor the number of deaths for both arms of the study to ensure that mortality is consistent with historical data. Ali details of the conduct and responsibìlities of the study DMC will be described in the 'DMC Charter' whkh will be finalized prior to the start of patient enrolment. Final Revision No. 3, 20-March-2006 Stud y REP0 I 04 Repertaxin in Lung Transplantation Page 43 of 73 9. STATISTICAL ISSUES 9.1. SAMPLE SIZE The sample size has been estimated based on PaO2/FiO2 ratios immediately and at 24 hours from the results of 700 lung transplant operations at the Washington University. The database reports means and standard deviations for this endpoint of 329±137 mmHg at the initial ICU assessment and 315±117 mmHg at 24 hours. A sample size of 50 in each group will have a 81 % power to detect a difference in means of 70 mmHg (the difference between a placebo mean of 315 and a treated mean of 385, a 22% improvement) assuming that the common standard deviation is 137 using a two group t-test with a 0.05 one-sided significance leve!. Altematively, a sample size of 50 in each group will have a 56% power to detect a difference in means of 50 mmHg (the difference between a placebo mean of 315 and a treated mean of 365, a 15% improvement) assuming that the common standard deviation is 137 using a two group t-test with a 0.05 one-sided significance leve!. There is no currently available datato provide a knowledgeable estimate of the effect size in this study. If the effect size is smaller than 15%, the power of this study will be reduced and the chance of a Type II error is increased. Under such circumstances, this study would stili provide valuable data regarding the effect size in the primary outcome variable as well as the results in ali of the secondary, exploratory endpoints and subgroup analysis. 9.2. RANDOMIZATION Patient will be randomized in a 1: 1 fashion to either repertaxin or placebo. Treatment will be balanced within centers. A master randomization list will be prepared, randomizing an excess of patients to allow flexible/competitive recruitment within each center. The randomization list will be prepared by according to the current versi on of the Standard Operating procedure (SOP). The randomization code will be broken at completion of the main part of the study, i.e. when the last patient in has completed his/her 30 days follow-up visit, and once the database has been locked. 9.3. ANALYSIS POPULATION The Safety population will consist of ali patients who received any study medication, and will be based on the treatment actually received. The Safety population will be used to present the demographic and baseline data, and ali safety data. The Intent to Treat (ITT) population will consist of ali patients who recei ved any study medication and the transplant, and will be based on the treatment randomized, regardless of the treatment actually received. The Per-Protocol (PP) population will consist of ali patients in the ITT population who did not have any major protocol violations, defined to be: Final Revision No. 3, 20-March-2006 CONFIDENTIAL PPD Study REP0I04 Repertaxin in Lung Transplantation Page 44 of73 • Patient who started study drug infusion after reperlusion, i.e. after vascular de- clamping of the first allograft, • Patients who did not receì ve the study medicati on for at least 24 hours, • Patients without data for PaOiFiO 2 ratio at O and 24 hours, while on mechanical ventìlation, • Patients who receive Orthoclone OKT3 or Campath induction immunosuppression, The primary and secondary efficacy analyses will be presented for the ITT population, primarily, and confinned by the PP population. 9.4. STATISTICAL METHODOLOGY Appropriate descriptjve statistics will be produced, according to the variable. For continuous data the mean, standard deviation, medìan and range (minimum and maximum) will be presented. For categorica! data, frequencies and percentages wìll be presented. If appropriate, confidence intervals around the mean or the proportions will be presented. Ali patient data collected on the CRF will be listed by patient, treatment group and center. The data for the doubl.e-blind phase of the study (up to 30 days post-treatment) will be presented in the clinica! study report. Data collected during the open phase of the study (from 1 month up to 12 months) will be presented in an addendum to the clinica] study report. Unless otherwise specìfied, the significance leve] used for statistical testing will be 5% and two-sided tests will be used. 9.4.1. Demographic and baseline characteristics Demographic and baseline characteristics will be summarized for all patients in the Safety population, by treatment group. 9.4.2. Primary efficacy analysis The PaO2/FiO2 ratio will be analyzed separately, at the two time points: O hours (ICU admission) and 24 hours after ICU admission. Analysis of variance wìll be used, including terms for treatment and center. The impo1iance of the treatment by center interaction wìll be investigated but if this is not significant at the 10% level, this term will be excluded from the final model. If a center by treatment interaction is detected, alternative methods of presentation will be explored. Treatment effect will be primariJy compared using a one-sided 0.05 leve] test and then re-tested for consistency with a two-sided 0.05 level test. Type of transplant (single or bilateral) wi11 then be included in the model of the analysis to explore any possible, even unanticipated, differences due to the specific procedure. Primarily the data will be analyzed excluding missing data. As confinnatory analysis, the ANOV As wìll be performed substituting any missing values by the last observation ca1Tied forward (LOCF) method, in patients extubated before 24 hours. If the data are missing due to Final Revision No. 3, 2O-March-2006 ( • Study REPO l 04 Repel1axin ìn Lung Transplantation Page45 of73 patient death, the PaO2/FiO2 ratio will be given a zero va]ue. If the data are missing due to Jack of recording while on mechanical ventilation, these data will not be substituted. 9.4.3. Secondary efficacy analysis The PaO2/FiO2 ratio will be analyzed, as described for the p1imary efficacy endpoint, at 48 hours and 72 hours after ICU admission. Analysis of variance will be used, including te1ms for treatment and center. The ìmportance of the treatment by center interaction will be ìnvestigated but if this is not significant at the 10% level, this tenn will be excluded from the final model. If a center by treatment interaction is detected, alternati ve methods of presentation will be explored. Type of transplant (single or bilateral) wm then be inc1uded in the model of the analysis to ex.plore any possible, even unanticipated, differences due to the specific procedure. Analysis of PGD score will be performed separately at the four time poìnts (O, 24, 48, 72 hours) to test for differences between treatments. Two analyses will be perforrned at each time-poìnt, using Cochran-Mantel Haenszel statistics, with the first analysis usìng type of transplant as a stratification factor, and the second using center as a strafrfication factor. The time to freedom from rnechanical ventilation and the duration of ICU stay will be presented using Kaplan-Meier plots, and wiH be analyzed using the Median test, to test for differences between treatments. Time to freedom from mechanical ventilation and the duration of ICU stay will also be presented by type of transplant, ancl the Median test will be performed on each type of transplant, if there are sufficient patient numbers in both categories. Ali other secondary endpoints wìll be presented using appropriate descriptive statistics, and no formal statistica] testing will be performed. ICU mo1tality and the number of patients who die within the first 30 days post transplant will be tabulated. FEV1 and PVC at 1 month post-transplant will be presented by type of transplant. FEV I and FVC, BOS score, cumulative acute rejection episodes and patient survival at 6 and 12 months post-transplant will be presented in an addendum to the clinica! study report, usìng descriptive statistics. BOS score, cumulative acute rejection episodes and patient survival will also be presented by type of transplant. 9.4.4. Analysis of the endpoint targeted to the mechanism of action PMN count in BAL specimen will be presented using descriptive statistics. 9.4.5. Analysis of pharmacokinetic endpoints Plasma levels of repertaxin (total and unbound) and its major metabolite, DF2243Y, are recorded 24±6 hours anà 48 hours from the start of infusion, and then in the interval 2-6 hours after the end of infusion. Data will be summarised using descriptive statistics. Final Revision No. 3, 20-March-2006 Study REP0I04 Repertaxin in lung Transplantation Page 46 of73 9.4.6. Saf ety analysis Safety variables will be presented for the Safety population. AEs will be presented, by treatment group, in tenns of the incidence, severity and relationship to the study drug, overall and by body system and preferred term. SAEs will be presented in the smne way. Laboratory tests are pe1formed at screening and at hospital discharge and shìft tables will be used to present shifts from within/outside the norma} range between screening and discharge. Vital signs at each time point, the change in vital signs from screening to each time point, and cardiopulmonary physìologic measurements at each time point will be presented using descri pti ve statisti es. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxi11 in Lung Transplantation Page 47 of 73 10. ETHICAL CONSIDERATIONS 10.1. INSTITUTIONAL REVIEW BOARD/RESEARCH ETHICS BOARO lt is the responsibility of the Investigator to obtain approvai of the trial protocol/amendments frorn the Institutional Review Board/Research Ethics Board (IRB/REB). Prior to the initiation of the study, the Investigator wìl1 submit to the approptiate IRB/REB for approva] the followings: • the study protocol, • the Patìent Informed Consent Document and any other w1itten documents to be provided to the patient, • the current version of the Investigator's Brochure, • Investigator's current curriculum vitae, • patient recruitment procedures e.g. advertisements, • any other requested document(s), A copy of the IRB/REB approva} will be sent to PPD along with ali other correspondence with the IRB/REB, including the submissìon documents. The Investigator should file all con-espondence with the IRB/REB in the Investigator Site File. PPD wìll promptly send to Dompé a copy of the IRB/REB approval. The study will not be started until full written approva] has been obtaìned from the appropriate IRB/REB. The letter of approval should be dated, and should specify the type (e.g. protocol number) and the date of the documents which were reviewed and approved. The Investigator will submit any future amendment to the protocol to the IRB/REB which granted the original approval (and other local authorities, according to local regulations). Any amendment will be implemented only when full approvai has been obrained from the approptiate IRB/REB, except for those amendments which involve only .logistica! or administrative aspects of the study. The Investigator wiU send to the IRB/REB any updated Investigator's Brochure. A dated list of the voting mernbers of the JRB/REB who were present when the protocol/amendment was reviewed and approved, including their titles/occupations and institutional affiliations should be provided where possible by the Investigator to PPD prior to study initiation. The Investigator will make all attempts to obtain a staternent from the IRB/REB that it is constituted and operates in accordance with the ICH-GCP and any locai regulations. The Investigator will submit required progress reports to the IRB/REB which approved the protocol at least annually, as well as report any serious unexpected ADRs, life-threatening problems or deaths. The Investigator will also infonn the IRB/REB of repo11s of serious unexpected ADRs (provided to him/her by PPD ) occurred at other sites participating to this clinica! trial and/or in other clinicàl studies conducted with repertaxin. The Investigator must inform the IRB/REB of the tennination of the study. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 48 of 73 10.2. INFORMED CONSENT No study-related procedures (including non-invasive and diagnostic procedures) will be undertaken prior to completion of the consenting process. Each potentially eligible patient will be informed of the study's objectives and overall requirements. The Investigator will explain the study fully to him/her using the Patient Informed Consént Document. If the patient is willing to participate in the study, (s)he will be requested to give written informed consent after being given sufficient time to consider his/her participation and the opportunity to ask for further details. The Patient Informed Consent Document will be signed and personally dated by both the patient and the Investigator. A copy of the signed form will be provided to the patient and the originai signed Patient Informed Consent Document will be retained and filed in the Investigator Site File. Although nursing staff may be involved in describing the tria] to a patient, the Investigator must participate in discussions with the patient and sign and personally date the Patient Informed Consent Document. Individuai (i.e. site specific) Patient Informed Consent Forms will be based on a master document provided by and must be approved by prior to submission to the IRB/REB. Any changes requested by the IRB/REB must be approved by prior to the documents being used. A copy of the final, IRB/REB-approved Patient Informed Consent Document must be submitted to prior to initiation of this study. Where applicable, patients next on the waiting list (max 25 patients) will be pre-consented, i.e. consent is provided before the patient is called into the hospital for transplantation. 10.3. CONFIDENTIALITY The Investigator must ensure that the subjects' anonymity will be maintained. On the CRFs or other documents submitted to patients should NOT be identified by their names, but by the assigned patient number if randomized plus their initials and date of birth (if not randomized). If patient names are included on copies of documents submitted to the names (except for initials) will be obliterated or masked and the assigned patient number added to the document. The Investigator should keep a separate log (Patient Master List) of patient's codes, names, addresses, telephone number and hospital number (if applicable ). Documents that are collected but not required for submission to (e.g. signed Patient Informed Consent Forms) should be maintained by the Investigator in strict confidence. 10.4. COMPENSATI ON FOR MEDICINE-INDUCED INJURY AND INDEMNIFICATION Before the tria! formally starts, Dompé will take out an insurance contrae! covering the amount requested by the respective national/federal laws for patients participating in clinica] trials. The insurance contrae! will also cover the Investigator/Institution against claims arising from the tria!, except for claims that arise from malpractice and/or negligence, including violations of the study protocol. Final Revision No. 3, 20-March-2006 CONFIDENTIAL PPD PPD PPD PPD PPD PPD PPD Study REP0104 Repertaxin in Lung Transplantation Page49 of73 Should any patients suffer adverse effects as a direct consequence of the Investigational Product administration in accordance with the protocol, Dòmpé will reimburse for hospital medicai costs required for diagnosis and treannent. Medical treatment shall be provided primarily through the study center. Dompé will not provide for the costs of medica] care unrelated to the study. In case of questions about medical care, cost for medical care or insurance, patients can talk to their Investigator. Contact details will be given in the Patient Infonned Consent Document. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 50 of73 11. DATA HANDLING ANO RECORD KEEPING 11.1. CASE REPORT FORMS COMPLETION CRFs will be supplied by PPD •. CRFs are the sole property of PPD and should not be made available in any fonn to third parties, except for authorized representatives of appropriate Health/Regulatory Auth01ities, without w1itten perm.ission from PPD ------ A CRF is required and should be completed for each included patient. The Investigator will be responsìble for the accuracy of the data entered ìn the CRFs. All entries must be written in ENGLISH in black ink. Source documents should be available to support ali the data recorded in the CRF. Location of source data, including those for whìch the CRF might be accepted as being the sole source document, will be specified and listed at the center Inìtìation Visit. The CRF must be available for review/collection to designated PPD representatives at each scheduled rnonito1ing visit. 11.2. DATA MANAGE1\1ENT Data management of the CRFs will be perfonned by PPD distinct phases. and will be split into two For the blìnded (main) part of the study the CRF pages for all patients up to the month 1 visit wìl1 be data-entered (double data entry) into the study database, and the data will be verified for missing data, ìnconsistencies, and for any necessary medical clarifications. Queries arising from these checks will be sent to the Investigator for response and signature. All possible attempts should be made by the Investigator to complete and return the signed responses as instructed to PD within the requested timeframes. Once all data queries pertaining to the blinded part of the study have been resolved, the study will be declared to be "clean", and the study data will be locked ready for analysis. FoIJowing the data lock the study blind will be broken and each patient' s medicina] assignment (i .e. repertaxin or placebo) will be revealed to the appropriate Investigator. The CRF pages for both follow up visits i.e. months 6 and 12 will be entered ìnto a different location of the same database using the same procedure as described above. Furthermore when the last patient's month 12 visit has been "deaned" the database will be Iocked ready for the final analysis. After the database lock has been achieved, the Investigator may archive che copies of the CRFs retained at the center. The origina} CRFs collected by PPD will be archived by Dompé. Ali data management will be conducted in accordance with good clinical, scientific and data management principles and in compliance with current PPD SOP. Final Revision No, 3, 20-March-2006 ( Study REPO 104 Repertaxin in Lung Transplantation PageSl of73 11.3. DOCUMENTA TION REQUIRED PRIOR TO INITIATION In addition to the documents mentioned in Sections 10.1 and 12.1, the following will be required from the Investigator prior to the initiation visit: • • • • • • • • • 11.4. Current, signed and dated Curriculum Vjtae of Principal Investigator and any Sub-In vesti gators/co-workers, Normal ranges of all laboratory tests to be performed at the study site and a recent certificatìon or accreditation of established quality control (or other documentation of established quality contro! or extemal quality assessment or other validation), A signed origina] of the final protocol and any amendments, Indemnìty / certificate of Insurance, A signed originai of the study Financial Agreement/Clinical Study Agreement with Dompé, including Phannacy, radiology etc (i.e. all study specific costs), Fonn 1572 (USA and Canada), Qualified Investigator Undertaking, Research Ethics Board Attestation, Clinica! Trial Site Infonnation Form (Canada only). Financial disclosure fo1m 3455 from all persons listed on the 1572, Data protection form (HJP AA in USA) signed by the Investigator (givìng the Investigator's approval and the patient's consent for persona! data to be kept on file at PD ), List of delegated responsibility (Study Team Signature List / Delegatìon of Responsibilities fo1m). DOCUJ.\tlENTATION REQUIRED DURING THE STUDY The following will be required from the Investigator during the study: • CmTent, signed and dated Cuniculum Vitae of Principal Investigator and any Co-Investigators/co-workers who are delegated protocol related responsibilities after study initiation, • Updates of nonnal ranges (including the dates from which they become effective) of all laboratory tests to be pe1fom1.ed at the study site and updates in certification or accreditati on of established quality control ( or other documentation of established quality contro! or extema1 quahty assessment or other validation), • A signed original of any protocoJ amendments, • Copies of any new approvals from or correspondence with the IRB/REB, • Renewals of certificates of Insurance, as app]icable per country regulations, • Updated form 1572 (USA and Canada), Qualified Investigator Undertalcing, Research Ethics Board Attestation, Clinica! Trial Site Information Form (Canada only), • Financial disclosure fonn 3455 from all new persons listed on the 1572, • Updated list of delegated responsibility (Study Team Signature List / Delegation of Responsibilities fonn). Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 52 of73 11.5. ESSENTIAL DOCUMENT RETENTION The Investigator will retain copies of all the essential documents (as defined by ICH-GCP) unti] at Jeast 2 years after the last approvai of a marketing application in an ICH region, and until there are no pending or contemplateci marketing applications in an ICH regi on, or at least 2 years have elapsed since the formai discontinuation of clinica! development of the Investigational Product. These documents should be retained for a longer period however if required by the apphcable regulatory requirements. The Investigator should take measures to prevent accidental or premature destruction of these documents. The essential documents include at least: the signed protocol, copies of the completed CRFs, signed Patient Informed Consent Forms from ali patients who consented, hospital records, and other source documents, IRB/REB approval and all related correspondence, including approved documents, and all other documentation included in the Investigator Site File and Phannacyillispensing File. The Investigator will inform PPD of the storage location of these essential documents and rnust contact PPD before clisposing of any. If the Investigator wishes to assjgn the files to someone else or to remove them to another location, the PPD Medicai Director should be consulted about this change. Dompé will infonn the Investigator in writing when these documents no Jonger need to be retained. Fina! Revision No.3, 20-March-2006 Study REPO I 04 Repertaxin in Lung Transplantatìon Page 53 of 73 12. STUDY MANAGEMENT The study wiJl be performed in accordance with the protocol, the Declaration of Helsinki (Appendix 5), and ICH Harmonised Tripartite Guideline for Good Clinica! Practice (ICH- GCP) and any local regulations. 12.1. REGULATORY BODY APPROV AL The study will not be started until full written approval from the relevant reglllatory body (FDA and Health Canada) has been received by Dompé. PPD will then previde the Investigator with a copy of appropriate document on behalf of Dompé. PPD unde:rtakes to obtain (on behalf of Dornpé) the necessary approvai from the Health Canada p1ior to initìation of the study. The responsibìlity for gaining FDA approva] lies with an independent regulatory consultant employed by Dompé. 12.2. STAFF INFORMATION & RESPONSIBILITIES It is the responsibility of the Investigator to ensure that all personnel involved in the study are fully infonned of all relevant aspects of the study, including detailed knowledge of and training in all procedures to be followed. The Investigator will provide a list of delegated responsibility to PPD detailing the various study tasks to be performed by each member of hìs/her study staff. Bach staff mernber should sign in agreement to their perfonning each of the tasks delegateci to them on the list. 12.3. MONITORING Monitoring will be canied out by ..._P_D ___ _., according to the current version of PD SOP. Prior to study stait, the Investigator will be informed of the anticipated frequency of the monitoring visits. He/she will also receive a notification prior to each monitoring visit during the course of the study. It is expected that the Investigator and/or his/her sub-Investigator(s) and other appropriate staff will be available on the day of the visit to discuss study conduct and to cooperate with the monitor to ensure that auy problems detected during the course of these monitoring visits are resolved. The purpose of the monitoring visit .is to ve1ìfy that the rights and the wellbeing of the patient are protected, that the reported data are accurate, complete and verifiable from source documents and that the conduct of the tria! complìes with the currently approved protocol and any amendments, with ICH GCP, and with regulatory requirements. The CRA will complete a standard report following each Monitoring Visìt and provide this repo1t to the Project Leader for review within a specified tirneframe. Final Revision No. 3, 20-March-2006 Study REPO 104 Repertaxin in L1mg Transplantation Page 54 of73 12.3.1. Access to records The Investigator will a1low designated PPD representatives, designated Dompé representatives, and regulatory bodies to have direct access to the source documents to verify the data reported in the CRFs. Source documents are the originals of any documents used by the Investigator or hospital/institution that allow verification of the existence of the patient and substantiate the ìntegtity of the data collected during the tria!. Source documents shoulcl be available to support all the data recorded in the CRF. Location of source data, including those for which the CRF might be accepted as being the sole source document, will be specified and listed at the center Initiation Visit. 12.4. AUDIT AND INSPECTION Audìt activìties will be perlormed by PPD Quality Assurance group, except for audit of Protocol/ Amendments, Patient Informed Consent Document, and CRF, which will be perfonned by the Dompé Quality Assurance Unit. On one or more occasions the study site may be audited by PPD . The Investigator will be informed in advance of such a vìsit. All audits will be performed by PD personnel according to the current version of PPD SOP. Additionally the study site may be inspected by Dompé representatìves or a regulatory agency on one or more occasions. 12.5. PROTOCOL DEVIATIONS/Al\tIENDMENTS Changes to the Protocol wìll be implemented only when written amendments have been signed by all individuals who sìgned the protocol. Any amendment will be sent to the appropriate IRB/REB. No deviations from or changes to the protocol wilJ be implemented without documented approvai of an amendment from the IRB/REB which granted the originai approval, except where necessary to eliminate an immediate hazard(s) to tria! patient, or when the change(s) invoJves only logistica! or administrative aspects of the trial. The devìations from or changes to the protocol implemented to eliminate an immediate hazard to the tria! patient and the proposed amendrnent, if appropriate, should be submitted to the IRB/REB for review and approva! as soon as possible. Any other deviation from the protocol that has not been approved by PD and the IRB/REB could result in a dìscontinuation from the study at the center involved. Any written amendment will be sent to all recipients of the protocol and to the regulatory authorities (FDA/Health Canada). 12.6. DISCONTINUA TION OF THE STUDY Dompé reserves the right to stop the study at any time on the basis of new information regarding safety or efficacy, or if study progress is unsatisfactory, or for other valid administrative reasons. After such a decision is made, the Investigator must inform all relevant persons e.g. study staff, potential patients etc. within 2 weeks. All delivered study materials must be collected and ali CRFs completed to the extent possible. Study discontinuation will be notified to FDA/Health Canada within 5 days from decìsion. Final Revision No.3, 20-March-2006 ( Study REP0104 Repertaxin in Lung Transplantation Page 55 of 73 12.7. PUBLICATIONS Any manuscript, abstract or other publication or presentation of results or information arising in connection with the study must be prepared in conjunction with Dompé and must be submitted to the Dompé Medical Director for review and comment at least 45 days prior to submìssìon for publication or presentation. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 56 of73 13. REFERENCES Baggiolini M, Dewald B, Moser B. lnterleukin-8 and related chemotactic cytokines CXC and CC chemokines. Adv Immunol 1994; 55:97-179. Be1tìni R et al. Non-competitive al1osteric inhibitors of the inflammatory chemokine receptors CXCR1 and CXCR2: prevention of reperfusion injury. PNAS 2004; 101: 11791-11796. Christie ID, Bavatia JE, Palevsky Hl, Litzky L, Blumenthal NP, Kaiser LR et al. Primary graft failure following lung transplantation. Chest 1998; 114(1 ): 51-60. Christìe JD, Carby M, Bag R, Conis P, Hertz M, Weill D. Report of the ISHLT Working Group on Prìmary Lung Graft Dysfunction Part Il. Definition. A consensus Statement of the International Society of Heart and Lung Transplantation. J Heart Lung Transplant 2005; 24: 1454-1459. Christie ID at al., Impact of P1imary Graft Failure on outcomes following lung transplantation. Chest 2005; 127:161-165. Clinical safety data management: Definitions and Standards for Expedited Repo1ting (E2A - Approved by the CPMP in November 1994 for studies commencing after 1st June 1995 [Note for Guidance 3C614A]; Approved by FDA with effective date 1st March 1995 [60 FR 11284]). Cockcroft DW, Gault MH. Prediction of creatinine clearance from serum creatinine. Nephron 1976; 16:31-41. de Perrot M, Sekine Y, Fischer S, Waddell T, McRae K, Liu M, Wig1e DA, Keshavjee S. Interleukin-8 release during early reperfusion predicts graft function in human lung transplantation. Am J Respir Crit Care Med 2002; 165: 211-215. de Perrot M, Liu M, \.Vaddell TK, Keshavjee S. Ischemia-reperfusion-induced lung injury. Am J Respir Crit Care Med 2003; 167: 490-511. De Vries ME, Hosiawa KA, Cameron CM, Bosinger SE et al. The role of chemokine receptors in allogenic-independent anà allogenic-dependent transplantation injury. Sem Immunol 2003; 15:33-48. Estenne M, Maurer JR, Boehler A, et aJ. Bronchìolitis obliterans syndrome 2001: an update of the diagnostic criteria. J Heart Lung Transplant 2002; 21:297-310. Fischer S, Matte-Martyn A, de Perrot M, Waddel1 TK, Sekine Y, Hutcheon M et al. Low- potassium dextran preservation solution improves lung function after human lung transplantation. J Thorac Cardiovasc Surg 2001; 121(3):594-596. Fiser SM, Tribble CG, Long SM, Kaza AK, Kem JA, Jones DR, Robbins MK, Kron IL. Ischemia-reperfusion injury after lung transplantation increases risk of late bronchiolitis obliterans syndrome. Ann Thorac Surg 2002; 73:1041-1047. Final Revision No. 3, 20-March-2006 ( Study REP0104 Repertaxin in Lung Transplantation Page 57 of73 ICH Hannonised Tripartite Guidehne for Good Clinical Practice (June 1996), as recommended for adoption to the three regulatory parties to ICH. Approved on l ih July 1996 (CPMP/lCH/135/95). Approved by FDA on 9th May 1997 and effective 9th May 1997 (62 FR 25692). Accepted and published by Health Canada, 1997. Keshavjee SH, Davis RD, Zamora MR, Schulman LL, de Perrot M, Patterson GA. A randomized placebo controlled tria! of complement inhibition in ischemia-reperfusion ìnjury after lung transplantation in humans. In press King RC, Binns OA, Rochiguez F, Kanithanon RC, Daniel TM, Spotnitz WD et al. Reperfusion injury significantly impacts clinical outcome after pulmonary transplantation. Ann Thorac Surg 2000; 69(6): 1681-1685. Lau CL, Patterson GA. Current status of lung transplantation. Eur Respir J 2003; 47:57s-64s. Lefer AM, Johnson G 3rd, Ma XL, Tsao PS, Thomas OR. Cardioprotectìve and endothelial protective effects of [Ala-IL8]77 in a rabbìt model of myocardial ischaemia and reperfusion. Br J Pharmacol 1991; 103(1):1153-9. Matsumoto T, Ikeda K, Mukaida N, Harada A, Matsumoto Y, Yamashita J, Matsushima K. Prevention of cerebral edema and infarct in cerebral reperfusion injury by an antibody to interleukin-8. Lab Invest 1997; 77(2):119-25. McRae KM, Pulmonary transplantation. Curr Opin Anaesthesiol 2000; 13:53-59. Meade MO, Granton Jt, Matte-Martyn A, McRae K, Weaver B, Ctipps P, Keshavjee SH and the Toronto Lung Transplant Program. A randornized trial of inhaled nitric oxide after lung transplantation. Arn J Repsir Crit Care Med 2003; 167:1483-1489. Mìura M, Fu X, Zhang QW, Remick DG, Fairchild RL. Neutralization of Groa and macrophage inflamrnatory protein-2 attenuates renal ischemia/reperfusion injury. Aro J Pathol 2001; 159:2137-2145. Morita K, Miura M. Paolone DR, Engeman TM, Kapoor A, Remick DG, Fairchild RL. Early chemokine cascade in murine cardiac grafts regulate T celi recruitment and progressìon of acute allograft rejection. J Immunol 2001; 167: 2979-2984. Patterson GA, Cooper ID, Goldman B et al. Technique of successful clìnìcal double lung transplantation. Ann Thorac Surg 1988; 45:626-633. REP0l0l - Clìnical study ME0706; Dompé Internal Report (final). REP0102 - Clinical study 'ME0735; Dompé Interna] Report (final). REP0103 Clinica} stuyd ìv1E0757; Dompé Internal Report (final). REP0203 - Clinica} study ìv1E0761; Dompé Internal Report (final). Sekido N, Mukaida N, Harada A, Nakanishi I, Watanabe Y, Matsushima K. Prevention of lung reperfusion injury in rabbìts by a monoclonal antibody against interleukin-8. Nature. 1993; 365(6447):654-7. Final Revision No. 3, 20-March-2006 Study REPO 104 Reperta,,ù1 in Lung Transplantation Page 58 of73 Sommers KE, Giiffith BP, Hardesty RL, Keenan RJ. Early lung allograft function in twin recipients from the same donor: tisk factor analysis. Ann Thorac Surg 1996; 62(3):784-790. Souza DG, Bertinì R, Viera AT, Cunha Q, Poole S, Allegretti M, Calotta F, Teixeira M1'vL Repertaxin, a novel inhibitor of rat CXCR2 function, inhibits inflammatory responses that follow intestinal ischemia and reperfusion injury. Br J Pharmacol 2004; 143:132-142. Thabut G, Vinatier I, Stem J-B, Lesèche G, Loirat P, Fournier M, Mal H. Primary graft failure following lung transp1antation. Predictive factors of mortality. Chest 2002; 121: 1876-1882. Waddell TK, Gorczynsld RM, DeCampos KN, Patterson GA, Slutsky AS. Major histocompatibility complex expression and lung ischemia- reperfusion in rats. Ann Thorac Surg 1996; 62(3):866-872. Welbourn CR, Goldman G, Paterson IS, Valeri CR, Shepro D, Hechtman HB. Pathophysiology of ischaemia reperlusìon injury: centra} role of the neutrophil. Br J Surg. 1991; 78(6):651-5. Yamaguchi Y, Ohshiro H, Nagao Y, Odawara K, Okabe K, Hidaka H et al. Urinary trypsin inhibitor reduces C-X-C chemokine production in rat liver ischemia/reperfusion. J Surg Res 2000; 94:107-115. Yousem SA, Be1Ty GJ, Cagle PT, et al. A revìsion of the 1990 working formulation for the cJassification of pulmonary allograft rejection: Lung rejection study group (LRST). J Heart Lung Transplant 1996; 15:1-15. Zamora MR, Davis RD, Keshavjee SH, Schulman L, Levin J, Ryan U and Patterson GA. Complement ìnhibition attenuates human lung transplant reperfusion ìnjury. Chest 1999; l 16:46S. Final Revision No. 3, 20-March-2006 Study REPO 104 Repertaxin in Lung Transplantation Page 59 of73 14. APPENDICES 14.1. APPENDIX 1 - METHODOLOGICAL DETAILS 14.1.1. PGD score - ISHL T system for grading of severity of primary graft dysfunction [ Christie et al, 2004] Grade O PaOi/FiO2 >300 mmlfa; no radiograohic ìnfiltrates Grade 1 PaO2'FiO2 2:,300 rnmHg + radiographic infiltrntes consistent with pulmonarv oedema Grade 2 200 mmHg _:ç PaOz/FiO2 <300 mmHg + radiographic infiltrates consistent with oulmonarv oedema Grade 3 PaOi/FiO2 <200 mmHg + radiographìc infiltrates consìstent with pulmonarv oedema Any patient with no infiltrate on chest X-rays is automatically grade O, regardless of PaO2/FiO2 value. If the patìent is on nasal cannula for oxygen or FiO2 <0.3, the patient is graded as O or 1, based on chest X-rays. Any patient on extra corporea] membrane oxygenation is automatìcally grade 3. Any subject mechanically ventilated with FiO2 greater than 0.5 or requiring nitric oxide beyond 48 hours from the time of trnnsplant should be considered grade 3. If multiple blood gas values are available, the worst Pa0i/FiO2 ratio will be used for the purpose of this grading scheme. PGD score will be evaluated at the following time points: - Time-O (T0): defined as within 6 hours of final lung reperfusion. The first blood gas in ICU is idea! for this. ldeally, measurement should be done on FiO2 1.0 and PEEP =5 while stili on mechanìcal ventilation. - T24, T48, T72: Later times being measured at potentially multiple time points after 24 hours, up to 72 hours. Time will be measured following T0±6 hours. Chest X-rays will graded by an Investigator, and then validated by a (blinded) transplant radiologist. In patients with cJinical diagnosis of PGD additional time points will be considered up to the clinical recovery of the condition as per available data according to center standard of care. 14.1.2. Bronchoalveolar lavage methods BAL specimen BAL sample will be taken from the allograft (first implanted allograft, in case of bi-latera1 lung transplantation). Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 60of73 The dìstal bronchoscope will be wedged in a subsegmental bronchus of either the right middle or left lingular lobe. Ifa prominent, focal infiltrate is present on chest X-rays, BAL will be perf ormed in the area of maximal radiographic abnormality. Approximately 50 mL aliquots of sterile, pre-warmed, non-pyrogenic, isotonic sodium ch]oride solution will be instilJed through the bronchoscope and immediately recovered by gentle suctìon (60 to 80 mmHg). This "back and fo1th" is repeated for a maximum of 4 washes. The recovered fluid will be pooled and mixed thoroughly. Tota} recovered volume will be measured. BAL fluid will be transferred to the center laboratory on ice, but it can be transported at room temperature if processing wil1 be undertaken within 30 minutes. BAL processing The recovered ce11 suspension wìll be filtered through surgical cotton gauze to remove excess mucus. To determine the total cell yield, a small aliquot of the fluid will be used for counting the ce1Is on a hemacytometer. Red blood cells are not included in the total celi yield. The BAL fluid will be then placed in siliconized glass or non-cell-adherent plastic centrifuge tubes and will be centrifuged at 200 x g for 5 minutes. The supematant will be discharged, taking care not to disturb the cell pellet. The cells pel1et will be resuspended in and washed twice with Hanks' balanced salt solution (or another medium such as phosphate-buffered sa1t solution) without Ca2+ and Mg2+. To obtain differential cell counts the cell suspension (approximately lxl0 5 cells) will be cytocentrifuged at 800 g for 10 minutes. The slides will be stained as per center practice (May-Grunwald-Giemsa or Diff-Quick stain is suggested) and air dried at room temperature for 1 hour. Stained spot will be fixed (Entellan fixative is suggested), and slides covered by a microscope cover glass and stored until shìpment to Dompé pha.r.ma s.p.a. (see below). Detailed packaging and labeling instruction and shipment procedures will be communicated at each center at stucly initiation. Cell differentials wiH be petfonned by Dompé pha.r.ma s.p.a. - Preclinical Pharmacology Department. Two hundreds cells will be counted in each slide. 14.1.3. Handling of samples for pharmacokinetic assay Samples will be centrifuged ideally within 15 minutes at 2500g (3350 rpm) at 4°C for 10 minutes. If that is not possible then samples can be stored at room temperature for not more than 2 hours or at 4°C for not more than 24 hours before centrifugation. At least 3 m.L of plasma will be obtained and divided equally into 2 aliquots, one to be used for tota! repertaxin and DF2243 Y, the ot.her for the repertax.in unbound fraction. Plasma samples will be stored at approximately -80°C into stopped glass tubes, unti! shipment to the centra1ized analytical Iaboratory for assay. Samples will be shipped to the centralized laboratory in appropriate package in dry ice (solid C0 2) to maìntain frozen conditions. Detailed packaging and labelìng instruction and shipment procedures will be communicated at each center at study initiation. Analysis will be perlormed by a centralized laboratory according to standardized and validated methods as per Good Laboratory Practices. Final Revision No. 3. 20-March-2006 Study REPO l 04 Repertaxiti in Lung Transplantation Page 61 of73 PPD , has been identìfied as the centralized laboratory where phannacokìnetic analysis is pe1formed. Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 62 of73 14.2. APPENDIX 2- PACKAGING AND LABELJNG DETAILS A "Patient Box" will be prepared for each patient. Each Patient Box will contain: • Two Treatment Boxes, each with 25 x 10 mL ampoules - either containing repertaxin or placebo. • Two (p]us one reserve) sterile empty infusion bags • Instructions to the Phannacy All items will have labels (English and French language forali, except ampoules); specimens of these are provided below. Only the Iabel of the infusion bag will be a "double - tear off' label. The Investigational Product already packaged to the date of thìs protocol revision, whether sent to the site or stored at Aptuìt, WILL NOT BE RE-LABELED. Labels of any additional future supplies will have the same format/information, apart from the name of the sponsor that will read "Dompé pha.r.ma s. p.a. ". NOTE: + Patient No. XXYY where XX= Study site YY = Patient sequential number within the site (The first patient at Study Site No.1 will have the number 0101). lnfusion Bag - "Preparation" date and time, as well as "Use by" date and time will be entered by the Phannacist. Use by date/time will be calculated as date/time of preparati on+ 72 hours (expiry date/time of the dosing solution). Patient Box and Infusion Bag - Patient initials will be entered by the Pharmacist. Specimen Label for exterior of each Patient Box STUDY (ESSAI) REP0104 Sponsor (Promotew'): Dompe s.p.a.~ Via Campo di Pile, L'Aquila -- ltaly PATIENT BOX (BOITE pour PATIENT} PATIENT No. XXYY PATIENTN° PATIENT INITIALS 1-1-1-1 [to be entered by the Pharmadst] INITIALES DU PATIENT fàcomp!éterpar!ePharm~cienJ CONTAINS: 1WO TREATMENT BOXES [No. 1 (0·24 h); No. 2 (24-48 h)], 1WO STERILE EMPTY INFUSION BAGS (PLUS ONE RESERVE BAG), INSTRUCT1CNS TO THE PHARMACY CONTENfJ: DEUX 80/TES DE TRAITEMENT [N° 1 (0-24 h}; N° 2 (24-48 h}J, DEUX POC4ES DE' PE'RFUSION VIDES ET STERILES (PLUS UNE POCHE DE RE'SE.~VE}, LES INSTRUcnONS POUR lA PHARMAOE INVESTIGATIONAL PRODUCTS: REPERTAXIN OR PLACEBO INJECTABLE SOLUTION PRODUffS DE RECHERCHE: SOLIJTION INJECTABLE DE REPERTAXIN 0IJ DE PLACEBO BUNDED BATCH No. N" Oé LOT CODJffE For questions • CRO contact = Pour toute questton contacter la ORC= EXPIRY DATE mm/yyyy DATE Dé PERE'MP710N mm/aaaa I DO NOT STORE AT >30°C {86°F) NE PAS ENTREPOSER A >30°C (86•F) I DIRECTIONS: Use ampoules in Treatment Box No. 1 and Treatment Box No. 2 to prepare the dosing solution of Investigational Product for the 0-24 hours and 24-48 hours periods, respectively. INSTRllCTIONS: Utiliser respectivement !es ampoufes de la Bolle de Traltement N° 1 et de la Bofte de Ttattement N" 2 pour préparer !es doses de so!utlons du Produits de Recherche durant !es périodes de 0-2·1 heures et 2448 /Jevres. CAUTION: New Drug-Umited by Federai law to investigational use. Investigational drug to be used by a qualified investigator only. ATTENTION: Nouvelle substance • Umitée à la recherche par la foi Fédérate. Produft de recherche, ne dolt étre urìliSé que par un tnYestlgateur quali!Jé. Final Revision No.3, 20-March-2006 Study REPOJ04 Repertaxin in Lung Transplantation Page 63 of73 Specimen Label for Treatment Box No. 1 [time period 0-24 hours] STUDY (ESSAI) REP0104 Sponsor (Promoteur''): Dompé s.p.a.; Via Campo di Pile, L'Aquila - Italy TREATMENT BOX No. 1 {0·24 hours} BOITE DE TRAITEMENT N" 1 (0-24 heures} PATIENT No. XXYY PATIENTN~ CONTAINS: 25 AMPOULES OF 10 ml REPERTAXIN (33 mg/ml) OR PLACEBO FOR INJECTABLE SOLUTION CONTENU: 25 AMPOUlE5 OE 10 ml DE SOLUTION INJECTABLé DE REPERTAXIN (JJ mg/ml) OU Oé PLACEBO BLINDED BATCH No. N" DE LOT CO[JlfiE EXPIRY DATE mrn/yyyy VATE DE PERENPTION mm/aaaa DO NOT STORE AT >30°C (86°F) NE PAS ENTREPOSER A >30°C {86~F) For questions - CRO contact "' Pour toute question contacter la ORC:= PPo.---------------------- DJRECTIONS: Transfer into infusion BAG No. 1 the contents of 23 AMPOULES + 370 ml of 0.9% Naa, according to procedures detailed !n the "Jnstructions to the Pharrnac/'. Report preparation date and time, and use by date and time (72 hours from preparat!on) on the bag !abel. NOTE: This box contains 2 AMPOULES ìn excess that are to be used as RESERVE ONLY. INSTRUCTIONS: Transférer le contenu de 23 ampoules dans la Pod1e de perfusion N° 1 + 370 ml de NaCI à 0.9%, comme Jnd!qué dans la procédure déloiliée « Instructions pour la Pharmade ». écrire /éJ dc1te et /'Mure de préparatlon ainsl que la date et l'heure de péremption (72 heures à partir de fa préparation) s11r l'étlquette de la poche. NOTE: Celte boite conaent 2 AMPOULES supplémeotat,--es à n'utiliser que COM/IIE RESERVE. CAUTION: New Drug-Limited by Federai law to investigational use. Investigalional drug to be used by a qualified investlgator on!y. A TTENTION: Nouvelle substance - Umitée à la recherche par /éJ tal Fédérale. Produit de recherei/e, ne doit etre uti!isé que par un lnvestigateur qua/itìé. Specimen Label for Treatment Box No.2 [time period 24-48 hours] STUDY (ESSAI) REP0104 Sponsor (Promoteur'): Dornpé s.p.a.; Via campo di Pile, L'Aquila - Jtaly TREATMENT BOX No.2 (24-48 hours) BOITE DE TRAITEMENT N° 2 (24-48 heures} PATIENT No. XXYY PATIENTN° CONTAINS: 25 AMPOULES OF 10 ml REPERTAXIN (33 mg/ml) OR PLACEBO FOR INJECTABlE SOLUTION CONTENU: 2S AMPOUlES DE 10 ml DE SOl/JTlON /NJECTABLE DE REPERTAXIN {33 mg/ml} OU DE PLACEBO BLINDED BATCH No. N° DE LOT CODIFIE For questions • CRO contact = Pour toute question contacter là ORC= EXPIRY DATE mm/y,fYY DATE DE PERéMPTION mm/a<1aa PD DO NOT STORE AT >30°C (86°F) NE PAS ENTREPOSER A >.30°C {86°F) OIRECTIONS: Transfer into infusion BAG No. 2 the contents of 23 AMPOULES + 370 ml of 0.9% NaO, according to procedures detaileò in the "Instructìons to the Pharmacy". Report preparation date and time, and use by date and time (72 hours frorn preparation) on the bag label. NOTE: This box contains 2 AMPOULES In excess that are te be used as RESERVE ONLY. INSTRUCTIONS: Transférer le contenu de 23 r1mpo11les d,ms la Poche de perfusion N° 2 + 370 ml de Naa iJ 0.9%, comme im:liqué dans la procédure détatlfée « Instructions pour la Phannacie .», ferire la date et l'heure de prép,m1aon ainsi que fa date et l'heure de péremption (72 lleures à partir de la préparation) sur l'étiquette de la poche. NOTE: Cette bcite contient 2 AMPOlJLES supplémentaires à n'utilìser que COMME RESERVE. CAUTION: New Drug-Limited by Federai law to investigalional use. Investigational drug to be used by a qualified investigator only. A TTENTION: Nouvelle substance • Limltée à fa recherclle par la loi rédérafe. Produit de recherche, ne doit étre utìlisé q11e par un investigateur quatifié. Final Revision No.3, 20-March-2006 Study REPO 104 Repertaxin in Lung Transplantation Page 64 of73 Specimen double tear off Label for Infusion Bag No. 1 [time period 0-24 hours] STUDY (ESSAI) REP0104 Sponsor (Promoteur): Dompé s.p.a.; Via campo dì Pile, L'Aquila - Italy INFUSION BAG No. 1 (0-24 hours) - POCHE DE PERFlJSION N° 1 (0-24 heures} PATIENT No. XXYY PATIENTN° PATIENT INITIALS 1-1-1-1 [to be entered by the Pharmadst] INITIALES DU PA TIENT {ii compléter par te Pflarmacien] CONTAINS: 600 ml OF REPERTAXIN (12.65 mg/ml) OR PLACEBO INJECTABLE DOSING SOLUTION CONTENU: 600 ml DE SOllfflON !NJECTABLE DE REPERrAX!N (12.65 mg/ml) ou DE PLACEBO PREPARED ON I_I_I/ l_!_I/ I_I_I_I_! PREPAREE LE dd (jj) / mm I WYY (aaaa) USE BY [_l_l/l_l_l/l_l_l_i_l lJTIUSERAVANTLE dd (ji) / mm / WYY(aaaa) For questions • CRO contact "' Pour toute question contàcter la ORC"' PD I_I_I : !_I_! [to be entered by the PharmacistJ h : min [à compléter par le Pharmacien] DO NOT STORE AT >30°C {86°F) NE PAS ENTREPOSER A >30°C (86°F) 1-1-1 : I_J_I [to be entered by the Pharmacist] h min [à compléter par le Phannacien] DIRECTIONS: Administer as a continuous i.v. infusion into a (high flow) centrai vein, by an infusion pump. Infusion wil! start approximately 2 hours before reperfusion. Pump rate for the loading (0-30 min ìnfusion) and maintenance (30 min - 24 hours infusion) dose will be adjusted according to patient body weight as per Appendix 3 of Study Protocol. INSTRUCTIONS: Admfnlstrer à /'a/de d'une pompe 8 fnfusion en fntusion continue i. v, dens une veine centrò/e (haut debit}. L 7nfusio11 doit commencer environ 2 heurcs 1want fa reperfusion. Le nux de la pompe pour la dose de charge (infusion 0-30 min) et d'équl!ibre (infusion 30 min - 24 heures) devra §tre a/usté en fonctfon du poids du patlent comme !nd!qué dans !'Appendice 3 du Protocole d'Essai. CAUTION: New Drug-Lirnited by Federai law to investigational use. Investigationa! drug to be used by a qualified investigator only. A TTENTION: Nouvelle svbstance - Llmltée a la recherche par la /of Fédérale. Prodult de recherche, ne doit etre utifisé que par un investigateur qu,1/ifié. Final Revision No.3, 20-March-2006 Sludy REP0104 Repertaxin in Lung Transplantation Page65 of73 Specimen double tear off Label for Infusion Bag No.2 [time period 24-48 bours] STUDY (ESSAI) REP0104 Sponsor (Promoteuf): Dompé s.p.a.; Via Campo di Pile, L'Aquila - ItalY INFUSION BAG No. 2 (24-48 hours) - POCHE DE PERFUSION N" 2 {24-48 heures) PATIENT No. XXYY PATIENTN" PATIENT INITIALS 1-1-1-1 [to be entered by the Pharmacist] INITIALES DU PA TIENT fa compléter par le Pharmacien] CONTAINS: 600 ml OF REPERTAXIN (12.65 mg/ml) OR PLACEBO INJECTABLE DOSING SOLUTION CONTENU: 500 mL DE SOLU7TON lNJECTABLE DE REPERTAXJN (1.l.65 mg/ml) OU DE PLACEBO PREPARED ON l_!_l/l_l_l/!_I_I_I_I PREPAREE LE dd Ui) / mm / WYY (aaaa) USE BY I_I_I/I_I_I/I_I_I_I_I UTILISERAVANTLE dd (jj) / mm / WYY (aaaa) For questions - CRO contact = Pour toute question contader la ORC= I_I_I : !_I_I [to be entered by the Pharmacist] h : min [à compléter par le PtJarmacien) DO NOT STORE AT >30°C (86°F) N& PAS ENTREPOSER A >30°C {86°F) LJ_I : I_I_I [to be entered by the Pharmacist] h min [à complérer par le Pharmaden] DIRECTIONS: Administer as a continuous i.v. infuslon into a (high flow) centrai vein, by an infusion pump. Pump rate far the maintenance dose (24-48 hours infusion) will be adjusted according to patient body weight as per Appendix 3 of Study Protoco!. INSTRUCTIONS: Administrer à /'a/de d'une pompe J infusion en infusion conanue i.v. dans une vetne centrale (haut déblt), Le /lux oc liJ pompe pçur mainter1ir te doSège (infusion 1448 heures) devra ètre aJusté en f'onction di.I polds du patient comme incliqué dill1$ l'Appendice 3 du Protocole d'Essai. CAUTION: New Drug-Limited by Federai law to investigational use. Investigational drug to be used by a qua!ified tnvestigator only. A rrENTION: Nouvelle substance • Umftée à la recherche par la loi Fédérale. Produit de recherche, ne doit étre utillsé que par un r,,vestigateur qua/ifié, Specimen label for each ampoule - repertaxin or placebo STUOY REP0104 Dompé s.p.a., Jtaly PATIENT No. XXYY 10 ml of repertaxin (33 mg/ml) or placebo DO NOT STORE AT >30°C (86°F) Blindecl batch No. Expiry date mm/yyyy New Drug•Llmlted by Federai law to investigational use. lmrestigational drug to be used by a qualified investlgator onlv. Final Revision No. 3, 20-March-2006 Study REP0 I 04 Repertaxin in Lung Transplantation Page 66 of 73 14.3. APPENDIX 3 • STUDY DRUG INFUSI ON RA TE BY BODY WEIGHT Loading dose = 4.488 mg/kg /hour Dosing solution (repertaxin) = 12.65 mg/mL Maintenance dose= 2.772 mg/kg /hour BODY WEIGHT(kg) LOADING DOSE MAINTENANCE DOSE (each figure is from 0.00 to 0.99) (mL/hour x 30 min) (mL/hour x 47.5 hours) 30 10,6 6,6 31 11,0 6,8 32 11,4 7,0 33 11,7 7,2 34 12,1 7,5 35 12,4 7,7 36 12,8 7,9 37 13,1 8,1 38 13,5 8.3 39 13.8 8,5 40 14,2 8,8 41 14,5 9,0 42 14~9 9,2 43 15,3 9,4 44 15,6 9,6 45 16,0 9,9 46 16,3 10,1 47 16,7 10,3 48 17,0 10,5 49 17,4 10,7 50 17,7 11,0 51 18,1 11,2 52 18,4 11,4 53 18,8 11,6 54 19,2 11,8 55 19.5 12,1 56 19,9 12.3 57 20,2 12,5 58 20,6 12,7 59 20,9 12,9 Final Revisìon No. 3, 20-March-2006 Study REP0I04 Repertaxin in Lung Tra11spla11tation Page 67 of73 BODY WEIGHT(kg) LOADING DOSE MAINTENANCE DOSE (each ffaure is from 0.00 to 0.99) (mL/hour x 30 min) (mL/hour x 47.5 hours) 60 21,3 13.1 61 21.6 13.4 62 22,0 13.6 63 22,4 13.8 64 22.7 14,0 65 23.1 14.2 66 23.4 14.5 67 23.8 14.7 68 24.1 14.9 69 24.5 15.1 70 24.8 15.3 71 25.2 15.6 72 25.5 15.8 73 25.9 16.0 74 26.3 16.2 75 26,6 16.4 76 27.0 16.7 77 27.3 16.9 78 27.7 17.1 79 28.0 17,3 80 28.4 17.5 81 28.7 17.7 82 29.1 18.0 83 29.4 18.2 84 29.8 18.4 85 30.2 18.6 86 30.5 18.8 87 30.9 19.1 88 31.2 19.3 89 31,6 19.5 90 31.9 19.7 91 32.3 19.9 92 32.6 20.2 Final Revisìon No.3, 20-March-2006 Study REP0104 Repertaxin in Lw1g Transplantation. Page 68 of73 BODY WEIGHT(kg) LOADING DOSE MAINTENANCE DOSE (each tiiwre is from 0.00 to 0.99) (mL/hour x 30 min) (mL/hour x 47.S hours) 93 33.0 20.4 94 33.3 20.6 95 33.7 20.8 Final Revision No. 3, 20-March-2006 Study REP0104 Reperwxin in Lung Tra11splcmtation Page 69 of73 14.4. APPENDIX 4 - STUDY FLOW CHART ICU Test/Examination Screening Tx ICU Time after ICU admission Hospital 1 month 6 months admission 12h 24 h 36h 48 h 54 h 60h 72h disc.harge discharj:!e 12 months Informed consent X Inclusion/exclusion criteria 1 X Laboratorv tests X X Renal/hepatic function2 X x2 xi x2 Studv d~ug administration 3 X---------· .... -:------- ·---·---------·-----·-·· • •• ·-X 2 Pharmacokinetic samoling 2 Xl. xz xz PaOi/FiO 2 ratio X X X X Chest X-rav - X X X X Vital signs (BP & HR) 4 X X X X X X X X X Cardiopulmonary measurements 4 X X X X X X X X BAL5 xs Vital status X X X X X FEV 1 &FVC X X X BOS score X X Cumulative acute reiection X X Adverse Events 6 6 X----·················-·····--·······-····-·········---------·---------------------··········-··········--·- --X X ••••• ·-·· •••• ····----·--· --------·-•• X6 Prior and concomìtant X---------------------···--------• ----•••• ••••• ····-···-······ --• -----·· ----------· ••••• ---------· -----· ---···· -·--· ---------·X 7 X----· ------·-·· ---·-··-· • ------·----· X 7 medications 7 I = ConfirmalOry screening to take piace upon admission to the center prior to randomìzation 2 = (shadowed boxes) Time is frorn the start of study <lrug infosion. The '24 hours' sample is ta.ken @ 24±6 hours; the '54 hours' samplc is taken in the interval 2-6 hours after tbe infusion end 3 "'Study drug infusion will start approximately 2 hours before the anticipated time of reperfusion 4 = Vita! signs and cardiopulmonary measurements (when a PA catheter is present) will be measured up to ICU discharge or up to 5 days after ICU admission, whichever occurs earlicr 5 "'BAL will be performed during mechanìcal vencilation, in thc interval between 12 and 24 hours afler rcperfusion 6::: All AEs will be reported up to 10 days or hospital disch.arge. Thereafter, only untoward events that the lnvestigator assesses as at least pos,ibly rclated to lhe study drug will be rccorded 7 = After IO days or hospital discharge, only drugs used for immunosuppression, infection prophylaxis, and AEs possibly related to the srudy drug (scc 6 above) will be recorded Final Revision No. 3, 20-March-2006 Study REP0104 Repertaxìn in Lung Transplantatian Page 70of73 14.5. APPENDIX 5 - WORLD MEDICAL ASSOCIA TION DECLARA TION OF HELSINKI Ethical Principles for Medical Research Involving Human Subjects Adopted by the 18th WMA Genera! Assembly Helsinki, Finland, June 1964 and amended by the 29th WMA Genera! Assembly, Tokyo, Japan, October 1975 35th WMA Generai Assembly, Venice, Italy, October 1983, 41st WMA Generai Assembly, Hong Kong, September 1989, 48th WMA Generai Assembly, Somerset West, Republic of South Africa, October 1996 and the 52nd WMA Generai Assembly, Edinburgh, Scotland, October 2000 Note of clarification on Paragraph 29 added by WMA Generai Assembly, Washington 2002 A. INTRODUCTION The World Medicai Association has developed the Declaration of Helsinki as a statement of ethical principles to provide guidance to physicians and other participants in medicai research involving human subjects. Medicai research involving human subjecl':l includes research on identifiab\e human material or idenlifiable data. It is the duty of the physician to promote and safeguard the health of the people. The physician's knowledge and conscience are dedicated to the fulfilment of this duty. The Declaration of Geneva of the World Medicai Association binds the physìcian with the words, "The health of my patient will be my fìrst consideration," and the Intemational Code of Medìcal Ethics declares that, "A physician shall act only in the patient's interest when providing medica! care which might have the effect of weakening the physical and menta! conditi on of the patient." Medicai progress is based on research which uitimately must rest in part on experimentation involving human subjects. In medicai research on human subjects, considerations related to the well-being of the human subject should take precedence over the interests of science and society. The primary purpose of medicai research involving human subjects is to improve prophylactic, diagnostic and therapeutic procedures and the understanding of the aetiology and pathogenesis of disease. Even the best proven prophylactic, diagnostic, and therapeutic methods must continuously be challenged through research for their effectiveness, efficiency, accessibility and quality. In current medicai practice and in medica\ research, most prophytactic, diagnostic and therapeutic procedures involve risks and burdens. Medicai research is subject to ethical standards that promote respect for ali human beings and protect their health and rights. Some research populations are vulnerable and need special protection. The particular needs of the economically and medically disadvantaged must be recognised. Special attention is also required for those who cannot give or refuse consent for themselves, for those who may be subject to giving consent under duress, for those who will not benefit personally from the research and for those for whom the research is combined with care. Research Investigators should be aware of the ethical, lega! and regulatory requirements for research on human subjects in their own countries as well as applicable international requirements. No national ethical, legai or regulatory reguirement should be allowe<l to reduce or eliminate any of the protections for human subjects set forth in this Declaralion. B. BASIC PRINCIPLES FOR ALL MEDICAL RESEARCH It is the duty of the physician in medica! research to protect the life, health, privacy, and dignity of the human subject. Medica\ research involving human subjects must confonn to generally accepted scientific principles, be based on a thorough knowledge of the scientific literature, other relevant sources of information, and on adequate laboratory and, where appropriate, animai experimentation. Appropriate caution must be exercised in the conduct of research which may affect the environmem, and the welfare of animals used for research must be respected. Final Revision No.3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 71 of73 The design and performance of each experimental procedure involving human subjects should be clearly formulated in an experimental protocol. This protocol should be submitted for consideration, cornment, guidance, and where appropriate, approvai to a specially appointed ethical review commìttee, which must be independent of the investigator, the sponsor or any other kind of undue influence. Thìs ìndependent committee should be in confomùty with the laws and regulations of the country in which the research experìment ìs performed. The committee has the right to monitor ongoìng trials. The researcher has the obligation to provide morùtoring information to the committee, especially any serious adverse evencs. The researcher should also submit to the committee, for review, information regardìng funding, sponsors. institutional affiliatioos, other potential conflicts of interest and incentives for subjects. The research protocol should always contaìn a statement of the ethtcal considerations involved and should indicate that there is compliance with the principles enunciated in this Declaration. Medicai research involving human subjects should be conducted only by scientifically qualified persons and under the supervision of a clinically competent medica! person. The responsibility tor the human subject must always rest with a rnedically qualified person and never rest on the subject of the research, even though the subject has given consent. Every medicai research project involvìng human subjects should be preceded by careful assessment of predictable risks and burdens in comparison with foreseeable benefits to the subject or to others. This does nor preclude the participation of healthy volunteers in medicai research. The design of ali studies should be publicly available. Physicians should abstain from engaging in research projects involving human subjects unless they are confident that the risks involved have been adequately assessed and can be satisfactorily managed. Physicians should cease any investigation if the risks are found to outweigh the potential benefits or if Ll-iere is conclusive pmof of positive and beneficiai results. Medicai research involving hurnan subjects should only be conducted if the importance of the objective outweighs the inherent risks and burdens to the subject. This is especial1y important when the human subjects are healthy volunteers. Medica! research is only justified if there is a reasonable likelihood that the populations in which thc rcsearch is carried out stand to benefit from the results of the research. The subjects must be volunteers and informed particìpants in che research project. The right of research subjects to safeguard theìr integrity must always be respected. Every precaution should be taken to respect the privacy of the subject, the confidentiality of the patìent's information and to minimise the impact of the study on the subject's physical and mental integrìty and on the personality of the subject. 1n any research on human beings, each potential subject must be adequately informed of the aims, rnethods, sources of funding, any possible cont1icts of interest, institutional affiliations of the researcher, the anticipated benefits and potential risks of the study and the discomfort it may entail. The subject should be informed of the right to abstain from participation in the study or to withdraw consent to participate at any time without reprisal. After ensuring thai the subject has understood the infomiation, the physician should then obtain the subject's freely-given informed consent, preferably in writing. If t11e consent cannot be obtained in writing, the non-written consent must be formally documented and witnessed. When obtaining informed consent for the research project the physician should be particularly cautious if the subject is in a dependent relationship with the physicìan or may consent under duress. In that case the informed consent should be obtained by a well-informed physician who is not engaged in lhe investigation and who is cornpletely independent of this relationship. For a research subject who is iegally incompetent, physically or mentally incapable of giving consent or is a legally incompetent minor, the investigator must obtain informed consent from the legally authorised representative in accordance with applicable law. These groups should not be included in research unless the research is necessary to promote the health of the population represented and thìs research cannot instead be performed on legally competent persons. When a subject deemed iegally incompetent, such as a minor child, is able co give assent to decisions about participalion in research, the investigator must obtain that assent in addition to the consenc of the legally authorised representati ve. Research on individuals from whorn ìt is noc possible to obtain consent, including proxy or advance consent, should be done only if the physical/mental condition that prevents obtaining informed consent ìs a necessary characteristic of the research population. The specific reasons for involving research subjects with a condìtion that renders them unable to give informed consent should be stated in the experimental protocol for consideration and approvai of the review committee. The protocol should state that consent to remain in the research should be obtained as soon as possible from the ìndividual or a legally authorised surrogate. Final Revision No. 3, 20-March-2006 Study REP0 104 Repertaxin in Lung Transplantatio11 Page 72 of73 Both authors and publishers have ethical obligations. In publication of the results of research, the investigators are obliged to preserve the accuracy of the results. Negative as well as positive results should be published or otherwise publicly available. Sources of funding, institutional affiliations and any possible conflicts of interest should be declared in the publìcation. Reports of experimentation not in accordance with the principles laìd down in this Declaration should not be accepted for publication. C. ADDITIONAL PRINCIPLES FOR MEDICAL RESEARCH COMBINED WITH MEDICAL CARE The physician may combine medicai research with medical care, only to the extent that the research is justified by its potential prophylactic, diagnostic or therapeutic value. When medica! research is combined wilh medical care, additional standards apply to protect the patients who are research subjects. The benefits, risks, burdens and effectiveness of a new method should be tested against those of the best current prophylactic, diagnostic, and therapeutic methods. This does not exclude the use of placebo, or no treatment, in studies where no proven prophy!actic, diagnostic or therapeutic method exists. At the conclusion of the study, every patient entered into the study should be assured of access to the best proven prophylactic, diagnostic and therapeutic methods identified by the study. The physician should fully infonn the patient which aspects of the care are related to the research. The refusal of a patient to participate in a study must never interfere with the patient-physic.ian relationship. In the treatment of a patient, where proven prophylactic, diagnostic and therapeutic methods do not exist or have been ineffective, the physician, with informed consent from the patient, must be free to use unproven or new prophylactic, diagnostic and therapeutic measures, if in the physician's judgement it offers hope of saving life, re-establishing health or alleviating suffering. Where possible, these measures should be made the object of research, designed to evaluate their safety and effìcacy. In all cases, new information should be recorded and, where appropriate, published. The other relevant guidelines of this Declaration should be followed. Note of ç)arification on Paragi:aph 29 of the WMA Declaration of Helsinki The WMA hereby reaffirms ìts position that extreme care must be taken in making use of placebo-controlled trials and that in generai this methodology should only be used in the absence of existing proven therapy. However. a placebo-controlled tria! may be ethically acceptable, even if proven therapy is available, under the following circumstances: or • Where for compelling and scientifically sound methodological reasons its use is necessary to detenni ne the efficacy or safety of a prophylactic, dìagnostic or therapeutic method • Where a prophylactic, diagnostic or therapeutic method is being investigated fora minor condition and the patients who receive placebo will not be subject to any additional risk or serious irreversìble harm. All other provisions of be Declarntion of Helsinki must be adhered to, especially the need for appropriate ethìcal and scientific review. (6.10.02) Final Revisìon No. 3, 20-March-2006 Study REP0104 Repertaxin in Lung Transplantation Page 73 of73 14.6. APPENDIX 6-ACCEPTABLE TIME WJNDOWS FOR ASSESSMENTS/ PROCEDURES Infusion Bag 1 begins approximately 2 hrs prior to re erfusìon > l hour prior to reperfusion Change 1oading dose to maintenance dose @ 30 30_35 mìnutes after statt of infusion mins Change Infusion Bag 1 to Infusion Bag 2 @ 24 hrs after start of infusion End of Infusìon @ 48 hrs PaO2/FiO2 ratio measurements ± 1 hour ± 1 hour @ ICU Admission ± 2 hour @ 24 hrs ± 2 hour @ 48 hrs ± 4 hours @ 72 hrs ± 4 hours 1---------------------t, ====~= X-ray to calculate PGD Score @ ICU Admission @ 24 hrs @ 48 hrs @ 72 hrs Vitals & Cardiopulmonary measurements @ ICU admission and every 12 hours after ICU admission for 5 days or ICU discharge (whichever occurs earJier) Renal & Hepatic Function after infusion start @ 12 hrs @ 24 hrs @ 36 hrs PKsamples After infusion start Just rior to end of infusion Post infusion end Mechanical Ventilation/Discharge @ 8 am ever rnornin Hospital discharge blood sample Month 1 visit post transplant Month 6 & Month 12 visits Final Revision No. 3, 20-March-2006 ±4 hour ± 4 hours ± 8 hours ± 8 hours ± 4 hours ±4 homs Up to 24 hours prior to dìscharge 30-35 days ± 2 weeks SAP_001.pdf: PPD PPD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian Statistica! Analysis Pian Approvai Sheet REP0104 A phase 2, multi-center, randomized, double-blind, placebo-controlled, parallel group study of repertaxin In the preventlon of prlmary graft dysfunctlon after lung transplantation. Month 1 / PD 1.1 {Final) ____ .... The undersigned have reviewed this and find it to be consistent with the study requirements as it applies to their respective function: Version date: 29 Sep 2006 Page 1 of25 ; 1 .. Statistical Analysls Pian REP0104 Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica! Ana/ysis Pian A phase 2, multi-center, randomized, double-blind, placebo-controlled, parallel group study of repertaxin in the preventlon of primary graft dysfunction after lung transplantation. Month 1 Prepared for: Dompé pha.r.ma s.p.a. Version Number 1.0 1.1 Version date: 29 Sep 2006 Prepared by: CONFIDENTIAL Author PD Page 2 of25 PD 1 1.1 1.2 1.3 2 3 3.1 3.2 3.3 4 4.1 4.2 4.2.1 4.2.2 4.3 5 6 6.1 6.2 6.3 7 8 8. i 8.2 9 9.1 9.2 9.3 9.4 9.4.1 9.4.2 9.4.3 9.4.4 9.4.5 9.5 9.5.1 9.5.2 9.6 9.6.1 9.6.2 9.6.3 9.6.4 9.6.5 9.6.6 10 11 12 13 lntroduction Generai Changes from Protocol Changes from previous versions of SAP Study Objectives Study Design Overvìew Sample Size Considerations Randomization Study variables and covarìates Primary variable Secondary variables Efficacy Safety Predetermined Covariates and Prognostic Factors Definitions Analysis Populations lntention-to-treat (ITT) Per protocol (PP) Safety Interim Analyses Data Revìew Data handling and Transfer Data Review Meeting Statistica! Methods Generai Principles Patient Disposìtion Protocol deviations Demographic and Baseline Characteristics Demographic data Recipient lnformation Significant Medicai History Donar lnformation Transplantation Efficacy analyses Primary Variable Secondary Variables Safety analyses Extent of Study Drug Exposure Adverse events Serious Adverse Events Laboratory data Concomitant Medications Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian 5 5 5 5 5 5 5 6 7 7 7 7 7 7 8 8 Vital Signs, Physìcal Findings and Other Observations Relateci lo Safety Validatìon 10 10 10 11 11 11 11 11 11 11 11 12 12 12 12 12 12 12 12 13 14 15 15 15 16 16 17 17 17 17 20 21 List of tables, Figures and listings for Month 1 Report Data Presentation Table Shells Version date: 29 Sep 2006 Page 3 of25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysìs Pian GLOSSARY OF ABBREVIATIONS AE ALT APTT AST ATC BAL BOS BP CMV CO CRF CSR CVP CXCL8 DMC Dompé FEV1 FiO2 FVC HR PaO2 PCWP PGD PK PMN PP PT PVR SAP SAE soc SVR Adverse Event Alanine Aminotransferase Actìvated Partial Thromboplastin Time Aspartate Aminotransferase Anatomie Therapeutic Chemical Classification Bronchoalveolar Lavage Bronchiolitis Obliterans Syndrome Blood Pressure Cytomegalovirus Cardiac Output Case Report Form Clinica! Study Report Right Atrial Pressure CXC ligand 8 [formerly interleukin (IL)-8} Data Monitoring Committee Dompé pha.r.ma s.p.a Forced Expiratory Volume in One Second Fraction of lnspired Oxygen Forced Vital Capacity Heart Rate lntravenous Intensive Care Unit lntention-To-Treat Millimeters mercury PPD Pulmonary Artery Partial Pressure of Arteria! Oxygen Partial Capillary Wedge Pressure Primary Graft Dysfunction Pharmacokinetic Polymorphonuclear Leukocyte Per Protocol Prothrombin Time Pulmonary Vascular Resistance Statistica! Analysis Pian Serious Adverse Event System Organ Class Systemic Vascular Resistance Version date: 29 Sep 2006 Page 4 of25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian 1 INTRODUCTION 1.1 Generai This statistica! analysis pian (SAP) describes the statistica! methods to be used during the analysis and reporting of data collected under Dompé pha.r.ma s.p.a. (Dompé} Protocol REP0104. The study is splil into two phases: screening to one month post-transplant and greater than one month to 12 months post-transplant. This SAP applies to all outputs, in-text or post-text, produced for inctusion in the clinica! study report (CSR) far the Month 1 analyses. A separate SAP will be produced for the Month 12 analyses. This SAP should be read in conjunction with the study protocol and case report form (CRF). This version of the pian has been developed using the revised protocol dated 30 August 2005; the CRF dateci 01 March 2005 and the amended pages relating to the 30 August protocol amendment. Any further changes to the protocol or CRF may necessitate updates to the SAP. At the time of writing this version of the SAP, approximately 60 patients have been randomized into the study. The analysis pian will be finalized and approved by the sponsor and 'PD --,--,-------- p rio r to database lock. ------------------- 1.2 Changes from Protocol None. 1.3 Changes from previous versions of SAP For the primary analysis, the covariance matrix for the final model was wrongly defined as the matrix with the highest value for the Al<aike's lnformation Criterion. The selection criterion has been changed to the matrix with the lowest value for the Akaike's lnformation Crìterion. 2 STUDY OBJECTIVES To evaluate whether CXCL8 inhibìtion with repertaxin leads to reduced severity of primary graft dysfunction (PGD}, as the result of improved functional and clinica! outcomes in lung transplantation patients. The safety of repertaxin in the specific clinica! setting wìll also be evaluated. The ability of repertaxin to reduce target cells (PMN) infiltration into the grafi will be evaluated lo confìrm its mechanism of action. Plasma levels of repertaxin and its major metabolite will be measured al steady state conditions lo previde population pharmacokinetic (PK) profile in this dinical setting. 3 STUDY DESIGN 3.1 Overview This is a phase 2, multi-center, randomized, double blind, placebo-controlled, parallel group study of repertaxin in the prevention of PGD after lung transplantation. The study will involve 100 Version date: 29 Sep 2006 Page5 of 25 PPD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian patients undergoing single or bilatera! lung transplant who will be randomized to receive either repertaxin or placebo via continuous intravenous (i.v.) infusion administered aver 48 hours. Patients randomized into the study who do not go on to receive study drug or lung transplant will be replaced therefore the tota! number of patients randomized may exceed 100. Patients will be followed for 12 months post-transplant but the majority of study assessrnents are performed during the intensive care unit (ICU) and hospital stay immediately after surgery. Patients will be selected far entry into the study from those listed on the center's lung transplant waiting list. lnclusion/exclusion criteria (as listed in protocol sections 5.1 and 5.2), disease history, extrapulmonary site of infection, significant medicai history, vita! signs, renai and hepatic function, laboratory safety tests and donor information will al! be assessed prior to randomìzatìon lo ensure patient eligibility. The lung transplant surgery will be performed according to the center's standard practice. The study drug infusion will start approximately 2 hours before the anticipated time of reperfusion of the first allograft. A loading dose will be administered tor the fìrst 30 minutes, followed by a maintenance dose tor the remaining 47.5 hours of administration. After completion of surgery, patlents will be admìtted to an ICU and blood gases (PaO2, FiO2 and PaO2/FiO2 ratio) will be measured on admission (time O) and at 24, 48 and 72 hours post ICU admission or unti! extubation whichever occurs earlier. Chest x-rays, renai and hepatic functlon and PK sampling will be measured at defined time poinls within the 72 hours following ICU admission. Vital slgns (BP and HR) and cardiopulrnonary measurements will be assessed at defined time points within the 5 days post ICU admission or unti! ICU discharge (or PA catheter removed) whichever occurs earlier. Further patient assessments are made at ICU discharge and hospital discharge. Patients will return to the center at least 30 days after the transplant for the Month 1 assessment and at 24-28 weeks and 50-54 weeks post transplant tor the Month 6 and Month 12 assessments respectìvely. Once data up to and including the Month 1 assessment has been collected, entered and cleaned, the Month 1 database will be locked, the study will be unblinded and the primary analysis of safety and effìcacy data will be performed. Data collected during the open phase of the study i.e. at Months 6 and 12 will be presented in an addendum to the main CSR. An independent Data Monitoring Committee (DMC) has been established to ensure patient safety, to consider patient risks agaìnst the potential for meeting tria! objectives, and to previde recommendations to Dompé with respect to the conduct and analysis of the trial. p PD will be responsible for providing the DMC statisticlan with listings of data relating to disposmon {randomization, completion and withdrawal), demography, adverse events (AEs) and occurrence of clinically diagnosed PGD. Full details of the responsibilities of the DMC are provided in the DMC Charter. 3.2 Sample Size Considerations Under the foliowing assumptions: 1. A single prirnary endpoint - PaO,2'FiO2 ratio 2. One prirnary comparison: repertaxin versus placebo at 24 hours post ICU admissìon (Ho: µR = µp; H,: PR > µp ). 3. A one-sided two group t-test of statistica! significance 4. a-leve! or probability of Type I error associated with the test of 0.05 5. A placebo mean of 315 mmHg 6. A common standard deviation of 137 mrnHg 7. A sample size of 50 in each group Version date: 29 Sep 2006 Page 6 of 25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 statistica! Ana/ysis Pian the study has 81% power to detect a difference in means of 70 mmHg (µR = 385 mmHg) and 56% power to detect a difference in means of 50 mmHg (µR = 365 mmHg). The data for assumptions 5 and 6 1s taken from the results of 700 lung transplant operations at the Washington University. However, there are currently no data available to provide an estimate of the treatment effect size for this study. 3.3 Randomization Prior to surgery, patients who meet all the entry criteria and who previde written informed consent will be randomized to receive either repertaxin or placebo according to a 1:1 randomization. A master randomization list was prepared far 352 patìents (44 at each of 8 centers) by lnsight Statistica! Consulting on behalf of PP07. A dummy randomization was reviewed by statisticians at both PD and Dompé prior toproduction of the final version. Since each patient should only ever require a single drug pack, the randomization number is identica! to the patient number. The randomization number is assigned sequentially at each site as patients are found to be elìgible tor entry to the study and are enrolled. Patients who are enrolled to replace patients who withdraw from the study prior to study drug infusìon or !ung transplantation wìlt be issued a new randomization/patient number. lf a patìent is allocateci a randomization number and either the transplant does not happen or the study drug is not administered, the relevant drug box should be annulled to avoid its re-use with another patient. 4 STUDY VARIABLES AND COVARIATES 4.1 Primary variable The primary variable within the study is PaO2'FiO2 ratio measured at ICU admission (time O) and 24 hours post ICU admission. 4.2 Secondary variables 4.2.1 Efficacy • • • • • • • • • • 4.2.2 • • • • • • Time profile of PaO2/FiO2 ratio measured ICU admission and at 24, 48 and 72 hours post ICU admìssion or until extubated whichever occurs earlier. PGD score Time to freedom from mechanical ventilation Duration of ICU stay ICU mortality Mortality in the first 30 days post-transplant FEV 1 and FVC at Months 1, 6 and 12 post-transplant 8OS score at Months 6 and 12 post-transplant Cumulative acute rejection eplsodes at months 6 and 12 post-transplant Patient survival rate evaluated at Months 6 and 12 post-transplant Safety Study drug dosìng lncidence of adverse events lncidence of clinically significant results and shifts (relative to normai ranges) in hematology, clinica! chemistry and coagulation test results lncidence of concomitant medication use Vital signs Cardiopulmonary measurements Version date: 29 Sep 2006 Page 7 of 25 Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian 4.3 Predetermined Covariates and Prognostic Factors • Type of transplant (single or double) Data recorded only at Months 6 and 12 wHI not be covered within this document but will be discussed in the Month 12 SAP. Data for PMN count in bronchoalveolar lavage (BAL) and plasma levels of repertaxin and its major metabolita DF2243Y will not be analyzed fPD These data will be provided in separate stand alone reports by the responsible laboratories. These reports will be included as appendices to the CSR. 5 DEFINITIONS Time O This is defìned as date and time of ICU admission. Month 1 This is defined as 30-35 days after date of lung transplant. Month 6 This is defined as 24 - 28 weeks ( 168 - 196 days) after date of lung transplant. Month 12 This is defined as 50 - 54 weeks (350- 378 days} after date of lung transplant. Age The integer age of the patient will be derived relative to the date of the screening visit usìng the following SAS code: age = INTCK('YEAR', dob, dos) - {(MONTH(dos) < MONTH{dob)) OR (MONTH(dos) = MONTH(dob) ANO DAY(dos) < DAY(dob))); where dob = date of birth and dos = date of screening visit. Transplant type This is defined as either single or double where a single transplant is defined as a single left lung or a single right lung and a double transplant is defined as a bilatera! sequential lung or an en• bloc double lung. Duration of cold ischemia This ìs the interval in hours between onset of organ ischemie time (date and time of cross•clamp on donor organ) and lhe removal of the donor organ from cold storage and ìs derìved as: (Date/time of organ removal from cold storage- date/time of cross clamp on donor organ)/3600 Duration of warm ischemia This is the interval in hours between removal of donor organ from cold storage and the start of reperfusion or vascular de-clamping of the allograft and is derived as: (Date/time of start of reperfusion - date/lime of organ removal from cold storage)/3600 Duration of cold and warm ischemia This is the interval in hours between onset of organ ischemie time and the start of repertusion and is derived as: (Date/time of start of reperfusion - date/time of cross clamp on donar organ)/3600 Time to freedom from mechanical ventilation This will be measured in hours and wìll be derived as: Version date: 29 Sep 2006 Page8 of 25 PPD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian (date/time of extubation - date/time of ICU admission)/3600. The resu!t will be rounded to the nearest integer. Duration of ICU stay This is the interval in hours between ICU admission and ICU discharge and will be derived as: (date/time of ICU discharge- date/time of ICU admission)/3600. The result will be rounded to the nearest integer. PGD Score This is graded as an integer between O and 3 according to the following definitions: Grade O no radiographic infiltrates Grade 1 PaOzfFiO2 :::300 mmHg + radìographic infìltrates consistent with pulmonary oedema Grade 2 200 mmHg PaO2/FiO2 <300 mmHg + radiographic ìnfiltrates consistent with pulmonary oedema Grade 3 PaO2'FiO2 <200 mmHg + radiographic infiltrates consistent with pulmonary oedema Duration of study drug infusion This is measured in hours and derived as follows: (date/time of termination of infusion - date/time of start of infusion}/3600. The duration should be adjusted for documented interruptions of at least one hour or cumulative interruptions of at least 2 hours. The CRF does not previde a piace to record inteiruptions so the clock times of infusion interruptions and infusion re-start will be provided via filenotes where applicable. Amount of study drug infused This is measured in milliliters and will be derived as the sum of each of the dosing intervals multiplied by the relevant infusion rate. Assuming that dosing is performed per protocol, the formula will be: (date/time of dose switch - date/time of start of infusion)/3600 x infusion rate tor loading dose + (date/time of bag switch- date/time of dose switch)/3600 x infusion rate for maintenance dose + (date/time of end of infusion - date/time of bag switch)/3600 x infusion rate for bag 2. lf the dose swìtch is not performed then it will be: (date/time of bag switch - date/time of start of infusion)/3600 x infusion rate far loading dose + (date/time of end of infusion - date/time of bag switch)/3600 x infusion rate for bag 2. lf lhe bag switch is not performed then it will be: date/time of dose switch - date/time of start of infusion)/3600 x infusion rate for loading dose + (date/time of end of infusion - date/time of dose switch)/3600 x infusion rate for maintenance dose. lf neither the dose switch nor the bag switch are performed then the formula will be: {date/time of end of ìnfusion - date/time of start of infusion)/3600 x infusion rate for loading dose. For repertaxin, the amount of sludy drug in milligrams will be derived as 12.65 x tota! volume transfused in milliliters. Treatment-emergent adverse events Version date: 29 Sep 2006 Page 9 of 25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian Treatment-emergent adverse events are those which first occur or increase in severity after the first dose of study drug. Adverse events with a start date greater than or equal to the start date of study drug infusion will be considered to be treatment emergent. lf the onset date is missing then the event will be assumed to be treatment-emergent. lf the start date is partial and only the month and year are populated then all events starting in the same month as the study drug infusion will be assumed to be treatment-emergent. lf the start date is partial and only the year is populated then all events starting in the same year as the study drug infusion will be assumed to be treatment-emergent. Systemic Vascular Resistance (SVR) This is measured in dyn·s·cm-5 and is derived as: 80 x (MAP - CVP)/CO where MAP = mean arteria! pressure = DBP + (SBP - DBP}/3. Pulmonary Vascular Resistance (PVR) This is measured in dyn·s·cm-5 and is derived as: 80 x {MPAP - PCWP)/CO where MPAP = mean pulmonary arteria! pressure = systolic PA pressure + (2 x diastolic PA pressure)/3. BOS Score This is an integer between O and 3 based on FEV1 categories defined as follows: BOS O FEV1 ~80% of baseline value 8OS 1 FEV1 66-80% of baseline value BOS 2 FEV1 5i-65% of baseline value BOS 3 FEV 1 s50% of baseline value The baseline value will be defined as per center practice as the best FEV 1 value following surgery. 6 ANAL YSIS POPULATIONS The primary analysis of the primary efficacy variable will be presented for the intention-to-treal (ITT) population. A secondary confirmatory analysis will be performed using the per protocol (PP) population. 6, 1 lntention-to-treat (ITT} The ITT population wìll consist of all randomized patients who received any study medication and a lung transplant. Patients will be analyzed according to the treatmen1 randomized regardless of which treatment was actually received. 6.2 Per protocol (PP) The PP population will consist of all patients in the ITT population who do noi meel any of the following criteria: • Started study drug infusìon after reperfusion i.e. after vascular de-clamping of the first allograft • Received study drug infusìon for less than 24 hours • Missing data for PaO2'FiO2 ratio at both time O and 24 hours post ICU admission while on mechanical ventilation • Received Orthoclone OKT3 or Campath induction immunosuppression at time of transplant. This will be recorded in the Month 1 Concomitant Medication pages if used. Further criteria for exc!usion from the PP population may be identified during the data review meeting held prior to database lock. Version date: 29 Sep 2006 Page 10 of 25 PPD 6.3 Safety Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysìs Pian The safety population will consist of ali patients who received any study medication and wlll be based on the treatment actually received. 7 INTERIM ANAL YSES No formai interim analyses of efflcacy are planned for this study. However, safety data will be reviewed on an ongoing basis by a DMC. Full details of the activities and responsibilities of the DMC are provided in the study DMC charter. 8 DATA REVIEW 8.1 Data handling and Transfer The data from this study will be entered, verified and cleaned by the Data Management group at PD in accordance with the study data handling manual and data clarification policies. The data will be provided to the staUstician as SAS datasets. The preferred term and system organ classification (SOC) from the relevant version of the MedDRA dictìonary will be provided tor all adverse events. The ATC group and sub-group from the relevant version of the WHO-DRUG dictionary will be provided for all prior and concomitant medications. 8.2 Data Review Meeting Programming of analysis datasets, tables, fìgures and listings wìll be ongoing during the data management of the study. When the Month 1 database is considered clean by data management, a set of blinded listings (using a dummy randomization) will be produced and distributed to the study team for review. A meeting will be held to review any data values requiring investigation or correction and also protocol deviations. Once ali data issues have been resolved, the Month 1 data will be locked. Treatment code unblinding and the full run of outputs will then take piace. 9 STATISTICAL METHODS 9.1 Generai Principles The statistica! package SAS (Version 8.1) will be used to produce all summary tables, graphs and listings. In generai, categorical data will be presented using co1.,mts and percentages, whìlst continuous variables will be presented using the mean, standard deviation, medlan, minimum, maximum and number of patients. Minima and maxima will usually be reported to the same leve! of accuracy as the raw data; means, medians and standard deviations will be presented to one further decimai piace; standard errors (if presented) will be presented to 2 decimai places more than the raw data. Percentages will be rounded to the nearest integer. In tabulations, denominators for calculation of percentages wil! be taken as the number of non- missing responses in the specified analysis population and treatment group unless otherwise stateci. Ali data recorded in the CRF wìll be listed. 9.2 Patient Disposition The number and percentage of patients screened, randomized and treated in the study will be presented, together with the number and percentage of patients who withdrew from the study prematurely prior to Month 1 and a breakdown of the corresponding reasons for withdrawal. The number and percentage of patìents included in each of the ITT, PP and safety analysis populations will also be included. Screening data recorded far patients who do not proceed to transplant with study drug administration will be listed separately in the report. The exact format of the listings will be discussed with Dompé when the total number of patients randomized but not treated/transplanted is known. Version date: 29 Sep 2006 Page 11 of 25 PPD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica! Analysis Pian A tabulation of the number and percentage of patients randomized at each center will be presented. AII data relating to study completion or withdrawal and inclusion in the analysis populations wìll also be listed. 9.3 Protocol deviations Protocol deviations which exclude patients from the ITT or PP populations (see Sections 6.1 and 6.2 for definitions) will be summarized by treatment group. 9.4 Demographic and Baseline Characteristics Summaries of baseline data will be produced using the safety population and will be presented by treatment group and overall. Data for parameters measured at screenìng that are also measured at subsequent time points post-randornization (e.g. vita! signs, renai and hepatic function) will not be presented in this section. AII baseline data will also be listed. 9.4.1 Demographic data Summary statistics for age (see Section 5 far definition of derivation}, height and weight at screening will be presented, together with frequency counts and percentages for ethnic origin, race and gender. 9.4.2 Reclpient lnformation Frequency counts and percentages will be presented for transplant procedure type, reasons tor transplant, medicai condition and CMV status. A footnote will be added to the iabie to clarify ihat multiple reasons for transplant may be indicated. Data relating to extra respiratory tract of infection will also be included. Frequency counts and percentages will be presented for blood culture results, fever and other signs of systemic sepsis syndrome. 9.4.3 Slgnificant Medicai Hìstory The tota! number of patìents reporting any significant medicai history and the tota! number of patients reporting significant occurrences ongoing at screening will be presented. In addition, the number and percentage of patients reporting an item of signifìcant medicai history within each of the following 15 body systems will also be presented: Generai Appearance; Head, Ears, Eyes, Nose and Throat; Cardiovascular; Pulmonary; Gastrointestinal; Liver; Renai; Genitourinary; Endocrine; lmmune/Allergy; Psychiatric; Centrai Nervous System; Dermatologie; Musculoskeletal; Other. 9.4.4 Donor lnformation Summary statistics will be presented tor the age of the donor, together with frequency counts and percentages of the gender of the donor, whether the donor was a brain dead heart beating donor and a breakdown of the reasons for brain death. lf multiple reasons are provided than a footnote should be added to the table explaining that the number of reasons gìven will exceed the number of patients. The number and percentage of donors whose transplant organ was flushed will be presented by type of solution (Perfadex or other) and category of flush (anterograde, retrograde, additional) and the number and percentage of donors whose transplant organ was stored in either Perfadex or other solution will also be presented. 9.4.5 Transplantation The data for first and second allograft will be presented separately. For each allografl, the following data will be presented: summary statistics tor the duration of cold ischemia, the duration of warm ischemia and the duration of both cold and warm ischemia (see section 5 for definitions} will be presented, together with frequency counts and percentages for the number of patients who had cardiopulmonary bypass performed. Ali olher data relating to transplant will be listed only. 9.5 Efficacy analyses Version date: 29 Sep 2006 Page 12 of 25 PP 9.5.1 PrJmary Variable Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Anafysis Pian The PaO2/FiO2 ratio will be analyzed at two time points: O hours (time of ICU admission) and 24 hours after ICU admission. These analyses will be performed using both the ITT and PP populations with the ITT analysìs considered as the primary result. As this is nota pivotal study and the contrast estimates will be obtained from a repeated measurements model fitted for data at both time points, adjustments for multiplicity are not considered necessary. Primary analx:sis The ratio at the two time points will be analyzed using a repeated measurements model using PROC MIXED within SAS®. The model will include fixed effect terms for center, time point and treatment. Time point will be specified as a repeated measurement. In order to se!ect an appropriate matrix for the observations within each patient, 3 models will be fitted using the compound symmetry, Huynh-Feldt and unstructured structures. The matrix for the final model will be selected using Akaike's lnformation Criterion where lhe lowest value indicates the best fit Type lii sums of squares from the mixed procedure within SAS® will be used to assess the sìgnifìcance of individuai terms within the model using the selected matrix structure. The importance of the treatment-by-center interaction will be investigateci but if the term is not significant at the 10% leve!, it will be excluded from subsequent models. lf there are insufficient data within each center and treatment combination to allow estimation of effects. centers will be pooled based on similar surgical practices. The adjusted least squares means wìll be estimated for each combination of time point and treatment. The treatment effect within each time point will be compared using a one sided test at the 5% level. Missing data will be excluded. The primary analysis will be performed for the ITT population. The tests of the fixed effects will be presented, together with the estimateci least squares means and summary statistìcs of the raw PaO2/FiO2 ratio for each of the two treatments at each time point. The estimateci treatment difference between repertaxin and placebo at each time point will be presented together with the corresponding 95% confidence interval. The confidence interval will be generated using a=0.1 o and the lower limìt will be replaced by -oo. Sensitivity Analyses The primary analysis wìll be repeated using the PP population. The primary analysis will be repeated using a two-sided test at the 5% leve!. The primary analysis wìll be repeated usìng the last observation carried forward (LOCF) method to substitute a missing PaO21'FiO 2 ratio tor patients extubated wìthin 24 hours after ICU submission. Far patients who dìed wìthin 24 hours of ICU admissìon a ratio of O will be used at the 24 hour time point. Data far patients with a ratio mìssing for any other reason will not be substituted. The prìmary anatysis will be performed including the type of transplant (single or double) as a fixed effect covariate whose importance will be assessed at the 5% level. The ìnteraction term far type of transplant and treatment will also be fitted but if this is not signifìcant at the 10% level il will be excluded from the final model. The randomization has not been stratified by the type of transplant and therefore this analysis will only be performed ìf there are sufficient data wìthin each treatment, center and type of transplant combinatlon. In summary, the following presentations of PaOJFiO2 ratio at time O and 24 hours will be produced: Repeated Measurements Analysis using a one-sided test far the ITT population with missing data excluded. Repeated Measurements Analysis using a one-sided test for the PP population with missing data excluded. Version date: 29 Sep 2006 Page 13 of 25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian Repeated Measurements Analysis using a two-sided test for the ITT population with missing data excluded. Repeated Measurements Analysìs using a one-sided test for the ITT population w\th substitution of missing data. Repeated Measurements Analysis using a one-sided test far the ITT population including type of transplant but with missing data excluded. Repeated Measurements Analysis using a one-sided test for the PP population ìncluding type of transplant but with missing data excluded. Additional exploratory analyses may be performèd to further investigate the robustness of the primary efficacy data. 9.5.2 Secondary Variables 9.5.2.1 PaO2/FiO2 ratio at 48 hours and 72 hours after ICU admission These data wm be analyzed as described for the primary analysis using a one-sided test at the 5% level and wìth missing data excluded. A further analysis including type of transplant again using a one-sided test at the 5% leve! and with missing data excluded will also be performed provided there are sufficient data recorded. Both these analyses will be performed on the ITT and PP analysis populations. 9.5.2.2 PGD score Analysis of PGD score will be performed separately at time O and at 24, 48 and 72 hours after ICU admission. Two analyses, using Cochran-Mantel-Haenszel statistics frorn PROC FREQ within SAS, wi\l be performed at each time point. The first will use type of transplant (single or double) as a stratificatlon factor and will use the following SAS code: Proc freq data=data1 (where=(time=x}) noprint; table tran_typ*PGD*treat/cmh out=ct_typ; output out=cmhtype cmh ; run; As the randomization did not include type of transplant it is possible that that there will be insufficient data within each of the cells to perform the analysis. This will be assessed after the Month 1 database lock. The second analysìs will use center as a stratificatìon factor. Both analyses will be performed for bo1h the ITT and PP populations. Far each analysis, the number and percentage of patients within each table cell will be presented, together with the second of the three p-values provided by PROC FREQ {labeled as Row Mean Scores Differ). and the corresponding Mantel-Haenzsel odds ratio and the associateci 95% confidence interval. 9.5.2.3 Time to freedom from mechanical ventilatìon Time to freedom from mechanical ventilation will be derived as described in Section 5. Kaplan- Meier summary statistics and plots will be used to illustrate time to freedom from mechanical ventilatìon for each treatment group. The analysis will be performed using PROC LIFETEST within SAS: proc lifetest plots=(s, Is, 11s) data=data2 method=km outsurv=estimates; time freetime•censor{1); strata treatment ; ods output ProductlimitEstìmates=file1 ; run; Patients who die while stili on mechanical ventilatlon will be censored at the time of death. Version date: 29 Sep 2006 Page 14 of 25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 statistica! Analysis Pian The risk set and number of events occurring within each 12-hour interval will be presented together with the event probability and standard errar. The estimated difference in event probability and corresponding 95% confidence interval will also be presented together with the log-rank and Wilcoxon tests for differences between treatments. The analysis will also be performed by treatment and type of transplant. Both analyses will be presented for both the ITT and PP populatìons. 9.5.2.4 Duration of ICU stay The duration of ICU stay will be derìved as described in Section 5. This duration is equivalent to time to ICU discharge. The duration of ICU stay will be analyzed as descrìbed far time to freedom from mechanical ventilation. 9.5.2.5 Mortality Frequency counts and percentages for the number of patients who die while in ICU and also the number and percentage of patients who die within the first 30 days post-transplant (derived as latest date of repertusion + 30) will be presented by treatment group. This table will be presented for the ITT populatìon only. 9.5.2.6 Pulmonary Function Tests Summary slatistics for FEV1 and FVC measured at Month 1 will be presented for the ITT population by treatment group and aiso for each treatment group and transplant type combinations. 9.5.2.7 Acute Rejection Episodes The number and percentage of patients reporting at least one acute rejection episode up to and including Month 1 will be reported by treatment group and also by treatment group and transplant type. The total number of episodes will also be reported for each treatment group and each treatment group and transplant type combination. This summary will be presented for the ITT population only. 9.6 Safety analyses 9.6.1 Extent of Study Drug Exposure Summary statistics far duration of study drug infusion and amount of study drug infused (see Section 5 for definition) will be presented by treatment group. 9.6.2 Adverse events A summary of treatment-emergent adverse events (see Section 5 for definition} reported between start of infusion and the Month 1 visit, including the tota( number of events reported, the number and percentage of patients reporting at least one adverse event, the number and percentage of patients withdrawing prematurely due to an adverse event, the nurnber and percentage of patients with at least one serious adverse event and the number and percentage of deaths, will be presented by treatment. In addition, a sumrnary of the event by each category of drug relationship will be presented for adverse events and also for serious adverse events. A breakdown of the number and percentage of patients reporting each adverse event, categorized by body system and preferred term coded according to the MedDRA dictionary, will be presented. Note that counling will be of patients not events and patients are only counted once within each preferred term. A further tabulation of these data, broken down by relatìonshìp to study drug, will be presented. Patients with multiple events within a particular body system or preferred term will be counted under the category of their most drug-related event within that body system or preferred term, Version date: 29 Sep 2006 Page 15 of 25 PPD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian Relationship lo study drug ìs categorized as none, unlìkely, possible, probable and highly probable as recorded on the CRF. Each category of relatìonship will be presented on a separate page. A summary of events reported, broken down by severlty (mild, moderate, severe, life threatening), will also be provided. Patients with multiple events within a particular body system or preferred term will be counted under the ca1egory of their most severe event within that body system or preferred term. Each category of severity will be presented on a separate page. A summary of adverse events leading to premature withdrawal wìll be provided, grouped by body system and preferred term. AII adverse events recorded on the CRF will be listed. 9.6.3 Serious Adverse Events A breakdown of the number and percentage of patients reporting each serious adverse event (SAE) between the start of infusion and the Month 1 vislt, categorized by body system and preferred term coded according to the MedDRA dictionary, will be presented. A further tabulation of these data, broken down by relationship to study drug, will be presented. Patients with multiple SA Es within a particular body system or pref erred term will be counted under the category of their most drug-related SAE withln that body system or preferred term. Relationship to study drug is categorized as none, unlikely, possible, probable and highly probable as recorded on the CRF. Each category of reiationship will be presented on a separate page. A summary of SAEs reported, broken down by severity (mild, moderate, severe, life threatening), will also be provided. Patients with multiple SAEs withìn a partìcuiar body system or preferred term will be counted under the category of their most severe SAE withìn that body system or preferred term. Each category of severity will be presented on a separate page. A summary of SAEs leading to premature withdrawal will be provided, grouped by body system and preferred term. 9.6.4 Laboratory data Samples for safety laboratory tests for hematology, clinica! chemistry and coagulation are taken at Screening and hospital discharge. The following parameters will be included in the summary data presentaUons: Hematology: hematocrit, hemoglobin, red blood cells, while blood cells, neutrophils, lymphocytes, monocytes, eosinophils, basophils Clinica! chemistry: sodiurn, potassium, chlorlde, bicarbonate, blood urea nitrogen (BUN), total bilirubin, alanine aminotransferase {ALT), aspartate aminotransferase (AST), serum creatinine Coagulation: activated partial thromboplastin time (APTT), prothrombin time (PT), INR Note: patients will either have a value for PT or INR at each time point. Results for each parameter at screening and hospital discharge will be assessed as being below the lower limìt of the normai range, within the normai range or above the upper limit of the normai range. The number and percentage of patients with out-of-range results and the number and percentage of patients with clinically significant out-of-range results will be presented for each parameter al each ![me point, together with the number of patients with results for that parameter and time point. Shifts in laboratory parameters during the hospital stay will also be presented for each parameter. Creatinina clearance is calculated using Cockcroft-Gault formula at Screening and at 12, 24 and 36 hours post ICU admission for the assessment of renai and hepatic function during the study. Version date: 29 Sep 2006 Page 16 of25 PD Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Ana/ysis Pian These data are used for assessment of patient during the study conduct phase and will be listed only. 9.6.5 Concomitant Medications Ali medications reported in the CRF as being taken prior to the Month 1 visit, categorized by medication group and subgroup according to WHO DRUG, will be summarized. The number and percentage of patients using at least one medication within each medication group and subgroup will be presented. 9.6.6 Vital Signs, Physical Flndìngs and Other Observations Related to Safety Vital signs Vita! signs, induding systolic and diastolic blood pressure and heart rate, are measured at Screening, ICU admission, every 12 hours post ICU admission unti! 120 hours post ICU admission or until ICU discharge or PA catheter removed whichever occurs earliest. Summary statistics for each of these three parameters will be presented by treatment group at each time point. Summary statistics for the change from screening lo each post-screening time point will also be presented. Cardiopulmonary measurements Cardiopulmonary measurements consist of CVP, PCWP, CO and PA Pressure (systolic/diastolic) andare measured at ICU admission, every 12 hours post ICU admission untii 120 hours post ICU admission or until ICU discharge or PA catheter removed whichever occurs earliest. These data will be summarized by treatment group al each time point. Summary statistics for the derìved variables SVR and PVR (see Section 5 for detinitions) will also be provided. 10 VALIDATION Ali summary tables and figures will be validated by independent programming within SAS. Any numerica! discrepancies will be resolved prior to finalization of the tables and figures. AII outputs will have the following checks performed: • Tìtles and footnotes are correct • No errors or wamings in SAS log file • Expected number of observations in dataset used for final output • Matches the specifications documented in this pian 11 LIST OF TABLES, FIGURE$ ANO LISTINGS FOR MONTH 1 REPORT Output numbering may be further sub-divided in arder to allow tor logica! presentation of the data. Tables 1.1 1.2 1.3 1.4 1.5 1.6 1.7 1.8 2.1.1 2.1.2 Patient Disposition (AII Screened Patients) Patient Randomization by Center (AII Randomized Patients) Protocol Deviations (Safety Population) Demographic Data (Safety Population) Recipient lnformation (Safety Population) Significant Medicai History (Safely Popu!ation} Donar lnformation (Safety Population) Transplantation (Safety Population) Repeated Measurements Anatysis of PaOdFiO2 ratio at O and 24 hours after ICU Admission using a one-sided test excluding mìssing data (ITT population) Repeated Measurements Analysis of PaO2/FiO2 ratio at O and 24 hours Version date: 29 Sep 2006 Page 17 of 25 PPD 2.1.3 2.1.4 2.1.5 2.1.6 2.2.1 2.2.2 2.2.3 2.2.4 2.3.1. 2.3.2 2.3.3 2.3.4 2.4.1 2.4.2 2.4.3 2.4.4 2.5.1 2.5.2 2.5.3 2.5.4 2.6 2.7 2.8 3.1 3.2.1.1 3.2.1.2 3.2.1.3 3.2.1.4 3.2.1.5 3.2.2.1 Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian after ICU Admission using a one-sided test excluding missing data (PP populatìon) Repeated Measurements Analysis of PaO2'FiO2 ratio at O and 24 hours after ICU Admission using a two-sided test excluding missing data (ITT population) Repeated Measurements Analysis of PaO2/FiO2 ratio at O and 24 hours after ICU Admission using a one-sided test and subslitution of missing data {ITT population) Repeated Measurements Analysis of PaO2'FìO2 ratio at O and 24 hours after ICU Admission including type of transplant using a one-sided test and excluding missing data {ITT population) Repeated Measurements Analysìs of PaO2'FìO2 ratio at O and 24 hours after ICU Admission includìng type ot transplant using a one-sided test and excluding mìssing data (PP population) Repeated Measurements Analysis of PaO2/FiO2 ratio at 48 and 72 hours after ICU Admission using a one-sided test excluding missing data (ITT population) Repeated Measurements Analysis of PaO:/FiO2 ratio at 48 and 12· hours after ICU Admission using a one-sided test excluding missing data (PP population) Repeated Measurernents Analysis of PaO2/FiO2 ratio at 48 and 72 hours after ICU Admission including Type of Transplant using a one-sided test excluding missing data (ITT population) Repeated Measurements Analysis of PaO2/FiO2 ratio at 48 and 72 hours after ICU Admission including Type of Transplant using a one-sided test excluding missing data (PP population) Analysìs of PGD Score stratified by Type of Transplant (ITT Population). Analysis of PGD Score stratified by Type of Transplant (PP Population} Analysis of PGD Score stratified by Centre (ITT Population) Analysis of PGD Score stratified by Centra (PP Population) Time to Freedom from Mechanìcal Ventilatìon (ITT Populatìon) Time to Freedom from Mechanical Ventilation (PP Population) Time to Freedom from Mechanical Ventilation by Type of Transplant (ITT Population) Time to Freedom from Mechanical Ventìlation by Type of Transplant (PP Population) Duration of ICU Stay (ITT Population) Duration of ICU Stay (PP Population) Duration of ICU Stay by Type of Transplant (ITT Population) Duratìon of ICU Stay by Type of Transplant (PP Population) Mortality up to 30 Days Post-Transplant (ITT Poputation) Pulmonary Function Tests at Month 1 (ITT Population) Acute Rejectìon Episodes by Month 1 (ITT Population) Extent of Study Drug Exposure (Safety Population) Summary of Treatment-Emergent Adverse Events (Safety Population) Treatment-Emergent Adverse Events by Body System and Preferred Term (Safety Populatìon) Treatment-Emergent Adverse Events by Body System, Preferred Terrn and Relationship to Study Drug (Safety Population) Treatment-Emergent Adverse Events by Body System, Preferred Term and Severity of Event (Safety Population) Treatment-Emergent Adverse Events Leading to Premature Withdrawal (Safety Population) Treatment-Emergent Serious Adverse Events by Body System and Preferred Term (Safety Population} Version date: 29 Sep 2006 Page 18 of 25 PD 3.2.2.2 3.2.2.3 3.2.2.4 3.3.1.1 3.3.1.2 3.3.1.3 3.3.2.1 3.3.2.2 3.3.2.3 3.4 3.5.1.1 3.5.2 3.5.3 3.6 Figures 1.1 1.2 i.3 1.4 2.1 2.2 2.3 2.4 Listlngs 2.1.1 2.1.2 2.1.3 2.1.4 2.2 2.5.1 2.5.2 2.6.1 2.6.2 2.6.3 2.6.4 2.6.5 2.6.6 2.7.1 2.7.2 2.8.1 2.8.2 2.8.3 Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian Treatment-Emergent Serious Adverse Events by Body System, Preferred Term and Relationship to Study Drug (Safety Population) Treatment-Emergent Serious Adverse Events by Body System, Preferred T erm and Severity of Event (Safety Population) Treatment-Emergent Serious Adverse Events Leading to Premature Withdrawal (Safety Population) Clinically Significant Results for Hematology (Safety Populalion) Clinically Significant Results tor Clinica! Chemistry (Safety Population) Clinically Significant Results for Coagulation (Safety Population) Shifts in Hematology Parameters (Safety Population) Shìfts in Clìnical Chemistry Parameters (Safety Population) Shifts on Coagulation Parameters (Safety Population) Prior and Concomitant Medications (Safety Population) Vital signs - Systolic Blood Pressure (Safety Population) Vita! signs - Diastolic Blood Pressure (Safety Population) Vital signs - Heart Rate (Safety Population) Cardiopulmonary Measurements (Safety Population) Kaplan-Meier Plot of Time to Freedom from Mechanìcal Ventilation (ITT Population) Kaplan-Meier Plot of Time to Freedom from Mechanical Ventilation (PP Population) Kaplan-Meier Plot of Time to Freedom from Mechanical Ventilation by Type of Transplant (ITT Population) Kaplan-Meier Plot of Time to Freedom from Mechanical Ventilation by Type of Transplant (PP Population) Kaplan-Meier Plot of Duration of ICU Stay (ITT Population) Kaplan-Meìer Plot of Duration of ICU Stay (PP Population) Kaplan-Meier Plot of Duration of ICU Stay by Type of Transplant (ITT Population) Kaplan-Meìer Plot of Duration of ICU Stay by Type of Transplant (PP Population) Patìent Dispositìon (Ali Randomized Pa1ients) Protocol Deviations (Ali Randomized Patients) Month 1 Completion and Reasons for Premature Withdrawal from Study (AII Randomized Patients) Reasons Screened but not Randomìzed (AII Screened Paiients) Demographìc Data Study Treatment Administration - Dates and Times Study Treatment Administration - Termination of lnfusion Individuai Efficacy Response Data - Pa0:2/FiO2 ratio Individuai Efficacy Response Data - PGD Score Individuai Efficacy Response Data - Time to Freedom from Mechanical Ventilation Individuai Efficacy Response Data - Duration of Hospìtal Stay Vital Status Pulmonary Function Tests Adverse Events Reported up to Month 1 Serious Adverse Events Reported up to Month 1 Laboratory Data: Hematology - Patìent Profiles Laboratory Data: Clinical Chemistry - Patient Profiles Laboratory Data: Coagulation - Patient Profìles Version date: 29 Sep 2006 Page 19 of25 PD 4.1 4.2.1 4.2.2 4.3.1 4.3.2 4.4 4.5.1 4.5.2 4.6.1 4.6.2 4.7 4.8 4.9 4.10 4.11.1 4.11.2 4.11.3 4.11.4 4.12 4.13 4.14 4.15 4.15.1 4.15.2 4.15.3 4.15.4 4.15.5 4.16 4.17.1 4.17.2. 4.18 4.19 4.20 4.21.x Dompé pha.r.ma s.p.a. REP0104 Month 1 Statistica/ Analysis Pian Visit Dates lnclusion Criteria Exclusion Criteria Recipient lnformation - Transplant type Recipient lnformatìon - CMV Status and Extrapulrnonary site of infection Significant Medicai History Donor lnformation - Demography and Cause of Death Donor lnformation - Lung Flush Solutions Transplant lnformation - Dates and Tìmes Transplant lnformation - Procedures and Complications Preparation of lnfusion Bags ICU Admission Bronchoalveolar Lavage ICU Discharge and Mechanical Ventilation Weaning Tracking Clinica! Diagnosis of PGD PaO2/FiO2 Ratio for Patients Diagnosed with PGD PGD Score for Patients Diagnosed with PGD Bronchoalveolar Lavage or Biopsy Patients Diagnosed with PGD First Extubation and ICU Discharge ICU Readmission and Hospital Discharge Patient Status at Month 1 Death Report Laboralory Data: Hematology - Variable Profiles Laboralory Data: Clinica! Chemistry- Variabte Profiles Laboratory Data: Coagulation - Variable Proftles Creatinine Clearance Renai and Hepatic Function Prior and Concomitant Medications Reported up to Month 1 Vital Signs and Cardiopulmonary Measuremenls Cardiopulmonary Measurements for Patients Dìagnosed with PGD Pharmacokinetic Sample Colleclion lnvestigator's Declaration lnvestigator Comments Screening data for Patients Randomised but Not Treated/Transplanted 12 DATA PRESENTATION Ali outputs will be produced using the following settings: Orientatlon Marains Font Headers Landscape Top: 1in/2.54cm SAS Monospace (Centered) Bottom: 1 in/2.54cm 8pt Protocol Number Left: 0.5in/1.27cm Table/Listing Riaht: 0.5in/1.27cm Number and title Titles will be as follows: Oompé pha.r.ma s.p.a. Protocol REP0104 Table tabno tabtitle Version date: 29 Sep 2006 Footers (Left) Path and Filename Creation date Page20 of25 PD 13 TABLE SHELLS Tables 2.1.x and 2.2.x Repeated Measurements Analysìs Source Center Ti1_11e Treatment Center-by-treatment Version date: 29 Sep 2006 Tests of Fixed Effects NDF DDF X X X X X X X X Type III F p X.XX o.xxxx x.xx o.xxxx X.XX o.xxxx x.xx O.xxxx Dompé pha.r.ma s.p.a. REP0104 statistica/ Analysis Pian Page 21 of25 PD Time o 24 Version date: 29 Sep 2006 Sum~ary Statistics Repertaxin Placebo Time=O Least Squares Mean xx.xx xx.xx Mean xx.xx xx.xx so xx. xx xx.xx Median xx.xx xx.xx Range (xx.x; XX.X) (xx.x; XX.X) N X X Time=24 Least Squares Mean xx.xx xx.xx Mean xx.xx xx.xx so xx.xx xx.xx r.tedian xx.xx xx.xx Range (xx.x; XX.X) (xx.x; XX.X) N X X Treatment Comparison Difference SE of 959., confidence Compari son between Means dHference interval Repertaxin Placebo x.xxx X.XXX (-x.xx, X.XX) Repertaxin Placebo x.xxx X.XXX (-X.XX, X.XX) Dompé pha.r.ma s.p.a. REP0104 Statistica/ Analysis Pian P-value o.xxxx o.xxxx Page 22 of25 PD Table 2.3.x Analysis of PGD Score Repertaxìn N= N % Grade o xx xx Grade xx xx Grade 2 xx xx Grade 3 xx xx 111 Odds ratio is Aepertaxin:Placebo Placebo Odds N= Ratio N % l 1 J xx xx X.XX xx xx xx xx xx xx 95% CI for Odds Ratio (x.xx, x.xx) Dompé pha.r.ma s.p.a. REP0104 Statistica/ Ana/ysis Pian P-value [2] o.xxx {2} The p-value is obtained froAI a Cochran-Mantel-Haenszel test of the treatment difference. Table 2.4.x Time to Freedom frorn Mechanical Ventilation TIME POINT 24 hrs 48 hrs 72 hrs 96 hrs etc Censored (N) Log-rank Repertaxìn N:a: Number of cumulative Patients Number of Remaining Events xx Version date: 29 Sep 2006 Event Probabìlìty S.E. Placebo N" Number of Cumulative Patients Nu111ber of Event Remainìng Events Probability xx S.E. Oifference in Event Prob. Chi-sq statistic 95% CI for Dìfference P-value Page23 of25 PD Examples of adverse event presentations Table 3.2.1 .1 Summary of Treatment-Emergent Adverse Events (TEAEs) Total number of TEAEs Nu~ber of Patients with: At least one TEAE Withdrawing prematurely due to TEAE At least one Serious TEAE TEAE leading to death TEAEs - Most serious Relationship to Study Drug Unrelated Unlikely Possible Probable Highly Probable Serious TEAEs - Most Serious Relationship to Study Drug Unrelated Unlikely Possible Probable Highly Probable Version date: 29 Sep 2006 Repertaxin xx xx (XX%) xx (XX%) xx (XX 96) xx (XX%) xx (XX%) xx {XX%) xx {XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) Dompé pha.r.ma s.p.a. REP0104 Statistica/ Analysis Pian Treatment Placebo xx xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX%) xx (XX9o) Page 24 of 25 PPD Dompé pha.r.ma s.p.a. REP0104 Statìstical Analysis Pian Table 3.2.1.2 Treatment-Einergent Adverse EvHnts by Body Systea and Preferred Term Treatment MedDRA Body System & Preferred Term Repertaxin Placebo PAIN xx (XX%) xx (XX%) Pain- other xx (XX%) xx (XX%) Bone Pain xx (XX%) xx (XX%) Abdominal xx (XX%) xx (XX%) Headache xx (XX%) xx (XX%) Chest pain xx (XX%) xx (XX~ò) GASTROINTESTINAL xx (XX%) xx (XX~,;) Constipation xx (XX%) xx (XX%) Naw~ea xx (XX%) xx (XX%) Anorexia xx (XX%) xx (XX%) Vomiting xx (XX%) xx (XX%) Diarrhea xx (XX%) xx {XX%) Version date: 29 Sep 2006 Page 25 of25
2
arm 1: Both repertaxin and placebo were aqueous solutions packaged into identical clear glass Type I ampoule single dose units. Each ampoule contained 10 mL of either repertaxin or placebo.The dosing solution for infusion was prepared aseptically at the designated Pharmacy within each center and dispensed as 12.65 mg/mL solution in appropriate size infusion bags. An initial 'loading dose' of Repertaxin of 4.488 mg/kg body weight/hour was administered over 30 minutes followed by a maintenance dose of 2.772 mg/kg body weight/hour lasting 47.5 hours.The loading and maintenance doses were administered using the same dosing solution (Repertaxin 12.65 mg/ml), but with the pump rate altered to provide an infusion rate of approximately 0.35 mUkg/hour and 0.22 ml/kg/hour, respectively. arm 2: Both repertaxin and placebo were aqueous solutions packaged into identical clear glass Type I ampoule single dose units. The dosing solution for infusion was prepared aseptically at the designated Pharmacy within each center and dispensed as 12.65 mg/mL solution in appropriate size infusion bags. ampoule contained 10 mL of either repertaxin or placebo. An initial 'loading dose' of 9 mg/ml sodium chloride (NaCl) solution was administered over 30 minutes, followed by a maintenance dose lasting 47.5 hours.
[ 0, 2 ]
2
[ 0, 10 ]
intervention 1: An initial 'loading dose' of repertaxin was administered over 30 minutes by intravenous infusion followed by a maintenance dose lasting 47.5 hours.The infusion was to start approximately 2 hours prior to the anticipated time of reperfusion and was to continue during the ICU/hospital stay. The study medication was administered as a continuous intravenous infusion into a (high flow) central vein, by an infusion pump adequate to provide reliable infusion rates , as per treatment schedule. Total infusion volume was not to exceed 500 mL/24 hours. intervention 2: An initial 'loading dose' of placebo was administered over 30 minutes by intravenous infusion followed by a maintenance dose lasting 47.5 hours.The infusion was to start approximately 2 hours prior to the anticipated time of reperfusion and was to continue during the ICU/hospital stay. The placebo was administered as a continuous intravenous infusion into a (high flow) central vein, by an infusion pump adequate to provide reliable infusion rates , as per treatment schedule. Total infusion volume was not to exceed 500 mL/24 hours.
intervention 1: Repertaxin intervention 2: Placebo
6
Los Angeles | California | United States | -118.24368 | 34.05223 Denver | Colorado | United States | -104.9847 | 39.73915 St Louis | Missouri | United States | -90.19789 | 38.62727 Durham | North Carolina | United States | -78.89862 | 35.99403 Cleveland | Ohio | United States | -81.69541 | 41.4995 Toronto | Ontario | Canada | -79.39864 | 43.70643
0
NCT00224406
[ 5 ]
221
NON_RANDOMIZED
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
The majority of asthma patients are not well controlled, despite the availability of asthma medication that could effectively treat the disease. In this study uncontrolled patients who are steroid-naive or on low dose inhaled corticosteroids will be treated with Seretide (salmeterol/fluticasone combination, SFC) 50/250 µg twice daily. The asthma control test (ACT) will be used to detect differences in the level of asthma control during treatment. The study aims to show a correlation between improvements of ACT und the level of asthma control which will be reached by the patients. The aim of the study is to show that most of symptomatic asthma patients can reach 'well controlled asthma' with SFC. We get information about ACT in daily practice and physicians are trained to use the asthma control test as a screening tool and for follow up of asthma management. Correlations are expected between the improvements in ACT, Quality of Life and asthma control according to the Gaining Optimal Asthma controL (GOAL) criteria.
null
Asthma
Asthma Asthma Control Test SERETIDE
null
1
arm 1: Participants received the combination product, fluticasone 250 microgram (mcg) plus salmeterol 50 mcg (SFC 50/250 mcg) for 12 weeks. Study treatment was received using DISKUS™ powder inhalers, one dose in morning and evening. Study medication was dispensed at visits 3, 4, and 5 for 30 days each. The participants were provided with salbutamol rescue medication if they developed acute asthmatic symptoms. This medication was provided in metered dose inhalers containing at least 200 puffs of 100 mcg salbutamol. Use of rescue medications was recorded in the participant's asthma diaries. Stable dosages of other concomitant medications were allowed if they had no impact on the outcome criteria.
[ 0 ]
3
[ 0, 0, 1 ]
intervention 1: Participant received salmeterol/fluticasone combination 50/250 mcg using DISKUS™ powder inhalers. intervention 2: This medication was provided in metered dose inhalers containing at least 200 puffs of 100 mcg salbutamol. intervention 3: Participant received salmeterol and fluticasone using DISKUS™ powder inhalers.
intervention 1: Salmeterol/Fluticasone 50/250 mcg intervention 2: Salbutamol 100 mcg intervention 3: DISKUS™ powder inhalers
32
Erlangen | Bavaria | Germany | 11.00783 | 49.59099 Kaufbeuren | Bavaria | Germany | 10.62192 | 47.88238 Landsberg am Lech | Bavaria | Germany | 10.88282 | 48.04819 Munich | Bavaria | Germany | 11.57549 | 48.13743 Munich | Bavaria | Germany | 11.57549 | 48.13743 Rednitzhembach | Bavaria | Germany | 11.07997 | 49.30095 Uttenreuth | Bavaria | Germany | 11.07216 | 49.59675 Würzburg | Bavaria | Germany | 9.95121 | 49.79391 Cottbus | Brandenburg | Germany | 14.32888 | 51.75769 Wedel | City state of Hamburg | Germany | N/A | N/A Bad Arolsen | Hesse | Germany | 9.01445 | 51.37982 Eschwege | Hesse | Germany | 10.05329 | 51.18386 Kassel | Hesse | Germany | 9.5 | 51.31667 Kassel | Hesse | Germany | 9.5 | 51.31667 Marburg | Hesse | Germany | 8.77069 | 50.80904 Braunschweig | Lower Saxony | Germany | 10.52673 | 52.26594 Buchholz | Lower Saxony | Germany | 9.56287 | 53.00884 Hanover | Lower Saxony | Germany | 9.73322 | 52.37052 Hanover | Lower Saxony | Germany | 9.73322 | 52.37052 Chemnitz | Saxony | Germany | 12.92922 | 50.8357 Dresden | Saxony | Germany | 13.73832 | 51.05089 Geesthacht | Schleswig-Holstein | Germany | 10.3779 | 53.43575 Greiz | Thuringia | Germany | 12.19918 | 50.65778 Sonneberg | Thuringia | Germany | 11.17463 | 50.3592 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Hamburg | N/A | Germany | 9.99302 | 53.55073
0
NCT00363480
[ 4 ]
22
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
Multicentric, open-label, randomized, pilot comparative study in parallel groups comparing 1 group of subjects receiving 0.057 milligram/kilogram/day (mg/kg/day) or 0.40 mg/kg/week of Saizen® during 1 year to 1 group receiving 0.035 mg/kg/day (0.24 mg/kg/week) of Saizen® during 1 year after an initial 3-year treatment of recombinant human growth hormone (r-hGH) therapy with 0.057 mg/kg/day in both groups.
null
Small Gestational Age (SGA)
null
2
arm 1: Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.057 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the Height-Standard Deviation Score (H-SDS) at this time (less than \[\<\] -2 SDS or greater than \[\>\] -2 SDS) arm 2: Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.035 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the H-SDS at this time (\< -2 SDS or \> -2 SDS)
[ 0, 0 ]
2
[ 0, 0 ]
intervention 1: Saizen® \[somatropin (recombinant deoxyribonucleic acid \[rDNA\] origin) for injection\], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone. intervention 2: Saizen® \[somatropin (rDNA origin) for injection\], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone.
intervention 1: Saizen® intervention 2: Saizen®
0
null
0
NCT00249821
[ 5 ]
318
NON_RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.
null
Growth Hormone Deficiency Turner Syndrome
null
2
arm 1: None arm 2: None
[ 0, 0 ]
2
[ 0, 0 ]
intervention 1: Subjects with TS will receive SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month intervention 2: Subjects with GHD will receive SAIZEN® as subcutaneous injection at a dose of 0.035 mg/kg of body weight per day (within the recommended dosage 0.025-0.035 mg/kg body weight) for a period of 1 month.
intervention 1: Saizen intervention 2: Saizen
13
Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Sydney | N/A | Australia | 151.20732 | -33.86785 Vienna | N/A | Austria | 16.37208 | 48.20849 Mississauga | N/A | Canada | -79.6583 | 43.5789 Paris | N/A | France | 2.3488 | 48.85341 Munich | N/A | Germany | 11.57549 | 48.13743 Rome | N/A | Italy | 12.51133 | 41.89193 Oslo | N/A | Norway | 10.74609 | 59.91273 Russia | N/A | Russia | N/A | N/A Singapore | N/A | Singapore | 103.85007 | 1.28967 Madrid | N/A | Spain | -3.70256 | 40.4165 Stockholm | N/A | Sweden | 18.06871 | 59.32938 Feltham | N/A | United Kingdom | -0.41388 | 51.4462
0
NCT00256126
[ 2, 3 ]
23
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
The reason for this study will be to find the safest maximum tolerated dose of oral vorinostat in combination with erlotinib \[Tarceva (TM)\] that can be given to patients with lung cancer who have relapsed or failed other therapy for the disease. Once the safest maximum tolerated dose of vorinostat is determined, patients enrolled in the clinical trial will continue vorinostat and erlotinib for up to 8 months. Safety and effectiveness will also be evaluated.
null
Carcinoma, Non-Small-Cell Lung
Relapsed Non-Small-Cell Lung Cancer Refractory Non-Small-Cell Lung Cancer
null
4
arm 1: Vorinostat 200 mg twice a day for 3 days a week + erlotinib 150 mg once a day was evaluated in the Phase I portion of the study and determined to be the MTD and therefore the recommended Phase II dose. Of the 16 patients treated at this dose level, 4 were assigned to the Phase I portion of the study and 12 were assigned to the Phase II portion arm 2: Vorinostat 300 mg once a day for 3 days a week + erlotinib 150 mg once a day was evaluated in the Phase I portion of the amended study and exceeded the MTD. All patients treated at this dose level were assigned to the Phase I portion of the study. arm 3: Vorinostat 300 mg twice a day for 3 days a week + erlotinib 150 mg once a day was evaluated in the Phase I portion of the study and exceeded the MTD. All patients treated at this dose level were assigned to the Phase I portion of the study. arm 4: Vorinostat 400 mg once a day for 21 out of 28 days + erlotinib 150 mg once a day was evaluated in the Phase I portion of the original study and exceeded MTD. This cohort was then amended (Amendment 1) to identify a more tolerable once daily vorinostat dosing regimen. All patients treated at this dose level were assigned to the Phase I portion of the study.
[ 0, 0, 0, 0 ]
5
[ 0, 0, 0, 0, 0 ]
intervention 1: Vorinostat 200 mg twice a day for 3 days a week. intervention 2: Vorinostat 300 mg once a day for 3 days a week. intervention 3: Vorinostat 300 mg twice a day for 3 days a week. intervention 4: Vorinostat 400 mg once a day for 21 out of 28 days. intervention 5: erlotinib 150 mg once a day.
intervention 1: Vorinostat intervention 2: Vorinostat intervention 3: Vorinostat intervention 4: Vorinostat intervention 5: erlotinib
0
null
1
NCT00251589
[ 5 ]
537
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
The purpose of this study is to evaluate the effect of the Dialogues Time to Talk program in subjects treated with Venlafaxine Extended Release (ER). Dialogues Time to Talk Program is a patient management program, which aims to help patients achieve successful outcomes by reinforcing physician treatment efforts, providing feedback to treating physicians, and encouraging better physician-patient communications.
null
Depressive Disorder, Major
Major Depressive Disorder Depression Anxiety
null
0
null
null
2
[ 0, 5 ]
intervention 1: None intervention 2: None
intervention 1: Venlafaxine ER intervention 2: Dialogues Time to Talk Program
44
Birmingham | Alabama | United States | -86.80249 | 33.52066 Birmingham | Alabama | United States | -86.80249 | 33.52066 Mesa | Arizona | United States | -111.82264 | 33.42227 Little Rock | Arkansas | United States | -92.28959 | 34.74648 San Jose | California | United States | -121.89496 | 37.33939 Avon | Connecticut | United States | -72.83065 | 41.80982 Cromwell | Connecticut | United States | -72.64537 | 41.5951 Wilmington | Delaware | United States | -75.54659 | 39.74595 Ocala | Florida | United States | -82.14009 | 29.1872 Pinellas Park | Florida | United States | -82.69954 | 27.8428 Stuart | Florida | United States | -80.25283 | 27.19755 Atlanta | Georgia | United States | -84.38798 | 33.749 Cartersville | Georgia | United States | -84.80231 | 34.16533 Rome | Georgia | United States | -85.16467 | 34.25704 Idaho Falls | Idaho | United States | -112.03414 | 43.46658 Newburgh | Indiana | United States | -87.40529 | 37.94449 Waterloo | Iowa | United States | -92.34296 | 42.49276 Metairie | Louisiana | United States | -90.15285 | 29.98409 Benzonia | Michigan | United States | -86.09926 | 44.62139 Interlochen | Michigan | United States | -85.7673 | 44.64472 Kalamazoo | Michigan | United States | -85.58723 | 42.29171 Chesterfield | Missouri | United States | -90.57707 | 38.66311 Omaha | Nebraska | United States | -95.94043 | 41.25626 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Charlotte | North Carolina | United States | -80.84313 | 35.22709 Charlotte | North Carolina | United States | -80.84313 | 35.22709 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Columbus | Ohio | United States | -82.99879 | 39.96118 Kettering | Ohio | United States | -84.16883 | 39.6895 Mason | Ohio | United States | -84.30994 | 39.36006 Mogadore | Ohio | United States | -81.39789 | 41.04645 Allentown | Pennsylvania | United States | -75.49018 | 40.60843 Newtown | Pennsylvania | United States | -74.93683 | 40.22928 Reading | Pennsylvania | United States | -75.92687 | 40.33565 Warwick | Rhode Island | United States | -71.41617 | 41.7001 Anderson | South Carolina | United States | -82.65013 | 34.50344 Greer | South Carolina | United States | -82.22706 | 34.93873 Bristol | Tennessee | United States | -82.18874 | 36.59511 Dallas | Texas | United States | -96.80667 | 32.78306 Fort Worth | Texas | United States | -97.32085 | 32.72541 Plano | Texas | United States | -96.69889 | 33.01984 San Antonio | Texas | United States | -98.49363 | 29.42412 Salt Lake City | Utah | United States | -111.89105 | 40.76078 Charleston | West Virginia | United States | -81.63262 | 38.34982
1
NCT00401726
[ 3, 4 ]
439
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This is a Phase II/III, randomized, double-blind, parallel group, placebo controlled, multicenter study to evaluate the safety and efficacy of rituximab in adults with PPMS. The study will enroll approximately 435 subjects at up to 60 sites in the United States and Canada.
null
Multiple Sclerosis
PPMS Primary progressive MS Primary progressive multiple sclerosis Rituxan MS
null
2
arm 1: None arm 2: None
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: Intravenous repeating dose intervention 2: Intravenous repeating dose
intervention 1: placebo intervention 2: rituximab
0
null
0
NCT00087529
[ 3 ]
3
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
null
This is a multicenter, open label extension study. Subjects who have completed treatment in the parent study of pertuzumab, either alone or with a combination agent, and who received at least one dose of pertuzumab in the parent study are eligible for inclusion in this trial if they are continuing to receive clinical benefit.
null
Solid Cancers
Advanced solid cancers HER2 EGFR
null
1
arm 1: Participants received the same dose of pertuzumab that they received in their parent Phase II trial, either 420 mg or 1050 mg, intravenously on Day 1 of every 3 week cycle until disease progression.
[ 0 ]
1
[ 0 ]
intervention 1: Pertuzumab was supplied as a single-use liquid formulation.
intervention 1: Pertuzumab
0
null
0
NCT00096941
[ 5 ]
179
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
The purpose of this trial is to study the safety of treating patients with multiple myeloma and at least one bone lesion with zoledronic acid 4mg intravenously (IV) every 3 - 4 weeks for 2 years. Patients will receive a zoledronic acid infusion for 15 minutes or 30 minutes.
null
Multiple Myeloma
Z-MAX, multiple myeloma, zoledronic acid, bone metastases
null
2
arm 1: Participants received 4 mg zoledronic acid intravenously, in 250 mL of fluid, every 3-4 weeks for up to 24 months, over a 15-minute infusion time, but increasing to a 30-minute infusion time if they experienced a clinically relevant rise in serum creatinine that resolved in less than 12 weeks arm 2: Participants received 4 mg zoledronic acid intravenously, in 250 mL of fluid, every 3-4 weeks for up to 24 months, over a 30-minute infusion time, but increasing to a 45-minute infusion time if they experienced a clinically relevant rise in serum creatinine that resolved in less than 12-weeks.
[ 0, 0 ]
1
[ 0 ]
intervention 1: 4 mg zoledronic acid in 250 mL of calcium-free solution (i.e., 0.9% sodium chloride or 5% glucose) administered intravenously.
intervention 1: zoledronic acid
68
Huntsville | Alabama | United States | -86.58594 | 34.7304 Glendale | Arizona | United States | -112.18599 | 33.53865 Tucson | Arizona | United States | -110.92648 | 32.22174 Little Rock | Arkansas | United States | -92.28959 | 34.74648 Little Rock | Arkansas | United States | -92.28959 | 34.74648 Anaheim | California | United States | -117.9145 | 33.83529 Bakersfield | California | United States | -119.01871 | 35.37329 Campbell | California | United States | -121.94996 | 37.28717 Concord | California | United States | -122.03107 | 37.97798 Fresno | California | United States | -119.77237 | 36.74773 La Jolla | California | United States | -117.2742 | 32.84727 Lancaster | California | United States | -118.13674 | 34.69804 Long Beach | California | United States | -118.18923 | 33.76696 Mission Hills | California | United States | -120.43683 | 34.68609 Orange | California | United States | -117.85311 | 33.78779 Rancho Mirage | California | United States | -116.41279 | 33.73974 Sunnyvale | California | United States | -122.03635 | 37.36883 West Hollywood | California | United States | -118.36174 | 34.09001 Greeley | Colorado | United States | -104.70913 | 40.42331 Fort Meyers | Florida | United States | N/A | N/A Hollywood | Florida | United States | -80.14949 | 26.0112 Kissimmee | Florida | United States | -81.41667 | 28.30468 Miami | Florida | United States | -80.19366 | 25.77427 New Port Richey | Florida | United States | -82.71927 | 28.24418 Ormond Beach | Florida | United States | -81.05589 | 29.28581 Pensacola | Florida | United States | -87.21691 | 30.42131 Rockledge | Florida | United States | -80.72533 | 28.35084 Augusta | Georgia | United States | -81.97484 | 33.47097 Tucker | Georgia | United States | -84.21714 | 33.85455 New Albany | Indiana | United States | -85.82413 | 38.28562 Hutchinson | Kansas | United States | -97.92977 | 38.06084 New Orleans | Louisiana | United States | -90.07507 | 29.95465 Scarborough | Maine | United States | -70.32172 | 43.57814 Bethesda | Maryland | United States | -77.10026 | 38.98067 Clinton | Maryland | United States | -76.89831 | 38.76511 Columbia | Maryland | United States | -76.83942 | 39.24038 Lambertville | Michigan | United States | -83.62799 | 41.76588 Southfield | Michigan | United States | -83.22187 | 42.47337 Kansas City | Missouri | United States | -94.57857 | 39.09973 Saint Joseph | Missouri | United States | -94.84663 | 39.76861 St Louis | Missouri | United States | -90.19789 | 38.62727 Omaha | Nebraska | United States | -95.94043 | 41.25626 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Cherry Hill | New Jersey | United States | -75.03073 | 39.93484 East Brunswick | New Jersey | United States | -74.41598 | 40.42788 Hackensack | New Jersey | United States | -74.04347 | 40.88593 Voorhees Township | New Jersey | United States | -74.49062 | 40.4795 Santa Fe | New Mexico | United States | -105.9378 | 35.68698 Bay Shore | New York | United States | -73.24539 | 40.7251 New York | New York | United States | -74.00597 | 40.71427 Syracuse | New York | United States | -76.14742 | 43.04812 Kettering | Ohio | United States | -84.16883 | 39.6895 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 West Reading | Pennsylvania | United States | -75.94743 | 40.3337 Cranston | Rhode Island | United States | -71.43728 | 41.77982 Providence | Rhode Island | United States | -71.41283 | 41.82399 Charleston | South Carolina | United States | -79.93275 | 32.77632 Knoxville | Tennessee | United States | -83.92074 | 35.96064 Dallas | Texas | United States | -96.80667 | 32.78306 Dallas | Texas | United States | -96.80667 | 32.78306 Lubbock | Texas | United States | -101.85517 | 33.57786 Ogden | Utah | United States | -111.97383 | 41.223 Salem | Virginia | United States | -80.05476 | 37.29347 Lacey | Washington | United States | -122.82319 | 47.03426 Seattle | Washington | United States | -122.33207 | 47.60621 Appleton | Wisconsin | United States | -88.41538 | 44.26193
0
NCT00104104
[ 3, 4 ]
46
RANDOMIZED
PARALLEL
0TREATMENT
3TRIPLE
false
0ALL
null
This study will determine the effectiveness of different treatments for panic disorder symptoms in individuals who still have symptoms after initial treatment with medication.
Panic disorder is a serious condition that may cause significant psychological and physical distress. Many patients with panic disorder remain symptomatic despite initial intervention. Unfortunately, little data is available to guide health care providers in "next-step" treatment approaches. This study will evaluate the effectiveness of treatments for individuals with panic disorder that is resistant to initial treatment with selective serotonin reuptake inhibitors (SSRIs). This study will last 24 weeks and will comprise three phases. In Phase 1, participants will receive the SSRI sertraline for 6 weeks. Phase 1 will be used to determine participants' resistance to treatment. During Phase 1, participants will begin a medication schedule and symptom diary and will have weekly study visits to assess regimen adherence and any side effects they may be experiencing. In Phase 2, participants will be randomly assigned to 6 weeks of one of two treatments: sertraline at an elevated dose from that given in Phase 1 or a sertraline and placebo regimen. During Phase 2, participants will have 3 study visits. Self-report scales and diary entries will be used to assess panic disorder symptoms and medication side effects. In Phase 3, participants will be randomly assigned to receive either cognitive behavioral therapy (CBT) or sertraline and clonazepam for 12 weeks. All participants will have weekly study visits during Phase 3. Questionnaires and self-report scales will be used to assess participants at the end of Phase 3. Study hypothesis: Combined selective serotonin reuptake inhibitors (SSRIs) and benzodiazepine treatment, increasing the dose of SSRI, and the addition of cognitive behavioral therapy (CBT) each may have benefits for patients with panic disorder who remain symptomatic after initial treatment with SSRIs.
Panic Disorder
Selective serotonin reuptake inhibitor Anxiety Pharmacotherapy Cognitive behavioral therapy CBT
null
2
arm 1: Participants in phase II will receive sertraline, or an equivalent medication, up to 100 mg plus a placebo pill. Participants in phase III will receive the same medication with cognitive behavioral therapy. arm 2: Participants in phase II will receive sertraline, or equivalent medication, up to 200 mg. Participants in phase III they will receive the same medication with flexible clonazepam augmentation.
[ 0, 0 ]
3
[ 0, 0, 5 ]
intervention 1: Participants will receive clonazepam. intervention 2: Participants will receive sertraline. intervention 3: Participants will receive cognitive behavioral therapy
intervention 1: Clonazepam intervention 2: Sertraline intervention 3: Cognitive behavioral therapy
1
Boston | Massachusetts | United States | -71.05977 | 42.35843
0
NCT00118417
[ 4 ]
235
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
false
This is a double-blind placebo-controlled clinical investigation into the efficacy and tolerability of inhaled treprostinil in patients with severe pulmonary arterial hypertension. The primary outcome is the change in 6-minute walk distance from baseline to week 12.
Patients who have been on a stable dose of 125 mg twice daily (bid) of bosentan or any stable dose of sildenafil for at least three months prior to study start were randomized to either treprostinil inhalation solution or matching placebo. Administration of study medication was performed by inhalation with the OPTINEB™ ultrasonic nebulizer. The proposed dosing regimen was four times daily-upon awakening, at midday, evening (dinner time) and bedtime. After a patient has completed the twelve-week study period, they were given the option of enrolling into an open-label extension study.
Pulmonary Hypertension
Pulmonary Arterial Hypertension
null
2
arm 1: 0.9 mg/mL treprostinil for inhalation supplied in 2.9mL ampoules for use in ultra sonic nebulizer arm 2: Placebo inhalation solution for use in ultrasonic nebulizer
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: Doses are titrated to 9 breaths four times daily. Each breath produces an 18 mcg dose of inhaled treprostinil. intervention 2: Doses are titrated to 9 breaths four times daily.
intervention 1: Inhaled treprostinil intervention 2: Placebo inhalation solution
30
Birmingham | Alabama | United States | -86.80249 | 33.52066 Tucson | Arizona | United States | -110.92648 | 32.22174 La Jolla | California | United States | -117.2742 | 32.84727 Torrance | California | United States | -118.34063 | 33.83585 Aurora | Colorado | United States | -104.83192 | 39.72943 Orlando | Florida | United States | -81.37924 | 28.53834 Kansas City | Kansas | United States | -94.62746 | 39.11417 Baltimore | Maryland | United States | -76.61219 | 39.29038 Boston | Massachusetts | United States | -71.05977 | 42.35843 Ann Arbor | Michigan | United States | -83.74088 | 42.27756 New York | New York | United States | -74.00597 | 40.71427 Durham | North Carolina | United States | -78.89862 | 35.99403 Pittsburgh | Pennsylvania | United States | -79.99589 | 40.44062 Nashville | Tennessee | United States | -86.78444 | 36.16589 Dallas | Texas | United States | -96.80667 | 32.78306 Wein | Vienna | Austria | N/A | N/A Graz | N/A | Austria | 15.45 | 47.06667 Brussels | N/A | Belgium | 4.34878 | 50.85045 Leuven | N/A | Belgium | 4.70093 | 50.87959 Paris | N/A | France | 2.3488 | 48.85341 Geißen | N/A | Germany | 12.00052 | 50.8617 Dublin | N/A | Ireland | -6.24889 | 53.33306 Haifa | N/A | Israel | 34.99928 | 32.81303 Jerusalem | N/A | Israel | 35.21633 | 31.76904 Petach Tikvah 49100 | N/A | Israel | N/A | N/A Bologna | N/A | Italy | 11.33875 | 44.49381 Barcelona | N/A | Spain | 2.15899 | 41.38879 Cambridge | N/A | United Kingdom | 0.11667 | 52.2 Glasgow GII 6NT | N/A | United Kingdom | N/A | N/A London | N/A | United Kingdom | -0.12574 | 51.50853
0
NCT00147199
[ 4 ]
187
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
true
Schizophrenia is a brain disease. The primary features of schizophrenia are characterized by Positive symptoms (symptoms that should not be there, inability to think clearly, to distinguish reality from fantasy i.e., hearing voices) and Negative symptoms (a reduction or absence of normal behaviors or emotions, i.e., unable to manage emotions, make decisions and relate to others). Other symptoms include reduced ability to recall and learn new information, difficulty with problem solving, or maintaining productive employment. The symptoms of schizophrenia may be due to an imbalance in chemicals in the brain, primarily dopamine and serotonin, which enables brain cells to communicate with each other. The clinical development of asenapine, as described in the 2007 IDB appears to have antipsychotic activity with superior symptomatic control compared to placebo and an improved safety profile compared to currently available neuroleptics. Its fast dissolving formulation may further add to treatment compliance. While various titration schedules have been used in previous studies, dose increases at 5 mg BID (twice daily) up to 10 mg BID have been well tolerated. Therefore, further exploration in a larger group of subjects with acute exacerbation of schizophrenia using an asenapine flexible dosing design ( 5 or 10 mg BID) will mimic actual clinical practice in a long-term 52-week extension trial.
null
Schizophrenia
null
3
arm 1: Haloperidol in original study (NCT00156104) and in current long-term extension. arm 2: Asenapine in original study and asenapine in current long-term extension. arm 3: Double-Blind subjects randomized to only placebo medication for 6 weeks in the short-term 041023 asenapine trial, were randomized (double-blind) into the long-term 041513 asenapine extension trial and received asenapine 5 mg BID for Week 1. After Week 1, subjects received asenapine (either 5 mg BID or 10 mg BID) for the remainder of the 52-week trial.
[ 1, 0, 0 ]
3
[ 0, 0, 0 ]
intervention 1: 2-8 mg BID intervention 2: 5 or 10 mg BID intervention 3: 5 or 10 mg BID
intervention 1: Haloperidol intervention 2: Asenapine intervention 3: Asenapine
0
null
0
NCT00156065
[ 5 ]
203
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
This is an efficacy and safety study of Vytorin (ezetimibe (+) simvastatin) compared to atorvastatin (ezetimibe/simvastatin) at week 6 in primary hypercholesterolemia patients in Korea. The primary hypothesis being tested is that daily administration of Vytorin will result in a greater reduction of low density lipoprotein cholesterol (LDL-C) concentration from baseline after 6 weeks treatment compared to atorvastatin.
null
Hypercholesterolemia
null
2
arm 1: Ezetimibe 10 mg/Simvastatin 20 mg arm 2: Atorvastatin 10 mg
[ 0, 1 ]
2
[ 0, 0 ]
intervention 1: simvastatin/ezetimibe 10/20 mg intervention 2: atorvastatin 10 mg
intervention 1: ezetimibe (+) simvastatin intervention 2: atorvastatin
0
null
0
NCT00166504
[ 5 ]
125
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
null
1FEMALE
null
The purpose of this study is compare treatment with valsartan with the possible addition of a diuretic, hydrochlorothiazide, on high blood pressure with the drug amlodipine with the possible addition of a diuretic, hydrochlorothiazide. In particular, the effect of treatment on the stiffness of the blood vessels will be studied.
null
Hypertension
hypertension postmenopausal valsartan amlodipine hydrochlorothiazide
null
2
arm 1: None arm 2: None
[ 0, 0 ]
3
[ 0, 0, 0 ]
intervention 1: Patients received 160 mg valsartan for 4 weeks. Patients were then up-titrated to receive either 320 mg valsartan for the following 8 weeks. intervention 2: Patients received 5 mg amlodipine for four weeks. Patients were then up-titrated to receive 10 mg amlodipine for the following 8 weeks. intervention 3: At Week 12, patients who did not meet target Blood Pressure for both Systolic Blood Pressure \< 140 mmHg and Diastolic Blood Pressure \< 90 mmHg were eligible to receive 12.5 mg open-label Hydrochlorothiazide for the following 26 weeks.
intervention 1: Valsartan 320 mg intervention 2: Amlodipine 10 mg intervention 3: Hydrochlorothiazide
1
Basel | N/A | Switzerland | 7.57327 | 47.55839
0
NCT00171054
[ 5 ]
47
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
This study evaluates two different immunosuppression drug regimens in patients with a recent kidney transplant. Patients initially received a regimen of Sirolimus, Tacrolimus and Prednisone and then randomized to discontinue either Tacrolimus or Prednisone.
null
Kidney Failure Graft vs Host Disease
Kidney Transplant
null
2
arm 1: None arm 2: None
[ 0, 1 ]
3
[ 0, 0, 0 ]
intervention 1: None intervention 2: None intervention 3: None
intervention 1: Tacrolimus intervention 2: Sirolimus intervention 3: prednisone
1
São Paulo | N/A | Brazil | -46.63611 | -23.5475
0
NCT00195429
[ 5 ]
40
RANDOMIZED
PARALLEL
0TREATMENT
1SINGLE
false
0ALL
false
Ketamine, an FDA approved anesthetic agent, is becoming the sedative/analgesic of choice for emergency sedation in children because it causes deep sedation with minimal respiratory depression in comparison to other available agents. However, emergence reactions are an important adverse effect of ketamine, characterized by transient changes in cognitive function, dissociation and mild schizophrenia-like symptoms. These cognitive and behavioral effects are dose-dependently induced by ketamine and other antagonists of the N-methyl-D-aspartate (NMDA) glutamate receptor. NMDA receptor hypofunction can disinhibit excitatory (cholinergic/glutamatergic) projections in key areas of the brain, and this has been proposed to explain key features of schizophrenia. Several treatments that block excessive excitatory transmitter release have also been shown to prevent cognitive and behavioral effects of ketamine-induced NMDA receptor hypofunction in humans. Alpha-2 adrenergic agonists, which can presynaptically inhibit acetylcholine release, can prevent mild ketamine-induced behavioral and cognitive symptoms in healthy human adults. However, this prevention strategy has not been evaluated in children. Children currently receive clinically-indicated treatment with the NMDA antagonist, ketamine, and this age group is an important target for pharmacological strategies aimed at the prevention of schizophrenia. This application proposes a double-blind, placebo-controlled, randomized trial to test the safety and effectiveness of dexmedetomidine, an FDA approved alpha-2 adrenergic agonist, in preventing ketamine-induced mental symptoms in children. Planned primary analyses will evaluate effects of the hypothesized prevention treatment on clinical and cognitive variables using analysis of variance (ANOVA). The proposed experiments are relevant to future prevention trials for individuals at risk for schizophrenia, and to preventing adverse effects of NMDA antagonist anesthetic agents (ketamine, nitrous oxide).
The proposed study will be conducted using existing dedicated clinical and research space in St. Louis Children's Hospital's Emergency Department, Pediatric Clinical Research Center (PCRC), and Orthopedic Clinic. This project has 3 major aims and 1 exploratory aim addressed by a prospective randomized blinded placebo controlled drug trial to test whether a pharmacological strategy can prevent NMDA receptor hypofunction-induced behavioral and cognitive dysfunction in pre- and post-pubertal children. Based on previous preclinical and clinical research on the effects and blockade of the effects of ketamine and similar compounds, the study investigators have carefully selected a dose of the alpha-2 adrenergic agonist dexmedetomidine that will permit this study to be conducted with low risk to enrolled subjects who are undergoing clinically-indicated ketamine sedation for forearm fracture reduction. General Experimental Design: This project will test the safety and effectiveness of dexmedetomidine for preventing ketamine-induced behavioral and cognitive symptoms in healthy human children undergoing clinically indicated ketamine sedation for forearm fracture reduction. Aims 1 and 2 will be addressed by randomized, blinded administration of dexmedetomidine or saline placebo to ketamine-sedated subjects to test the efficacy of dexmedetomidine in preventing ketamine-induced behavioral and cognitive changes during recovery from sedation. Aim 3 will be addressed by comparing between the subjects randomized to receive dexmedetomidine or saline placebo measurements of distress and frequency of adverse cardiopulmonary effects during sedation, fracture-reduction, and recovery.
Psychoses, Substance-Induced
NMDA antagonist-induced psychosis Children Memory Impairment Decreased cognitive function Increased memory impairment
null
2
arm 1: Ketamine without dexmedetomidine arm 2: Ketamine infusion plus dexmedetomidine
[ 2, 0 ]
2
[ 0, 0 ]
intervention 1: Ketamine without dexmedetomidine intervention 2: Ketamine plus dexmedetomidine
intervention 1: Ketamine intervention 2: Dexmedetomidine
1
St Louis | Missouri | United States | -90.19789 | 38.62727
0
NCT00205712
[ 4 ]
559
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This is a Phase III, randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy of retreatment with rituximab in subjects with active rheumatoid arthritis (RA) who are receiving Methotrexate (MTX).
null
Rheumatoid Arthritis
null
2
arm 1: 1000 mg rituximab intravenous initial treatment on day 1 and day 15 plus 10-25 mg/week methotrexate followed by re-treatment during weeks 24 -40 consisting of two additional doses of 1000 mg rituximab 14 days apart plus 10-25 mg/week methotrexate. arm 2: 1000 mg rituximab intravenous initial treatment on day 1 and day 15 plus 10-25 mg/week methotrexate followed by retreatment during weeks 24 -40 consisting of two doses of placebo 14 days apart plus 10-25 mg/week methotrexate.
[ 0, 2 ]
4
[ 0, 0, 0, 0 ]
intervention 1: Intravenous repeating dose intervention 2: Intravenous repeating dose intervention 3: Oral or parenteral repeating dose intervention 4: Intravenous repeating dose
intervention 1: placebo intervention 2: rituximab intervention 3: methotrexate intervention 4: folate
0
null
0
NCT00266227
[ 3 ]
20
NON_RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
Intracavitary injection of low dose alteplase into loculated abdominopelvic abscesses will reduce the duration of percutaneous drainage and increase the proportion of successful drainages.
The use of fibrinolytics for the percutaneous drainage of loculated pleural effusions has been shown to reduce the catheter dwell time and to improve drainage of the effusions. Abscesses in the abdomen and pelvis are often loculated which makes percutaneous drainage difficult. We hypothesize that the infusion of alteplase via a drainage catheter into the loculated abscess collections of the abdomen and pelvis will similarly decrease catheter dwell time and improve overall abscess drainage. The direct injection of Activase into abscess cavities utilizes a very low dose of drug within a closed environment which should not be associated with any significant risk of hemorrhage. To date, there does not appear to be a significant risk of systemic hemorrhagic complications associated with the use of intracavitary thrombolytics for the drainage of abdominopelvic abscesses although only a few such studies have been reported. We hope to prove that the use alteplase for intracavitary thrombolysis improves outcomes associated with percutaneous catheter drainage of loculated abscess collections without increasing complications or costs. The design of the trial will be as a single-center, prospective, open-label, randomized trial comparing the infusion of Activase versus saline for treatment of loculated abdominopelvic abscesses requiring percutaneous drainage. Patients with loculated abdominopelvic abscesses who are referred for percutaneous drainage will be eligible for this study. Patients will undergo standard placement of a 10-12 french percutaneous drain into their abscess cavity under computed tomography guidance. If the entire contents of the abscess cavity cannot be aspirated at the time of initial catheter placement, the abscess will be assumed to be loculated. The patient will then be randomized to have their abscess catheter irrigated twice a day with a volume of fluid approximately equal to the one-half the residual volume of the abscess. In the control group, the normal saline will be fluid instilled into the abscess cavity. The study group will receive Activase reconstituted in sterile water and then diluted to the appropriate volume with normal saline.
Abdominal Abscess Pelvic Abscess
null
2
arm 1: Multiple Alteplase injection into the abscess collection to improve percutaneous drainage arm 2: Multiple normal saline injection into the abscess collection to improve percutaneous drainage
[ 0, 2 ]
2
[ 0, 10 ]
intervention 1: 2mg or 4mg given twice daily for three days into loculated abscess intervention 2: saline injection twice daily for three days
intervention 1: Alteplase intervention 2: saline
1
Honolulu | Hawaii | United States | -157.85833 | 21.30694
0
NCT00284739
[ 5 ]
725
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This study will last up to 6 weeks. Subjects will visit the clinic up to 5 times. Certain clinic visits will include a physical examination, medical history review, and lung function tests. All study related medications and medical examinations will be provided at no cost to the subject. The drugs used in this study are approved for the age group under study.
A Multicenter, Randomized, Double-Blind, Triple-Dummy, Placebo-Controlled, Parallel Group, Four-Week Study Assessing the Efficacy of Fluticasone Propionate Aqueous Nasal Spray 200mcg QD versus Montelukast 10mg QD in Adolescent and Adult Subjects with Asthma and Seasonal Allergic Rhinitis Who are Receiving ADVAIR DISKUS® 100/50mcg BID or Placebo BID
Asthma
Allergic Rhinitis Asthma
null
4
arm 1: Fluticasone propionate/salmeterol DISKUS combination product (FSC) twice daily (BID) plus vehicle placebo nasal spray once daily (QD) plus montelukast capsule 10mg (MON) QD arm 2: FSC BID plus vehicle placebo nasal spray QD plus placebo capsule QD arm 3: Fluticasone propionate/salmeterol DISKUS combination product (FSC)100/50mcg BID plus fluticasone propionate aqueous nasal spray 200mcg (FPANS) QD plus placebo capsule QD arm 4: Placebo DISKUS BID plus vehicle placebo nasal spray QD plus MON QD
[ 1, 1, 1, 1 ]
7
[ 0, 0, 0, 0, 0, 0, 0 ]
intervention 1: fluticasone propionate/salmeterol DISKUS combination intervention 2: montelukast capsule intervention 3: fluticasone propionate aqueous nasal spray intervention 4: vehicle placebo nasal spray intervention 5: ADVAIR DISKUS intervention 6: placebo capsule intervention 7: placebo DISKUS
intervention 1: fluticasone propionate/salmeterol (FSC) intervention 2: montelukast (MON) intervention 3: fluticasone propionate (FP) intervention 4: placebo nasal intervention 5: ADVAIR DISKUS intervention 6: placebo capsule intervention 7: placebo DISKUS
121
Birmingham | Alabama | United States | -86.80249 | 33.52066 Glendale | Arizona | United States | -112.18599 | 33.53865 Scottsdale | Arizona | United States | -111.89903 | 33.50921 Tucson | Arizona | United States | -110.92648 | 32.22174 Hot Springs | Arkansas | United States | -93.05518 | 34.5037 Berkeley | California | United States | -122.27275 | 37.87159 Huntington Beach | California | United States | -117.99923 | 33.6603 Long Beach | California | United States | -118.18923 | 33.76696 Los Angeles | California | United States | -118.24368 | 34.05223 Rancho Mirage | California | United States | -116.41279 | 33.73974 Riverside | California | United States | -117.39616 | 33.95335 Roseville | California | United States | -121.28801 | 38.75212 San Diego | California | United States | -117.16472 | 32.71571 San Diego | California | United States | -117.16472 | 32.71571 San Jose | California | United States | -121.89496 | 37.33939 San Jose | California | United States | -121.89496 | 37.33939 Stockton | California | United States | -121.29078 | 37.9577 Vista | California | United States | -117.24254 | 33.20004 Boulder | Colorado | United States | -105.27055 | 40.01499 Colorado Springs | Colorado | United States | -104.82136 | 38.83388 Fort Collins | Colorado | United States | -105.08442 | 40.58526 Lakewood | Colorado | United States | -105.08137 | 39.70471 Brandon | Florida | United States | -82.28592 | 27.9378 Coral Gables | Florida | United States | -80.26838 | 25.72149 Ocala | Florida | United States | -82.14009 | 29.1872 Pensacola | Florida | United States | -87.21691 | 30.42131 Tallahassee | Florida | United States | -84.28073 | 30.43826 Albany | Georgia | United States | -84.15574 | 31.57851 Atlanta | Georgia | United States | -84.38798 | 33.749 Columbus | Georgia | United States | -84.98771 | 32.46098 Gainesville | Georgia | United States | -83.82407 | 34.29788 Lawrenceville | Georgia | United States | -83.98796 | 33.95621 Savannah | Georgia | United States | -81.09983 | 32.08354 Savannah | Georgia | United States | -81.09983 | 32.08354 Chicago | Illinois | United States | -87.65005 | 41.85003 Springfield | Illinois | United States | -89.64371 | 39.80172 Indianapolis | Indiana | United States | -86.15804 | 39.76838 South Bend | Indiana | United States | -86.25001 | 41.68338 Iowa City | Iowa | United States | -91.53017 | 41.66113 Overland Park | Kansas | United States | -94.67079 | 38.98223 Lexington | Kentucky | United States | -84.47772 | 37.98869 Louisville | Kentucky | United States | -85.75941 | 38.25424 Owensboro | Kentucky | United States | -87.11333 | 37.77422 Baton Rouge | Louisiana | United States | -91.18747 | 30.44332 Covington | Louisiana | United States | -90.10042 | 30.47549 Lafayette | Louisiana | United States | -92.01984 | 30.22409 Shreveport | Louisiana | United States | -93.75018 | 32.52515 Sunset | Louisiana | United States | -92.06845 | 30.41131 Baltimore | Maryland | United States | -76.61219 | 39.29038 North Andover | Massachusetts | United States | -71.13506 | 42.6987 Minneapolis | Minnesota | United States | -93.26384 | 44.97997 Jackson | Mississippi | United States | -90.18481 | 32.29876 Jefferson City | Missouri | United States | -92.17352 | 38.5767 Rolla | Missouri | United States | -91.77127 | 37.95143 St Louis | Missouri | United States | -90.19789 | 38.62727 Warrensburg | Missouri | United States | -93.73605 | 38.76279 Lincoln | Nebraska | United States | -96.66696 | 40.8 Omaha | Nebraska | United States | -95.94043 | 41.25626 Omaha | Nebraska | United States | -95.94043 | 41.25626 Omaha | Nebraska | United States | -95.94043 | 41.25626 Papillion | Nebraska | United States | -96.04224 | 41.15444 Forked River | New Jersey | United States | -74.19014 | 39.83984 Summit | New Jersey | United States | -74.36468 | 40.71562 Rochester | New York | United States | -77.61556 | 43.15478 Asheville | North Carolina | United States | -82.55402 | 35.60095 Raleigh | North Carolina | United States | -78.63861 | 35.7721 Canton | Ohio | United States | -81.37845 | 40.79895 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Parma | Ohio | United States | -81.72291 | 41.40477 Oklahoma City | Oklahoma | United States | -97.51643 | 35.46756 Bend | Oregon | United States | -121.31531 | 44.05817 Portland | Oregon | United States | -122.67621 | 45.52345 Pittsburgh | Pennsylvania | United States | -79.99589 | 40.44062 Upland | Pennsylvania | United States | -75.38269 | 39.85261 Providence | Rhode Island | United States | -71.41283 | 41.82399 Charleston | South Carolina | United States | -79.93275 | 32.77632 Charleston | South Carolina | United States | -79.93275 | 32.77632 Greenville | South Carolina | United States | -82.39401 | 34.85262 Orangeburg | South Carolina | United States | -80.85565 | 33.49182 Simpsonville | South Carolina | United States | -82.25428 | 34.73706 Spartanburg | South Carolina | United States | -81.93205 | 34.94957 Chattanooga | Tennessee | United States | -85.30968 | 35.04563 Germantown | Tennessee | United States | -89.81009 | 35.08676 Knoxville | Tennessee | United States | -83.92074 | 35.96064 Savannah | Tennessee | United States | -88.2492 | 35.2248 Austin | Texas | United States | -97.74306 | 30.26715 Dallas | Texas | United States | -96.80667 | 32.78306 Dallas | Texas | United States | -96.80667 | 32.78306 Dallas | Texas | United States | -96.80667 | 32.78306 Dallas | Texas | United States | -96.80667 | 32.78306 El Paso | Texas | United States | -106.48693 | 31.75872 El Paso | Texas | United States | -106.48693 | 31.75872 Houston | Texas | United States | -95.36327 | 29.76328 Houston | Texas | United States | -95.36327 | 29.76328 Kerrville | Texas | United States | -99.14032 | 30.04743 San Antonio | Texas | United States | -98.49363 | 29.42412 San Antonio | Texas | United States | -98.49363 | 29.42412 San Antonio | Texas | United States | -98.49363 | 29.42412 Waco | Texas | United States | -97.14667 | 31.54933 Salt Lake City | Utah | United States | -111.89105 | 40.76078 West Jordan | Utah | United States | -111.9391 | 40.60967 Danville | Virginia | United States | -79.39502 | 36.58597 Richmond | Virginia | United States | -77.46026 | 37.55376 Kirkland | Washington | United States | -122.20874 | 47.68149 Winnipeg | Manitoba | Canada | -97.14704 | 49.8844 Ajax | Ontario | Canada | -79.03288 | 43.85012 Brampton | Ontario | Canada | -79.76633 | 43.68341 Greater Sudbury | Ontario | Canada | -80.99001 | 46.49 Kanata | Ontario | Canada | -75.91606 | 45.3001 Mississauga | Ontario | Canada | -79.6583 | 43.5789 Niagara Falls | Ontario | Canada | -79.06627 | 43.10012 Ottawa | Ontario | Canada | -75.69812 | 45.41117 Ottawa | Ontario | Canada | -75.69812 | 45.41117 Québec | Quebec | Canada | -71.21454 | 46.81228 Trois-Rivières | Quebec | Canada | -72.5477 | 46.34515 Tallinn | N/A | Estonia | 24.75353 | 59.43696 Tartu | N/A | Estonia | 26.72509 | 58.38062 Bialystok | N/A | Poland | 23.16433 | 53.13333 Bialystok | N/A | Poland | 23.16433 | 53.13333 Krakow | N/A | Poland | 19.93658 | 50.06143 Lodz | N/A | Poland | 19.47395 | 51.77058
0
NCT00296491
[ 3 ]
30
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
true
The purpose of this study is to determine how GMP IV Artesunate is metabolized and cleared by individuals with uncomplicated malaria infection and to determine how fast it eliminates malaria infection from the body.
This is an unblinded non-randomized phase II pharmacokinetic study of a new GMP formulation of intravenous artesunate. Artesunate has been used throughout Asia and Africa for many years. Its overall efficacy associated with the ability to lower parasitemia is well established. To date, pharmacokinetic studies have not been done in Africa using GMP (Good Manufacturing Practices)-produced drug. The objective of this study is to show that GMP IV artesunate rapidly clears parasites in Adult Kenyan populations with malaria and that the pharmacokinetic profile of the drug approximates other populations of adults tested (Asians and North Americans).
Malaria
Uncomplicated Malaria GMP artesunate
null
1
arm 1: Subject are given intravenous Artesunate once a day for 3 days. Following completion of Artesunate treatment, all subjects received Malarone follow-on therapy to ensure parasitologic cure.
[ 0 ]
2
[ 0, 0 ]
intervention 1: Intravenous Artesunate (2.4 mg/kg) once a day for three days intervention 2: (proguanil/atovaquone) follow-on therapy (4 tablets once daily for three days)
intervention 1: Artesunate intervention 2: Malarone
1
Kisumu | New Nyanza | Kenya | 34.76171 | -0.10221
0
NCT00298610
[ 3 ]
97
RANDOMIZED
PARALLEL
0TREATMENT
1SINGLE
false
0ALL
true
Functional constipation (FC) is common with 14.3% estimated prevalence in Hong Kong, but treatment for this condition in conventional medicine is suboptimal. Complementary and alternative medicines, especially Chinese herbal medicine (CHM) are used frequently by patients with FC, but there is little research evidence about these commonly used CHM. The purpose of the study is evaluate the efficacy and safety of CHM, as well as determining the optimal dosage.
Functional constipation (FC) is a common complaint in clinical practice, with the estimated prevalence 14.3% in Hong Kong, as nearly affecting 1 million Hong Kong People in different extent. It is comparable with western population, which is 15% in North America. By the definition of Rome II criteria, FC comprises a group of functional disorders, which presents as persistent difficult, infrequent or seemingly incomplete defecation. Constipation is often perceived to be benign, easily treated condition with short-term treatment being relatively straightforward. However, the fact is the management of FC is perplexed as some subjects complain of constipation more than decade. Moreover, chronic constipation can develop into more serious bowel complaints, such as faecal impaction, incontinence and bowel perforations. There is also accumulating evidence shown that constipated subjects have significantly higher anxiety and depression scores and lower quality of life. Therefore, the demand of effective agents to normalize bowel function is extremely large. Conventional treatment for constipation mainly relies on dietary fibre and laxatives. Although there is no credible evidence that any serious problem is associated with their prolonged use, the treatment of it has been suboptimal. First, a recent systematic review pointed out that there were paucity of trials for many commonly used agents, therefore, their use might not be well validated. Second, many patients with severe constipation do not respond adequately or lose of effectiveness after a short period of time. Third, many patients who intake dietary fibre complain of flatulence, distension, bloating and poor taste. As a result, the compliance is low as about 50%. Fourth, the use of osmotic laxatives, such as polyethylene glycol, become increasingly popular due to fewer side effects and better taste, however, the prices are much more expensive than other medications. Many constipation sufferers seek help from alternative medicine, especially from Chinese herbal medicine. For example, according to a telephone survey in Hong Kong, more than 85% of constipated subjects seek for coping strategies, such as asking for medical consultations, taking prescribed medicine and seeking for alternative therapy, involving Chinese medicine. Traditional Chinese medicine (TCM) is particularly attractive as their effectiveness in treating functional disorders and retaining balance of body functions. The CHM used in study is derived from classic text of Chinese medicine (Shang Han Lun, Discussion of Cold-induced Disorders), which can "moisten the intestines, drain heat, promote the movement of qi and unblock the bowel". It is well known that randomized controlled trial (RCT) is the gold standard to test the efficacy of intervention, thus in this project, we attempt to follow the basic requirements of RCT to testify the efficacy and safety of CHM on FC, as well as to determine the optimal dosage. We believe such study will benefit the advancement of CHM, or even as the foundation of research study in future.
Constipation
Randomized Controlled Trials Medicine, Chinese Traditional Medicine, Herbal Constipation
null
3
arm 1: MaZiRenWan (MZRW) Low dose 2.5g sachet by mouth, twice daily for 8 weeks arm 2: MaZiRenWan (MZRW) Median dose 5.0g sachet by mouth, twice daily for 8 weeks arm 3: MaZiRenWan (MZRW) High dose 7.5g sachet by mouth, twice daily for 8 weeks
[ 0, 0, 0 ]
3
[ 0, 0, 0 ]
intervention 1: Dissolved MaZiRenWan (MZRW) granule (2.5g/sachet) in 150 ml hot water, take orally twice daily for 8 weeks intervention 2: Dissolved MaZiRenWan (MZRW) granule (5.0g/sachet) in 150 ml hot water, take orally twice daily for 8 weeks intervention 3: Dissolved MaZiRenWan (MZRW) granule (7.5g/sachet) in 150 ml hot water, take orally twice daily for 8 weeks
intervention 1: MaZiRenWan (MZRW) Low dose intervention 2: MaZiRenWan (MZRW) Median dose intervention 3: MaZiRenWan (MZRW) High dose
1
Hong Kong | N/A | China | 114.17469 | 22.27832
0
NCT00299975
[ 5 ]
216
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
null
To determine if duloxetine 60 mg once daily can work up to 6 months in treating pain from Diabetic Neuropathy.
null
Diabetic Neuropathies
null
1
arm 1: All subjects receive 30 mg once daily (QD), by mouth (per os - PO) for 1 week followed by duloxetine 60 mg QD, PO for 7 weeks, then maintenance at 60 mg QD, PO for responders to 6 months and rescue at 120 mg QD, PO for non-responders to 6 months. Patients beginning maintenance at the 60 mg QD dose could be increased to the 120 mg QD level if they did not maintain appropriate level of response throughout the maintenance period.
[ 0 ]
1
[ 0 ]
intervention 1: None
intervention 1: Duloxetine
19
Fortaleza | N/A | Brazil | -38.54306 | -3.71722 Porto Alegre | N/A | Brazil | -51.23019 | -30.03283 Angers | N/A | France | -0.55202 | 47.47156 Annecy | N/A | France | 6.12565 | 45.90878 Bron | N/A | France | 4.91303 | 45.73865 Dijon | N/A | France | 5.01667 | 47.31667 Narbonne | N/A | France | 3.00141 | 43.18396 Nevers | N/A | France | 3.159 | 46.98956 Nice | N/A | France | 7.26608 | 43.70313 Chemitz | N/A | Germany | N/A | N/A Dresden | N/A | Germany | 13.73832 | 51.05089 Mainz | N/A | Germany | 8.2791 | 49.98419 Münster | N/A | Germany | 7.62571 | 51.96236 Schkeuditz | N/A | Germany | 12.22141 | 51.39678 Siegen | N/A | Germany | 8.02431 | 50.87481 Würzburg | N/A | Germany | 9.95121 | 49.79391 Genova | N/A | Italy | 11.87211 | 45.21604 Perugia | N/A | Italy | 12.38878 | 43.1122 Rome | N/A | Italy | 12.51133 | 41.89193
0
NCT00322621
[ 4 ]
584
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
false
The purpose of this study is to compare the efficacy of cethromycin to clarithromycin for the treatment of mild to moderate community-acquired pneumonia (CAP).
Lower respiratory tract infections remain one of the leading causes of death worldwide. Increasing rates of antibiotic resistance and newer, more pervasive pneumonia-causative pathogens contribute to this statistic. Currently available macrolide antibiotics for the treatment of community-acquired pneumonia are slowly losing effectiveness, resulting in the need to develop newer drugs to fight resistant infections. In this study, we compare the safety and efficacy of a common macrolide, clarithromycin, to a new ketolide, cethromycin.
Pneumonia
Pneumonia Respiratory Infection Infectious Advanced Life Sciences Lung Pulmonary Cethromycin Restanza Clarithromycin Biaxin
null
2
arm 1: None arm 2: None
[ 1, 0 ]
2
[ 0, 0 ]
intervention 1: Cethromycin 300 mg once per day (QD) for 7 days, administered orally intervention 2: Clarithromycin 250 mg twice per day (BID) for 7 days, administered orally
intervention 1: Cethromycin intervention 2: Clarithromycin
3
Woodridge | Illinois | United States | -88.05034 | 41.74697 Woodridge | Illinois | United States | -88.05034 | 41.74697 Woodridge | Illinois | United States | -88.05034 | 41.74697
0
NCT00336505
[ 3 ]
16
RANDOMIZED
PARALLEL
0TREATMENT
3TRIPLE
false
0ALL
true
The purposes of this study in United Network for Organ Sharing (UNOS) Status 1B (or country equivalent) cardiac transplant candidates are to assess the safety and efficacy of Natrecor (nesiritide). The study will evaluate the drug's ability to prevent clinical worsening when administered as a 28-day continuous intravenous infusion in patients receiving standard care and continuous intravenous infusion of dobutamine or milrinone.
Endogenous B-type natruretic peptide (BNP) concentrations correlate with heart failure (HF) severity and are significantly elevated in severe end-stage HF patients. However, normal biologic compensatory responsiveness to BNP is attenuated in patients with severe HF. Patients who are UNOS 1B cardiac transplant candidates have relatively few options for therapy, despite the fact that they constitute a very ill patient population. These options include getting a transplant, if one is available, receiving intravenous inotropes and/or vasodilators, or being implanted with a circulatory support device (e.g. Left Ventricular Assist Device). Natrecor (Nesiritide) is a human recombinant form of BNP approved for use in the treatment of acute decompensated HF. Limited information is available from controlled clinical trials on the effects of Natreor (nesiritide) infusion for longer than 72 hours. However, based on reports in the literature, Natrecor (nesiritide) has been used by independent investigators for much longer durations in end-stage HF patients, including UNOS Status 1B cardiac transplant candidates, who are refractory to standard care. In these patients, Natrecor (nesiritide) has been safely administered by continuous infusion for more than 2 and up to 210 days. These experiences showed that continuous Natrecor (nesiritide) infusion was well tolerated, uniformly improved hemodynamics (circulation of the blood and the forces involved), maintained stable renal (kidney) function, and reduced the need for ventricular assist devices during the prolonged wait for heart transplant. In controlled clinical trials reported in literature, Natrecor (nesiritide) was given concomitantly with several other standard HF therapeutic agents, such as diuretics, angiotensin-converting enzyme (ACE) inhibitors, beta-blockers, dopamine, dobutamine, anticoagulants, digoxin, and oral nitrates. Concomitant dosing was well tolerated, and no drug interactions were observed. No randomized controlled trial has been conducted to evaluate the safety of combination nesiritide and milrinone therapy. Several investigations were undertaken because of concerns over the potential additive hypotensive (lowering blood pressure) effect that these two agents may exert when administered concurrently. The study investigators reported that concurrent dosing of milrinone and nesiritide appeared to be safe and beneficial, without evidence of an additive hypotensive (lowering blood pressure) effect. The TMAC trial will investigate the safety and efficacy of Natrecor (nesiritide), compared with placebo, when added to standard care in the management of end-stage refractory HF.This prospective, randomized(patients are assigned different treatments based on chance), parallel, multicenter, double-blind (neither the doctor nor the patient knows whether the patient is being administered a placebo or the test drug), placebo-controlled study will be conducted with UNOS Status 1B (or equivalent) cardiac transplant candidates who are receiving standard care and continuous IV infusion of dobutamine or milrinone while awaiting heart transplantation. Approximately 120 patients will participate in this study. Enrolled patients will be randomized 1:1 to receive Natrecor (nesiritide) by continuous infusion in addition to standard care and one inotrope (drugs such as dobutamine or milrinone used to increase the heart rate and improve the force of cardiac contraction), or to receive placebo by continuous infusion in addition to standard care and one inotrope (dobutamine or milrinone). Patients will be managed as inpatients or outpatients, according to the guidelines in this protocol and study center practices. All patients will continue to receive their usual long-term cardiac medications (excluding commercial nesiritide). Study drug dose adjustments may be made according to the guidelines in this protocol and the investigator's clinical judgment, and in accordance with the dosing guidelines.Study drug will be administered as a continuous intravenous infusion under the control of a programmable ambulatory infusion pump for the 28-day treatment period. Patients will be hospitalized for study drug initiation and titration to ensure safety, observe tolerability, and achieve the recommended study drug dose. Day 0 (Screening) will entail completing required procedures and randomization. Baseline and screening procedures may occur on the same day. A window of up to 7 days will be permitted between Day 0 (Screening) and Day 1 (Baseline) visits if circumstances delay initiation of study drug dosing. The following day, Day 1 (Baseline), continuous infusion of study drug will begin. Patients will be hospitalized for a minimum of 3 days after starting the study drug for titration and dose optimization. A dose optimization period of up to 5 days will be permitted. Patients will be eligible for discharge following completion of the study procedures on the 4th dosing day (Day 4). Patients will return to the clinic for weekly visits (± 2 days) through Day 28 (Termination of Treatment) and again at Day 35 for a 7-day post dosing safety visit. On the Day 28 visit (Termination of Treatment), Patients may be hospitalized for up to 24 hours at the investigator's discretion to undergo end of treatment study procedures. This study includes two substudies: the Pulmonary Function Testing Substudy and the Glomerular Filtration Rate Substudy. All TMAC Patients will be asked to participate in these substudies. Participation is optional and does not affect enrollment or participation in TMAC. The Study Hypothesis is that continuous administration of Natrecor (nesiritide) over 28 days when added to standard care (including inotropes) is safe and provides a clinical benefit when compared to placebo added to standard care (including inotropes) therapy. Study drug will be administered continuously as a fixed-rate infusion for 28 days without a bolus dose. Natrecor (nesiritide) dosing will begin at 0.005 mcg/kg/min and may be titrated to a maximum dose of 0.015 mcg/kg/min.
Congestive Heart Failure Cardiac Transplantation Renal Insufficiency Renal Failure
Congestive heart failure cardiac transplantation renal insufficiency renal failure dialysis ultrafiltration vasodilator Natrecor (nesiritide)
null
2
arm 1: Natrecor (nesiritide)+Standard Care+dobutamine or milrinone 28-day continuous infusion no bolus 3-hour 0.005 mcg/kg/min may be titrated to 0.015 mcg/kg/min arm 2: Placebo+Standard Care+dobutamine or milrinone 28-day continuous infusion no bolus 3-hour 0.005 mcg/kg/min may be titrated to 0.015 mcg/kg/min
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: 3-hour 0.005 mcg/kg/min, may be titrated to 0.015 mcg/kg/min intervention 2: 28-day continuous infusion, no bolus
intervention 1: Natrecor (nesiritide)+Standard Care+dobutamine or milrinone intervention 2: Placebo+Standard Care+dobutamine or milrinone
14
Stanford | California | United States | -122.16608 | 37.42411 Tampa | Florida | United States | -82.45843 | 27.94752 Chicago | Illinois | United States | -87.65005 | 41.85003 Maywood | Illinois | United States | -87.84312 | 41.8792 Baltimore | Maryland | United States | -76.61219 | 39.29038 Boston | Massachusetts | United States | -71.05977 | 42.35843 St Louis | Missouri | United States | -90.19789 | 38.62727 New York | New York | United States | -74.00597 | 40.71427 Cleveland | Ohio | United States | -81.69541 | 41.4995 Columbus | Ohio | United States | -82.99879 | 39.96118 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 Charleston | South Carolina | United States | -79.93275 | 32.77632 Houston | Texas | United States | -95.36327 | 29.76328 Seattle | Washington | United States | -122.33207 | 47.60621
0
NCT00338455
[ 4 ]
28
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
This study evaluated the safety and efficacy of an increased frequency of octreotide acetate injections or an increase in dose in partially responsive acromegalic patients with persistently uncontrolled disease.
null
Acromegaly
acromegaly octreotide acetate partial responder patients
null
2
arm 1: Patients received octreotide 30 mg every 21 days intramuscularly (im) for 6 months, a total of 8 doses. At each study visit, octreotide was administered only after completion of all scheduled efficacy and safety evaluations for that visit. Octreotide was injected im into the right or left gluteal regions. The injections were initially administered by a trained and authorized member of the investigational team. When no study visit at the investigational site was required, the injections were given by a trained nurse or the family doctor. arm 2: Patients received octreotide 60 mg every 28 days intramuscularly (im) for 6 months, a total of 6 doses. Octreotide mg was administered as two 30 mg injections. At each study visit, octreotide was administered only after completion of all scheduled efficacy and safety evaluations for that visit. Octreotide was injected im into the right or left gluteal regions. The injections were initially administered by a trained and authorized member of the investigational team. When no study visit at the investigational site was required, the injections were given by a trained nurse or the family doctor.
[ 0, 0 ]
1
[ 0 ]
intervention 1: Each vial of study medication contained octreotide acetate 30 mg in a microencapsulated biodegradable polymer, poly (DL-lactide-co-glycolide) (D-(+)glucose), with 17% w/w mannitol in an approximate octreotide:polymer ratio of 1:20. The vehicle contained 0.5% sodium carboxymethylcellulose.
intervention 1: Octreotide acetate 30 mg suspension
1
Brescia | N/A | Italy | 10.21472 | 45.53558
0
NCT00372697
[ 2 ]
16
NON_RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
This is a clinical study to evaluate the safety and pharmacokinetics of an overseas determined maximum tolerated dose (MTD) of MK-0683 (vorinostat) in a Japanese patient population with solid tumors.
null
Tumors
null
2
arm 1: 600 mg daily (300 mg twice daily \[b.i.d.\]) for 3 consecutive days followed by 4 days of rest. arm 2: 400 mg once daily (400 mg q.d.) continuous daily dosing for 21 days.
[ 0, 0 ]
2
[ 0, 0 ]
intervention 1: 600 mg daily (300 mg twice daily \[b.i.d.\]) for 3 consecutive days followed by 4 days of rest. intervention 2: 400 mg once daily (400 mg q.d.) continuous daily dosing for 21 days.
intervention 1: Vorinostat intervention 2: Vorinostat
0
null
0
NCT00373490
[ 4 ]
300
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This study will examine the efficacy and safety of Xalacom comparing with those of Xalatan in Japanese patients with POAG or OH, in order to show superiority of Xalacom over Xalatan in efficacy and similarity of safety between Xalacom and Xalatan.
null
Glaucoma Ocular Hypertension
null
2
arm 1: None arm 2: None
[ 0, 0 ]
2
[ 0, 0 ]
intervention 1: latanoprost 0.005%, one rop once daily in evening intervention 2: latanoprost 0.005% adn timolol 0.5%, one drop, once daily
intervention 1: Xalatan intervention 2: Xalacom
54
Ichinomiya | Aichi-ken | Japan | 136.8 | 35.3 Ichinomiya | Aichi-ken | Japan | 136.8 | 35.3 Nagoya | Aichi-ken | Japan | 136.90641 | 35.18147 Nagoya | Aichi-ken | Japan | 136.90641 | 35.18147 Nagoya | Aichi-ken | Japan | 136.90641 | 35.18147 Narashino | Chiba | Japan | 140.04152 | 35.68184 Sapporo | Hokkaido | Japan | 141.35 | 43.06667 Sapporo | Hokkaido | Japan | 141.35 | 43.06667 Sapporo | Hokkaido | Japan | 141.35 | 43.06667 Sapporo | Hokkaido | Japan | 141.35 | 43.06667 Koube | Hyōgo | Japan | N/A | N/A Kanazawa | Ishikawa-ken | Japan | 136.61667 | 36.6 Fujisawa | Kanagawa | Japan | 139.47666 | 35.34926 Kamakura | Kanagawa | Japan | 139.54698 | 35.31085 Sagamihara | Kanagawa | Japan | 139.24167 | 35.56707 Yokohama | Kanagawa | Japan | 139.65 | 35.43333 Sapporo | Kokkaido | Japan | 141.35 | 43.06667 Uji | Kyoto | Japan | 135.80325 | 34.89044 Miyakonojō | Miyazaki | Japan | 131.06667 | 31.73333 Kumagaya | Saitama | Japan | 139.39004 | 36.13497 Tokorozawa | Saitama | Japan | 139.46903 | 35.79916 Fuji | Shizuoka | Japan | 138.68333 | 35.16667 Fuji | Shizuoka | Japan | 138.68333 | 35.16667 Mishima | Shizuoka | Japan | 138.91667 | 35.11667 Susono | Shizuoka | Japan | 138.90691 | 35.17388 Bunkyo-ku | Tokyo | Japan | N/A | N/A Chiyoda City | Tokyo | Japan | 139.75056 | 35.68449 Chiyoda-ku | Tokyo | Japan | N/A | N/A Hachiōji | Tokyo | Japan | 139.32389 | 35.65583 Hamura | Tokyo | Japan | 139.31952 | 35.76232 Katsushika-ku | Tokyo | Japan | 139.85 | 35.73333 Kiyose | Tokyo | Japan | 139.53014 | 35.77952 Koto-ku | Tokyo | Japan | N/A | N/A Meguro City | Tokyo | Japan | 139.70174 | 35.6322 Meguro-ku | Tokyo | Japan | N/A | N/A Musashino | Tokyo | Japan | 139.55944 | 35.70611 Musashino | Tokyo | Japan | 139.55944 | 35.70611 Ōta-ku | Tokyo | Japan | 139.71605 | 35.56126 Setagaya City | Tokyo | Japan | 139.64715 | 35.64188 Shinjuku | Tokyo | Japan | 139.70854 | 35.69115 Tachikawa | Tokyo | Japan | 139.41891 | 35.7091 Taito-ku | Tokyo | Japan | N/A | N/A Fujuoka | N/A | Japan | N/A | N/A Fukuoka | N/A | Japan | 130.41667 | 33.6 Fukuoka | N/A | Japan | 130.41667 | 33.6 Fukuoka | N/A | Japan | 130.41667 | 33.6 Gifu | N/A | Japan | 136.76039 | 35.42291 Hiroshima | N/A | Japan | 132.45 | 34.4 Niigata | N/A | Japan | 139.04125 | 37.92259 Osaka | N/A | Japan | 135.50107 | 34.69379 Osaka | N/A | Japan | 135.50107 | 34.69379 Osaka | N/A | Japan | 135.50107 | 34.69379 Shizuoka | N/A | Japan | 138.38333 | 34.98333 Sugita | N/A | Japan | 139.61713 | 35.37609
0
NCT00383019
[ 4 ]
173
RANDOMIZED
CROSSOVER
0TREATMENT
2DOUBLE
false
0ALL
false
This is a randomized, double-blind, placebo-controlled, crossover, two-attack, out-patient, early-intervention evaluation of subjects who have migraine with or without aura and who discontinued use of short acting triptan(s) within the past year due to non-response or intolerance. Subjects will treat 2 separate migraine attacks during the mild phase of each attack; one attack will be treated with one tablet of the Combination Product (sumatriptan succinate and naproxen sodium) and the other attack with one tablet of placebo (crossover design). \[Study 1 of 2\]
This is a randomized, double-blind, placebo-controlled, crossover, two-attack, out-patient, early-intervention evaluation of subjects who have migraine with or without aura and who discontinued use of short acting triptan(s) within the past year due to non-response or intolerance. Subjects will treat 2 separate migraine attacks during the mild phase of each attack; one attack will be treated with one tablet of the Combination Product (sumatriptan succinate and naproxen sodium) and the other attack with one tablet of placebo (crossover design); however, the order of these treatments will be randomized. A minimum 1-week washout period is required between study medication treatment of the first and second migraine attacks. Each subject will have two visits: (1) a Screening visit at study entry and (2) a Final visit 4-10 days after the second (or last) attack. A telephone contact will also be required 1-3 days after the first attack, and then once per month until the Final visit. The primary study objective is to assess efficacy as measured by sustained pain-free (SPF) relief of Combination Product compared to placebo in treating migraine subjects who have previously discontinued treatment with short acting triptans (rizatriptan, sumatriptan, almotriptan, zolmitriptan, and eletriptan).
Migraine Disorders
Combination Product naproxen sodium sumatriptan succinate short acting triptan migraine, acute Poor response
null
2
arm 1: Combination product (sumatriptan and naproxen sodium) \[Attack 1\] followed by placebo \[Attack 2\] arm 2: Placebo \[Attack 1\] followed by Combination Product (sumatriptan and naproxen sodium) \[Attack 2\]
[ 5, 5 ]
2
[ 0, 0 ]
intervention 1: Bilayer tablet containing 85mg sumatriptan (as 119mg sumatriptan succinate; fast disintegrating/rapid release formulation) active ingredient in one layer, and 500mg naproxen sodium active ingredient in the second layer. intervention 2: Matching placebo tablet.
intervention 1: Combination Product (sumatriptan succinate / naproxen sodium) intervention 2: Placebo
26
Fresno | California | United States | -119.77237 | 36.74773 Newport Beach | California | United States | -117.92895 | 33.61891 Santa Monica | California | United States | -118.49138 | 34.01949 Walnut Creek | California | United States | -122.06496 | 37.90631 Fairfield | Connecticut | United States | -73.26373 | 41.14121 Stamford | Connecticut | United States | -73.53873 | 41.05343 Aventura | Florida | United States | -80.13921 | 25.95648 Tallahassee | Florida | United States | -84.28073 | 30.43826 Tampa | Florida | United States | -82.45843 | 27.94752 Chicago | Illinois | United States | -87.65005 | 41.85003 Northbrook | Illinois | United States | -87.82895 | 42.12753 South Bend | Indiana | United States | -86.25001 | 41.68338 Lenexa | Kansas | United States | -94.73357 | 38.95362 St Louis | Missouri | United States | -90.19789 | 38.62727 Omaha | Nebraska | United States | -95.94043 | 41.25626 Orchard Park | New York | United States | -78.74392 | 42.76756 Greensboro | North Carolina | United States | -79.79198 | 36.07264 Matthews | North Carolina | United States | -80.72368 | 35.11681 Raleigh | North Carolina | United States | -78.63861 | 35.7721 Tabor City | North Carolina | United States | -78.87669 | 34.14878 Fargo | North Dakota | United States | -96.7898 | 46.87719 Dallas | Texas | United States | -96.80667 | 32.78306 Houston | Texas | United States | -95.36327 | 29.76328 Alexandria | Virginia | United States | -77.04692 | 38.80484 Roanoke | Virginia | United States | -79.94143 | 37.27097 Virginia Beach | Virginia | United States | -75.97799 | 36.85293
0
NCT00383162
[ 5 ]
50
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
false
Both morphine and hydromorphone are pain medications commonly used after surgery. It is thought at the institution that hydromorphone causes less side effects but this has not been studied. The study proposes to treat the patients with either morphine or hydromorphone and determine how much nausea, vomiting, and itching they have with each drug
null
Post Operative Pain
pain morphine hydromorphone nausea vomiting pruritis
null
2
arm 1: Patients receive morphine 1mg/dose PCA for postsurgical pain; max 10 mg/hr; lockout 6 minutes. arm 2: Patients receive hydromorphone 0.2mg/dose PCA for postsurgical pain; max 10mg/hr; lockout 6 minutes.
[ 1, 1 ]
2
[ 0, 0 ]
intervention 1: Morphine 1mg/mL, dose 1mL, lockout 6 minutes, max 10mL intervention 2: hydromorphone PCA 0.2mg/lml, dose 1ml, lockout 6min, max 10ml
intervention 1: Morphine PCA intervention 2: Hydromorphone PCA
1
New York | New York | United States | -74.00597 | 40.71427
0
NCT00385541
[ 3 ]
56
RANDOMIZED
CROSSOVER
0TREATMENT
4QUADRUPLE
false
0ALL
true
This is a multicenter, randomized, double-blind, cross-over study to compare the pharmacodynamic response in subjects with Acute Coronary Syndrome receiving a 10-mg maintenance dose (MD) of prasugrel compared with a 150-mg maintenance dose of clopidogrel, following a 900-mg loading dose (LD) of clopidogrel.
null
Acute Coronary Syndrome
null
2
arm 1: Open label lead-in one time dose of Clopidogrel 900 mg oral tablets (a single or cumulative dose) and 250 mg to 500 mg aspirin loading dose (LD), either orally or intravenously. Patients are then assigned to maintenance dose (MD) prasugrel 10 mg and two placebo tablets, matched to clopidogrel, and 100 mg aspirin, all taken orally once a day for 14 days. Patients cross-over to MD of clopidogrel two 75 mg tablets and one placebo matched to prasugrel and 100 mg aspirin, all taken orally once a day for the next 14 days. arm 2: Open label lead-in one time dose of Clopidogrel 900 mg oral tablets (a single or cumulative dose) and 250 mg to 500 mg aspirin loading dose (LD), either orally or intravenously. Patients are then assigned to maintenance dose (MD) Clopidogrel two 75 mg and one placebo tablet, matched to prasugrel, and 100 mg aspirin, all taken orally once a day for 14 days. Patients cross-over to MD of prasugrel one 10 mg tablet and two placebo tablets matched to clopidogrel and 100 mg aspirin, all taken orally once a day for the next 14 days.
[ 0, 1 ]
2
[ 0, 0 ]
intervention 1: Prasugrel 10-mg tablet taken orally as a daily maintenance dose for a 14-day treatment period. intervention 2: Clopidogrel two 75-mg tablets taken orally as a daily maintenance dose for a 14-day treatment period.
intervention 1: Prasugrel intervention 2: Clopidogrel
2
Créteil | N/A | France | 2.46569 | 48.79266 Paris | N/A | France | 2.3488 | 48.85341
0
NCT00385944
[ 4 ]
218
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
The primary purpose of the study is to evaluate the impact of treatment with fentanyl buccal tablets on the anxiety symptoms commonly associated with chronic pain in patients with breakthrough pain (BTP). Other purposes are to assess the management of BTP, to evaluate patient functioning, and to determine any influences on the successful dose achieved.
null
Pain Chronic Pain Breakthrough Pain
null
1
arm 1: Successful dose strength for each participant was determined during a titration period of no more than 10 days. Participants used the successful dose of 100, 200, 400, 600, or 800 mcg during the four week open-label treatment period.
[ 0 ]
1
[ 0 ]
intervention 1: Fentanyl buccal tablets (FBT) placed in the buccal cavity above a rear molar until disintegrated, approximately 14-25 minutes. Dose titration: Participants self-administered FBT, starting at 100, 200 or 400 mcg (depending on analgesic used pre-study) and titrated to 600 and 800 mcg as needed. For each breakthrough pain (BTP) episode, participants took a dose, and did not take further study drug if adequate pain relief was achieved. If pain was not controlled within 30 minutes, the same dose level was repeated. If pain relief was inadequate 30 minutes after the second dose, usual rescue medication was taken for that BTP episode. Doses were adjusted until pain relief was adequate and side effects were tolerated. Open-label: Once the successful dose of FBT was identified (100, 200, 400, 600, or 800 mcg), participants were dispensed a 4-week supply at the successful dose. Participants were not to use FBT for more than 6 BTP episodes or take more than 8 FBT daily.
intervention 1: Fentanyl Buccal Tablets
33
Birmingham | Alabama | United States | -86.80249 | 33.52066 Phoenix | Arizona | United States | -112.07404 | 33.44838 Anaheim | California | United States | -117.9145 | 33.83529 Bakersfield | California | United States | -119.01871 | 35.37329 Beverly Hills | California | United States | -118.40036 | 34.07362 National City | California | United States | -117.0992 | 32.67811 Santa Monica | California | United States | -118.49138 | 34.01949 Westminster | Colorado | United States | -105.0372 | 39.83665 New Haven | Connecticut | United States | -72.92816 | 41.30815 Clearwater | Florida | United States | -82.8001 | 27.96585 Largo | Florida | United States | -82.78842 | 27.90979 Sarasota | Florida | United States | -82.53065 | 27.33643 Tampa | Florida | United States | -82.45843 | 27.94752 Dawsonville | Georgia | United States | -84.11908 | 34.42121 Bloomington | Illinois | United States | -88.99369 | 40.4842 Chicago | Illinois | United States | -87.65005 | 41.85003 Elkhart | Indiana | United States | -85.97667 | 41.68199 Indianapolis | Indiana | United States | -86.15804 | 39.76838 Valparaiso | Indiana | United States | -87.06114 | 41.47309 Overland Park | Kansas | United States | -94.67079 | 38.98223 Shreveport | Louisiana | United States | -93.75018 | 32.52515 Missoula | Montana | United States | -113.994 | 46.87215 Henderson | Nevada | United States | -114.98194 | 36.0397 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Stony Brook | New York | United States | -73.14094 | 40.92565 Eugene | Oregon | United States | -123.08675 | 44.05207 Altoona | Pennsylvania | United States | -78.39474 | 40.51868 Duncansville | Pennsylvania | United States | -78.4339 | 40.42341 Greer | South Carolina | United States | -82.22706 | 34.93873 Hendersonville | Tennessee | United States | -86.62 | 36.30477 Richardson | Texas | United States | -96.72972 | 32.94818 Richardson | Texas | United States | -96.72972 | 32.94818 Salt Lake City | Utah | United States | -111.89105 | 40.76078
0
NCT00387010
[ 3 ]
1
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
null
RATIONALE: Everolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. PURPOSE: This phase II trial is studying how well everolimus works in treating patients with advanced or metastatic colorectal cancer that did not respond to previous therapy.
OBJECTIVES: * Determine response rate, time to tumor progression, and survival of patients with advanced or metastatic refractory colorectal cancer and mutations in the PI3K gene treated with everolimus. * Determine the toxicity profile of this drug in these patients. * Measure the signaling pathways activated in these patients. * Determine the pharmacodynamic effects of this drug in these patients. OUTLINE: This is an open-label study. Patients receive oral everolimus once daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo tumor biopsies and normal skin biopsies at baseline and after the first course of study treatment. Tumor tissue is examined for biological markers (e.g., epidermal growth factor receptor, ERK, Akt, p70s6k, p27, and Rb protein) by immunohistochemistry; apoptosis quantification by TUNEL assay; Ki-67 quantification and Ki-index; gene expression; and c-fos and p27 expression by reverse-transcriptase polymerase chain reaction. PROJECTED ACCRUAL: A total of 37 patients will be accrued for this study.
Colorectal Cancer
adenocarcinoma of the colon adenocarcinoma of the rectum recurrent colon cancer stage III colon cancer stage IV colon cancer recurrent rectal cancer stage III rectal cancer stage IV rectal cancer
null
0
null
null
10
[ 0, 3, 3, 3, 3, 3, 3, 3, 3, 3 ]
intervention 1: None intervention 2: None intervention 3: None intervention 4: None intervention 5: None intervention 6: None intervention 7: None intervention 8: None intervention 9: None intervention 10: None
intervention 1: everolimus intervention 2: antiangiogenesis therapy intervention 3: biopsy intervention 4: diagnostic procedure intervention 5: gene expression analysis intervention 6: immunohistochemistry staining method intervention 7: laboratory biomarker analysis intervention 8: mutation analysis intervention 9: protein tyrosine kinase inhibitor therapy intervention 10: reverse transcriptase-polymerase chain reaction
1
Baltimore | Maryland | United States | -76.61219 | 39.29038
0
NCT00390364
[ 4 ]
83
RANDOMIZED
CROSSOVER
2DIAGNOSTIC
1SINGLE
false
0ALL
false
The purpose of this study is to determine if the contrast agent is effective and safe in the Magnetic Resonance Imaging (MRI) of vascular diseases in patients of Chinese origin.
The study has previously been posted by Schering AG, Germany. Schering AG, Germany has been renamed to Bayer HealthCare AG, Germany. Bayer HealthCare AG, Germany is the sponsor of the trial.
Vascular Diseases
Gadovist Gadavist MRI Imaging vascular diseases Chinese
null
2
arm 1: Period 1: Participant received Gadobutrol 1.0 M (iv: intravenous injection), at a dose of 0.2 mL/kg BW, up to 0.3 mL/kg BW if 3 Fields of View (FOVs) to be imaged; Period 2: Participant received Gadopentate 0.5 M (iv), at a dose of 0.4 mL/kg BW, up to 0.6 mL/kg BW if 3 FOVs to be imaged arm 2: Period 1: Participant received Gadopentate 0.5 M (iv), at a dose of 0.4 mL/kg BW, up to 0.6 mL/kg BW if 3 FOVs to be imaged; Period 2: Participant received Gadobutrol 1.0 M (iv: intravenous injection), at a dose of 0.2 mL/kg BW, up to 0.3 mL/kg BW if 3 Fields of View (FOVs) to be imaged
[ 0, 0 ]
2
[ 0, 0 ]
intervention 1: Participant received a single intravenous injection with Gadobutrol (1.0 M) at a volume of 0.2 mL/kg BW (dose = 0.2 mmol/kg BW) or up to 0.3 mL/kg BW when 3 Fields of View were imaged (up to dose = 0.3 mmol/kg BW) intervention 2: Participant received a single intravenous injection with Gadopentate (0.5 M) at a volume of 0.4 mL/kg BW (dose = 0.2 mmol/kg BW) or up to 0.6 mL/kg BW when 3 Fields of View were imaged (up to dose = 0.3 mmol/kg BW)
intervention 1: Gadobutrol (Gadavist, Gadovist, BAY86-4875) intervention 2: Gadopentate dimeglumine (Magnevist, BAY86-4882)
3
Chengdu | Sichuan | China | 104.06667 | 30.66667 Beijing | N/A | China | 116.39723 | 39.9075 Shanghai | N/A | China | 121.45806 | 31.22222
0
NCT00395733
[ 3 ]
50
RANDOMIZED
CROSSOVER
0TREATMENT
2DOUBLE
false
0ALL
false
The study was designed to obtain data about the efficacy and safety of 3 doses of indacaterol (150, 300, and 600 µg) in Japanese patients with chronic obstructive pulmonary disease (COPD) so that optimal dose(s) could be chosen for testing in later studies.
null
Chronic Obstructive Pulmonary Disease
chronic obstructive pulmonary disease COPD QAB149 indacaterol long acting β2-agonist
null
4
arm 1: In treatment period, 1 patients received 2 placebo capsules; in treatment period 2, patients received 1 indacaterol 150 μg capsule + 1 placebo capsule; in treatment period 3, patients received 1 indacaterol 300 μg capsule + 1 placebo capsule; and in treatment period 4, patients received 2 indacaterol 300 μg capsules. There was a washout period of 14-28 days between each treatment period. Patients received each treatment only once. The short-acting β2-agonist salbutamol was available for rescue use throughout the study. arm 2: In treatment period 1, patients received 1 indacaterol 150 μg capsule + 1 placebo capsule; in treatment period 2, patients received 2 indacaterol 300 μg capsules; in treatment period 3, patients received 2 placebo capsules; and in treatment period 4, patients received 1 indacaterol 300 μg capsule + 1 placebo capsule. There was a washout period of 14-28 days between each treatment period. Patients received each treatment only once. The short-acting β2-agonist salbutamol was available for rescue use throughout the study. arm 3: In treatment period 1, patients received 1 indacaterol 300 μg capsule + 1 placebo capsule; in treatment period 2, patients received 2 placebo capsules; in treatment period 3, patients received 2 indacaterol 300 μg capsules; and in treatment period 4, patients received 1 indacaterol 150 μg capsule + 1 placebo capsule. There was a washout period of 14-28 days between each treatment period. Patients received each treatment only once. The short-acting β2-agonist salbutamol was available for rescue use throughout the study. arm 4: In treatment period 1, patients received 2 indacaterol 300 μg capsules; in treatment period 2, patients received 1 indacaterol 300 μg capsule + 1 placebo capsule; in treatment period 3, patients received 1 indacaterol 150 μg capsule + 1 placebo capsule; and in treatment period 4 patients received 2 placebo capsules. There was a washout period of 14-28 days between each treatment period. Patients received each treatment only once. The short-acting β2-agonist salbutamol was available for rescue use throughout the study.
[ 0, 0, 0, 0 ]
3
[ 0, 0, 0 ]
intervention 1: Indacaterol 150 μg was provided in powder filled capsules with a single dose dry powder inhaler (SDDPI). intervention 2: Indacaterol 300 μg was provided in powder filled capsules with a single dose dry powder inhaler (SDDPI). intervention 3: Placebo to indacaterol was provided in powder filled capsules with a single dose dry powder inhaler (SDDPI).
intervention 1: Indacaterol 150 μg intervention 2: Indacaterol 300 μg intervention 3: Placebo
8
Kasukabe | N/A | Japan | 139.74966 | 35.98308 Kishiwada | N/A | Japan | 135.36667 | 34.46667 Kurume | N/A | Japan | 130.51667 | 33.31667 Obihiro | N/A | Japan | 143.20444 | 42.91722 Sagamihara | N/A | Japan | 139.24167 | 35.56707 Sapporo | N/A | Japan | 141.35 | 43.06667 Tokyo | N/A | Japan | 139.69171 | 35.6895 Wakayama | N/A | Japan | 135.16667 | 34.23333
0
NCT00403845
[ 5 ]
278
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This 16 week study will examine the ability of olmesartan medoxomil to lower the blood pressure of patients with moderate to severe high blood pressure in comparison to placebo. The medication being tested has been approved by the FDA for the treatment of high blood pressure.
This study was to randomize an equal number of participants to either an olmesartan medoxomil based treatment or to placebo. The titration scheme was as follows: * First 3 weeks (wks), all participants - olmesartan medoxomil 20 mg or placebo * Next 3 wks, participants whose blood pressure was not controlled - olmesartan medoxomil 40 mg or placebo * Next 3 wks, participants whose blood pressure was not controlled - olmesartan medoxomil 40 mg/HCT 12.5 mg or placebo * Final 3 wks, participants whose blood pressure was not controlled - olmesartan medoxomil 40 mg/HCT 25 mg or placebo * Subjects with a mean BP of \<120/80 mmHg at any visit were considered responders and were not titrated up to the next dose level. However, they remained in the study at their currently assigned dose of study medication. * Subjects with a mean office SBP ≥120 mmHg or a mean office DBP ≥80 mmHg at any subsequent visit(s) were considered 'uncontrolled' and were titrated to the next dose level according to the titration scheme above. * Subjects who reach the highest dose (ie, olmesartan medoxomil 40 mg/HCT 25 mg) remained on that dose until study exit at Visit 8, unless safety concerns caused discontinuation of treatment.
Hypertension
Hypertension Angiotensin Receptor Blocker Calcium Channel Blocker Angiotensin Converting Enzyme Inhibitor Hydrochlorothiazide Stage I and II Hypertension
null
2
arm 1: Olmesartan medoxomil, plus hydrochlorothiazide, if necessary arm 2: Placebo tablets were taken once daily for 12 weeks
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: olmesartan medoxomil + hydrochlorothiazide, if necessary. Oral tablets administered for once daily for 12 weeks intervention 2: Oral tablets administered for once daily for 12 weeks
intervention 1: olmesartan medoxomil + hydrochlorothiazide, if necessary intervention 2: Placebo
29
Birmingham | Alabama | United States | -86.80249 | 33.52066 Mobile | Alabama | United States | -88.04305 | 30.69436 Muscle Shoals | Alabama | United States | -87.66753 | 34.74481 Searcy | Arkansas | United States | -91.73625 | 35.25064 Carmichael | California | United States | -121.32828 | 38.61713 Spring Valley | California | United States | -116.99892 | 32.74477 Coral Gables | Florida | United States | -80.26838 | 25.72149 Deerfield Beach | Florida | United States | -80.09977 | 26.31841 DeLand | Florida | United States | -81.30312 | 29.02832 Fort Lauderdale | Florida | United States | -80.14338 | 26.12231 Hialeah | Florida | United States | -80.27811 | 25.8576 Hollywood | Florida | United States | -80.14949 | 26.0112 Pembroke Pines | Florida | United States | -80.22394 | 26.00315 Chicago | Illinois | United States | -87.65005 | 41.85003 Elizabeth | New Jersey | United States | -74.2107 | 40.66399 Winston-Salem | North Carolina | United States | -80.24422 | 36.09986 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Oklahoma City | Oklahoma | United States | -97.51643 | 35.46756 Eugene | Oregon | United States | -123.08675 | 44.05207 Downingtown | Pennsylvania | United States | -75.70327 | 40.0065 Charleston | South Carolina | United States | -79.93275 | 32.77632 Nashville | Tennessee | United States | -86.78444 | 36.16589 New Tazewell | Tennessee | United States | -83.59963 | 36.44258 Colleyville | Texas | United States | -97.15501 | 32.88096 San Antonio | Texas | United States | -98.49363 | 29.42412 Murray | Utah | United States | -111.88799 | 40.66689 Manassas | Virginia | United States | -77.47527 | 38.75095 Olympia | Washington | United States | -122.90169 | 47.04491 Madison | Wisconsin | United States | -89.40123 | 43.07305
0
NCT00430638
[ 5 ]
266
RANDOMIZED
PARALLEL
0TREATMENT
1SINGLE
false
0ALL
null
Patients with glaucoma or ocular hypertension currently being treated with latanoprost 0.005%, and in need of additional IOP lowering, will be randomized to receive either bimatoprost 0.03% or travoprost 0.004% in place of latanoprost 0.005%
null
Glaucoma Ocular Hypertension
null
2
arm 1: None arm 2: None
[ 0, 1 ]
2
[ 0, 0 ]
intervention 1: bimatoprost 0.03% 1 drop nightly for 3 months intervention 2: travoprost 0.004% 1 drop nightly for 3 months
intervention 1: bimatoprost 0.03% eye drops intervention 2: travoprost 0.004% eye drops
1
San Diego | California | United States | -117.16472 | 32.71571
0
NCT00440011
[ 5 ]
191
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
The purpose of this study is to assess real-world effectiveness of montelukast in children (2 to 14 years) with asthma and allergic rhinitis.
null
Asthma Allergic Rhinitis
null
2
arm 1: Participants were treated for 12 months after randomization: Participants 2 to 5 years of age took one 4 mg chewable tablet and 6 to 14 years of age took one 5 mg chewable tablet daily in the evening. If participants had exacerbated from mild to moderate within 12 weeks, inhaled corticosteroids (ICS) was added to Montelukast and ICS, respectively and those participants were excluded in the efficacy evaluation at 12 weeks. arm 2: Participants were treated for 12 months after randomization: Each participant's physician selected the ICS agent, dose, and regimen. If participants had exacerbated from mild to moderate within 12 weeks, ICS was added to Montelukast and ICS, respectively and those participants were excluded in the efficacy evaluation at 12 weeks.
[ 0, 1 ]
2
[ 0, 0 ]
intervention 1: Montelukast 4/5 mg tablet (oral chewable), once daily, 12 weeks to up to 12 months intervention 2: Inhaled corticosteroid solution, 1-4 puffs daily, 12 weeks to up to 12 months
intervention 1: montelukast sodium intervention 2: inhaled corticosteroid
0
null
0
NCT00442559
[ 2 ]
43
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
The purpose of this research study is to determine how safe and effective subconjunctival injections of CGC-11047 are in subjects with wet age related macular degeneration at two different dosing intervals.
The purpose of this research study is to determine how safe and effective subconjunctival injections of CGC-11047 are in subjects with wet age related macular degeneration at two different dosing intervals. Half of the participants in the study will receive CGC-11047 every two weeks and half of the participants in the study will receive CGC-11047 every four weeks. If your doctor determines that you are eligible for the study based on his/her judgement and according to the entry requirements set by the sponsor of the research, you will be randomly assigned (by chance, like the flip of a coin), to receive CGC-11047 every two weeks or every four weeks. Whether you receive drug every two weeks or every four weeks, you will still need to come to the clinic approximately every two weeks for the first three months, and approximately four times after that until it has been at least 12 months since you received your first treatment of study drug. Two studies (this one and another one) are occurring to test CGC-11047 in patients with wet age-related macular degeneration for the first time. However, this drug (CGC-11047) is being studied in cancer patients at doses much higher than will be given to any subjects in either of these studies. The cancer patients have tolerated the drug well with the exception of one cancer patient who had two reactions to the drug (pancreatitis and hypotension). However, this patient had advanced cancer (non-Hodgkin's lymphoma with a life expectancy of less than 3 months) and received 38X the dose to be administered in this AMD study. Aside from this one patient, there have not been any serious side effects related to the drug. This study will involve about 100 subjects at about 15 different sites internationally. The study will take place over 12 months and will include about 12 office visits to the study doctor.
Age Related Macular Degeneration
null
2
arm 1: 16.5 mg CGC-11047 as a subconjunctival injection once every two weeks. arm 2: 16.5 mg CGC-11047 as a subconjunctival injection once every four weeks.
[ 0, 0 ]
1
[ 0 ]
intervention 1: 16.5 mg (3.3%) subconjunctival injection
intervention 1: CGC-11047
8
Baltimore | Maryland | United States | -76.61219 | 39.29038 Mexico City | District Federal | Mexico | -99.12766 | 19.42847 Cheboksary | N/A | Russia | 47.25194 | 56.13222 Moscow | N/A | Russia | 37.61556 | 55.75222 Rostov-on-Don | N/A | Russia | 39.72328 | 47.23135 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863
0
NCT00446654
[ 4 ]
60
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
true
2MALE
false
A research study to determine if acetaminophen (APAP) given intravenously (IV-a liquid given through a needle into a vein in your arm) is safe and effective in controlling fever when compared to placebo. Acetaminophen given this way is the investigational part of this study.
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Single-Dose Study of the Efficacy and Safety of Intravenous Acetaminophen (IVAPAP) Versus Placebo for the Treatment of Endotoxin-Induced Fever in Healthy Adult Males
Fever
null
2
arm 1: 1 g of acetaminophen in 100 mL of intravenous solution arm 2: 100 mL of intravenous placebo solution
[ 0, 2 ]
3
[ 0, 0, 2 ]
intervention 1: IV Placebo intervention 2: Intravenous acetaminophen solution 1 g / 100 ml intervention 3: Administration of Reference Standard Endotoxin (RSE) to induce fever. Applicable to both study arms: Administration of a 1 ng/kg body weight test dose of RSE to test for fever response. Observation period of at least 60 minutes to ensure no exaggerated systemic responses, followed by administration of a 4 ng/kg of RSE to induce fever.
intervention 1: IV Placebo intervention 2: IV Acetaminophen intervention 3: Reference Standard Endotoxin (RSE)
1
Austin | Texas | United States | -97.74306 | 30.26715
0
NCT00493311
[ 4 ]
185
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
true
0ALL
false
This study is a multicenter, randomized, double-blind, placebo-controlled, parallel group, dose-comparison to determine the efficacy and safety of a standard-dose of colchicine (4.8 mg) versus low-dose colchicine (1.8 mg) or placebo for acute gout flares.
This study is a multi-center, randomized, double-blind, placebo-controlled, parallel group trial to compare the efficacy and safety of standard-dose colchicine (STD)(total dose = 4.8 mg) versus low-dose colchicine (total dose 1.8 mg) or placebo for the treatment of acute gout flares. Eight hundred and thirteen patients with a confirmed diagnosis of gout were screened. 238 of the screened patients failed screening; 235(98.7%) failed because they did not meet inclusion/exclusion criteria. The 575 eligible patients were randomly assigned (1:1:1) to one of three treatment groups . At the randomization visit the investigator dispensed a blister card containing eight identical looking capsules (in a combination of active drug and placebo capsules) in a double blind fashion for use during their next gout flare. Patients were instructed to self-initiate treatment with the study medication within 12 hours of a gout flare onset. Gout flares were determined by calling a Gout Flare Call Center established for this purpose. At Investigator discretion, rescue medication could also be provided, but patients were encouraged not to use rescue medication within the first 24 hours after starting treatment with study drug. Of the 575 study participants, 185 had a qualifying gout flare and 390 did not. Patients used a diary to record study drug administration, pain score, the presence or absence of gastrointestinal adverse events (nausea, vomiting, diarrhea, and abdominal pain) and the timing of any rescue medication use prior to beginning treatment and 1, 2, 3, 4, 5, 6, 7, 8, 16, 24, 32, 40, 48, 56, 64, and 72 hours after the start of dosing. The pain score was based on a scale of 1 - 10 where 1 was no pain and 10 was the worst pain imaginable. Efficacy was defined as a 50% reduction in pain score in the target joint at 24 hours in patients who did not use rescue medicine. The primary efficacy analysis was to be based on an Intent-to-Treat (ITT) population, defined as all patients who were randomized, contacted the Call Center, and were instructed to begin taking study drug. An otherwise qualified patient was excluded from the ITT population only if the patient returned a study drug blister pack completely unused. Secondary outcome measures compared the efficacy of STD dose colchicine to a low dose regimen and placebo using the same criteria for efficacy as for the primary outcome measure. Additional secondary outcome measures were time to 50% and 90% reduction in pain in the target joint analyzed by treatment group using Kaplan-Meier methods, and the change in mean pain intensity from 0 to 72 hours plotted by time point for each treatment group. All safety analyses were carried out using the safety population defined as all patients who received at least one dose of study medication regardless of authorization by the Call Center To determine the safety of colchicine when administered via two different dose regimens all patients who had a gout flare were seen by the investigator as soon as possible after onset and evaluated until the flare and any adverse events resolved. All adverse effects, whether recorded by the patient in the diary or obtained by systematic evaluation by the investigator were recorded and reported in tabular form. Treatment-emergent adverse events (TEAE) were summarized by MedDRA System Organ Class and preferred terms and tabulated according treatment arm, overall incidence, severity and relationship to study medication. Multiple events within a patient were counted once and at greatest severity and closest relationship to study medication.
Gout
null
3
arm 1: After confirmation of a gout flare, patients were to begin standard dosing of colchicine 4.8mg (two capsules (1.8mg) initially followed by additional one capsule doses (0.6mg) every hour for an additional 6 doses). arm 2: Within 12 hours of a confirmed gout flare, patients were to begin the low dose colchicine regimen consisting of a total dose of 1.8 mg - two colchicine capsules initially (1.2 mg)followed an hour later by a single additional capsule of active drug(0.6 mg)then by 5 additional hourly doses of an identical looking placebo capsules arm 3: None
[ 0, 0, 2 ]
3
[ 0, 0, 10 ]
intervention 1: At randomization, patients were given an identical looking blister pack containing (8) over encapsulated colchicine 0.6 mg tablets identical in appearance to placebo capsules. Patients were instructed to take 2 capsules initially (1.2 mg) followed by an additional capsule (0.6 mg) every hour for a total of six additional doses (total colchicine dose 4.8 mg) beginning within 12 hours of onset of a qualifying gout flare as confirmed by calling the gout flare call center. intervention 2: At randomization, patients were given an identical looking blister pack containing (3) over encapsulated colchicine 0.6 mg tablets identical in appearance to placebo capsules and five placebo capsules. Patients were instructed to take 2 capsules initially (0.6 mg x 2) followed by an additional capsule every hour for a total of six additional doses (one active (0.6 mg) and 5 placebo capsules), a total colchicine dose = 1.8 mg) beginning within 12 hours of onset of a qualifying gout flare as confirmed by calling the gout flare call center intervention 3: At randomization, patients were given an identical looking blister pack containing (8) placebo capsules identical in appearance to the study drug. Patients were instructed to take 2 capsules initially followed by an additional capsule every hour for a total of six additional doses beginning within 12 hours of onset of a qualifying gout flare as confirmed by calling the gout flare call center.
intervention 1: High Dose Colchicine (4.8 mg total dose) intervention 2: Low Dose Colchicine (1.8mg total dose) intervention 3: Placebo Control
83
Birmingham | Alabama | United States | -86.80249 | 33.52066 Birmingham | Alabama | United States | -86.80249 | 33.52066 Columbiana | Alabama | United States | -86.60721 | 33.17817 Huntsville | Alabama | United States | -86.58594 | 34.7304 Tucson | Arizona | United States | -110.92648 | 32.22174 Tucson | Arizona | United States | -110.92648 | 32.22174 Jonesboro | Arkansas | United States | -90.70428 | 35.8423 Little Rock | Arkansas | United States | -92.28959 | 34.74648 Irvine | California | United States | -117.82311 | 33.66946 La Jolla | California | United States | -117.2742 | 32.84727 Paramount | California | United States | -118.15979 | 33.88946 Rancho Cucamonga | California | United States | -117.59311 | 34.1064 San Diego | California | United States | -117.16472 | 32.71571 West Covina | California | United States | -117.93895 | 34.06862 Clearwater | Florida | United States | -82.8001 | 27.96585 Crystal River | Florida | United States | -82.5926 | 28.90248 Gainesville | Florida | United States | -82.32483 | 29.65163 Green Cove Springs | Florida | United States | -81.67815 | 29.99191 Jacksonville | Florida | United States | -81.65565 | 30.33218 Jupiter | Florida | United States | -80.09421 | 26.93422 Lake Mary | Florida | United States | -81.31784 | 28.75888 Lake Worth | Florida | United States | -80.07231 | 26.61708 Orange City | Florida | United States | -81.29867 | 28.94888 Ormond Beach | Florida | United States | -81.05589 | 29.28581 Port Orange | Florida | United States | -80.99561 | 29.13832 Tampa | Florida | United States | -82.45843 | 27.94752 Vero Beach | Florida | United States | -80.39727 | 27.63864 Winter Haven | Florida | United States | -81.73286 | 28.02224 Calhoun | Georgia | United States | -84.95105 | 34.50259 Decatur | Georgia | United States | -84.29631 | 33.77483 Lawrenceville | Georgia | United States | -83.98796 | 33.95621 Tifton | Georgia | United States | -83.5085 | 31.45046 Boise | Idaho | United States | -116.20345 | 43.6135 Meridian | Idaho | United States | -116.39151 | 43.61211 Libertyville | Illinois | United States | -87.95313 | 42.28308 Moline | Illinois | United States | -90.51513 | 41.5067 Cedar Rapids | Iowa | United States | -91.64407 | 42.00833 Elizabethtown | Kentucky | United States | -85.85913 | 37.69395 Louisville | Kentucky | United States | -85.75941 | 38.25424 Baton Rouge | Louisiana | United States | -91.18747 | 30.44332 Frederick | Maryland | United States | -77.41054 | 39.41427 Rockville | Maryland | United States | -77.15276 | 39.084 Wheaton | Maryland | United States | -77.05526 | 39.03983 Springfield | Massachusetts | United States | -72.58981 | 42.10148 Worcester | Massachusetts | United States | -71.80229 | 42.26259 Lansing | Michigan | United States | -84.55553 | 42.73253 Hattiesburg | Mississippi | United States | -89.29034 | 31.32712 Florissant | Missouri | United States | -90.32261 | 38.78922 St Louis | Missouri | United States | -90.19789 | 38.62727 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Reno | Nevada | United States | -119.8138 | 39.52963 Manalapan | New Jersey | United States | -74.39571 | 40.25733 Medford | New Jersey | United States | -74.8235 | 39.90095 Voorhees Township | New Jersey | United States | -74.49062 | 40.4795 Albany | New York | United States | -73.75623 | 42.65258 Brewster | New York | United States | -73.61707 | 41.39732 New York | New York | United States | -74.00597 | 40.71427 Rochester | New York | United States | -77.61556 | 43.15478 Syracuse | New York | United States | -76.14742 | 43.04812 Williamsville | New York | United States | -78.73781 | 42.96395 Belmont | North Carolina | United States | -81.0373 | 35.24292 Charlotte | North Carolina | United States | -80.84313 | 35.22709 Dayton | Ohio | United States | -84.19161 | 39.75895 Mayfield Village | Ohio | United States | N/A | N/A Middleburg Heights | Ohio | United States | -81.81291 | 41.36144 Duncansville | Pennsylvania | United States | -78.4339 | 40.42341 Harleysville | Pennsylvania | United States | -75.38712 | 40.27955 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 Orangeburg | South Carolina | United States | -80.85565 | 33.49182 Milan | Tennessee | United States | -88.75895 | 35.91979 New Tazewell | Tennessee | United States | -83.59963 | 36.44258 Arlington | Texas | United States | -97.10807 | 32.73569 Austin | Texas | United States | -97.74306 | 30.26715 Carrollton | Texas | United States | -96.89028 | 32.95373 Fort Worth | Texas | United States | -97.32085 | 32.72541 Houston | Texas | United States | -95.36327 | 29.76328 Irving | Texas | United States | -96.94889 | 32.81402 San Antonio | Texas | United States | -98.49363 | 29.42412 Sugarland | Texas | United States | N/A | N/A Ettrick | Virginia | United States | -77.42998 | 37.24015 Portsmouth | Virginia | United States | -76.29827 | 36.83543 Reston | Virginia | United States | -77.3411 | 38.96872 Suffolk | Virginia | United States | -76.58496 | 36.72836
0
NCT00506883
[ 4 ]
105
RANDOMIZED
PARALLEL
0TREATMENT
3TRIPLE
true
2MALE
false
To assess the rapidity of onset of antipyretic effect and the efficacy and safety of a single dose of IV acetaminophen (IV APAP) versus oral (PO) acetaminophen in the treatment of fever induced by a standard dose of endotoxin
A Phase III, Randomized, Double-Blind, Double-Dummy, Single-Dose Study of the Efficacy and Safety of Intravenous Acetaminophen Versus Oral Acetaminophen for the Treatment of Endotoxin-Induced Fever in Healthy Adult Males
Fever
null
2
arm 1: Administration of a 1 ng/kg body weight test dose of RSE to test for fever response. Observation period of at least 60 minutes to ensure no exaggerated systemic responses, followed by administration of a 4 ng/kg of RSE to induce fever. Randomization to receive 1 g of acetaminophen in 100 ml of intravenous solution and oral placebo. arm 2: Administration of a 1 ng/kg body weight test dose of RSE to test for fever response. Observation period of at least 60 minutes to ensure no exaggerated systemic responses, followed by administration of a 4 ng/kg of RSE to induce fever. Randomization to receive oral acetaminophen 1 g plus 100 ml of intravenous placebo solution.
[ 0, 1 ]
3
[ 0, 0, 2 ]
intervention 1: Single dose of 1 gm IV acetaminophen intervention 2: Single dose of 1 g PO APAP intervention 3: To subjects in both study arms: Administration of a 1 ng/kg body weight test dose of RSE to induce fever and test for fever response. Observation period of at least 60 minutes to ensure no exaggerated systemic responses, followed by administration of a 4 ng/kg of RSE to induce fever.
intervention 1: Intravenous acetaminophen plus oral placebo intervention 2: Oral acetaminophen plus IV placebo intervention 3: Reference standard endotoxin (RSE)
1
Knoxville | Tennessee | United States | -83.92074 | 35.96064
0
NCT00564629
[ 3 ]
50
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
true
RATIONALE: Erlotinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving erlotinib before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. PURPOSE: This phase II trial is studying how well erlotinib works in treating patients with breast cancer that can be removed by surgery.
OBJECTIVES: Primary * To determine the in situ antitumor effect of neoadjuvant erlotinib hydrochloride as measured by a reduction in Ki67 and/or an increase in terminal deoxynucleotidyl transferase-mediated deoxyuridine triphosphate-biotin nick end labeling (TUNEL)-positive tumor cells in patients with treatment-naive, operable breast cancer. Secondary * To identify a molecular profile, based on measurements of Estrogen Receptor (ER), Epidermal Growth Factor Receptor (EGFR), and a Human Epithelial Growth Factor Receptor-2(HER2), and protein expression profiles in patients with treatment-naïve, operable breast cancer that is responsive to erlotinib hydrochloride. * To correlate tumor concentrations of erlotinib hydrochloride with serum levels immediately before surgery. OUTLINE: This is a multi-center study. Patients receive oral erlotinib hydrochloride once daily for 5-14 days. Patients then undergo surgical resection within 24 hours after the last dose of erlotinib hydrochloride. Tumor tissue samples are collected at baseline and during surgery for correlative laboratory studies. Tissue samples are stained for ER, HER2, and EGFR levels, proliferation (Ki67), and apoptosis (TUNEL) by immunohistochemistry. Levels of erlotinib hydrochloride in tissue samples are measured by matrix-assisted laser desorption/ionization mass spectrometry. Blood samples are collected on the day of surgery. Levels of erlotinib hydrochloride in blood samples are measured by liquid chromatography/mass spectrometry. Patients are followed within 6 weeks after surgery.
Breast Cancer
stage I breast cancer stage II breast cancer stage IIIA breast cancer stage IIIB breast cancer
null
1
arm 1: None
[ 0 ]
9
[ 0, 6, 6, 10, 10, 10, 10, 10, 3 ]
intervention 1: Tarceva will be given orally at a dose of 150 mg/day for 5-14 days. Patients are to undergo surgical resection of their tumor within 24 hours of the last dose of Tarceva. intervention 2: Used to assess drug-induced changes in tumor cell proliferation and cell death in pre-therapy and surgical specimens intervention 3: Used to assess drug-induced changes in tumor cell proliferation and cell death in pre-therapy and surgical specimens intervention 4: Used to assess drug-induced changes in tumor cell proliferation and cell death in pre-therapy and surgical specimens intervention 5: Used to assess level of expression of genetic markers in pre-therapy and surgical specimens intervention 6: Used to determine blood plasma levels of Erlotinib on the day of surgery intervention 7: Used to determine blood plasma levels of Erlotinib on the day of surgery intervention 8: After treatment and following surgery, intervention will be used to determine Tarceva levels in tissue intervention 9: Surgical treatment will occur within 24-hours following completion of therapy.
intervention 1: erlotinib hydrochloride intervention 2: TUNEL assay intervention 3: protein expression analysis intervention 4: immunohistochemistry staining method intervention 5: laboratory biomarker analysis intervention 6: liquid chromatography intervention 7: mass spectrometry intervention 8: matrix-assisted laser desorption ionization mass spectrometry intervention 9: therapeutic conventional surgery
5
Birmingham | Alabama | United States | -86.80249 | 33.52066 Boston | Massachusetts | United States | -71.05977 | 42.35843 Chapel Hill | North Carolina | United States | -79.05584 | 35.9132 Nashville | Tennessee | United States | -86.78444 | 36.16589 Nashville | Tennessee | United States | -86.78444 | 36.16589
0
NCT00633750
[ 5 ]
614
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
This study evaluated safety, tolerability and efficacy of Fluvastatin XL® -extended release (80 mg once daily) in patients with metabolic syndrome
null
Metabolic Syndrome
Metabolic syndrome,dyslipidemia,fluvastatin extended release
null
1
arm 1: 80 mg once daily, at bedtime.
[ 0 ]
1
[ 0 ]
intervention 1: Fluvastatin extended release 80 mg
intervention 1: Fluvastatin XL®
1
Istanbul | N/A | Turkey (Türkiye) | 28.94966 | 41.01384
0
NCT00664742
[ 3 ]
120
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
true
The study is designed to assess safety and effects of vorapaxar, when added to standard of care (aspirin and clopidigrel), in Japanese subjects with acute coronary syndrome. The study may also provide information about the effect of vorapaxar on preventing heart attack and stroke in this subject population.
The study drug (loading dose) is administered at least 1 hour before catheterization for diagnostic imaging or percutaneous coronary interventions (PCI). The incidence of bleeding is thought to be an important index to assess the safety of this drug, therefore thrombolysis in myocardial infarction (TIMI) is evaluated.
Atherosclerosis Myocardial Ischemia Myocardial Infarction
null
5
arm 1: Vorapaxar 20 mg loading dose + daily 1 mg maintenance dose + standard of care (Aspirin + Ticlopidine) arm 2: Vorapaxar 20 mg loading dose + daily 2.5 mg maintenance dose + standard of care (Aspirin + Ticlopidine) arm 3: Vorapaxar 40 mg loading dose + daily 1 mg maintenance dose + standard of care (Aspirin + Ticlopidine) arm 4: Vorapaxar 40 mg loading dose + daily 2.5 mg maintenance dose + standard of care (Aspirin + Ticlopidine) arm 5: Placebo loading dose + daily placebo maintenance dose + standard of care (Aspirin + Ticlopidine)
[ 0, 0, 0, 0, 2 ]
4
[ 0, 0, 0, 0 ]
intervention 1: Oral tablets; single 20-mg or 40-mg loading dose on first day followed by daily 1-mg or 2.5-mg maintenance dose for 59 days intervention 2: Oral tablets; matching placebo for SCH 530348 loading and maintenance doses for 59 days intervention 3: Loading dose of 75-325 mg on Day 1, then 75-100 mg once daily for 60 days. intervention 4: 100 mg two or three times daily for 60 days.
intervention 1: Vorapaxar intervention 2: Placebo intervention 3: Aspirin intervention 4: Clopidogrel
0
null
0
NCT00684203
[ 3 ]
31
RANDOMIZED
PARALLEL
0TREATMENT
3TRIPLE
false
0ALL
false
This study was conducted: 1) to assess the clinical effect of Navarixin on the Psoriasis Activity and Severity Index (PASI), 2) to determine the effects of Navarixin on the Physician's Global Assessment (PGA), 3) to evaluate the safety and tolerability of Navarixin, and 4) to determine the multiple-dose pharmacokinetics of Navarixin.
null
Psoriasis
null
2
arm 1: Navarixin 30 mg administered orally once daily for 28 days. arm 2: Matching placebo to Navarixin administered orally once daily for 28 days.
[ 0, 2 ]
2
[ 0, 10 ]
intervention 1: Navarixin capsules orally, once daily for 28 days. intervention 2: Matching placebo capsules to Navarixin orally, once daily for 28 days.
intervention 1: Navarixin 10 mg intervention 2: Placebo
0
null
0
NCT00684593
[ 0 ]
147
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
true
0ALL
false
The purpose of this study is to determine whether rifaximin is effective in the treatment of tropical enteropathy in a population of African children at high risk for this disease.
null
Tropical Enteropathy
null
2
arm 1: None arm 2: None
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: 100mg of rifaxin for 7 consecutive days, twice daily intervention 2: twice daily for 7 consecutive days
intervention 1: Rifaximin intervention 2: Placebo
1
Limela | N/A | Malawi | 34.71667 | -14.88333
0
NCT00858988
[ 3 ]
124
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
false
This is a research study testing SABER-Bupivacaine (an experimental pain-relieving medication). SABER-Bupivacaine is designed to continuously deliver bupivacaine, a common local anesthetic, for a few days in order to treat local post-surgical pain. This study is testing SABER-Bupivacaine in people having surgery to repair a hernia. The purpose of the study is to measure and compare the safety (side effects), tolerability (ability to tolerate), and efficacy (how well it works) of two different volumes of SABER-Bupivacaine with SABER-Placebo.
null
Postoperative Pain Hernia Surgery
Post-operative pain hernia surgery opioid
null
3
arm 1: 2.5 mL SABER-Bupivacaine/Once arm 2: 5.0 mL SABER-Bupivacaine/Once arm 3: 2.5 mL or 5.0 mL SABER-Placebo/Once
[ 0, 0, 2 ]
3
[ 0, 0, 0 ]
intervention 1: Injectable Extended Release Solution; 2.5 mL SABER-Bupivacaine/Once intervention 2: Injectable Extended Release Solution; 5.0 mL SABER-Bupivacaine/Once intervention 3: Injectable Solution; 2.5 or 5.0 mL SABER-Placebo/Once
intervention 1: SABER-Bupivacaine intervention 2: SABER-Bupivacaine intervention 3: SABER-Placebo
5
Cairns | Queensland | Australia | 145.76613 | -16.92366 Sunnybank | Queensland | Australia | 153.06064 | -27.58003 Port Lincoln | South Australia | Australia | 135.87442 | -34.72625 Ringwood East | Victoria | Australia | 145.25 | -37.81667 Hamilton | N/A | New Zealand | 175.28333 | -37.78333
0
NCT00974350
[ 5 ]
16
RANDOMIZED
CROSSOVER
0TREATMENT
4QUADRUPLE
false
0ALL
false
This study examines the effects of pioglitazone on renal sodium handling in subjects prone to insulin resistance, i.e. diabetic and/or hypertensive subjects.
Aim: Glitazones are powerful insulin sensitizers prescribed for the treatment of type 2 diabetes. Their use is however associated with fluid retention and an increased risk of congestive heart failure. We previously demonstrated that pioglitazone increases proximal sodium reabsorption in healthy volunteers. This study examines the effects of pioglitazone on renal sodium handling in subjects prone to insulin resistance, i.e. diabetic and/or hypertensive subjects. Methods: In this double-blind, randomized, placebo-controlled, four-way, cross-over study, we examined the effects of pioglitazone (45mg daily during 6 weeks) or placebo on renal, systemic and hormonal responses to changes in sodium intake in 16 individuals, 8 with type 2 diabetic and 8 with hypertension.
Diabetes Hypertension
diabetes blood pressure pioglitazone sodium insulin resistance, renal function, sodium, pioglitazone
null
2
arm 1: placebo-controlled, randomized, cross-over study arm 2: placebo-controlled, randomized, cross-over study was to explore the effects of pioglitazone (45 mg q.d. for 6 weeks) on the renal, hormonal and blood pressure responses to changes in sodium intake in a population prone to insulin resistance
[ 2, 2 ]
2
[ 0, 0 ]
intervention 1: placebo-controlled, randomized, cross-over study was to explore the effects of pioglitazone (45 mg q.d. for 6 weeks) on the renal, hormonal and blood pressure responses to changes in sodium intake in a population prone to insulin resistance intervention 2: placebo-controlled, randomized, cross-over study was to explore the effects of pioglitazone (45 mg q.d. for 6 weeks) on the renal, hormonal and blood pressure responses to changes in sodium intake in a population prone to insulin resistance
intervention 1: Pioglitazone intervention 2: Metformin
1
Geneva | Canton of Geneva | Switzerland | 6.14569 | 46.20222
0
NCT01090752
[ 3 ]
127
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This was a randomized, double blind, placebo-controlled study in subjects who have undergone major surgery. Each subject's study participation consisted of a screening visit, a 2-day treatment period, and a follow-up visit. Following surgery, subjects were randomly assigned to receive intranasally (IN) ketorolac 10 mg, IN ketorolac 30 mg, or placebo when the pain intensity (PI) rating equaled at least 40 on a 100-mm visual analog scale (VAS). Thereafter, subjects received study drug every 8 hours, with the last dose given at 40 hours. For pain not relieved by the study drug, the subjects had access to morphine sulfate (MS) administered via patient controlled analgesia (PCA). The primary objective was to evaluate the analgesic efficacy of multiple intranasal (IN) doses of ketorolac over 2 days. The secondary objective was to evaluate the safety and tolerability of this dosing regimen.
null
Postoperative Pain
null
3
arm 1: None arm 2: None arm 3: None
[ 0, 0, 2 ]
3
[ 0, 0, 0 ]
intervention 1: 10 mg Intranasal (2 x 100 uL of a 5% solution) intervention 2: 30 mg Intranasal (2 x 100 uL of a 15% solution) intervention 3: Intranasal
intervention 1: Ketorolac tromethamine intervention 2: Ketorolac tromethamine intervention 3: Placebo
1
Hamilton | N/A | New Zealand | 175.28333 | -37.78333
0
NCT01351090
[ 4 ]
1,380
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
false
The purpose of this study is to investigate the efficacy and safety of telcagepant (MK-0974) compared to an approved medication for acute migraine. This study was conducted as a "triple-dummy" design; for each dose of study drug, participants each received 3 forms of study drug (2 capsules of active and/or placebo and 1 tablet of active and/or placebo) and were instructed to take one of each form of study drug at dosing time. The primary hypotheses of this study are that telcagepant is superior to placebo in Pain Freedom at 2 Hours Post-Dose, Pain Relief at 2 Hours Post-Dose, Absence of Photophobia at 2 Hours Post-Dose, Absence of Phonophobia at 2 Hours Post-Dose and Absence of Nausea at 2 Hours Post-Dose.
null
Migraine
null
4
arm 1: Participants receive telcagepant 150 mg capsules, one capsule administered orally at initial onset of moderate to severe migraine headache. If, after 2 hours post-dose, participants still have a moderate to severe migraine or migraine recurs, participants may receive an optional second dose of study drug (telcagepant 150 mg or placebo) or one dose of non-study rescue medication. arm 2: Participants receive telcagepant 300 mg capsules, one capsule administered orally at initial onset of moderate to severe migraine headache. If, after 2 hours post-dose, participants still have a moderate to severe migraine or migraine recurs, participants may receive an optional second dose of study drug (telcagepant 300 mg or placebo) or one dose of non-study rescue medication. arm 3: Participants receive zolmitriptan 5 mg tablets, one tablet administered orally at initial onset of moderate to severe migraine headache. If, after 2 hours post-dose, participants still have a moderate to severe migraine or migraine recurs, participants may receive an optional second dose of study drug (placebo) or one dose of non-study rescue medication. arm 4: Participants receive placebo matching capsules or tablets, one capsule or tablet administered orally at initial onset of moderate to severe migraine headache. If, after 2 hours post-dose, participants still have a moderate to severe migraine or migraine recurs, participants may receive an optional second dose of study drug (placebo) or one dose of non-study rescue medication.
[ 0, 0, 1, 2 ]
7
[ 0, 0, 0, 0, 0, 0, 0 ]
intervention 1: Telcagepant 150 mg liquid-filled soft gel capsules intervention 2: Telcagepant 300 mg liquid-filled soft gel capsules intervention 3: Zolmitriptan 5 mg tablets intervention 4: Placebo to match telcagepant 150 mg liquid-filled soft gel capsules intervention 5: Placebo to match tecagepant 300 mg liquid-filled soft gel capsules intervention 6: Placebo to match zolmitriptan 5 mg tablets intervention 7: If moderate or severe migraine headache pain continues or recurs 2 hours after dose of study drug, participants are allowed to take an optional second dose of study drug or their own non-study rescue migraine medication, which may include analgesics (e.g., nonsteroidal anti-inflammatory drugs \[NSAIDs\] or opiates), anti-emetics, or zolmitriptan. Triptans other than zolmitriptan and ergot derivatives are prohibited for 24 hours following the last dose of study drug.
intervention 1: Telcagepant potassium 150 mg intervention 2: Telcagepant potassium 300 mg intervention 3: Zolmitriptan 5 mg intervention 4: Placebo to telcagepant 150 mg intervention 5: Placebo to tecagepant 300 mg intervention 6: Placebo to zolmitriptan 5 mg intervention 7: Rescue medication
0
null
0
NCT00442936
[ 3 ]
145
RANDOMIZED
PARALLEL
0TREATMENT
null
false
0ALL
false
This clinical trial is a multi centre, randomised, single-blind, parallel group, placebo-controlled, single oral dose study with a positive control arm. Patients previously scheduled for 3rd molar tooth extraction, who are otherwise healthy, will be recruited. Upon completion of surgery, e.g. prior to established pain, patients will be randomised to treatment (SB-706598, placebo or co-codamol) and dosed with the study medication
null
Toothache
Acute Pain Dental Pain Vanilloid VR1 TRPV1 Inflammation
null
4
arm 1: Eligible participants received a single dose of SB705498 matching placebo capsules (4 placebo capsules) via oral route and were followed up to a maximum of 14 days. arm 2: Eligible participants received a single dose of SB705498 400 milligram (mg) capsules (2 x 200 mg capsules plus 2 placebo capsules) via oral route and were followed up to a maximum of 14 days. arm 3: Eligible participants received a single dose of SB705498 1000 mg capsules (2 x 200 mg capsules plus 2 x 300 mg capsules) via oral route and were followed up to a maximum of 14 days. arm 4: Eligible participants received a single dose of Co-codamol capsules (2 x Paracetamol Ph Eur 500 mg, codeine phosphate hemihydrate Ph Eur 12.8 mg plus two placebo capsules) via oral route and were followed up to a maximum of 14 days.
[ 0, 0, 0, 0 ]
4
[ 0, 0, 0, 0 ]
intervention 1: SB705498 400 mg intervention 2: SB705498 1000 mg intervention 3: Placebo intervention 4: Co-Codamol
intervention 1: SB705498 400 mg intervention 2: SB705498 1000 mg intervention 3: Placebo intervention 4: Co-Codamol
5
Verona | Veneto | Italy | 10.9938 | 45.43854 Seoul | N/A | South Korea | 126.9784 | 37.566 Croydon | Surrey | United Kingdom | -0.1 | 51.38333 Leeds | N/A | United Kingdom | -1.54785 | 53.79648 Manchester | N/A | United Kingdom | -2.23743 | 53.48095
0
NCT00281684
[ 3 ]
23
RANDOMIZED
PARALLEL
2DIAGNOSTIC
null
false
0ALL
false
GSK233705 is a high-affinity specific muscarinic receptor (mAChR) antagonist which is being developed for the treatment of chronic obstructive pulmonary disease. This is a randomised, double-blind, placebo-controlled, dose ascending, parallel group study to examine the safety, tolerability, pharmacokinetics and pharmacodynamics of twice daily inhaled doses of GSK233705B for 7 days, in COPD subjects.
null
Pulmonary Disease, Chronic Obstructive
muscarinic receptor, bronchodilators Chronic obstructive pulmonary disease,
null
0
null
null
1
[ 0 ]
intervention 1: None
intervention 1: GSK233705B
4
Eindhoven | N/A | Netherlands | 5.47778 | 51.44083 Harderwijk | N/A | Netherlands | 5.62083 | 52.34167 Utrecht | N/A | Netherlands | 5.12222 | 52.09083 Zuidlaren | N/A | Netherlands | 6.68194 | 53.09417
0
NCT00453479
[ 3 ]
136
RANDOMIZED
PARALLEL
0TREATMENT
3TRIPLE
false
0ALL
false
This study is a multicenter, double-blind, study to evaluate the safety and effectiveness of treatment with LY2127399 (in addition to the standard of care treatment, methotrexate) for participants with Rheumatoid Arthritis. Participants will receive three intravenous doses of LY2127399 or placebo. Participants will participate in 10 or more visits to the study site, over 6 months. Evaluation of safety and efficacy will be conducted throughout the study.
null
Arthritis, Rheumatoid
null
2
arm 1: None arm 2: None
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: 30 mg, 60 mg or 160 mg, IV (in the vein) in weeks 0, 3 and 6. Treatment duration: 6 weeks. intervention 2: IV (in vein) in weeks 0, 3 and 6. Treatment duration: 6 weeks.
intervention 1: LY2127399 intervention 2: Placebo
14
Bacau | N/A | Romania | 26.91384 | 46.56718 Baia Mare | N/A | Romania | 23.56808 | 47.65729 Brasov | N/A | Romania | 25.60613 | 45.64861 Brăila | N/A | Romania | 27.97429 | 45.27152 Bucharest | N/A | Romania | 26.10626 | 44.43225 Cluj-Napoca | N/A | Romania | 23.6 | 46.76667 Constanța | N/A | Romania | 28.63432 | 44.18073 Craiova | N/A | Romania | 23.8 | 44.31667 Iași | N/A | Romania | 27.6 | 47.16667 Sf. Gheorghe | N/A | Romania | N/A | N/A Sibiu | N/A | Romania | 24.15 | 45.8 Târgovişte | N/A | Romania | 25.4567 | 44.92543 Târgu Mureş | N/A | Romania | 24.55747 | 46.54245 Timișoara | N/A | Romania | 21.22571 | 45.75372
0
NCT00308282
[ 4 ]
40
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
false
The purpose of this study is to test the safety and effectiveness of Losartan as compared to Losartan/HCTZ in pediatric patients (6 to 17 years) with high blood pressure.
Participants who meet eligibility requirements will enter the single-blind Filter Period. Participants with either sitting systolic blood pressure (SiSBP) or sitting diastolic blood pressure (SiDBP) \>=95th percentile for gender/age/height will be administered either losartan 25 mg or losartan 50 mg depending on body weight (\<50 kg and \>=50 kg, respectively). After 3 weeks, if SiSBP or SiDBP is \>=95th percentile, participants will titrate to either losartan 50 mg or 100 mg according to weight. Participants with both SiSBP and SiDBP \<95th percentile will be excluded from the study. After an additional 3 weeks, participants whose blood pressure (either SiSBP or SiDBP) remains \>=95th percentile will then enter the double-blind phase. Participants who meet all entry criteria will be randomized in a 1:1 fashion to either losartan or losartan/hydrochlorothiazide (HCTZ) for 4 weeks and will be stratified according to body weight (\<50 kg and \>=50 kg). Participants whose weight is \<50 kg will be randomized to losartan 50 mg or losartan50 mg/HCTZ 12.5 mg. Participants weighing \>=50 kg will be randomized to losartan 100 mg or losartan/HCTZ 100 mg/12.5 mg. At the end of double-blind treatment, participants may enter a 20-week double-blind extension and will continue on their randomized therapy with losartan or losartan/HCTZ. 40 participants were enrolled and screened; the trial was terminated before any participants were randomized into the double-blind treatment period.
Hypertension
null
4
arm 1: Losartan 50 mg tablet, oral, once daily for 4 weeks. Participant also will be co-administered placebo for losartan 50 mg/hydrochlorothiazide (HCTZ) 12.5 mg once daily for 4 weeks arm 2: Losartan 100 mg tablet, oral, once daily for 4 weeks. Participant also will be co-administered placebo for losartan 100 mg/hydrochlorothiazide (HCTZ) 12.5 mg once daily for 4 weeks arm 3: Losartan 50 mg/hydrochlorothiazide (HCTZ) 12.5 mg tablet, oral, once daily for 4 weeks. Participant also will be co-administered placebo for losartan 50 mg once daily for 4 weeks arm 4: Losartan 100 mg/hydrochlorothiazide (HCTZ) 12.5 mg tablet, oral, once daily for 4 weeks. Participant also will be co-administered placebo for losartan 100 mg once daily for 4 weeks
[ 1, 1, 0, 0 ]
4
[ 0, 0, 0, 0 ]
intervention 1: losartan/hydrochlorothiazide 12.5mg tablet po qd; for a 4 week study period. intervention 2: Losartan 50mg titrating up to Losartan 100mg tablet po qd; for a 4 week study period intervention 3: None intervention 4: losartan/hydrochlorothiazide 12.5mg Pbo tablet po qd; for a 4 week study period.
intervention 1: hydrochlorothiazide (+) losartan potassium intervention 2: losartan potassium intervention 3: Placebo for Losartan intervention 4: Placebo for Losartan/HCTZ
0
null
0
NCT00447603
[ 4 ]
371
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
null
The purpose of this study is to determine if lacosamide (SPM 927) is safe if taken for a longer period of time and whether it continues to work well to treat pain. Subjects will receive lacosamide at a dose that will be individually determined to be the one that provides most pain relief with the least side effects. The maximum dose will be 600mg/day. Subjects may participate in this trial until October 2007. This time may be extended to allow them to participate until lacosamide is commercially available. If a subject meet the requirements for the study at Visit 1 and after a two weeks phase without trial medication, s/he enters a Titration Phase to determine the personal optimal dose of lacosamide. When this dose is reached s/he will enter the Maintenance Phase and will be asked to return for visits every 4 weeks for the first 24 weeks and every 12 weeks thereafter.
null
Painful Diabetic Neuropathy
Painful diabetic neuropathy Lacosamide
null
1
arm 1: Open label active treatment
[ 0 ]
1
[ 0 ]
intervention 1: Lacosamide film-coated tablets; two times per day; up to 400 mg/day for 2.75 years
intervention 1: Lacosamide
1
Monheim | N/A | Germany | 10.85834 | 48.84389
0
NCT00220337
[ 3 ]
30
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
true
The purpose of this study is to evaluate the safety of combination therapy of radiotherapy and temozolomide ("concomitant radiotherapy phase"), and then temozolomide monotherapy ("monotherapy phase"), in patients with newly diagnosed glioblastoma multiforme. Progression free survival and response rate will also be calculated.
null
Glioblastoma
null
1
arm 1: It is the only arm of the study. Subjects receive a combination of radiotherapy and temozolomide, and then temozolomide monotherapy.
[ 0 ]
2
[ 4, 0 ]
intervention 1: Radiotherapy will be administered in combination with temozolomide during the concomitant radiotherapy phase. Radiotherapy will consist of a conventionally fractioned regimen, delivering a total dose of 60 Gy in 6 weeks, in a once daily schedule of 2 Gy per fraction, for a total of 30 fractions. Radiation will be provided by a linear accelerator of x ray energy of 4 MV or higher. intervention 2: During the concomitant radiotherapy phase (6 weeks), temozolomide will be administered in combination with radiotherapy, once daily at 75 mg/m2/day. Then, during the monotherapy phase, subjects will receive 6 cycles of temozolomide alone. Each cycle will last 28 days, and temozolomide will be administered once daily from Day 1 to Day 5 of each cycle. The dose of temozolomide in the first cycle will be 150 mg/m2/day, and may be increased to 200 mg/m2/day for Cycle 2 and subsequent cycles depending on nonhematologic toxicity observed and neutrophil and platelet count values. Capsules containing 5 mg, 20 mg, or 100 mg of temozolomide will be combined to achieve each subject's calculated dose.
intervention 1: Radiotherapy intervention 2: Temozolomide
0
null
0
NCT00684567
[ 4 ]
4,469
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
true
The objective is to compare the safety and efficacy of the following three treatment regimens in previously untreated adult subjects with chronic hepatitis C infected with Genotype 1: (1) PegIntron 1.5 µg/kg/wk in combination with weight based REBETOL (800-1400 mg/day); (2) PegIntron 1µg/kg/wk in combination with weight based REBETOL (800-1400 mg/day); and (3) PEGASYS 180 µg/wk plus COPEGUS 1000-1200 mg/day.
PegIntron Dose will be administered once weekly subcutaneously on the same day of the week: Screening 2 Weight 40-50 kg Volume to Inject (mL) 0.22; Screening 2 Weight 51-60 kg Volume to Inject (mL) 0.28; Screening 2 Weight 61-75 kg Volume to Inject (mL) 0.33; Screening 2 Weight 76-85 kg Volume to Inject (mL) 0.41; Screening 2 Weight 86-104 kg Volume to Inject (mL) 0.48; Screening 2 Weight 105-125 kg Volume to Inject (mL) 0.58 from two vials REBETOL Dosage (for Use With PegIntron): Screening 2 Weight 40-65 kg Daily Dose 800 mg; Screening 2 Weight \>65-85 kg Daily Dose 1000 mg; Screening 2 Weight \>85-105 kg Daily Dose 1200 mg; Screening 2 Weight \>105-125 kg Daily Dose 1400 mg The PEGASYS dose of 1 mL (180 µg) will be administered once weekly subcutaneously on the same day of the week COPEGUS Dosage (for Use With PEGASYS): Screening 2 Weight \<75 kg Daily Dose 1000 mg; Screening 2 Weight \> or = 75 kg Daily Dose 1200mg NOTE: Double Blind for PegIntron; Open Label for REBETOL, PEGASYS and COPEGUS NOTE: REBETOL is the Schering-Plough brand name for ribavirin. COPEGUS is the Hoffman-La Roche brand name for ribavirin.
Hepatitis C, Chronic
null
3
arm 1: PegIntron (peginterferon alfa-2b; SCH 54031) 1.5 ug/kg/week in combination with weight-based REBETOL (ribavirin; SCH 18908) 800-1400 mg/day administered for 48 weeks with 24-week post-treatment follow-up arm 2: PegIntron (peginterferon alfa-2b; SCH 54031) 1.0 ug/kg/week in combination with weight-based REBETOL (ribavirin; SCH 18908) 800-1400 mg/day administered for 48 weeks with 24-week post-treatment follow-up arm 3: PEGASYS (peginterferon alfa-2a) 180 ug/week plus COPEGUS (ribavirin) 1000-1200 mg/day administered for 48 weeks with 24-week post-treatment follow-up
[ 0, 0, 1 ]
5
[ 2, 2, 0, 2, 0 ]
intervention 1: 1.5 ug/kg/week subcutaneously (SC) for 48 weeks intervention 2: 1.0 ug/kg/week SC for 48 weeks intervention 3: weight based dose 800-1400 mg/day orally (PO) for 48 weeks intervention 4: 180 ug/week SC administered for 48 weeks intervention 5: 1000-1200 mg/day PO for 48 weeks
intervention 1: PegIntron (peginterferon alfa-2b; SCH 54031) intervention 2: PegIntron (peginterferon alfa-2b; SCH 54031) intervention 3: REBETOL (ribavirin; SCH 18908) intervention 4: PEGASYS (peginterferon alfa-2a) intervention 5: COPEGUS (ribavirin)
0
null
1
NCT00081770
[ 2 ]
151
NON_RANDOMIZED
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
To evaluate the safety and tolerability of oral SKI-606 (bosutinib) administered on a daily schedule to subjects with advanced malignant solid tumors and to define a maximum tolerated dose (MTD) in this subject population.
null
Neoplasms
Solid tumors
null
4
arm 1: Dose finding study of monotherapy bosutinib in patients with advanced solid tumors. arm 2: Enroll 30 patients at RP2D to further evaluate safety and efficacy in subgroup population. arm 3: Enroll 30 patients at RP2D to further evaluate safety and efficacy in subgroup population. arm 4: Enroll 30 patients at RP2D to further evaluate safety and efficacy in subgroup population.
[ 0, 0, 0, 0 ]
4
[ 0, 0, 0, 0 ]
intervention 1: Dose levels evaluated 50mg, 100mg, 200mg, 300mg, 400mg, 500mg and 600mg. 500mg was identified as MTD, however due to GI toxicities at that dose, 400mg was selected as the RP2D. Drug was administered as long as tolerable and disease under study did not worsen. intervention 2: 400mg QD bosutinib, as long as tolerated and disease under study does not worsen. intervention 3: 400mg QD bosutinib, as long as tolerated and disease under study does not worsen. intervention 4: 400mg QD bosutinib, as long as tolerated and disease under study does not worsen.
intervention 1: bosutinib intervention 2: bosutinib intervention 3: bosutinib intervention 4: bosutinib
19
Birmington | Alabama | United States | N/A | N/A Scottsdale | Arizona | United States | -111.89903 | 33.50921 Los Angeles | California | United States | -118.24368 | 34.05223 Tampa | Florida | United States | -82.45843 | 27.94752 Atlanta | Georgia | United States | -84.38798 | 33.749 Indianpolis | Indiana | United States | N/A | N/A Baltimore | Maryland | United States | -76.61219 | 39.29038 Detroit | Michigan | United States | -83.04575 | 42.33143 Lansing | Michigan | United States | -84.55553 | 42.73253 New York | New York | United States | -74.00597 | 40.71427 New York | New York | United States | -74.00597 | 40.71427 Charlotte | North Carolina | United States | -80.84313 | 35.22709 Charlotte | North Carolina | United States | -80.84313 | 35.22709 UNC Chapel Hill | North Carolina | United States | N/A | N/A UNC Chapel Hill | North Carolina | United States | N/A | N/A Cleveland | Ohio | United States | -81.69541 | 41.4995 San Antonio | Texas | United States | -98.49363 | 29.42412 Tyler | Texas | United States | -95.30106 | 32.35126 Seattle | Washington | United States | -122.33207 | 47.60621
1
NCT00195260
[ 4 ]
145
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
null
0ALL
true
The purpose of the study is to assess the effect of everolimus initiation together with reduction or discontinuation of calcineurin inhibitor (CNI) on renal function in maintenance liver transplant recipients with CNI-related renal impairment, while maintaining efficacy.
null
Liver Transplantation
Liver transplantation, everolimus, calcineurin inhibitor, renal function
null
2
arm 1: Reduced CNI dose + everolimus (1.5 mg twice daily (b.i.d)) ± steroids arm 2: Standard CNI dose ± MPA/AZA ± steroids
[ 1, 0 ]
4
[ 0, 0, 0, 0 ]
intervention 1: 1.5 mg bid adjusted in order to achieve a trough level between 3 and 8 ng/mL while in combination with CNI and between 6 and 12 ng/mL after CNI discontinuation intervention 2: None intervention 3: None intervention 4: None
intervention 1: Everolimus intervention 2: Calcineurin inhibitors (CNI) intervention 3: Mycophenolate acid (MPA)/ Azathioprine (AZA) intervention 4: Steroids
2
Germany | N/A | Germany | N/A | N/A Basel | N/A | Switzerland | 7.57327 | 47.55839
1
NCT00267189
[ 4 ]
107
NON_RANDOMIZED
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
true
The primary objective is to assess the safety, efficacy and tolerability of the combination of PEG-Intron plus REBETOL in pediatric subjects with chronic hepatitis C. The secondary objective is to measure the multiple-dose pharmacokinetics of PEG-Intron and REBETOL in pediatric subjects with chronic hepatitis C.
This global, multicenter, open-label Phase 3 study will evaluate the safety, efficacy and tolerability of PEG-Intron plus REBETOL in previously untreated pediatric subjects, ages 3 through 17 years, with chronic hepatitis C.
Hepatitis C, Chronic
null
1
arm 1: PEG2b 1.5 μg/kg/wk given subcutaneously (once weekly) and RBV 400-1200 mg/day by mouth divided in 2 daily doses (administered twice daily with food, dosed 12 hours apart) for 48 weeks. Subjects treated up to 48 weeks and followed for additional 24 weeks after the end of treatment (total of 72 weeks study participation).
[ 0 ]
2
[ 2, 0 ]
intervention 1: PEG2b 1.5 μg/kg/wk given subcutaneously (once weekly) for 48 weeks. intervention 2: 15 mg/kg/day for up to 48 weeks
intervention 1: peginterferon alfa-2b (PEG2b) (SCH 54031) intervention 2: ribavirin (SCH 18908)
0
null
0
NCT00104052
[ 5 ]
50
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
true
The purpose of this study is to examine the safety and efficacy of quetiapine for generalized anxiety disorder patients who remain symptomatic despite treatment with paroxetine CR.
Generalized anxiety disorder (GAD) is a relatively common condition affecting 5% of the population, with a typically chronic course and associated with significant psychosocial impairment and decreased quality of life (Schweizer, 1995). Although a number of therapeutic agents demonstrate some efficacy in the treatment of generalized anxiety disorder, only a minority of anxious patients experience remission with initial treatment. The purpose of this study is to examine the efficacy of one strategy, the addition of quetiapine, for the treatment of patients with GAD who remain refractory despite an adequate treatment trial with a selective serotonin reuptake inhibitor (SSRI). This is an investigator-initiated augmentation study of an already approved drug for a different indication. Quetiapine is a novel antipsychotic agent with potent effects at the serotonergic, as well as dopaminergic receptor, and a more favorable side effect profile than standard neuroleptics, including a low potential to cause extrapyramidal symptoms. This is a two phase, 18-week research study in which participants who remain symptomatic at the end of one phase (10 weeks) enter into the next phase. In phase I, all participants receive paroxetine CR (Paxil CR) for 10 weeks. Participants who continue to have anxiety symptoms will enter the 8-week Phase II, in which they continue taking Paxil CR and they will also be randomly assigned (by chance, like a flip of a coin) to receive quetiapine (Seroquel) or placebo (contains no active medication).
Anxiety Disorder
generalized anxiety disorder pharmacotherapy treatment refractory double-blind
null
2
arm 1: Eleven individuals were randomized to plaecbo augmentation of continued paroxetine CR at the week 10 dose level. In the first phase of the study, individuals started at 12.5 mg/day of paroxetine and flexibly titrated up to a maximum of 62.5 mg/day by week 10. Individuals who did not achieve remission and were randomized into the placebo group received placebo augmentation of continued paroxetine CR at the week 10 dose level. arm 2: Eleven individuals were randomized to quetiapine augmentation of continued paroxetine CR at the week 10 dose level. In the first phase of the study, individuals started at 12.5 mg/day of paroxetine and flexibly tirated up to a maximum of 62.5 mg/day by week 10. Individuals who did not receive remission and were randomized to receive quetiapine started at 25 mg at bedtime for the first week, then flexibly dosed based on response and tolerability to a maximum of 200 mg BID by week 16.
[ 1, 0 ]
3
[ 0, 0, 0 ]
intervention 1: None intervention 2: None intervention 3: None
intervention 1: Continued Paroxetine CR intervention 2: Quetiapine intervention 3: Placebo
2
Boston | Massachusetts | United States | -71.05977 | 42.35843 Durham | North Carolina | United States | -78.89862 | 35.99403
0
NCT00113295
[ 3 ]
175
RANDOMIZED
PARALLEL
9OTHER
4QUADRUPLE
false
0ALL
null
Recent studies have found that poor oral hygiene may foster the colonization of the oropharynx by potential respiratory pathogens in mechanically-ventilated (MV), intensive care unit (ICU) patients. Thus, improvements in oral hygiene in MV-ICU patients may prevent ventilator-associated pneumonia (VAP). The specific aims of this investigation are: 1) to organize the necessary infrastructure to develop and perform a pilot clinical trial to evaluate alternative oral hygiene procedures to prevent VAP; 2) to use this organization to perform a pilot clinical trial to determine if the use of oral topical chlorhexidine gluconate (CHX) will prevent dental plaque, oropharyngeal colonization by respiratory pathogens, and VAP in MV-ICU patients.
Recent studies have found that poor oral hygiene may foster the colonization of the oropharynx by potential respiratory pathogens in mechanically-ventilated (MV), intensive care unit (ICU) patients. Thus, improvements in oral hygiene in MV-ICU patients may prevent ventilator-associated pneumonia (VAP). The Specific Aims of this investigation are: 1) to organize the necessary infrastructure to develop and perform a pilot clinical trial to evaluate alternative oral hygiene procedures to prevent VAP; 2) to use this organization to perform a pilot clinical trial to determine if the use of oral topical chlorhexidine gluconate (CHX) will prevent dental plaque, oropharyngeal colonization by respiratory pathogens, and VAP in MV-ICU patients. This pilot longitudinal, double blind intervention study will consider the appropriate frequency of delivery of CHX to improve oral hygiene in MV-ICU patients. Preliminary data from these pilot studies will also allow accurate sample size calculations to be made for a large-scale multi-center clinical trial; and 3) to perform molecular epidemiological studies to identify and genetically type bacteria cultured from lower airway secretions of MV-ICU patients with or without VAP and compare them to isolates of the same species from their dental plaque. This pilot study will enable this multidisciplinary team of investigators to organize the infrastructure, patient recruitment and methodologic protocols, and data management and analysis procedures necessary to perform a multi-center, controlled clinical trial to assess the efficacy and generalizability of this intervention to improve oral hygiene in MV-ICU and prevent VAP.
Pneumonia
Ventilator-associated pneumonia
null
3
arm 1: Delivered Twice a day arm 2: Delivered twice a day arm 3: Delivered once a day, placebo once a day
[ 2, 0, 0 ]
2
[ 0, 0 ]
intervention 1: chlorhexidine gluconate oral rinse intervention 2: None
intervention 1: chlorhexidine gluconate oral rinse (0.12%) intervention 2: placebo
1
Buffalo | New York | United States | -78.87837 | 42.88645
0
NCT00123123
[ 4 ]
41
RANDOMIZED
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
null
The purpose of this study is to characterize the PK profile, safety and tolerability of single and multiple doses of pantoprazole in children aged 1 through 11 years with endoscopically proven GERD.
null
Gastroesophageal Reflux
Gastrointestinal Reflux Disease
null
0
null
null
1
[ 0 ]
intervention 1: None
intervention 1: pantoprazole for approximately 9 weeks.
23
Little Rock | Arkansas | United States | -92.28959 | 34.74648 Orange | California | United States | -117.85311 | 33.78779 San Diego | California | United States | -117.16472 | 32.71571 Washington D.C. | District of Columbia | United States | -77.03637 | 38.89511 Gainesville | Florida | United States | -82.32483 | 29.65163 Miami | Florida | United States | -80.19366 | 25.77427 Pensacola | Florida | United States | -87.21691 | 30.42131 Park Ridge | Illinois | United States | -87.84062 | 42.01114 Indianapolis | Indiana | United States | -86.15804 | 39.76838 Louisville | Kentucky | United States | -85.75941 | 38.25424 Boston | Massachusetts | United States | -71.05977 | 42.35843 Boston | Massachusetts | United States | -71.05977 | 42.35843 Brockton | Massachusetts | United States | -71.01838 | 42.08343 Detroit | Michigan | United States | -83.04575 | 42.33143 Flint | Michigan | United States | -83.68746 | 43.01253 Jackson | Mississippi | United States | -90.18481 | 32.29876 Kansas City | Missouri | United States | -94.57857 | 39.09973 New York | New York | United States | -74.00597 | 40.71427 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Memphis | Tennessee | United States | -90.04898 | 35.14953 Temple | Texas | United States | -97.34278 | 31.09823 Salt Lake City | Utah | United States | -111.89105 | 40.76078 Milwaukee | Wisconsin | United States | -87.90647 | 43.0389
0
NCT00141817
[ 2 ]
7
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
The purpose of this study is to see how vincristine, when placed in an oil droplet called a liposome (VSLI), is absorbed, distributed (moved around) and excreted from the the body (pharmacokinetics). This study will also assess the safety of VSLI and to see if VSLI will slow the growth or shrink tumors in patients with metastatic melanoma that has resulted in liver impairment, and who have relapsed after previous therapies.
OBJECTIVES: Primary: To assess the pharmacokinetics of VSLI administered intravenously to patients with malignant melanoma and hepatic dysfunction secondary to metastases. Secondary: To assess the safety and antitumor activity of VSLI in this population.
Malignant Melanoma
null
1
arm 1: Single armed study; all subjects received VSLI
[ 0 ]
1
[ 0 ]
intervention 1: Marqibo (VSLI) 1.0 mg/m2 delivered by intravenous infusion over 1 hour every 2 weeks.
intervention 1: Vincristine Sulfate Liposomes Injection
1
Houston | Texas | United States | -95.36327 | 29.76328
0
NCT00145041
[ 0 ]
96
RANDOMIZED
PARALLEL
0TREATMENT
1SINGLE
true
0ALL
false
This investigation will evaluate the subjective effect on postoperative pain of three catheter placements in the knee: 1. intraarticular infusion only, 2. patellar tendon harvest site only, 3. both intraarticular and patellar tendon harvest site.
There will be 96 subjects recruited into this study. Based on power analysis for repeated measures analysis of variance, the sample size required for this investigation is 24 subjects per group based on a 90% confidence interval and alpha of 0.05. There will be 24 subjects in Group 1 (intraarticular bupivicaine infusion only). There will be 24 subjects in Group 2 (patellar tendon harvest site only with bupivicaine). There will be 24 subjects in Group 3 (both intraarticular and patellar tendon harvest site with bupivicaine). There will be 24 subjects in the control, Group 4 (intraarticular infusion physiologic saline only). All patients will be recruited under the discretion of the principal investigator. The primary outcome measure for this investigation will be the Postoperative Patient Diary. This document records patient's subjective evaluations of pain, comfort, ability to sleep, activity level and quality of life. The Postoperative Patient Diary will be administered daily on the day of surgery and each morning and each evening before the patient retires for 3 postoperative days. All pain medications taken during the 3 days of the postoperative evaluation will be recorded on the Postoperative Patient Diary. The investigational treatment for this investigation is the use of a Pain Care 3000 continuous infusion regional anesthesia device and accepted pharmacologic modalities for the control of postoperative pain with continuous infusion of a local anesthetic agent (bupivicaine 0.5%) intraarticularly only (Group 1), at the patellar tendon harvest site (Group 2) and combined continuous infusion at the patellar tendon harvest site and intraarticular infusion (Group 3). Patients randomly assigned to the control group (Group 4) will receive intraarticular infusion physiologic saline only. The catheter delivers 2 cc of bupivicaine 0.5% per hour total. In patients in Group 3, with 2 catheters (1 in the patellar tendon harvest site and 1 intraarticular infusion), the catheters are connected by a "Y" connector. The dosage of bupivicaine will be split among the two sites. The device does not allow delivery of more than 2 cc per hour. The patient cannot adjust or alter the flow rate. A standard general anesthetic protocol will be employed at the discretion of the supervising anesthesiologist. Prior to surgery, all patients will be instructed on both the use of the continuous infusion device and on the methods for completing the study questionnaires. Catheters will be placed at the end of the surgical procedure according to the randomization schedule. Patients will then complete the study questionnaires on the day of surgery and on three consecutive days following surgery. The clinical evaluation will last 3 postoperative days. During the postoperative period, sufficient additional pain medications will be available to all patients to sufficiently control postoperative pain. No analgesics or local anesthetics, other than those specified should be taken during the postoperative period by any subject. Other medications such as "rescue analgesics" considered to be necessary to the patient's welfare will be given at the discretion of the principal investigator. If the medication provided is for pain relief, the patient must request it. The administration of all medication, from premedication until discharge from the hospital, must be recorded in the patient diary if it has a direct bearing on the study outcome. Any medication taken for pain following hospital discharge within the established post discharge follow up period must be recorded in the patient diary. The investigator is responsible for assuring that there are procedures and expertise available to cope with medical emergencies that may occur during the study. In case of emergency, symptomatic treatment will be provided according to hospital routine. The reason for the emergency may constitute a serious adverse event. After surgery, a patient would be discontinued from the study if it is discovered that they show a previously unrecognized allergy to the medication (bupivicaine). This would be rare. If it occurs, the catheter would be immediately removed, thus ending the infusion of the medication and the patient would no longer participate in the study. Standard of Care Procedures: Opioid based analgesics or their derivatives have been the standard of care in managing postoperative pain for this procedure. These medications have been administered orally, intramuscularly, or intravenously. There is no difference in the surgical procedure except for the insertion of the catheter at the surgical site upon closure of the surgical wound. All patients will receive Toradol 30 mg parenterally q6h x2, followed by Toradol 10 mg PO QID for 3 days. This anti-inflammatory has an analgesic effect and is not used prn. Any narcotic is used only as needed and the use of this will be tracked for this study. All patients (including those in the study) will be kept in the hospital overnight following surgery and will receive a patient controlled analgesia (PCA) device for intravenous administration of morphine through the night of the hospital stay. Morphine usage during this period will be recorded. Patients undergoing this knee surgery are admitted to the 23 hour unit, stay overnight, and are discharged the following morning usually before 10:00 am. This is the standard of care and will be the same for those participating in the study.
Pain, Postoperative
anterior cruciate ligament reconstruction Pain Care 3000 Catheter Placement Pain Breg, Inc
null
4
arm 1: Breg Pain Care 3000 Catheter;Bupivicaine 0.5%;Intraaticular Only Continuous infusion of a local anesthetic agent (bupivicaine 0.5%) intraarticularly only via Breg Pain Care 3000 Catheter arm 2: Breg Pain Care 3000 Catheter;Bupivicaine 0.5%;Patellar Tendon Site Only Continuous infusion of a local anesthetic agent (bupivicaine 0.5%) at the patellar tendon harvest site via Breg Pain Care 3000 Catheter arm 3: Breg Pain Care 3000 Catheter;Bupivicaine 0.5%; Intraarticular and Patellar Tendon Sites Continuous infusion of a local anesthetic agent (bupivicaine 0.5%) at the Patellar Tendon Harvest Site and Intraarticular infusion via Breg Pain Care 3000 Catheter arm 4: Breg Pain Care 3000 Catheter with Placebo Receive liquid with no pain medication (placebo) through a catheter in one part of the operative knee via Breg Pain Care 3000 Catheter
[ 0, 0, 0, 2 ]
3
[ 1, 0, 0 ]
intervention 1: Device: Breg Pain Care 3000 Catheter intervention 2: Drug: Bupivicaine 0.5 % intervention 3: Drug: Placebo
intervention 1: Breg Pain Care 3000 Catheter intervention 2: Bupivicaine 0.5% intervention 3: Placebo
1
Rochester | New York | United States | -77.61556 | 43.15478
0
NCT00178178
[ 3 ]
84
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
null
The purpose of the study is to describe the tumor response rates for the two regimens being studied, and to determine how long patients live after receiving the treatment, how long patients are without return of their disease after they receive treatment, and how long the response they get from the treatment lasts. The amount and type of side effects/toxicities of each regimen will also be evaluated. The regimen including Oxaliplatin + 5FU/Folinic Acid is a current standard of care.
null
Colorectal Cancer
null
2
arm 1: Avastin + Gemcitabine + 5-Fluorouracil (5FU)/Folinic Acid arm 2: Avastin + Oxaliplatin + 5-Fluorouracil (5FU)/Folinic Acid
[ 0, 1 ]
4
[ 0, 0, 0, 0 ]
intervention 1: 900 mg/m2 IV over 90 minutes after Folinic Acid weekly for 6 weeks every 8 weeks until progression. intervention 2: 5 milligrams per kilogram (mg/kg) intervention 3: Folinic Acid: 100 milligrams per square meter (mg/m2) intravenous (IV) over 60 minutes (A+FFG). Folinic Acid: 200 mg/m2 as a 2-hr infusion on Days 1 and 2 of a 14-day cycle (A+FOLFOX 4). 5-Fluorouracil: 450 mg/m2 as an IV bolus in middle of Folinic Acid (A+FFG). 5-Fluorouracil: 400 mg/m2 bolus plus a 600 mg/m2 22-hour infusion on Days 1 and 2 of a 14-day cycle (A+FOLFOX 4). intervention 4: 85 mg/m2 as a 2-hour infusion on Day 1 of a 14 day cycle.
intervention 1: Gemcitabine intervention 2: avastin intervention 3: 5FU/folinic acid intervention 4: oxaliplatin
15
Tucson | Arizona | United States | -110.92648 | 32.22174 Beverly Hills | California | United States | -118.40036 | 34.07362 Palm Springs | California | United States | -116.54529 | 33.8303 Stockton | California | United States | -121.29078 | 37.9577 Kansas City | Kansas | United States | -94.62746 | 39.11417 Springfield | Massachusetts | United States | -72.58981 | 42.10148 Lansing | Michigan | United States | -84.55553 | 42.73253 Tupelo | Mississippi | United States | -88.70464 | 34.25807 Stony Brook | New York | United States | -73.14094 | 40.92565 The Bronx | New York | United States | -73.86641 | 40.84985 Burlington | North Carolina | United States | -79.4378 | 36.09569 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Springfield | Ohio | United States | -83.80882 | 39.92423 Marshfield | Wisconsin | United States | -90.1718 | 44.66885 Milwaukee | Wisconsin | United States | -87.90647 | 43.0389
0
NCT00192075
[ 4 ]
753
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
The purpose of this study is to investigate whether a long-acting injectable formulation of risperidone provides better effectiveness over 2 years, as measured by the time to relapse, compared with quetiapine tablets in a routine psychiatric care setting. Aripiprazole will be investigated in a descriptive manner.
Although many schizophrenia patients currently take oral antipsychotic medications, it is estimated that up to 75% of them have difficulty adhering to the daily oral regimen. Long-acting injectable formulations may eliminate the need for daily medication and enhance patient compliance with the treatment regimen. This is an open-label (all people involved know the identity of the intervention), randomized (study drug assigned by chance) study of a formulation of risperidone (coated microspheres) injected into the muscle at 2 week intervals over 104 weeks in stable patients with schizophrenia or schizoaffective disorder, who are being treated with oral risperidone, olanzapine, or other conventional antipsychotic agents. A comparator group will receive tablets of quetiapine to be taken 2 or 3 times daily, depending on the optimal dosage. In countries where aripiprazole is available, aripiprazole was also included in a descriptive manner. Reasons for switching symptomatically stable patients from their current antipsychotic treatment include insufficient effectiveness of the medication on symptoms, adverse events, or a patient's request. The principal measure of effectiveness of the drug is the time to relapse. Assessments of effectiveness also include: Positive and Negative Syndrome Scale (PANSS), which measures the symptoms of schizophrenia; overall severity of illness measured by the Clinical Global Impression subscale (CGI-S); patient's condition measured by the Clinical Global Impression condition subscale (CGI-C); quality of life assessed by the SF-12 survey. Safety evaluations include incidence of adverse events, Extrapyramidal Symptoms Rating Scale (ESRS), clinical laboratory tests (biochemistry, haematology, and urinalysis), and vital signs (pulse, blood pressure). The study hypothesis is that treatment with long-acting risperidone injected intramuscularly every 2 weeks provides better effectiveness than quetiapine, as measured by time to relapse, in patients with schizophrenia or schizoaffective disorder. Risperidone injections 25mg biweekly for 104 weeks, increasing or decreasing (increments of 12.5mg) at investigator's discretion. Risperidone tablets (2mg daily for 2 days) for patients starting on risperidone. Quetiapine and Aripiprazole used according to package insert.
Schizophrenia Psychotic Disorders
Schizophrenia, relapse prevention Antipsychotic agents Long-acting risperidone Quetiapine Aripiprazole
null
3
arm 1: Risperidone Long Acting Injectable (LAI) 25 mg injection every 2 weeks until week 104. Dosage may be increased or decreased in steps of 12.5 mg. Additional oral risperidone can be administered as required until a dose increase becomes effective. arm 2: Quetiapine Oral tablets are titrated from 50 mg daily to 300-400 mg daily in first 4 days. Subsequently treatment is maintained for 104 weeks and dosage can be adjusted with increments or decrements of 25 to 50 mg. arm 3: Aripiprazole 10-30 mg oral once daily for 104 weeks
[ 0, 1, 5 ]
3
[ 0, 0, 0 ]
intervention 1: 10-30 mg oral once daily for 104 weeks intervention 2: 25 mg injection every 2 weeks until week 104. Dosage may be increased or decreased in steps of 12.5 mg. Additional oral risperidone can be administered as required until a dose increase becomes effective. intervention 3: Oral tablets are titrated from 50 mg daily to 300-400 mg daily in first 4 days. Subsequently treatment is maintained for 104 weeks and dosage can be adjusted with increments or decrements of 25 to 50 mg.
intervention 1: Aripiprazole intervention 2: Risperidone Long Acting Injectable (LAI) intervention 3: Quetiapine
96
Hall in Tirol | N/A | Austria | 11.51667 | 47.28333 Linz | N/A | Austria | 14.28611 | 48.30639 Neunkirchen | N/A | Austria | 16.08107 | 47.72096 Pleven | N/A | Bulgaria | 24.61667 | 43.41667 Sofia | N/A | Bulgaria | 23.32415 | 42.69751 Sofia Sofia | N/A | Bulgaria | N/A | N/A Osijek | N/A | Croatia | 18.69389 | 45.55111 Rijeka | N/A | Croatia | 14.44241 | 45.32673 Split | N/A | Croatia | 16.43915 | 43.50891 Zagreb | N/A | Croatia | 15.97798 | 45.81444 Brno | N/A | Czechia | 16.60796 | 49.19522 Lnáře | N/A | Czechia | 13.78406 | 49.4579 Opava | N/A | Czechia | 17.90257 | 49.93866 Pardubice | N/A | Czechia | 15.77659 | 50.04075 Plzen Czechia | N/A | Czechia | N/A | N/A Prague | N/A | Czechia | 14.42076 | 50.08804 Praha 2 N/A | N/A | Czechia | N/A | N/A Uherský Brod | N/A | Czechia | 17.64715 | 49.02513 Ústí nad Labem | N/A | Czechia | 14.03227 | 50.6607 Middelfart | N/A | Denmark | 9.73054 | 55.50591 Vordingborg | N/A | Denmark | 11.91057 | 55.00801 Pÿrnu N/A | N/A | Estonia | N/A | N/A Tallinn | N/A | Estonia | 24.75353 | 59.43696 Tartu | N/A | Estonia | 26.72509 | 58.38062 Bron | N/A | France | 4.91303 | 45.73865 Brumath | N/A | France | 7.71095 | 48.73398 Créteil | N/A | France | 2.46569 | 48.79266 Dieppe | N/A | France | 1.07772 | 49.9216 Hénin-Beaumont | N/A | France | 2.96485 | 50.41359 Mont-Saint-Martin | N/A | France | 5.78337 | 49.54363 Poitiers | N/A | France | 0.34348 | 46.58261 Reims | N/A | France | 4.02853 | 49.26526 Roubaix | N/A | France | 3.17456 | 50.69421 Toulouse | N/A | France | 1.44367 | 43.60426 Augsburg | N/A | Germany | 10.89851 | 48.37154 Berlin | N/A | Germany | 13.41053 | 52.52437 Bochum | N/A | Germany | 7.21648 | 51.48165 Duisburg | N/A | Germany | 6.76516 | 51.43247 Düsseldorf | N/A | Germany | 6.77616 | 51.22172 Karlstadt am Main | N/A | Germany | 9.77239 | 49.96034 Krefeld | N/A | Germany | 6.55381 | 51.33645 München | N/A | Germany | 13.31243 | 51.60698 Oranienburg | N/A | Germany | 13.24197 | 52.75577 Stralsund | N/A | Germany | 13.0818 | 54.30911 Heraklion -Crete | N/A | Greece | N/A | N/A Thessalonikis | N/A | Greece | N/A | N/A Budapest | N/A | Hungary | 19.04045 | 47.49835 Gyõr | N/A | Hungary | N/A | N/A Gyula | N/A | Hungary | 21.28333 | 46.65 Kistarcsa | N/A | Hungary | 19.26247 | 47.54757 Szeged | N/A | Hungary | 20.14824 | 46.253 Vác | N/A | Hungary | 19.13612 | 47.77591 Cork | N/A | Ireland | -8.47061 | 51.89797 Dublin | N/A | Ireland | -6.24889 | 53.33306 Kerry | N/A | Ireland | N/A | N/A Sligo | N/A | Ireland | -8.46943 | 54.26969 Jerusalem | N/A | Israel | 35.21633 | 31.76904 Ramat Gan | N/A | Israel | 34.81065 | 32.08227 Tel Aviv | N/A | Israel | 34.78057 | 32.08088 Jelgava | N/A | Latvia | 23.71278 | 56.65 Riga | N/A | Latvia | 24.10589 | 56.946 Alytus | N/A | Lithuania | 24.04142 | 54.39635 Kaunas | N/A | Lithuania | 23.90961 | 54.90272 Klaipėda | N/A | Lithuania | 21.13912 | 55.7068 Šiauliai | N/A | Lithuania | 23.31667 | 55.93333 Vilnius | N/A | Lithuania | 25.2798 | 54.68916 Choroszcz | N/A | Poland | 22.98889 | 53.14332 Gdynia Na | N/A | Poland | N/A | N/A Poznan | N/A | Poland | 16.92993 | 52.40692 Warsaw | N/A | Poland | 21.01178 | 52.22977 Coimbra | N/A | Portugal | -8.41955 | 40.20564 Porto | N/A | Portugal | -8.61099 | 41.14961 Bucharest | N/A | Romania | 26.10626 | 44.43225 Craiova | N/A | Romania | 23.8 | 44.31667 Iași | N/A | Romania | 27.6 | 47.16667 Khobar | N/A | Saudi Arabia | 50.20833 | 26.27944 Košice | N/A | Slovakia | 21.25808 | 48.71395 Zvolen | N/A | Slovakia | 19.15324 | 48.57442 Begunje na Gorenjskem | N/A | Slovenia | 14.21667 | 46.38333 Ljubljana | N/A | Slovenia | 14.50513 | 46.05108 Ormož | N/A | Slovenia | 16.15444 | 46.41139 Madrid | N/A | Spain | -3.70256 | 40.4165 Oviedo (Asturias) | N/A | Spain | -5.84476 | 43.36029 Seville | N/A | Spain | -5.97317 | 37.38283 Valencia | N/A | Spain | -0.37966 | 39.47391 Gothenburg | N/A | Sweden | 11.96679 | 57.70716 Trollhättan | N/A | Sweden | 12.28864 | 58.28365 Ankara Turkey | N/A | Turkey (Türkiye) | N/A | N/A Bakirkoy/Istanbul N/A | N/A | Turkey (Türkiye) | N/A | N/A Istanbul | N/A | Turkey (Türkiye) | 28.94966 | 41.01384 Izmir | N/A | Turkey (Türkiye) | 27.13838 | 38.41273 Barnet | N/A | United Kingdom | -0.2 | 51.65 Barnsley | N/A | United Kingdom | -1.48333 | 53.55 Hull | N/A | United Kingdom | -0.33525 | 53.7446 Llantrissant | N/A | United Kingdom | N/A | N/A Swansea | N/A | United Kingdom | -3.94323 | 51.62079
0
NCT00216476
[ 4 ]
460
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
To compare the efficacy and safety of aliskiren in combination with losartan compared to losartan on the regression of the increased size of the left ventricle in overweight patients with high blood pressure.
null
Hypertension Left Ventricular Hypertrophy Overweight
null
3
arm 1: Patients in this arm initially received 150 mg of aliskiren for two weeks and were then force-titrated up to 300 mg of aliskiren where they remained for 34 weeks. In order to adequately blind the study, patients were required to take a total of 2 tablets and 1 capsule of study medication or placebo per day. In the first 2 weeks, patients took 1 tablet of aliskiren 150 mg, 1 tablet of aliskiren 150 mg placebo, and 1 capsule of losartan placebo. In the remaining 34 weeks, patients took 2 tablets of aliskiren 150 mg and 1 capsule of losartan placebo. Each dose was to be taken by mouth with water at approximately 8:00 AM, except on the morning of study visit when the dose was taken after all procedures and assessments had been completed. arm 2: Patients in this arm initially received 50 mg of losartan for two weeks and were then force-titrated up to 100 mg of losartan where they remained for 34 weeks. In order to adequately blind the study, patients were required to take a total of 2 tablets and 1 capsule of study medication or placebo per day. In the first 2 weeks, patients took 2 tablets of aliskiren 150 mg placebo and 1 capsule of losartan 50 mg. In the remaining 34 weeks, patients took 2 tablets of aliskiren 150 mg placebo and 1 capsule of losartan 100 mg. Each dose was to be taken by mouth with water at approximately 8:00 AM, except on the morning of study visit when the dose was taken after all procedures and assessments had been completed. arm 3: Patients in this arm initially received 150 mg of aliskiren in combination with 50 mg of losartan for two weeks and were then force-titrated up to 300 mg of aliskiren in combination with 100 mg of losartan where they remained for 34 weeks. In order to adequately blind the study, patients were required to take a total of 2 tablets and 1 capsule of study medication or placebo per day. In the first 2 weeks, patients took 1 tablet of aliskiren 150 mg, 1 tablet of aliskiren 150 mg placebo, and 1 capsule of losartan 50 mg. In the remaining 34 weeks, patients took 2 tablets of aliskiren 150 mg and 1 capsule of losartan 100 mg. Each dose was to be taken by mouth with water at approximately 8:00 AM, except on the morning of study visit when the dose was taken after all procedures and assessments had been completed.
[ 0, 1, 0 ]
4
[ 0, 0, 0, 0 ]
intervention 1: Aliskiren 150 mg tablets intervention 2: Losartan 50 or 100 mg capsules intervention 3: Aliskiren 150 mg placebo tablet intervention 4: Losartan 50/100 mg placebo capsules
intervention 1: Aliskiren 150/300 mg intervention 2: Losartan 50/100 mg intervention 3: Aliskiren placebo intervention 4: Losartan 50/100 mg placebo
9
East Hanover | New Jersey | United States | -74.36487 | 40.8201 Argentina | N/A | Argentina | -62.26693 | -29.53532 Colombia | N/A | Colombia | -74.8015 | 3.37606 Finland | N/A | Finland | N/A | N/A Germany | N/A | Germany | N/A | N/A Italy | N/A | Italy | N/A | N/A Russia | N/A | Russia | N/A | N/A Spain | N/A | Spain | N/A | N/A Sweden | N/A | Sweden | N/A | N/A
0
NCT00219141
[ 3 ]
94
RANDOMIZED
CROSSOVER
0TREATMENT
4QUADRUPLE
false
0ALL
true
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
This is a multi-center phase II trial of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Subjects will undergo two baseline assessments over 4 to 6 week period, then will be randomized to treatment or placebo for the next six months. All subjects will then be placed on active treatment for the subsequent six month period. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Subjects will undergo two baseline assessments over a four to six week period, followed by one year active treatment with VPA and carnitine. Outcome measures are performed every 3 to 6 months, and include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) SMA Type 2 SMA Type 3
null
3
arm 1: Patients in Cohort 1a - Placebo Comparator, will be on a placebo for 6 months and then will switch to the active treatment. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form. arm 2: Cohort 1b - Active Comparator will be on treatment throughout the study. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form. arm 3: Cohort 2 pts are on open-label treatment throughout. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.
[ 2, 1, 0 ]
2
[ 0, 0 ]
intervention 1: VPA,sprinkle cap; Levocarnitine, syrup; dosage is by weight intervention 2: None
intervention 1: Valproic Acid and Levocarnitine intervention 2: Placebo
6
Baltimore | Maryland | United States | -76.61219 | 39.29038 Detroit | Michigan | United States | -83.04575 | 42.33143 Columbus | Ohio | United States | -82.99879 | 39.96118 Salt Lake City | Utah | United States | -111.89105 | 40.76078 Madison | Wisconsin | United States | -89.40123 | 43.07305 Montreal | Quebec | Canada | -73.58781 | 45.50884
0
NCT00227266
[ 3 ]
9
NA
SINGLE_GROUP
1PREVENTION
0NONE
false
0ALL
false
This study is designed to confirm the safety of the proposed dose and schedule of ABI-007 for hemodialysis patients with vascular access device failure, and to obtain preliminary data on the effectiveness of such treatment.
This is an open-label, pilot, phase II study to assess the feasibility of administering ABI-007 intravenously \[IV\] over 3-5 minutes to patients with hemodialysis graft dysfunction (i.e., either a thrombosed polytetrafluoroethylene (PTFE) graft, or a patent but dysfunctional PTFE graft). Patients with graft dysfunction who are successfully treated with angioplasty will start treatment after angioplasty or during their first dialysis through the graft following intervention, and will receive 3 subsequent treatments of ABI-007 at 35 mg/m\^2 on weeks 5, 13 and 21.
Hemodialysis Graft Dysfunction
Vascular Access Graft Failure Venous Neointimal Hyperplasia(VNH) Hemodialysis
null
1
arm 1: ABI-007 35 mg/m\^2 given intravenously (IV) into the arteriovenous (AV) graft within 96 hours after angioplasty, followed by repeat treatment during weeks 5, 13 and 21
[ 0 ]
1
[ 0 ]
intervention 1: None
intervention 1: ABI-007
4
Peoria | Illinois | United States | -89.58899 | 40.69365 Shreveport | Louisiana | United States | -93.75018 | 32.52515 Rochester | New York | United States | -77.61556 | 43.15478 Cincinnati | Ohio | United States | -84.51439 | 39.12711
0
NCT00249002
[ 3 ]
212
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
1FEMALE
null
The primary objective of this study is to assess the effect of AMG 162 (denosumab) treatment on the lumbar spine BMD at month 12 and safety profile across the dose range tested (14, 60 and 100 mg SC once every 6 months) in Japanese postmenopausal osteoporotic subjects compared with those treated with placebo over 12 months.
null
Osteoporosis
RANKL RANK denosumab AMG 162 osteoporosis bone turnover bone mineral density Japanese
null
4
arm 1: None arm 2: None arm 3: None arm 4: None
[ 1, 1, 2, 1 ]
4
[ 0, 0, 0, 0 ]
intervention 1: 100 mg AMG 162 (denosumab) SC every 6 months intervention 2: 60 mg AMG 162 (denosumab) SC every 6 months intervention 3: 14 mg AMG 162 (denosumab) SC every 6 months intervention 4: Placebo SC every 6 months
intervention 1: 100 mg AMG 162 intervention 2: 60 mg AMG 162 intervention 3: 14 mg AMG 162 intervention 4: Placebo
0
null
0
NCT00306189
[ 4 ]
327
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
false
The primary objective of this trial is to assess the maintenance of efficacy of gabapentin enacarbil (GEn, XP13512) taken once daily in the long-term treatment of patients suffering from Restless Legs Syndrome (RLS).
This study was a multicenter, blinded, randomized withdrawal study in subjects with primary Restless Legs Syndrome (RLS). Eligible subjects were initially enrolled in a 24-week single blind (SB) treatment period during which they received XP13512. Subjects who completed the initial treatment period and met the responder criteria were then randomized 1:1 to receive either XP13512 or placebo during the 12-week double-blind (DB) treatment period. The primary study objective was to assess the maintenance of efficacy of XP13512 1200 mg taken once daily in the long-term treatment of subjects with primary RLS. The secondary study objectives were to assess maintenance of improvements in sleep outcomes and quality of life, and to assess the safety and tolerability of XP13512 in the treatment of primary RLS subjects.
Restless Legs Syndrome
null
2
arm 1: GEn (XP13512) 1200 mg arm 2: Placebo
[ 0, 2 ]
3
[ 0, 0, 0 ]
intervention 1: 1200 mg GEn (XP13512) orally, once daily for 24 weeks followed by either 1200 mg GEn (XP13512) or placebo, orally, once daily for an additional 12 weeks intervention 2: 1200 mg GEn (XP13512), orally, once daily for 24 weeks followed by either 1200 mg GEn (XP13512) or placebo, orally, once daily for an additional 12 weeks intervention 3: Placebo, orally, once daily for 12 weeks following single blind 24-week phase
intervention 1: GEn (XP13512) intervention 2: GEn (XP13512) intervention 3: Placebo
0
null
0
NCT00311363
[ 3 ]
30
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
true
2MALE
true
Studies demonstrate that methamphetamine (meth) use is associated with high-risk sexual behavior among MSM, putting meth-using MSM at extraordinarily high risk for transmitting or acquiring HIV. No studies have tested the feasibility and acceptability of conducting pharmacologic interventions to reduce meth use and meth-associated sexual risk behavior among MSM. The purpose of this pilot study is to determine the feasibility enrolling and retaining meth-dependent MSM into a pharmacologic study of bupropion vs. placebo and measuring the tolerability of and adherence to medication among these participants.
The high rate of meth use among MSM is paralleled by evidence of rises in sexual risk behavior and HIV infection among this population. The MSM meth epidemic, and its link with HIV transmission, underscores the need to pilot test new, innovative modalities to reduce meth use and meth-associated sexual risk behavior. Ultimately, a pharmacologic treatment for meth use may not only serve to improve outcomes among those who are accessing current treatment services, but might also benefit those who are not willing or able to utilize such services. While studies show that MSM who enter substance use treatment decrease both their substance use and sexual risk behavior, current behavioral meth treatment programs report low rates of success in treating meth dependence among MSM. We believe the time has come to test the acceptability of pharmacologic interventions to reduce meth use among MSM, and to assess the feasibility of conducting such trials among sexually active, meth-dependent MSM, whose meth-associated sexual behavior use places them at extraordinarily high risk for transmitting or acquiring HIV. In this pilot study, we will provide meth-dependent MSM with placebo or daily bupropion XL (extended-release), a well-tolerated dopamine agonist that has potential to reduce meth use. The specific aims of this study are: 1. To assess the feasibility of enrolling and retaining meth-dependent MSM into a randomized, double-blind study of bupropion versus placebo with biologic (urine meth testing) and behavioral (sexual risk) measures. 2. To explore the tolerability of bupropion and placebo among meth-dependent MSM, as determined by the number of adverse clinical events in the bupropion and placebo arms. 3. To describe the acceptability of bupropion and placebo among meth-dependent MSM, by measuring (via electronic pill caps) medication adherence to bupropion and placebo. This randomized, double-blind, placebo-controlled, two-arm pilot study will enroll 30 meth-dependent MSM assigned to receive 3 months of bupropion XL 300 mg daily or placebo. We will include both HIV- and HIV-INFECTED MSM, because meth use is common in both groups. We will enroll meth-dependent MSM because they are the most likely population to benefit from this potential treatment. Participants will be seen weekly for urine specimen collection and substance-use counseling. Clinical exams, medical history, specimen collection, and behavioral assessments will be performed at baseline and at the 1, 2, and 3 month visits. Interim visits will be scheduled whenever indicated by signs or symptoms. Our decision to maintain participants on 3 months of bupropion is based on the smoking literature, which demonstrated bupropion's efficacy in treating nicotine addiction within similar time periods; we anticipate that any future efficacy trial will maintain participants on bupropion for this duration.
Substance Abuse HIV Infections
Methamphetamine HIV
null
2
arm 1: buproprion XL 300mg daily arm 2: placebo 300mg daily
[ 1, 2 ]
2
[ 0, 0 ]
intervention 1: None intervention 2: None
intervention 1: Bupropion intervention 2: Placebo
1
San Francisco | California | United States | -122.41942 | 37.77493
0
NCT00318409
[ 4 ]
1,355
NON_RANDOMIZED
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
This study is a long-term follow-up protocol for patients who participated in study NK-104-3.01EU or study NK-104-3.02EU.
Patients from the NK-104-3.01EU and NK-104-3.02EU studies will continue to receive either pitavastatin or the comparator statin for 1 year.
Hypercholesterolemia Dyslipidemia
Kowa Hypercholesterolemia dyslipidemia pitavastatin NK-104 Hypercholesterolemia or combined dyslipidemia
null
1
arm 1: Pitavastatin 4 mg once daily
[ 0 ]
1
[ 0 ]
intervention 1: Pitavastatin 4 mg once daily
intervention 1: Pitavastatin
186
Copenhagen | N/A | Denmark | 12.56553 | 55.67594 Copenhagen | N/A | Denmark | 12.56553 | 55.67594 Copenhagen Nv | N/A | Denmark | N/A | N/A Frederiksberg | N/A | Denmark | 12.53463 | 55.67938 Hellerup | N/A | Denmark | 12.57093 | 55.73204 Vejle | N/A | Denmark | 9.5357 | 55.70927 Helsinki | N/A | Finland | 24.93545 | 60.16952 Helsinki | N/A | Finland | 24.93545 | 60.16952 Helsinki | N/A | Finland | 24.93545 | 60.16952 Tampere | N/A | Finland | 23.78712 | 61.49911 Turku | N/A | Finland | 22.26869 | 60.45148 Bad Soden/Taunus | N/A | Germany | N/A | N/A Beckum | N/A | Germany | 8.04075 | 51.75571 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin-Spandau | N/A | Germany | N/A | N/A Chemnitz | N/A | Germany | 12.92922 | 50.8357 Dresden | N/A | Germany | 13.73832 | 51.05089 Frankfurt am Main | N/A | Germany | 8.68417 | 50.11552 Goch | N/A | Germany | 6.15895 | 51.67873 Hamburg | N/A | Germany | 9.99302 | 53.55073 Heidelberg | N/A | Germany | 8.69079 | 49.40768 Lampertheim | N/A | Germany | 8.4725 | 49.59786 Leipzig | N/A | Germany | 12.37129 | 51.33962 Mainz | N/A | Germany | 8.2791 | 49.98419 Mannheim | N/A | Germany | 8.46694 | 49.4891 Melcherstaette | N/A | Germany | N/A | N/A Messkirch | N/A | Germany | 9.11479 | 47.99457 Offenbach/M | N/A | Germany | N/A | N/A Weinheim | N/A | Germany | 8.66697 | 49.54887 Wiesbaden | N/A | Germany | 8.24932 | 50.08258 Worpswede | N/A | Germany | 8.93333 | 53.21667 Andhra Pradesh | N/A | India | N/A | N/A Andhra Pradesh | N/A | India | N/A | N/A Bangalore | N/A | India | 77.59369 | 12.97194 Chennai | N/A | India | 80.27847 | 13.08784 Gujarat | N/A | India | N/A | N/A Hyderabad | N/A | India | 78.45636 | 17.38405 Hyderabad | N/A | India | 78.45636 | 17.38405 Karnataka | N/A | India | N/A | N/A Maharashtra | N/A | India | N/A | N/A Maharashtra | N/A | India | N/A | N/A Maharashtra | N/A | India | N/A | N/A Maharashtra | N/A | India | N/A | N/A Maharashtra | N/A | India | N/A | N/A Maharashtra | N/A | India | N/A | N/A Mumbai | N/A | India | 72.88261 | 19.07283 New Delhi | N/A | India | 77.2148 | 28.62137 Tamil Nadu | N/A | India | N/A | N/A Tamil Nadu | N/A | India | N/A | N/A Beersheva | N/A | Israel | N/A | N/A Haifa | N/A | Israel | 34.99928 | 32.81303 Holon | N/A | Israel | 34.77918 | 32.01034 Jerusalem Ein Kerem | N/A | Israel | N/A | N/A Kfar Saba | N/A | Israel | 34.90694 | 32.175 Mount Scopus Jerusalem | N/A | Israel | N/A | N/A Safed | N/A | Israel | 35.496 | 32.96465 Tel Aviv | N/A | Israel | 34.78057 | 32.08088 Tel Litwinsky | N/A | Israel | 34.84588 | 32.05096 Bologna | N/A | Italy | 11.33875 | 44.49381 Chieti | N/A | Italy | 14.16494 | 42.34827 Ferrara | N/A | Italy | 11.62057 | 44.83804 Genova | N/A | Italy | 11.87211 | 45.21604 Modena | N/A | Italy | 10.92539 | 44.64783 Napoli | N/A | Italy | 14.5195 | 40.87618 Palermo | N/A | Italy | 13.3636 | 38.1166 Parma | N/A | Italy | 10.32618 | 44.79935 Treviglio | N/A | Italy | 9.59102 | 45.52081 Trieste | N/A | Italy | 13.77678 | 45.64953 Breda | N/A | Netherlands | 4.77596 | 51.58656 Breda | N/A | Netherlands | 4.77596 | 51.58656 Eindhoven | N/A | Netherlands | 5.47778 | 51.44083 Eindhoven | N/A | Netherlands | 5.47778 | 51.44083 Groningen | N/A | Netherlands | 6.56667 | 53.21917 Groningen | N/A | Netherlands | 6.56667 | 53.21917 Hoorn | N/A | Netherlands | 5.05972 | 52.6425 Leiden | N/A | Netherlands | 4.49306 | 52.15833 Leiden | N/A | Netherlands | 4.49306 | 52.15833 Nijmegen | N/A | Netherlands | 5.85278 | 51.8425 Nijmegen | N/A | Netherlands | 5.85278 | 51.8425 Rotterdam | N/A | Netherlands | 4.47917 | 51.9225 Rotterdam | N/A | Netherlands | 4.47917 | 51.9225 Sliedrecht | N/A | Netherlands | 4.77639 | 51.82083 Velp | N/A | Netherlands | 5.97361 | 51.995 Velp | N/A | Netherlands | 5.97361 | 51.995 Wp Tiel | N/A | Netherlands | N/A | N/A Zoetermeer | N/A | Netherlands | 4.49306 | 52.0575 Zoetermeer | N/A | Netherlands | 4.49306 | 52.0575 Kongsberg | N/A | Norway | 9.65017 | 59.66858 Oslo | N/A | Norway | 10.74609 | 59.91273 Oslo | N/A | Norway | 10.74609 | 59.91273 Skedsmokorset | N/A | Norway | 11.03278 | 60.00459 Bialystok | N/A | Poland | 23.16433 | 53.13333 Gruziadz | N/A | Poland | N/A | N/A Katowice | N/A | Poland | 19.02754 | 50.25841 Siedlce | N/A | Poland | 22.29006 | 52.16772 Tarnów | N/A | Poland | 20.98698 | 50.01381 Tychy | N/A | Poland | 18.96641 | 50.13717 Warsaw | N/A | Poland | 21.01178 | 52.22977 Warsaw | N/A | Poland | 21.01178 | 52.22977 Warsaw | N/A | Poland | 21.01178 | 52.22977 Łosice | N/A | Poland | 22.71801 | 52.21129 Kemerovo | N/A | Russia | 86.08333 | 55.33333 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Novosibirsk | N/A | Russia | 82.94339 | 55.03442 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 San Juan | Alicante | Spain | -1.16667 | 39.53333 Villaroel | Barcelona | Spain | N/A | N/A Alicante | N/A | Spain | -0.48149 | 38.34517 Barcelona | N/A | Spain | 2.15899 | 41.38879 Barcelona | N/A | Spain | 2.15899 | 41.38879 Barcelona | N/A | Spain | 2.15899 | 41.38879 Córdoba | N/A | Spain | -4.77275 | 37.89155 Madrid | N/A | Spain | -3.70256 | 40.4165 Madrid | N/A | Spain | -3.70256 | 40.4165 Madrid | N/A | Spain | -3.70256 | 40.4165 Madrid | N/A | Spain | -3.70256 | 40.4165 Oviedo | N/A | Spain | -5.84476 | 43.36029 Santander | N/A | Spain | -3.80444 | 43.46472 Santiago de Compostela | N/A | Spain | -8.54569 | 42.88052 Seville | N/A | Spain | -5.97317 | 37.38283 Valencia | N/A | Spain | -0.37966 | 39.47391 Valencia | N/A | Spain | -0.37966 | 39.47391 Zaragoza | N/A | Spain | -0.87734 | 41.65606 Zaragoza | N/A | Spain | -0.87734 | 41.65606 Angelhom | N/A | Sweden | N/A | N/A Gothenburg | N/A | Sweden | 11.96679 | 57.70716 Helsingborg | N/A | Sweden | 12.69437 | 56.04673 Karineholm | N/A | Sweden | N/A | N/A Ludvika | N/A | Sweden | 15.18776 | 60.14959 Sandviken | N/A | Sweden | 16.76667 | 60.61667 Sandviken | N/A | Sweden | 16.76667 | 60.61667 Södertälje | N/A | Sweden | 17.62525 | 59.19554 Stockholm | N/A | Sweden | 18.06871 | 59.32938 Varberg | N/A | Sweden | 12.25078 | 57.10557 Bath | N/A | United Kingdom | -2.36172 | 51.3751 Bath | N/A | United Kingdom | -2.36172 | 51.3751 Bath | N/A | United Kingdom | -2.36172 | 51.3751 Bath | N/A | United Kingdom | -2.36172 | 51.3751 Birmingham | N/A | United Kingdom | -1.89983 | 52.48142 Bolton | N/A | United Kingdom | -2.43333 | 53.58333 Chesterfield | N/A | United Kingdom | -1.41667 | 53.25 Chorley | N/A | United Kingdom | -2.61667 | 53.65 Cornwall | N/A | United Kingdom | N/A | N/A Dronfield | N/A | United Kingdom | -1.47507 | 53.30221 Irvine | N/A | United Kingdom | -4.65508 | 55.6194 Liverpool | N/A | United Kingdom | -2.97794 | 53.41058 Maidenhead | N/A | United Kingdom | -0.71986 | 51.52279 Manchester | N/A | United Kingdom | -2.23743 | 53.48095 Nottingham | N/A | United Kingdom | -1.15047 | 52.9536 Plymouth | N/A | United Kingdom | -4.14305 | 50.37153 Reading | N/A | United Kingdom | -0.97113 | 51.45625 Sheffield | N/A | United Kingdom | -1.4659 | 53.38297 Sheffield | N/A | United Kingdom | -1.4659 | 53.38297 Southampton | N/A | United Kingdom | -1.40428 | 50.90395 Surrey | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Wiltshire | N/A | United Kingdom | N/A | N/A Yorkshire | N/A | United Kingdom | N/A | N/A
0
NCT00325780
[ 4 ]
747
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
false
This study, will compare pregabalin with placebo for the duration of 14 weeks to evaluate the efficacy and safety of pregabalin in patients with fibromyalgia.
null
Fibromyalgia
null
4
arm 1: None arm 2: None arm 3: None arm 4: None
[ 0, 0, 0, 2 ]
4
[ 0, 0, 0, 0 ]
intervention 1: 600mg/day intervention 2: 450mg/day intervention 3: 300mg/day intervention 4: placebo
intervention 1: pregabalin intervention 2: pregabalin intervention 3: pregabalin intervention 4: placebo
74
Warrawong | New South Wales | Australia | 150.88833 | -34.485 Maroochydore | Queensland | Australia | 153.09953 | -26.66008 Clayton | Victoria | Australia | 145.11667 | -37.91667 Fitzroy | Victoria | Australia | 144.97833 | -37.79839 Kelowna | British Columbia | Canada | -119.48568 | 49.88307 Winnipeg | Manitoba | Canada | -97.14704 | 49.8844 Bathurst | New Brunswick | Canada | -65.65112 | 47.61814 St. John's | Newfoundland and Labrador | Canada | -52.70931 | 47.56494 Hawkesbury | Ontario | Canada | -74.61595 | 45.60009 Toronto | Ontario | Canada | -79.39864 | 43.70643 Drummondville | Quebec | Canada | -72.48241 | 45.88336 Montreal | Quebec | Canada | -73.58781 | 45.50884 Pointe-Claire | Quebec | Canada | -73.81669 | 45.44868 Québec | Quebec | Canada | -71.21454 | 46.81228 Sherbrooke | Quebec | Canada | -71.89908 | 45.40008 Sherbrooke | Quebec | Canada | -71.89908 | 45.40008 Frederiksberg | N/A | Denmark | 12.53463 | 55.67938 Svendborg | N/A | Denmark | 10.60677 | 55.05982 Montpellier | Cedex 5 | France | 3.87635 | 43.61093 Lille | Cedex | France | 3.05858 | 50.63297 Clermont-Ferrand | N/A | France | 3.08682 | 45.77969 Paris | N/A | France | 2.3488 | 48.85341 Saint-Etienne | N/A | France | 4.39 | 45.43389 Bad Säckingen | N/A | Germany | 7.94612 | 47.55371 Berlin | N/A | Germany | 13.41053 | 52.52437 Berlin | N/A | Germany | 13.41053 | 52.52437 Bonn | N/A | Germany | 7.09549 | 50.73438 Mannheim | N/A | Germany | 8.46694 | 49.4891 München | N/A | Germany | 13.31243 | 51.60698 Indore | Madhya Pradesh | India | 75.8333 | 22.71792 Ludhiana | Punjab | India | 75.85379 | 30.91204 Ludhiana | Punjab | India | 75.85379 | 30.91204 Lucknow | Uttar Pradesh | India | 80.92313 | 26.83928 Bari | N/A | Italy | 16.86982 | 41.12066 Benevento | N/A | Italy | 14.77816 | 41.1307 Bologna | N/A | Italy | 11.33875 | 44.49381 Chieti Scalo | N/A | Italy | N/A | N/A Perugia | N/A | Italy | 12.38878 | 43.1122 Pisa | N/A | Italy | 10.4036 | 43.70853 Mexico | D. F. | Mexico | -98.43784 | 18.88011 León | Guanajuato | Mexico | -101.67374 | 21.12908 Monterrey | Nuevo León | Mexico | -100.31721 | 25.68435 San Luis Potosí City | N/A | Mexico | -100.97135 | 22.15234 Alkmaar | N/A | Netherlands | 4.74861 | 52.63167 Den Helder | N/A | Netherlands | 4.75933 | 52.95988 Leeuwarden | N/A | Netherlands | 5.80859 | 53.20139 Rotterdam | N/A | Netherlands | 4.47917 | 51.9225 Zwolle | N/A | Netherlands | 6.09444 | 52.5125 Lisbon | N/A | Portugal | -9.1498 | 38.72509 Lisbon | N/A | Portugal | -9.1498 | 38.72509 Lisbon | N/A | Portugal | -9.1498 | 38.72509 Lisbon | N/A | Portugal | -9.1498 | 38.72509 Suwon | Kyeongki-do | South Korea | 127.00889 | 37.29111 Seoul | N/A | South Korea | 126.9784 | 37.566 Seoul | N/A | South Korea | 126.9784 | 37.566 Barcelona | N/A | Spain | 2.15899 | 41.38879 Barcelona | N/A | Spain | 2.15899 | 41.38879 Córdoba | N/A | Spain | -4.77275 | 37.89155 Guadalajara | N/A | Spain | -3.16185 | 40.62862 Linköping | N/A | Sweden | 15.62157 | 58.41086 Mölndal | N/A | Sweden | 12.01378 | 57.6554 Örebro | N/A | Sweden | 15.2066 | 59.27412 Stockholm | N/A | Sweden | 18.06871 | 59.32938 Lausanne | N/A | Switzerland | 6.63282 | 46.516 Zurich | N/A | Switzerland | 8.55 | 47.36667 Zurich | N/A | Switzerland | 8.55 | 47.36667 Manchester | Greater Manchester | United Kingdom | -2.23743 | 53.48095 Greenock | Renfrewshire | United Kingdom | -4.76121 | 55.94838 North Shields | Tyne and Wear | United Kingdom | -1.44925 | 55.01646 London | N/A | United Kingdom | -0.12574 | 51.50853 Poole | N/A | United Kingdom | -1.98458 | 50.71429 Caracas | Distrito Federal | Venezuela | -66.87919 | 10.48801 Caracas | Distrito Federal | Venezuela | -66.87919 | 10.48801 Caracas | Miranda | Venezuela | N/A | N/A
0
NCT00333866
[ 3 ]
19
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
The objective of the study is to determine whether mifepristone (VGX-410) has anti-HIV-1 activity at doses of either 300 mg or 600 mg per day after oral administration for 14 days at each dose level.
VT004 is a double-blind, randomized, placebo-controlled Phase II study of two doses of orally administered mifepristone (VGX-410) (300 and 600 mg) taken as a twice daily (BID) dosage (150mg BID and 300mg BID) in a dose-escalating fashion for 14 days at each dose level to determine antiviral activity and safety in HIV-1-infected participants. At entry, 18 subjects will be randomized in a 5:1 fashion (15 on active drug: 3 on placebo) to receive escalating doses of VGX-410 or matching placebo. Patients will be randomized to receive 300 mg (as 150 mg BID dose) of VGX-410 or matching placebo for 14 days. In patients that complete this dose without significant safety concerns or side effects, the dose will be increased to 600 mg (as 300 mg BID dose) or matching placebo for 14 days. Patients must meet all inclusion/exclusion criteria listed below and be seen on a weekly basis by study personnel while on study drug. Patients must return \~ one month after finishing the study therapy for examination and safety labs.
HIV Infections
HIV-1
null
2
arm 1: 150mg twice daily of VGX-410 for 14 days and, if well tolerated, a dose escalation to 300mg twice daily of VGX-410 for 14 days arm 2: 150mg twice daily of placebo for VGX-410 for 14 days and, if well tolerated, a dose escalation to 300mg twice daily of placebo for VGX-410 for 14 days
[ 1, 2 ]
2
[ 0, 0 ]
intervention 1: None intervention 2: None
intervention 1: VGX-410 (Mifepristone) intervention 2: Placebo for VGX-410 (Mifepristone)
3
Washington D.C. | District of Columbia | United States | -77.03637 | 38.89511 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238
0
NCT00352911
[ 3 ]
125
NON_RANDOMIZED
PARALLEL
2DIAGNOSTIC
0NONE
true
0ALL
false
This phase II, open-label research study was conducted in 129 healthy volunteers. Each subject will be given one initial oral dose of one of 7 FDA-approved medications (probe drugs), followed by a 7 day period where subjects receive the study medication AEGR-733 at 10 or 60 mg. On study day 8 subjects will receive the second oral dose of the same probe drug that was given on day 1 and a last dose of AEGR-733 (total of 7 doses).Subjects will return in 1 week for a final safety visit. Each FDA- approved probe drug will be given to ten (10) or fifteen (15) subjects. Safety, pharmacokinetic and pharmacodynamic assessments will be performed.
Objectives: Primary: To evaluate the effects of low and high doses of AEGR-733 on the pharmacokinetics of 6 FDA-approved medications that are likely to be used in combination with AEGR-733 as assessed by: • Pharmacokinetic parameters: Cmax, Tmax, T1/2, and AUC (area under the curve). Secondary: To evaluate the safety of AEGR-733 in combination with other lipid lowering agents in healthy subject as assessed by: * Changes in associated liver enzymes AST, ALT and, Alkaline Phosphatase, \& Total Bilirubin. * Changes in all reported adverse events. * To evaluate the effects of AEGR-733 in combination with other lipid lowering agents on the following lipids and lipoproteins: TC, LDL-C, VLDL, TG, HDL-C, ApoB and ApoAI. 4.0 STUDY DESIGN AND RATIONALE 4.1 STUDY DESIGN This is a single-center, phase II, clinical trial consisting of a eight (8) day open-label phase to assess the pharmacokinetic drug interactions of AEGR-733 on 6 probe drugs in healthy volunteers, followed by a one week safety visit. 105 subjects will be enrolled into this fixed-sequenced research study. Eligible subjects based on the screening visit will come to the GCRC for an inpatient visit (25-36 hr depending on if they come in evening before study day 1 or morning of). On the morning of study day 1, subjects will be assigned to one of 6 probe drugs(A-H below) and will take one dose of this medication. Timed blood samples will be drawn just before the administration of the probe drug and during the following times after drug administration (1,2,3,4,5,6,8,10,12,18, and 24 hrs). Prior to discharge after the 24 h blood sample, subjects will take an oral dose of AEGR-733 at 10 mg or 60 mg. Subjects will be given a 5 day supply of AEGR-733 at 10 mg or 60 mg to be taken once daily in the morning for the next 5 days (through day 7). On study day 8, subjects will take a final dose of AEGR-733 at 10 mg or 60 mg (total doses= 7) simultaneously with the same probe drug they took on day 1. Timed blood samples will be drawn just before the administration of the probe drug and AEGR-733 as well as 1,2,3,4,5,6,8,10,12,18, and 24 hours after study drug administration. After the 24 hour blood sample, subjects will be discharged. 15 subjects who participate in this study will receive dextromethorphan as the probe drug, which requires urine collection for 8 hours post dose. Blood for pharmacokinetic samples will not be collected on these subjects. Subjects receiving dextromethorphan may leave after the 8 hour urine collection at visits 2 and 3 (referred to as the inpatient visits). All subjects will come back 1 week later for a final visit to check safety lab parameters including liver transaminases and total bilirubin. Subjects will be instructed to abstain from drinking any alcoholic beverages once screened until study completion. Subjects who are not willing to comply with these requests will not be enrolled. The FDA-approved lipid-lowering therapies will include: A) Atorvastatin, 20 mg (n=15)and AEGR-733 10 mg B) Ezetimibe, 10 mg (n=10)and AEGR-733 10 mg C) Simvastatin, 20 mg (n=15)and AEGR-733 10 mg D) Rosuvastatin, 20 mg (n=10)and AEGR-733 10 mg E) Micronized fenofibrate, 145 mg (n=10)and AEGR-733 10 mg F) Atorvastatin, 20 mg (n=15) and AEGR-733 60 mg G) Rosuvastatin, 20 mg (n=15) and AEGR-733 60 mg H) Dextromethorphan, 30 mg (n=15) and AEGR-733 60 mg I) Extended Release Niacin, 1000 mg (n=20) and AEGR-733 10 mg
Healthy
healthy volunteers
null
0
null
null
6
[ 0, 0, 0, 0, 0, 0 ]
intervention 1: Atrovastatin 20 mg and AEGR-733 10 mg or 60 mg intervention 2: Simvastatin 20 mg and AEGR-733 10 mg or 60 mg intervention 3: Ezetimibe 10 mg and AEGR-733 10 mg intervention 4: Micronized Fenofibrate 145 mg and AEGR-733 10 mg intervention 5: Dextromethorphan 30 mg and AEGR-733 10 mg intervention 6: 1000 mg ER niacin and AEGR-733 10 mg
intervention 1: atorvastatin intervention 2: simvastatin intervention 3: ezetimibe intervention 4: fenofibrate intervention 5: dextromethorphan intervention 6: Extended Release Niacin
1
Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238
0
NCT00359281
[ 4 ]
404
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
false
The purpose of this study is to evaluate the long-term safety of mometasone furoate/formoterol (MF/F) metered dose inhaler (MDI) 200/10 mcg twice-a-day (BID) and MF/F MDI 400/10 mcg BID and two doses of fluticasone/salmeterol combination (F/SC) (250/50 mcg BID and 500/50 mcg BID) in subjects with persistent asthma who require maintenance treatment on inhaled glucocorticosteroids (ICS); evaluator-blind. In addition, the extrapulmonary effects on 24-hour plasma cortisol area under curve (AUC), of MF/F MDI 200/10 mcg BID, MF/F MDI 400/10 mcg BID, F/SC MDI 250/50 mcg BID, and F/SC MDI 500/50 mcg BID will be evaluated.
null
Asthma
null
4
arm 1: None arm 2: None arm 3: None arm 4: None
[ 0, 0, 1, 1 ]
4
[ 0, 0, 0, 0 ]
intervention 1: MF/F 200/10 mcg via a metered dose inhaler (MDI) twice daily for 1 year intervention 2: MF/F 400/10 mcg via a metered dose inhaler (MDI) twice daily for 1 year intervention 3: F/SC 250/50 twice daily for 1 year intervention 4: F/SC 500/50 twice daily for 1 year
intervention 1: mometasone furoate combination MDI 200/10 mcg BID intervention 2: mometasone furoate combination MDI 400/10 mcg BID intervention 3: Fluticasone/Salmeterol 250/50 mcg BID intervention 4: Fluticasone/Salmeterol 500/50 mcg BID
0
null
0
NCT00379288
[ 4 ]
169
RANDOMIZED
CROSSOVER
0TREATMENT
2DOUBLE
false
0ALL
null
This is a randomized, double-blind, placebo-controlled, crossover, two-attack, out-patient, early-intervention evaluation of subjects who have migraine with or without aura and who discontinued use of short acting triptan(s) within the past year due to non-response or intolerance. Subjects will treat 2 separate migraine attacks during the mild phase of each attack; one attack will be treated with one tablet of the Combination Product (sumatriptan succinate and naproxen sodium) and the other attack with one tablet of placebo (crossover design). \[Study 2 of 2\]
This is a randomized, double-blind, placebo-controlled, crossover, two-attack, out-patient, early-intervention evaluation of subjects who have migraine with or without aura and who discontinued use of short acting triptan(s) within the past year due to non-response or intolerance. Subjects will treat 2 separate migraine attacks during the mild phase of each attack; one attack will be treated with one tablet of the Combination Product (sumatriptan succinate and naproxen sodium) and the other attack with one tablet of placebo (crossover design); however, the order of these treatments will be randomized. A minimum 1-week washout period is required between study medication treatment of the first and second migraine attacks. Each subject will have two visits: (1) a Screening visit at study entry and (2) a Final visit 4-10 days after the second (or last) attack. A telephone contact will also be required 1-3 days after the first attack, and then once per month until the Final visit. The primary study objective is to assess efficacy as measured by sustained pain-free (SPF) relief of Combination Product compared to placebo in treating migraine subjects who have previously discontinued treatment with short acting triptans (rizatriptan, sumatriptan, almotriptan, zolmitriptan, and eletriptan).
Migraine Disorders
migraine with or without aura naproxen sodium combination product sumatriptan succinate poor response short acting triptan migraine, acute
null
2
arm 1: Combination Product (sumatriptan and naproxen sodium) \[Attack 1\] followed by Placebo \[Attack 2\] arm 2: Placebo \[Attack 1\] followed by Combination Product (sumatriptan and naproxen sodium) \[Attack 2\]
[ 5, 5 ]
2
[ 0, 0 ]
intervention 1: Matching placebo tablet intervention 2: Bilayer tablet containing 85mg sumatriptan (as 119mg sumatriptan succinate; fast disintegrating/rapid release formulation) active ingredient in one layer, and 500mg naproxen sodium active ingredient in second layer.
intervention 1: Placebo intervention 2: Combination Product (sumatriptan succinate/naproxen sodium)
28
Jasper | Alabama | United States | -87.27751 | 33.83122 Oceanside | California | United States | -117.37948 | 33.19587 San Francisco | California | United States | -122.41942 | 37.77493 Hartford | Connecticut | United States | -72.68509 | 41.76371 Aventura | Florida | United States | -80.13921 | 25.95648 Augusta | Georgia | United States | -81.97484 | 33.47097 Savannah | Georgia | United States | -81.09983 | 32.08354 Northbrook | Illinois | United States | -87.82895 | 42.12753 Des Moines | Iowa | United States | -93.60911 | 41.60054 Shreveport | Louisiana | United States | -93.75018 | 32.52515 Pikesville | Maryland | United States | -76.72247 | 39.37427 Boston | Massachusetts | United States | -71.05977 | 42.35843 Worcester | Massachusetts | United States | -71.80229 | 42.26259 Ann Arbor | Michigan | United States | -83.74088 | 42.27756 St Louis | Missouri | United States | -90.19789 | 38.62727 Omaha | Nebraska | United States | -95.94043 | 41.25626 Stratford | New Jersey | United States | -75.01545 | 39.82678 Williamsville | New York | United States | -78.73781 | 42.96395 Toledo | Ohio | United States | -83.55521 | 41.66394 Westerville | Ohio | United States | -82.92907 | 40.12617 Allentown | Pennsylvania | United States | -75.49018 | 40.60843 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 Charleston | South Carolina | United States | -79.93275 | 32.77632 Memphis | Tennessee | United States | -90.04898 | 35.14953 Nashville | Tennessee | United States | -86.78444 | 36.16589 Dallas | Texas | United States | -96.80667 | 32.78306 San Antonio | Texas | United States | -98.49363 | 29.42412 Seattle | Washington | United States | -122.33207 | 47.60621
0
NCT00382993
[ 4 ]
422
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
null
The primary purpose of this study is to evaluate the blood pressure lowering effects of ramipril, an FDA-approved drug for the treatment of hypertension in adults, in children and adolescents aged 6 to 16 years with hypertension.
Information is needed on the treatment of hypertension in children and adolescents with antihypertensive drugs like ramipril. The study will assess the safety and blood pressure effects of several doses of the antihypertensive drug ramipril in children and adolescents age 6-16 years. Approximately 450 children will be given placebo, or 1 of the 3 doses of ramipril. The treatment assigned will be done by chance, like flipping a coin. Approximately 120 study centers throughout the world will participate in the trial. Each child will complete a 1- to 4-week Screening Period where they will stop taking their current blood pressure lowering drug(s), a 4-week Treatment Period where they will receive placebo or one of the ramipril doses, and a Follow-up visit 1 week after completion of the Treatment Period. Children diagnosed with hypertension according to the fourth report on the diagnosis, evaluation and treatment of high blood pressure in children and adolescents (U.S. report), will be included in the study if their blood pressure meets certain values. Each child will complete a minimum of 6 and up to 9 clinic visits over the course of the study during which procedures and assessments of blood pressure and safety will be performed. In addition, a child's parents/guardians will be instructed to measure their child's blood pressure at home between clinic visits. A planned interim analysis was performed after approximately 240 subjects completed the trial. The study was stopped, as permitted by protocol, after the analysis.
Hypertension
Hypertension/*etiology Blood Pressure Pediatric Adolescent Child
null
4
arm 1: once per day arm 2: 0.3125, 0.625, or 1.25 mg once a day, based on subject weight arm 3: 1.25, 2.5, or 5 mg once a day, based on subject weight arm 4: 5, 10, or 20 mg once a day, based on subject weight
[ 2, 0, 0, 0 ]
2
[ 0, 0 ]
intervention 1: once a day oral ramipril capsules given for 4 weeks intervention 2: once a day oral placebo capsule for 4 weeks
intervention 1: ramipril intervention 2: placebo
100
Little Rock | Arkansas | United States | -92.28959 | 34.74648 Little Rock | Arkansas | United States | -92.28959 | 34.74648 Beverly Hills | California | United States | -118.40036 | 34.07362 Los Angeles | California | United States | -118.24368 | 34.05223 Orange | California | United States | -117.85311 | 33.78779 Cocoa Beach | Florida | United States | -80.60922 | 28.32055 Lakeland | Florida | United States | -81.9498 | 28.03947 Atlanta | Georgia | United States | -84.38798 | 33.749 Augusta | Georgia | United States | -81.97484 | 33.47097 Honolulu | Hawaii | United States | -157.85833 | 21.30694 Park Ridge | Illinois | United States | -87.84062 | 42.01114 Tinley Park | Illinois | United States | -87.78449 | 41.57337 Louisville | Kentucky | United States | -85.75941 | 38.25424 Shreveport | Louisiana | United States | -93.75018 | 32.52515 Bridgeton | Missouri | United States | -90.41151 | 38.767 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Toms River | New Jersey | United States | -74.19792 | 39.95373 Syracuse | New York | United States | -76.14742 | 43.04812 Williamsville | New York | United States | -78.73781 | 42.96395 Cary | North Carolina | United States | -78.78112 | 35.79154 Chapel Hill | North Carolina | United States | -79.05584 | 35.9132 Durham | North Carolina | United States | -78.89862 | 35.99403 Sylva | North Carolina | United States | -83.22598 | 35.37371 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Columbus | Ohio | United States | -82.99879 | 39.96118 Norman | Oklahoma | United States | -97.43948 | 35.22257 Tulsa | Oklahoma | United States | -95.99277 | 36.15398 Portland | Oregon | United States | -122.67621 | 45.52345 Portland | Oregon | United States | -122.67621 | 45.52345 Philadelphia | Pennsylvania | United States | -75.16362 | 39.95238 Kingsport | Tennessee | United States | -82.56182 | 36.54843 Memphis | Tennessee | United States | -90.04898 | 35.14953 Beaumont | Texas | United States | -94.10185 | 30.08605 Houston | Texas | United States | -95.36327 | 29.76328 Salt Lake City | Utah | United States | -111.89105 | 40.76078 Charlottesville | Virginia | United States | -78.47668 | 38.02931 Midlothian | Virginia | United States | -77.64916 | 37.50598 Norfolk | Virginia | United States | -76.28522 | 36.84681 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 San Juan | N/A | Argentina | -68.52568 | -31.53726 Tucomán | N/A | Argentina | N/A | N/A Santiago | N/A | Chile | -70.64827 | -33.45694 Santiago | N/A | Chile | -70.64827 | -33.45694 Santiago | N/A | Chile | -70.64827 | -33.45694 Viña del Mar | N/A | Chile | -71.55183 | -33.02457 Barranquilla | N/A | Colombia | -74.78132 | 10.96854 Bogotá | N/A | Colombia | -74.08175 | 4.60971 Bogotá | N/A | Colombia | -74.08175 | 4.60971 Bogotá | N/A | Colombia | -74.08175 | 4.60971 Medellín | N/A | Colombia | -75.57151 | 6.245 Medellín | N/A | Colombia | -75.57151 | 6.245 Ahmedabad | N/A | India | 72.58727 | 23.02579 Ahmedabad | N/A | India | 72.58727 | 23.02579 Bangalore | N/A | India | 77.59369 | 12.97194 Chennai | N/A | India | 80.27847 | 13.08784 Delhi | N/A | India | 77.23149 | 28.65195 Delhi | N/A | India | 77.23149 | 28.65195 Delhi | N/A | India | 77.23149 | 28.65195 Hyderabaad | N/A | India | N/A | N/A Hyderabaad | N/A | India | N/A | N/A Ludhiana | N/A | India | 75.85379 | 30.91204 Mumbai | N/A | India | 72.88261 | 19.07283 Pune | N/A | India | 73.85535 | 18.51957 Trivandrum | N/A | India | 76.94924 | 8.4855 Gdansk | N/A | Poland | 18.64912 | 54.35227 Katowice | N/A | Poland | 19.02754 | 50.25841 Lodz | N/A | Poland | 19.47395 | 51.77058 Lodz | N/A | Poland | 19.47395 | 51.77058 Poznan | N/A | Poland | 16.92993 | 52.40692 Szczecin | N/A | Poland | 14.55302 | 53.42894 Szczecin | N/A | Poland | 14.55302 | 53.42894 Torun | N/A | Poland | 18.59814 | 53.01375 Warsaw | N/A | Poland | 21.01178 | 52.22977 Wroclaw | N/A | Poland | 17.03333 | 51.1 Benoni | N/A | South Africa | 28.32078 | -26.18848 Johannesburg | N/A | South Africa | 28.04363 | -26.20227 Port Elizabeth | N/A | South Africa | 25.61494 | -33.96109 Potchefstroom | N/A | South Africa | 27.1 | -26.71667 Pretoria | N/A | South Africa | 28.18783 | -25.74486 Pretoria | N/A | South Africa | 28.18783 | -25.74486 Pretoria | N/A | South Africa | 28.18783 | -25.74486 Pretoria | N/A | South Africa | 28.18783 | -25.74486 Sandton | N/A | South Africa | 28.054 | -26.104 Istanbul | N/A | Turkey (Türkiye) | 28.94966 | 41.01384 Istanbul | N/A | Turkey (Türkiye) | 28.94966 | 41.01384 Izmir | N/A | Turkey (Türkiye) | 27.13838 | 38.41273 Samsun | N/A | Turkey (Türkiye) | 36.3361 | 41.27976 Dnipro | N/A | Ukraine | 35.04066 | 48.46664 Kharkiv | N/A | Ukraine | 36.25475 | 49.98177 Kiev | N/A | Ukraine | 30.5238 | 50.45466 Kyiv | N/A | Ukraine | 30.5238 | 50.45466 Lviv | N/A | Ukraine | 24.02324 | 49.83826 Odesa | N/A | Ukraine | 30.74383 | 46.48572 Simferopol | N/A | Ukraine | 34.11079 | 44.95719 Uzhhorod | N/A | Ukraine | 22.2947 | 48.6242 Vinnitsa | N/A | Ukraine | 37.71861 | 49.84639
0
NCT00389519
[ 4 ]
372
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
The purpose of this study is to evaluate efficacy and safety of pregabalin in the treatment of postherpetic neuralgia in a dose-ranging manner.
null
Neuralgia, Postherpetic
null
4
arm 1: None arm 2: None arm 3: None arm 4: None
[ 2, 0, 0, 0 ]
4
[ 0, 0, 0, 0 ]
intervention 1: placebo, oral administration for 13 weeks (1 week titration and 12-week fixed dose). intervention 2: Pregabalin 150mg/day (75mg BID), oral administration for 13 weeks (1-week titration and 12-week fixed dose). intervention 3: Pregabalin 300mg/day (150mg/BID), oral administration for 13 weeks (1-week titration and 12-week fixed dose). intervention 4: Pregabalin 600 mg/day (300 mg/BID), oral administration for 13 weeks (1-week titration and 12-week fixed dose).
intervention 1: Placebo intervention 2: Pregabalin intervention 3: Pregabalin intervention 4: Pregabalin
39
Nagoya | Aichi-ken | Japan | 136.90641 | 35.18147 Ichikawa | Chiba | Japan | 139.9065 | 35.73413 Urayasu | Chiba | Japan | 139.90055 | 35.65879 Kasuga | Fukuoka | Japan | 130.4611 | 33.52594 Kasuya-gun | Fukuoka | Japan | N/A | N/A Maebaru-chūō | Fukuoka | Japan | 130.20148 | 33.55916 Maebashi | Gunma | Japan | 139.08333 | 36.4 Takasaki | Gunma | Japan | 139.01667 | 36.33333 Asahikawa | Hokkaido | Japan | 142.36489 | 43.77063 Sapporo | Hokkaido | Japan | 141.35 | 43.06667 Akashi | Hyōgo | Japan | 135.00687 | 34.65524 Amagasaki | Hyōgo | Japan | 135.41667 | 34.71667 Himeji | Hyōgo | Japan | 134.7 | 34.81667 Kobe | Hyōgo | Japan | 135.183 | 34.6913 Nishinomiya | Hyōgo | Japan | 135.33199 | 34.71562 Tsuchiura | Ibaraki | Japan | 140.21047 | 36.09047 Sagamihara | Kanagawa | Japan | 139.24167 | 35.56707 Yokohama | Kanagawa | Japan | 139.65 | 35.43333 Sendai | Miyagi | Japan | 140.86667 | 38.26667 Beppu | Ohita | Japan | 131.49751 | 33.27945 Kishiwada | Osaka | Japan | 135.36667 | 34.46667 Takatsuki | Osaka | Japan | 135.61678 | 34.84833 Kawaguchi | Saitama | Japan | 139.71072 | 35.80521 Kitamoto | Saitama | Japan | 139.53775 | 36.03322 Tokorozawa | Saitama | Japan | 139.46903 | 35.79916 Adachi-ku | Tokyo | Japan | N/A | N/A Arakawa-ku | Tokyo | Japan | N/A | N/A Bunkyo-ku | Tokyo | Japan | N/A | N/A Edogawa-ku | Tokyo | Japan | N/A | N/A Mitaka | Tokyo | Japan | 139.56002 | 35.68361 Nakano-ku | Tokyo | Japan | N/A | N/A Shinagawa-ku | Tokyo | Japan | N/A | N/A Shinjuku-ku | Tokyo | Japan | N/A | N/A Suginami-ku | Tokyo | Japan | N/A | N/A Toshima-ku | Tokyo | Japan | N/A | N/A Fukuoka | N/A | Japan | 130.41667 | 33.6 Fukushima | N/A | Japan | 140.46667 | 37.75 Osaka | N/A | Japan | 135.50107 | 34.69379 Saitama | N/A | Japan | 139.65657 | 35.90807
0
NCT00394901
[ 4 ]
394
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
The purpose of this study is to evaluate the effectiveness and safety of Extended Release Osmotic Controlled-Release Oral Delivery System (OROS) Paliperidone compared to placebo in patients with Schizophrenia. Olanzapine will be used as a reference drug in the study.
This is a multicenter, double blind (neither the patient nor the physician knows whether drug or placebo is being taken, or at what dosage), randomized (patients are assigned different treatments based on chance), placebo- and active-controlled, parallel-group study. Patients will be randomized into 1 of 3 treatment groups to receive oral dosages of Extended Release (ER) Osmotic Controlled-Release Oral Delivery System (OROS) paliperidone 6 mg, olanzapine 10 mg, or placebo. They will receive two capsules of Paliperidone ER 3 mg, placebo or Olanzapine 5 mg once daily after breakfast for 6 weeks. The study will include a screening period, followed by double-blind treatment for 6 weeks.
Schizophrenia
Schizophrenia JNS007ER Positive and Negative Syndrome Scale (PANSS) Paliperidone Extended Release Osmotic Controlled-Release Oral Delivery System (OROS) paliperidone
null
3
arm 1: Extended Release (ER) Osmotic Controlled-Release Oral Delivery System (OROS) paliperidone arm 2: None arm 3: None
[ 0, 2, 1 ]
3
[ 0, 0, 0 ]
intervention 1: Type= exact number, unit= mg, number= 3, form= tablet, route= oral use. Two tablets once daily for 6 weeks. intervention 2: Form= tablet, route= oral use. Two tablets once daily for 6 weeks. intervention 3: Type= exact number, unit= mg, number= 2.5, form= tablet, route= oral use. Four tablets once daily for 6 weeks.
intervention 1: ER OROS paliperidone intervention 2: Placebo intervention 3: Olanzapine
56
Aizu-Wakamatsu | N/A | Japan | 139.94546 | 37.49142 Fujioka | N/A | Japan | 139.07204 | 36.24624 Fujisawa | N/A | Japan | 139.47666 | 35.34926 Fukui | N/A | Japan | 135.54836 | 34.84214 Hadano | N/A | Japan | 139.22361 | 35.37111 Himeji | N/A | Japan | 134.7 | 34.81667 Hiratsuka | N/A | Japan | 139.33735 | 35.32785 Hiroshima | N/A | Japan | 132.45 | 34.4 Ibaraki | N/A | Japan | 135.56828 | 34.81641 Ichikawa | N/A | Japan | 139.9065 | 35.73413 Iida | N/A | Japan | 137.82074 | 35.51965 Inazawa | N/A | Japan | 136.78333 | 35.25 Itoman | N/A | Japan | 127.66918 | 26.12647 Kaizuka | N/A | Japan | 135.35 | 34.45 Kanzaki | N/A | Japan | 136.71667 | 35.63333 Kashihara | N/A | Japan | 135.61667 | 34.58333 Kitakyushu | N/A | Japan | 130.85034 | 33.85181 Kochi | N/A | Japan | 133.53333 | 33.55 Kōshi | N/A | Japan | 139.89769 | 35.02754 Kumamoto | N/A | Japan | 130.69181 | 32.80589 Kurayoshi | N/A | Japan | 133.81667 | 35.43333 Matsudo | N/A | Japan | 139.90144 | 35.77995 Matsusaka | N/A | Japan | 136.53706 | 34.57895 Moriguchi | N/A | Japan | 135.56667 | 34.73333 Morioka | N/A | Japan | 141.15 | 39.7 Nagoya | N/A | Japan | 136.90641 | 35.18147 Naha | N/A | Japan | 127.67851 | 26.213 Nakagami | N/A | Japan | 140.35564 | 39.151 Nankoku | N/A | Japan | 133.64937 | 33.56943 Nishinomiya | N/A | Japan | 135.33199 | 34.71562 Noda | N/A | Japan | 139.86793 | 35.94897 Numazu | N/A | Japan | 138.86667 | 35.1 Ohta | N/A | Japan | N/A | N/A Okinawa | N/A | Japan | 127.80139 | 26.33583 Oyama | N/A | Japan | 139.8 | 36.3 Ōita | N/A | Japan | 131.6 | 33.23333 Sakai | N/A | Japan | 135.46653 | 34.58216 Sapporo | N/A | Japan | 141.35 | 43.06667 Takasaki | N/A | Japan | 139.01667 | 36.33333 Takatsuki | N/A | Japan | 135.61678 | 34.84833 Tanba | N/A | Japan | 135.03914 | 35.16034 Tokyo | N/A | Japan | 139.69171 | 35.6895 Tottori | N/A | Japan | 134.67796 | 34.24877 Toyama | N/A | Japan | 137.21667 | 36.7 Toyoake | N/A | Japan | 136.99931 | 35.038 Toyonaka | N/A | Japan | 135.46932 | 34.78244 Tōgane | N/A | Japan | 140.36667 | 35.55 Tsuyama | N/A | Japan | 133.99885 | 35.05215 Ueda | N/A | Japan | 138.28161 | 36.40265 Urasoe | N/A | Japan | 127.73012 | 26.25902 Uruma | N/A | Japan | 127.85908 | 26.37609 Yanagawa | N/A | Japan | 130.4 | 33.16667 Yao | N/A | Japan | 135.6 | 34.61667 Yokkaichi | N/A | Japan | 136.61667 | 34.96667 Yokohama | N/A | Japan | 139.65 | 35.43333 Yokosuka | N/A | Japan | 139.66722 | 35.28361
0
NCT00396565
[ 3 ]
41
RANDOMIZED
CROSSOVER
0TREATMENT
4QUADRUPLE
false
0ALL
false
This study was designed to provide data about the safety and efficacy of 3 doses of indacaterol (150, 300, and 600 µg) in Japanese asthma patients so that an optimal dose, or doses, could be chosen for testing in later studies.
null
Asthma
asthma QAB149 indacaterol long acting β2-agonist bronchodilator
null
4
arm 1: In treatment period 1: patients received 2 placebo capsules; in treatment period 2: patients received 1 indacaterol (Ind) 150 μg capsule + 1 placebo capsule; in treatment period 3: patients received 1 indacaterol 300 μg capsule + 1 placebo capsule; and in treatment period 4: patients received 2 indacaterol 300 μg capsules. Two inhalation capsules of study drug were inhaled using a single dose dry powder inhaler (SDDPI) in the morning on day 1 of each treatment period at approximately the same time of day +/- 15 minutes. There was a washout period of 14-28 days between each treatment period. In open label treatment period 5: patients received 100 μg salmeterol (50 μg in the morning, 50 μg twelve hours post initial dose) inhaled via Diskus®, an inhalation device, on Day 1. Daily inhaled corticosteroid treatment (if applicable) was allowed to remain stable throughout the study. The short acting (beta) β2-agonist (SABA) salbutamol was available for rescue use throughout the study. arm 2: In treatment period 1: patients received 1 indacaterol (Ind) 150 μg capsule + 1 placebo capsule; in treatment period 2: patients received 2 indacaterol 300 μg capsules; in treatment period 3: patients received 2 placebo capsules; and in treatment period 4: patients received 1 indacaterol 300 μg capsule + 1 placebo capsule. Two inhalation capsules of study drug were inhaled using a single dose dry powder inhaler (SDDPI) in the morning on day 1 of each treatment period at approximately the same time of day +/- 15 minutes. There was a washout period of 14-28 days between each treatment period. In open label treatment period 5: patients received 100 μg salmeterol (50 μg in the morning, 50 μg twelve hours post initial dose) inhaled via Diskus®, an inhalation device, on Day 1. Daily inhaled corticosteroid treatment (if applicable) was allowed to remain stable throughout the study. The short acting (beta) β2-agonist (SABA) salbutamol was available for rescue use throughout the study. arm 3: In treatment period 1: patients received 1 indacaterol (Ind) 300 μg capsule + 1 placebo capsule; in treatment period 2: patients received 2 placebo capsules; in treatment period 3: patients received 2 indacaterol 300 μg capsules; and in treatment period 4: patients received 1 indacaterol 150 μg capsule + 1 placebo capsule. Two inhalation capsules of study drug were inhaled using a single dose dry powder inhaler (SDDPI) in the morning on day 1 of each treatment period at approximately the same time of day +/- 15 minutes. There was a washout period of 14-28 days between each treatment period. In open label treatment period 5: patients received 100 μg salmeterol (50 μg in the morning, 50 μg twelve hours post initial dose) inhaled via Diskus®, an inhalation, device on Day 1. Daily inhaled corticosteroid treatment (if applicable) was allowed to remain stable throughout the study. The short acting (beta) β2-agonist (SABA) salbutamol was available for rescue use throughout the study. arm 4: In treatment period 1: patients received 2 indacaterol (Ind) 300 μg capsules; in treatment period 2: patients received 1 indacaterol 300 μg capsule + 1 placebo capsule; in treatment period 3: patients received 1 indacaterol 150 μg capsule + 1 placebo capsule; and in treatment period 4: patients received 2 placebo capsules. Two inhalation capsules of study drug were inhaled using a single dose dry powder inhaler (SDDPI) in the morning on day 1 of each treatment period at approximately the same time of day +/- 15 minutes. There was a washout period of 14-28 days between each treatment period. In open label treatment period 5: patients received 100 μg salmeterol (50 μg in the morning, 50 μg twelve hours post initial dose) inhaled via Diskus®, an inhalation device, on Day 1. Daily inhaled corticosteroid treatment (if applicable) was allowed to remain stable throughout the study. The short acting (beta) β2-agonist (SABA) salbutamol was available for rescue use throughout the study.
[ 0, 0, 0, 0 ]
3
[ 0, 0, 0 ]
intervention 1: In the morning, powder filled capsules inhaled using a single dose dry powder inhaler (SDDPI). intervention 2: Placebo to indacaterol was provided in powder filled capsules with a single dose dry powder inhaler (SDDPI). intervention 3: Salmeterol 100 μg total dose taken on Day 1. 50 μg in the morning and 50 μg twelve hours post initial dose inhaled via Diskus®, an inhalation device for Salmeterol.
intervention 1: Indacaterol intervention 2: Placebo intervention 3: Salmeterol
8
Kasukabe | N/A | Japan | 139.74966 | 35.98308 Kishiwada | N/A | Japan | 135.36667 | 34.46667 Shimotsuga | N/A | Japan | N/A | N/A Suita | N/A | Japan | 135.51567 | 34.76143 Tokyo | N/A | Japan | 139.69171 | 35.6895 Tokyo | N/A | Japan | 139.69171 | 35.6895 Wakayama | N/A | Japan | 135.16667 | 34.23333 Yokohama | N/A | Japan | 139.65 | 35.43333
0
NCT00403754
[ 4 ]
547
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
false
This study will investigate the effectiveness of desloratadine in treating subjects with allergic rhinitis (AR) who meet the criteria for intermittent allergic rhinitis.
null
Rhinitis, Allergic, Seasonal Rhinitis, Allergic, Perennial
Allergic Rhinitis Intermittent Allergic Rhinitis
null
2
arm 1: None arm 2: None
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: 5-mg Desloratadine tablet, once daily for 15 days intervention 2: Placebo tablet, once daily for 15 days
intervention 1: 5-mg Desloratadine intervention 2: Placebo
0
null
0
NCT00406783
[ 4 ]
404
RANDOMIZED
SINGLE_GROUP
0TREATMENT
4QUADRUPLE
false
0ALL
false
The primary purpose of your participation in this study is to help answer the following research question, and not to provide you treatment for your condition--Whether duloxetine once daily can help patients with Chronic Low Back Pain.
null
Back Pain Without Radiation
null
4
arm 1: duloxetine 20 mg once a day (QD), by mouth (PO) for 13 weeks arm 2: duloxetine 30 mg once a day (QD), by mouth (PO) for 1 week then duloxetine 60 mg QD, PO for 12 weeks arm 3: duloxetine 30 mg once a day (QD), by mouth (PO) for 1 week followed by duloxetine 60 mg QD, PO for 1 week, then duloxetine 120 mg QD, PO for 11 weeks arm 4: placebo once a day (QD), by mouth (PO) for 13 weeks
[ 0, 0, 0, 2 ]
2
[ 0, 0 ]
intervention 1: None intervention 2: None
intervention 1: Duloxetine intervention 2: Placebo
4
Fort Myers | Florida | United States | -81.84059 | 26.62168 Orlando | Florida | United States | -81.37924 | 28.53834 South Miami | Florida | United States | -80.29338 | 25.7076 Tampa | Florida | United States | -82.45843 | 27.94752
0
NCT00408876
[ 4 ]
698
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This study will evaluate the safety and efficacy of the fixed combination of valsartan/amlodipine in adult patients with mild to moderate hypertension
null
Hypertension
Hypertension, valsartan, amlodipine, high blood pressure
null
2
arm 1: None arm 2: None
[ 0, 1 ]
2
[ 0, 0 ]
intervention 1: 1 valsartan/amlodipine 80/5 mg tablet and 1 placebo capsule matching amlodipine 5 mg to be taken with water at approximately 8:00 a.m. once daily, except on the morning of every scheduled study visit, when study drug was taken after the completion of all other study procedures. intervention 2: 1 amlodipine 5 mg capsule and 1 placebo tablet matching valsartan/amlodipine 80/5 mg to be taken with water at approximately 8:00 a.m. once daily, except on the morning of every scheduled study visit, when study drug was taken after the completion of all other study procedures.
intervention 1: Valsartan/amlodipine 80/5 mg intervention 2: Amlodipine 5 mg
12
Beijing | N/A | China | 116.39723 | 39.9075 Beijing | N/A | China | 116.39723 | 39.9075 Changsha | N/A | China | 112.97087 | 28.19874 Chongqing | N/A | China | 106.55771 | 29.56026 Chongqing | N/A | China | 106.55771 | 29.56026 Fuzhou | N/A | China | 119.30611 | 26.06139 Fuzhou | N/A | China | 119.30611 | 26.06139 Nanchang | N/A | China | 115.85306 | 28.68396 Shanghai | N/A | China | 121.45806 | 31.22222 Shanghai | N/A | China | 121.45806 | 31.22222 Shanghai | N/A | China | 121.45806 | 31.22222 Xi'an | N/A | China | 108.92861 | 34.25833
0
NCT00413049
[ 4 ]
1,134
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
This study evaluated the safety and efficacy of the fixed combination of valsartan/amlodipine in adult patients with mild to moderate hypertension.
null
Hypertension
Hypertension, valsartan, amlodipine, high blood pressure
null
3
arm 1: None arm 2: None arm 3: None
[ 0, 1, 1 ]
3
[ 0, 0, 0 ]
intervention 1: 1 valsartan/amlodipine 80/5 mg tablet, 1 placebo capsule to match valsartan once daily intervention 2: 1 valsartan 80 mg capsule, 1 placebo tablet to match valsartan/amlodipine 80/5 mg once daily intervention 3: 1 valsartan 160 mg capsule, 1 placebo tablet to match valsartan/amlodipine 80/5 mg once daily
intervention 1: Valsartan/amlodipine 80/5 mg intervention 2: Valsartan 80 mg intervention 3: Valsartan 160 mg
19
Beijing | N/A | China | 116.39723 | 39.9075 Beijing | N/A | China | 116.39723 | 39.9075 Beijing | N/A | China | 116.39723 | 39.9075 Hangzhou | N/A | China | 120.16142 | 30.29365 Hangzhou | N/A | China | 120.16142 | 30.29365 Hangzhou | N/A | China | 120.16142 | 30.29365 Nanjing | N/A | China | 118.77778 | 32.06167 Nanjing | N/A | China | 118.77778 | 32.06167 Shanghai | N/A | China | 121.45806 | 31.22222 Shanghai | N/A | China | 121.45806 | 31.22222 Shanghai | N/A | China | 121.45806 | 31.22222 Shanghai | N/A | China | 121.45806 | 31.22222 Shanghai | N/A | China | 121.45806 | 31.22222 Shenyang | N/A | China | 123.43278 | 41.79222 Shenyang | N/A | China | 123.43278 | 41.79222 Shijiazhuang | N/A | China | 114.47861 | 38.04139 Shijiazhuang | N/A | China | 114.47861 | 38.04139 Suzhou | N/A | China | 120.59538 | 31.30408 Suzhou | N/A | China | 120.59538 | 31.30408
0
NCT00413413
[ 4 ]
547
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
null
The purpose of this study is to evaluate the efficacy of Kadian NT (ALO-01) compared with placebo for treating moderate to severe chronic pain over a 12 week period.
The primary objective of this study is to evaluate the efficacy of ALO-01 compared with placebo for the treatment of chronic moderate to severe pain (focusing on osteoarthritis of the hip or knee) as measured by mean change in diary Brief Pain Inventory (BPI) score of average pain (daily scores of average pain averaged over 7 days) from randomization to 12 weeks following randomization.
Osteoarthritis Chronic Pain
hip knee pain osteoarthritis OA Kadian Alpharma naltrexone Opioid morphine Embeda ALO-01
null
2
arm 1: Up to 80 mg twice a day (bid) arm 2: Twice a day (bid)
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: capsules, up to 80 mg bid intervention 2: capsules, bid
intervention 1: ALO-01 (Morphine Sulfate Plus Naltrexone Hydrochloride Extended Release) intervention 2: Placebo
81
Birmingham | Alabama | United States | -86.80249 | 33.52066 Mobile | Alabama | United States | -88.04305 | 30.69436 Peoria | Arizona | United States | -112.23738 | 33.5806 Phoenix | Arizona | United States | -112.07404 | 33.44838 Tempe | Arizona | United States | -111.90931 | 33.41477 Anaheim | California | United States | -117.9145 | 33.83529 Anaheim | California | United States | -117.9145 | 33.83529 Anaheim | California | United States | -117.9145 | 33.83529 Buena Park | California | United States | -117.99812 | 33.86751 Burbank | California | United States | -118.30897 | 34.18084 Fair Oaks | California | United States | -121.27217 | 38.64463 Hawaiian Gardens | California | United States | -118.07284 | 33.8314 Irvine | California | United States | -117.82311 | 33.66946 La Jolla | California | United States | -117.2742 | 32.84727 La Jolla | California | United States | -117.2742 | 32.84727 Pico Rivera | California | United States | -118.09673 | 33.98307 Santa Monica | California | United States | -118.49138 | 34.01949 Upland | California | United States | -117.64839 | 34.09751 Denver | Colorado | United States | -104.9847 | 39.73915 Stamford | Connecticut | United States | -73.53873 | 41.05343 Trumbull | Connecticut | United States | -73.20067 | 41.24287 Hallandale | Florida | United States | -80.14838 | 25.9812 Hialeah | Florida | United States | -80.27811 | 25.8576 Jacksonville | Florida | United States | -81.65565 | 30.33218 Jupiter | Florida | United States | -80.09421 | 26.93422 Kissimmee | Florida | United States | -81.41667 | 28.30468 Largo | Florida | United States | -82.78842 | 27.90979 Merritt Island | Florida | United States | -80.69 | 28.359 Ormond Beach | Florida | United States | -81.05589 | 29.28581 Pembroke Pines | Florida | United States | -80.22394 | 26.00315 Port Orange | Florida | United States | -80.99561 | 29.13832 Tampa | Florida | United States | -82.45843 | 27.94752 Weston | Florida | United States | -80.39977 | 26.10037 Blue Ridge | Georgia | United States | -84.32409 | 34.86397 Decatur | Georgia | United States | -84.29631 | 33.77483 Marietta | Georgia | United States | -84.54993 | 33.9526 Marietta | Georgia | United States | -84.54993 | 33.9526 Boise | Idaho | United States | -116.20345 | 43.6135 Peoria | Illinois | United States | -89.58899 | 40.69365 Evansville | Indiana | United States | -87.55585 | 37.97476 Newburgh | Indiana | United States | -87.40529 | 37.94449 Overland Park | Kansas | United States | -94.67079 | 38.98223 Mandeville | Louisiana | United States | -90.06563 | 30.35825 New Orleans | Louisiana | United States | -90.07507 | 29.95465 Towson | Maryland | United States | -76.60191 | 39.4015 Fall River | Massachusetts | United States | -71.15505 | 41.70149 No Dartmouth | Massachusetts | United States | N/A | N/A Springfield | Massachusetts | United States | -72.58981 | 42.10148 Saginaw | Michigan | United States | -83.95081 | 43.41947 St Louis | Missouri | United States | -90.19789 | 38.62727 Henderson | Nevada | United States | -114.98194 | 36.0397 Las Vegas | Nevada | United States | -115.13722 | 36.17497 Voorhees Township | New Jersey | United States | -74.49062 | 40.4795 Mount Vernon | New York | United States | -73.83708 | 40.9126 Charlotte | North Carolina | United States | -80.84313 | 35.22709 Winston-Salem | North Carolina | United States | -80.24422 | 36.09986 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Toledo | Ohio | United States | -83.55521 | 41.66394 Oklahoma City | Oklahoma | United States | -97.51643 | 35.46756 Oklahoma City | Oklahoma | United States | -97.51643 | 35.46756 Oklahoma City | Oklahoma | United States | -97.51643 | 35.46756 Tulsa | Oklahoma | United States | -95.99277 | 36.15398 Allentown | Pennsylvania | United States | -75.49018 | 40.60843 Bensalem | Pennsylvania | United States | -74.95128 | 40.10455 Duncansville | Pennsylvania | United States | -78.4339 | 40.42341 Fleetwood | Pennsylvania | United States | -75.81798 | 40.45398 Souderton | Pennsylvania | United States | -75.32518 | 40.31177 Tipton | Pennsylvania | United States | -78.29585 | 40.6359 Clarksville | Tennessee | United States | -87.35945 | 36.52977 Cordova | Tennessee | United States | -89.7762 | 35.15565 Hendersonville | Tennessee | United States | -86.62 | 36.30477 Memphis | Tennessee | United States | -90.04898 | 35.14953 Milan | Tennessee | United States | -88.75895 | 35.91979 Austin | Texas | United States | -97.74306 | 30.26715 DeSoto | Texas | United States | -96.85695 | 32.58986 Killeen | Texas | United States | -97.7278 | 31.11712 San Antonio | Texas | United States | -98.49363 | 29.42412 San Antonio | Texas | United States | -98.49363 | 29.42412 San Antonio | Texas | United States | -98.49363 | 29.42412 Roanoke | Virginia | United States | -79.94143 | 37.27097
0
NCT00420992
[ 4 ]
698
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
false
The purpose of this study is to determine whether ceftaroline is effective and safe in the treatment of complicated skin infections in adults.
Additional purpose of the study is to compare ceftaroline effectivity versus Vancomycin plus Aztreonam in the treatment of complicated skin infections in adults.
Bacterial Infections
Abscess Antibacterial Antibiotic Antimicrobial Bacterial infection, skin Ceftaroline Ceftaroline acetate Cellulitis Cephalosporin Complicated skin and skin structure infection cSSSI Intravenous Methicillin-resistant Staphylococcus Aureus (MRSA) PPI-0903 Prodrug Skin disease, bacterial Skin infection Staphylococcal skin infection Staphylococcus aureus Streptococcal skin infection Surgical site infection TAK-599
null
2
arm 1: None arm 2: None
[ 0, 1 ]
2
[ 0, 0 ]
intervention 1: vancomycin at 1 g parenteral infused over 60 minutes followed by aztreonam 1 g infused over 60 minutes, every 12 hours, for 5 to 14 days. intervention 2: 600 mg parenteral infused over 60 minutes, every 12 hours for 5 to 14 days
intervention 1: IV Vancomycin plus IV Aztreonam intervention 2: Ceftaroline
52
Dothan | Alabama | United States | -85.39049 | 31.22323 Long Beach | California | United States | -118.18923 | 33.76696 Los Angeles | California | United States | -118.24368 | 34.05223 Sacramento | California | United States | -121.4944 | 38.58157 Sacramento | California | United States | -121.4944 | 38.58157 San Diego | California | United States | -117.16472 | 32.71571 San Francisco | California | United States | -122.41942 | 37.77493 Torrance | California | United States | -118.34063 | 33.83585 Savannah | Georgia | United States | -81.09983 | 32.08354 Naperville | Illinois | United States | -88.14729 | 41.78586 Indianapolis | Indiana | United States | -86.15804 | 39.76838 Shreveport | Louisiana | United States | -93.75018 | 32.52515 Columbus | Ohio | United States | -82.99879 | 39.96118 Landsdale | Pennsylvania | United States | N/A | N/A Tacoma | Washington | United States | -122.44429 | 47.25288 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Buenos Aires | N/A | Argentina | -58.37723 | -34.61315 Córdoba | N/A | Argentina | -64.18853 | -31.40648 Córdoba | N/A | Argentina | -64.18853 | -31.40648 Entre Ríos | N/A | Argentina | -65.63233 | -25.44524 Santa Fe | N/A | Argentina | -60.70868 | -31.64881 Curiuba-Parans | N/A | Brazil | N/A | N/A São Paulo | N/A | Brazil | -46.63611 | -23.5475 Santiago | N/A | Chile | -70.64827 | -33.45694 Viña del Mar | N/A | Chile | -71.55183 | -33.02457 Berlin | N/A | Germany | 13.41053 | 52.52437 Bochum | N/A | Germany | 7.21648 | 51.48165 Hanau | N/A | Germany | 8.91418 | 50.13423 Plauen | N/A | Germany | 12.13782 | 50.4973 Quedlinburg | N/A | Germany | 11.15006 | 51.78843 Chihuahua City | N/A | Mexico | -106.08889 | 28.63528 Lima | N/A | Peru | -77.02824 | -12.04318 Bytom | N/A | Poland | 18.93282 | 50.34802 Krakow | N/A | Poland | 19.93658 | 50.06143 Lublin | N/A | Poland | 22.56667 | 51.25 Sosnowiec | N/A | Poland | 19.10385 | 50.28682 Todz | N/A | Poland | N/A | N/A Bucharest | N/A | Romania | 26.10626 | 44.43225 Bucharest | N/A | Romania | 26.10626 | 44.43225 Timișoara | N/A | Romania | 21.22571 | 45.75372 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Moscow | N/A | Russia | 37.61556 | 55.75222 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Saint Petersburg | N/A | Russia | 30.31413 | 59.93863 Smolensk | N/A | Russia | 32.04371 | 54.77944 Dnipropetrovsk | N/A | Ukraine | 35.04066 | 48.46664 Ivano-Frankivsk | N/A | Ukraine | 24.71248 | 48.92312 Lviv | N/A | Ukraine | 24.02324 | 49.83826
0
NCT00424190
[ 4 ]
1,183
RANDOMIZED
PARALLEL
0TREATMENT
4QUADRUPLE
false
0ALL
null
This study was designed to compare the efficacy, tolerability, and safety of the combination valsartan/amlodipine 160/5 mg versus amlodipine 10 mg in patients with essential hypertension not adequately controlled (defined as mean sitting systolic blood pressure \[msSBP\] ≥ 130 mmHg and ≤ 160 mmHg) on amlodipine 5 mg alone. The study evaluated both the efficacy and tolerability of the treatments by providing data that assessed blood pressure and the proportion of patients developing peripheral edema.
null
Essential Hypertension
Essential hypertension blood pressure edema valsartan amlodipine combination treatment
null
2
arm 1: Twelve (12) weeks treatment with the combination of valsartan/amlodipine 160/5 mg. Together with the active medication, patients received a placebo that matched amlodipine 5 mg. The three capsules were taken by mouth with water once daily in the morning, regardless of meals. Patients were instructed not to take their study medication the morning of their study visits. Instead, they brought the study medication with them to the site and took it there as instructed by the investigator. arm 2: Eight (8) weeks of treatment with amlodipine 10 mg (two 5 mg capsules). Together with the active medication, the patients received a placebo that matched valsartan 160 mg. At Week 8, patients were switched and treated with the combination of valsartan/amlodipine 160/5 mg and a placebo that matched amlodipine 5 mg for an additional 4 weeks until the end of the study. The three capsules were taken by mouth with water once daily in the morning, regardless of meals. Patients were instructed not to take their study medication the morning of their study visits. Instead, they brought the study medication with them to the site and took it there as instructed by the investigator.
[ 0, 1 ]
3
[ 0, 0, 0 ]
intervention 1: None intervention 2: None intervention 3: capsules
intervention 1: Valsartan 160 mg capsules intervention 2: Amlodipine 5 mg capsules intervention 3: placebo
12
Agentina | N/A | Argentina | N/A | N/A Chile | N/A | Chile | N/A | N/A Ecuador | N/A | Ecuador | -79.8904 | -2.25741 Finland | N/A | Finland | N/A | N/A France | N/A | France | -0.84802 | 45.60366 Germany | N/A | Germany | N/A | N/A Italy | N/A | Italy | N/A | N/A Norway | N/A | Norway | N/A | N/A Spain | N/A | Spain | N/A | N/A Sweden | N/A | Sweden | N/A | N/A Switzerland | N/A | Switzerland | N/A | N/A Turkey | N/A | Turkey (Türkiye) | N/A | N/A
0
NCT00437645
[ 4 ]
243
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
null
0ALL
null
The study will evaluate the efficacy of cyclosporine at 2, 5 mg/kg/day bid (i.e. bis in a day), when administered twice a week compared to continuous administration, in patients with chronic plaque psoriasis.
null
Chronic Plaque Psoriasis
Cyclosporine, chronic plaque psoriasis, intermittent therapy
null
2
arm 1: Oral soft gelatin capsules of cyclosporine 10 mg, 25 mg, 50 mg or 100 mg administered twice a week for 24 weeks at the dosage of 5 mg/Kg/day in two daily administrations arm 2: Oral soft gelatin capsules of placebo matching cyclosporine administered twice a week for 24 weeks in two daily administrations
[ 1, 2 ]
2
[ 0, 0 ]
intervention 1: Oral soft gelatin capsules of Cyclosporine 10 mg, 25 mg, 50 mg or 100 mg administered twice a week for 24 weeks at the dosage of 5 mg/Kg/day in two daily administrations intervention 2: Oral soft gelatin capsules of placebo matching cyclosporine administered twice a week for 24 weeks in two daily administrations
intervention 1: Cyclosporine A microemulsion intervention 2: Placebo
1
Bari | N/A | Italy | 16.86982 | 41.12066
0
NCT00438360
[ 5 ]
120
RANDOMIZED
PARALLEL
0TREATMENT
0NONE
false
0ALL
true
To demonstrate efficacy and safety of TachoSil® in cardiovascular surgery
null
Haemorrhage Haemostasis Cardiovascular Surgery
Surgical treatment of haemorrhage
null
2
arm 1: None arm 2: Standard Treatment of haemorrhage in cardiovascular surgery
[ 1, 1 ]
2
[ 0, 0 ]
intervention 1: None intervention 2: Any haemostatic fleece material without additional active coagulation stimulating compounds (primarily Surgicel®)
intervention 1: fibrinogen (human) + thrombin (human) intervention 2: Standard haemostatic treatment in cardiovascular surgery
1
Roskilde | N/A | Denmark | 12.08035 | 55.64152
0
NCT00440401
[ 5 ]
132
RANDOMIZED
CROSSOVER
0TREATMENT
0NONE
false
0ALL
false
This study will compare the effects of high and low level sodium (salt) diets on blood pressure in patients with hypertension (high blood pressure) who are taking aliskiren 300 mg.
null
Hypertension
High blood pressure, hypertension, aliskiren, low sodium diet
null
2
arm 1: Patients on low sodium diet ( \<= 100 mmol/day) for the first 4 weeks and high sodium (\>= 200 mmol/day) diet for the next 4 weeks. \[with Aliskiren 300 mg\] arm 2: Patients on high sodium (\>= 200 mmol/day) diet for the first 4 weeks and on low sodium diet ( \<= 100 mmol/day) for the next 4 weeks. \[with Aliskiren 300 mg\]
[ 0, 0 ]
1
[ 0 ]
intervention 1: None
intervention 1: Aliskiren
19
Orangevale | California | United States | -121.22578 | 38.67851 Brooklyn Center | Minnesota | United States | -93.33273 | 45.07608 Butte | Montana | United States | -112.53474 | 46.00382 Missoula | Montana | United States | -113.994 | 46.87215 Lincoln | Nebraska | United States | -96.66696 | 40.8 Johnson City | New York | United States | -75.95881 | 42.11563 Asheboro | North Carolina | United States | -79.81364 | 35.70791 Shelby | North Carolina | United States | -81.53565 | 35.29235 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Kettering | Ohio | United States | -84.16883 | 39.6895 Downington | Pennsylvania | United States | N/A | N/A Erie | Pennsylvania | United States | -80.08506 | 42.12922 Pittsburgh | Pennsylvania | United States | -79.99589 | 40.44062 Dallas | Texas | United States | -96.80667 | 32.78306 Kingsport | Texas | United States | N/A | N/A Bountiful | Utah | United States | -111.88077 | 40.88939 Salt Lake City | Utah | United States | -111.89105 | 40.76078 Port Orchard | Washington | United States | -122.63625 | 47.54037 Madison | Wisconsin | United States | -89.40123 | 43.07305
0
NCT00441064
[ 3 ]
4
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
false
The purpose of this study is to evaluate the safety of a protein called BPI that is naturally made by the body's white blood cells to fight infection.
This study will initially evaluate an FDA approved investigational dosage and will measure the blood levels over time while BPI is being infused through a vein to see if the dose is potentially enough to obtain the desired effect. The drug will be started within 8 hours of burn injury and continued for 48 hours. Patients will be followed on admission and through infusion in the Burn ICU where they will be monitored by ICU standards. They will be seen regularly thereafter on post-burn days 1, 2, 3, 4, 7, 14 and 28 days while in the hospital. If the patient is discharged prior to the 28 day evaluation, they will be evaluated on or around the 28th day in the burn clinic. A blood sample will be obtained to look for potential genetic markers that may increase a burn patients' risk to develop infection in the post burn period.
Burns
null
1
arm 1: BPI will be infused by bolus for 30 minutes followed by continuous infusion for 47.5 hours
[ 0 ]
1
[ 0 ]
intervention 1: BPI will be give IV with an initial bolus given over 30 minutes and then a continuous infusion for the next 47.5 hours
intervention 1: BPI
1
Dallas | Texas | United States | -96.80667 | 32.78306
0
NCT00462904
[ 5 ]
52
NON_RANDOMIZED
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
null
This study will evaluate the safety and efficacy of daptomycin against complicated skin and skin-structure infections in adults
null
Staphylococcal Skin Infections
Complicated Skin and Skin-Structure Infections, Daptomycin, MRSA, Diabetic Foot, Abscess, Cellulitis Complicated skin and skin-structure infections
null
1
arm 1: 350 mg of Daptomycin was supplied as sterile lyophilized powder in glass vials. Each vial was to be reconstituted with 7 mL of normal saline or water for injection, to give a 50 mg/mL drug concentration. Daptomycin was to be administered as a 30-minute intravenous infusion, once daily for at least 7 days, at the dose of 4 mg/Kg, up to a maximum of 14 days.
[ 0 ]
1
[ 0 ]
intervention 1: None
intervention 1: Daptomycin
2
Novartis Italy | N/A | Italy | N/A | N/A Saronno | N/A | Italy | 9.03517 | 45.62513
0
NCT00463801
[ 4 ]
295
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
false
The purpose of the study is to evaluate sleep onset following administration of zolpidem tartrate sublingual tablet (Intermezzo) versus placebo in adult insomnia patients.
null
Insomnia
Insomnia
null
2
arm 1: None arm 2: None
[ 0, 2 ]
2
[ 0, 0 ]
intervention 1: 3.5 milligram zolpidem tartrate sublingual tablet taken as needed following a middle-of-the-night awakening over the 28-night study period. Participants placed the study drug under the tongue and allowed it to dissolve there for about 2 minutes, then swallowed after dissolved. intervention 2: Placebo sublingual tablet taken as needed following a middle-of-the-night awakening over the 28-night study period. Participants placed the study drug under the tongue and allowed it to dissolve there for about 2 minutes, then swallowed after dissolved.
intervention 1: zolpidem tartrate sublingual tablet intervention 2: Placebo
1
Durham | North Carolina | United States | -78.89862 | 35.99403
0
NCT00466193
[ 5 ]
680
RANDOMIZED
PARALLEL
0TREATMENT
2DOUBLE
false
0ALL
false
The primary objective of the study is to compare nighttime symptom relief of fluticasone furoate nasal spray versus oral fexofenadine
null
Rhinitis, Allergic, Seasonal
ragweed once daily fexofenadine fluticasone furoate allergic rhinitis
null
0
null
null
1
[ 0 ]
intervention 1: None
intervention 1: fluticasone furoate, fexofenadine
42
Little Rock | Arkansas | United States | -92.28959 | 34.74648 Albany | Georgia | United States | -84.15574 | 31.57851 Columbus | Georgia | United States | -84.98771 | 32.46098 Lawrenceville | Georgia | United States | -83.98796 | 33.95621 Chicago | Illinois | United States | -87.65005 | 41.85003 Normal | Illinois | United States | -88.99063 | 40.5142 Evansville | Indiana | United States | -87.55585 | 37.97476 Indianapolis | Indiana | United States | -86.15804 | 39.76838 Lafayette | Indiana | United States | -86.87529 | 40.4167 South Bend | Indiana | United States | -86.25001 | 41.68338 Iowa City | Iowa | United States | -91.53017 | 41.66113 Louisville | Kentucky | United States | -85.75941 | 38.25424 Owensboro | Kentucky | United States | -87.11333 | 37.77422 Metairie | Louisiana | United States | -90.15285 | 29.98409 Sunset | Louisiana | United States | -92.06845 | 30.41131 Novi | Michigan | United States | -83.47549 | 42.48059 Ypsilanti | Michigan | United States | -83.61299 | 42.24115 Minneapolis | Minnesota | United States | -93.26384 | 44.97997 Plymouth | Minnesota | United States | -93.45551 | 45.01052 Jackson | Mississippi | United States | -90.18481 | 32.29876 Columbia | Missouri | United States | -92.33407 | 38.95171 Rolla | Missouri | United States | -91.77127 | 37.95143 St Louis | Missouri | United States | -90.19789 | 38.62727 St Louis | Missouri | United States | -90.19789 | 38.62727 Warrensburg | Missouri | United States | -93.73605 | 38.76279 Lincoln | Nebraska | United States | -96.66696 | 40.8 Omaha | Nebraska | United States | -95.94043 | 41.25626 Omaha | Nebraska | United States | -95.94043 | 41.25626 Papillion | Nebraska | United States | -96.04224 | 41.15444 Canton | Ohio | United States | -81.37845 | 40.79895 Cincinnati | Ohio | United States | -84.51439 | 39.12711 Sylvania | Ohio | United States | -83.71299 | 41.71894 Oklahoma City | Oklahoma | United States | -97.51643 | 35.46756 Pittsburgh | Pennsylvania | United States | -79.99589 | 40.44062 Charleston | South Carolina | United States | -79.93275 | 32.77632 Orangeburg | South Carolina | United States | -80.85565 | 33.49182 Spartanburg | South Carolina | United States | -81.93205 | 34.94957 Knoxville | Tennessee | United States | -83.92074 | 35.96064 Waco | Texas | United States | -97.14667 | 31.54933 Greenfield | Wisconsin | United States | -88.01259 | 42.9614 Milwaukee | Wisconsin | United States | -87.90647 | 43.0389 West Allis | Wisconsin | United States | -88.00703 | 43.01668
0
NCT00502775
[ 0 ]
9
NA
SINGLE_GROUP
0TREATMENT
0NONE
false
0ALL
true
The current use of telepsychiatry, or psychiatric care using videoconferencing, is very limited. The present study investigates the use of this method with a depressed Chinese American population in a nursing home. It is believed that this population can benefit from telepsychiatric treatment when used in collaboration with the primary care they receive in the nursing home.
Individuals at the South Cove Manor nursing home will be referred to this study if they are experiencing depression. After meeting with the Principal Investigator (a psychiatrist), he will collaborate with the primary care physician and the nursing home staff to establish a treatment plan for the patient. Videoconferencing will be used by the P.I. for regular psychiatric visits with the patient as well as meetings with the nursing home staff to coordinate care for the patient. After collaboration between the two groups, treatment suggestions will be given to the patient's primary care physician (PCP) to implement in the primary care setting. The patient's symptomology, mood, satisfaction of life and side effects of medication will be recorded regularly to measure the effects of treatment for analysis.
Major Depressive Disorder
Major Depressive Disorder Telepsychiatry Chinese American Nursing Home
null
0
null
null
2
[ 0, 5 ]
intervention 1: Antidepressant medication, prescribed at discretion of psychiatrist intervention 2: Regular care visits from health workers
intervention 1: Psychotropic medication (at discretion of psychiatrist) intervention 2: Collaborative psychiatric and primary medical care
2
Boston | Massachusetts | United States | -71.05977 | 42.35843 Boston | Massachusetts | United States | -71.05977 | 42.35843
0
NCT00505518
[ 3 ]
20
RANDOMIZED
CROSSOVER
0TREATMENT
2DOUBLE
false
0ALL
false
This study will involve the use of a new compound, GW642444 that is being developed for the treatment of asthma and chronic obstructive pulmonary disease (COPD). It works by acting on cells in the lungs, causing some of the muscles around the lungs to relax and open up better (bronchodilation), making breathing easier. When a medicine is made into a form ready to be given to patients, the active ingredient is often prepared in the form of a salt, and inactive ingredients (excipients) are often added. Inactive ingredients might be used to help a medicine work better, to make it easier to produce the medicine, or to make it easier to get an accurate dose of medicine. In previous studies the study drug has been given as a dry powder in the form of either the 'H' salt (with the excipient lactose), or in the form of the 'M' salt (with the excipients lactose and cellobiose octaacetate). In this study the 'M' salt form of the study drug has been prepared with lactose and a new excipient called magnesium stearate (instead of cellobiose octaacetate). Participants in this study will receive both the 'H' salt (GW642444H) and the new 'M' salt (GW642444M) containing magnesium stearate. This study will be the first time the new 'M' salt form of the study drug will be given to COPD patients.
null
Pulmonary Disease, Chronic Obstructive
pharmacodynamics, Chronic Obstructive Pulmonary Disease (COPD) safety, COPD patients pharmacokinetics, tolerability, GW642444,
null
5
arm 1: None arm 2: None arm 3: None arm 4: None arm 5: None
[ 0, 0, 0, 0, 0 ]
3
[ 0, 0, 0 ]
intervention 1: drug intervention 2: drug intervention 3: None
intervention 1: GW642444M intervention 2: GW642444H intervention 3: placebo
4
Wiesbaden | Hesse | Germany | 8.24932 | 50.08258 Mainz | Rhineland-Palatinate | Germany | 8.2791 | 49.98419 Berlin | State of Berlin | Germany | 13.41053 | 52.52437 Berlin | State of Berlin | Germany | 13.41053 | 52.52437
0
NCT00519376