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2.1. Patient Characteristics | kidney stones, kidney abnormalities | KIDNEY STONE | This RCT was approved by the Institutional Review Board of Applied Science Private University (ASU) (protocol number 2020-PHA-16) and undertaken between October 2020 and December 2020. The clinical trial was conducted following the Helsinki Declaration. Each individual who was enrolled provided informed consent for thi... | PMC10005440 |
2.2. Intervention | Before and after the VD3 supplement, baseline and follow-up values of anthropometric and clinical parameters were collected. At the conclusion of the 8-week interventional phase, the subjects underwent a 2-week washout phase before and following VD3 administration. VD3 is a fat-soluble vitamin with a long half-life; a ... | PMC10005440 | ||
2.3. Anthropometric Measurement | This RCT was conducted throughout the winter of 2020 at the ASU Pharmacy school laboratories to minimize seasonal fluctuations in vitamin D assays in the blood [ | PMC10005440 | ||
2.4. Clinical Parameter Assays | ab181421, TNF-α, ab178013 | Serum assay of clinical parameters was collected into labeled Eppendorf tubes at Ibn Al-Haytham Hospital, Clinical Laboratories Department, Jordan.The chemiluminescence immunoassay LIAISON 25OHD assay (DiaSorin, Saluggia, Italy) measured total serum 25OHD. The assay quantifies serum 25OHD and is cross-reactive with 25O... | PMC10005440 | |
2.5. Statistical Analysis | SPSS version 27 for Windows was used to execute the statistical analysis. A paired | PMC10005440 | ||
3. Results | PMC10005440 | |||
3.1. Baseline Values of the Participants | A total of 75 out of 127 (59.1%) participants in the trial adhered to the protocol and finished the intervention period that lasted for 8 weeks. As indicated in Participants’ average age was (38.37 9.77). When measured at baseline, all other serum markers had mean values that were within normal limits. Descriptive anal... | PMC10005440 | ||
3.2. Baseline Clinical Characteristics | The baseline mean value for serum 25OHD was (17.29 ± 6.18) ng/mL (all participants were VD deficient). None of the participants presented in this trial with a baseline serum 25OHD level equal to or greater than 30 ng/mL. All participants’ baseline values of all serum parameters, including PTH, Ca, and PO | PMC10005440 | ||
3.3. Connection between Selected Cytokine Variables and 25OHD Concentrations | In this trial, selected proinflammatory cytokines (IL-1β, TNF-α, and IL-6) and anti-inflammatory cytokine (IL-10) showed statistically significant intercorrelations (The Pearson correlation analysis showed no intercorrelation between serum cytokine levels in the VD3 group, as shown in | PMC10005440 | ||
3.4. Changes in the Serum Levels of 25OHD and PTH | Paired sample | PMC10005440 | ||
3.5. Changes in the Serum Levels of Selected Cytokines Associated with Cytokine Storm at Baseline and 10-Week Follow-Up | At the end of this study, a paired | PMC10005440 | ||
3.6. Stepwise Regression Analysis | REGRESSION | The multivariate stepwise regression analysis revealed significant mediating factors (IDVs) on the circulatory levels of selected cytokines associated with CS at the 10-week follow-up supplementation of VDRegarding the TNF/IL-10 ratio, WHR only was selected by the stepwise regression model among all IVDs to be involved... | PMC10005440 | |
4. Discussion | chronic kidney disease, IBS, HD, chronic renal impairment, diabetic | CHRONIC RENAL IMPAIRMENT, FLUID OVERLOAD, INFLAMMATORY RESPONSES, CHRONIC DISEASES, INFLUENZA A, CHRONIC INFLAMMATION | At the end of the trial, high doses of VDResults of the current trial were consistent with a prior RCT [Previous clinical trials have typically been conducted under a treatment protocol close to or similar to our protocol, but they have been scarce. After extensive review, some clinical research studies were conducted ... | PMC10005440 |
5. Conclusions | High doses of VD | PMC10005440 | ||
Author Contributions | D.A.B.: Conceptualization, methodology, writing original article, data curation, investigation and review/editing the article. A.A. (Anas Abed): investigation, and review/editing. B.A.M.: methodology, writing and review/editing the article. A.A. (Ahmad Aljaberi): formal analysis and review/editing the article. A.S.: fo... | PMC10005440 | ||
Institutional Review Board Statement | Not applicable. | PMC10005440 | ||
Informed Consent Statement | Informed consent was obtained from all subjects involved in the study. | PMC10005440 | ||
Data Availability Statement | Not applicable. | PMC10005440 | ||
Conflicts of Interest | The authors declare no conflict of interest. | PMC10005440 | ||
References | tumor | TUMOR, VITAMIN D DEFICIENCY | Consort flow diagram for the study.Frequencies and percentages of anthropometric and lifestyle variables at baseline (Abbreviations: VDStatistical description of anthropometric parameters at baseline level (n = 75).Abbreviations: SD, standard deviation; BMI, body mass index; WHR, waist/hip ratio.Descriptive summary of ... | PMC10005440 |
Background | depression, PTSD, anxiety | ADVERSE EFFECTS | Educating dentists in treatment methods for dental anxiety would increase the patients’ access to treatments that are important to their oral health. However, to avoid adverse effects on comorbid symptoms, involvement by a psychologist has been considered necessary. The objective of the present paper was to evaluate wh... | PMC10288821 |
Methods | anxiety | A two-arm parallel randomised controlled trial was set in a general dental practice. Eighty-two patients with self-reported dental anxiety either completed treatment with dentist-administered cognitive behavioural therapy (D-CBT, | PMC10288821 | |
Results | PTSD, Depression, Anxiety, anxiety | -11 | An Intention-To-Treat analysis indicated reduced dental anxiety scores by the Modified Dental Anxiety Scale (median MDAS: 5.0 (-1,16)). The median scores on the Hospital Index of Anxiety and Depression (HADS-A/D) and the PTSD checklist for DSM-IV (PCL) were reduced as follows: HADS-A: 1 (-11, 11)/HADS-D: 0 (-7, 10)/PCL... | PMC10288821 |
Conclusions | depression, PTSD, anxiety | ADVERSE EFFECTS | The study findings support that a general dental practitioner may treat dental anxiety with Four Habits/Midazolam or D-CBT without causing adverse effects on symptoms of anxiety, depression or PTSD. Establishing a best practice for treatment of patients with dental anxiety in general dental practice should be a shared ... | PMC10288821 |
Trial registration | The trial was approved by REC (Norwegian regional committee for medical and health research ethics) with ID number 2017/97 in March 2017, and it is registered in clinicaltrials.gov 26/09/2017 with identifier: NCT03293342. | PMC10288821 | ||
Supplementary Information | The online version contains supplementary material available at 10.1186/s12903-023-03061-4. | PMC10288821 | ||
Keywords | Open access funding provided by University of Oslo (incl Oslo University Hospital) | PMC10288821 | ||
Background | phobias, phobia, anxiety, PTSD, depression | Dental anxiety has a high prevalence, and this combined with a high impact on oral health, constitutes a serious public health challenge [Cognitive behavioural therapy (CBT) is recognized as the treatment of choice for specific phobias, including the most severe form of dental anxiety, dental phobia [The daily manageme... | PMC10288821 | |
Methods | The trial was approved by REC (Norwegian regional committee for medical and health research ethics) with ID number 2017/97 and it is registered in clinicaltrials.gov with identifier: NCT03293342. | PMC10288821 | ||
Data | psychiatric, anxiety | ADVERSE REACTIONS, RECRUITMENT, TSD | The data presented in this article was collected in a general dental practice in a rural Norwegian town. A total of 96 patients were admitted to the study between September 2017 and March 2020. Recruitment was conducted by a research assistant. In a baseline session the research assistant provided more detailed informa... | PMC10288821 |
Sample of patients | anxiety | The inclusion criteria for the RCT study were as follows: (i) self-reported dental anxiety at a level of severity that affected the participant’s ability to receive dental treatment, and (ii) the ability to communicate fluently in Norwegian. The patients were mainly self-referred (In the present study, sample size calc... | PMC10288821 | |
One dentist treated all the patients | anxiety | Patients in both treatment conditions were treated by the same dentist, a 40-year-old Norwegian woman, who had completed her dental studies in Oslo 12 years prior to the study. Although she had no relevant specialisation, she had completed a theoretical exam on CBT treatment for dental anxiety (equivalent to a 40-h cou... | PMC10288821 | |
Video-evaluation | All sessions were videotaped. A random selection of tapes was evaluated by the co-authors (TW and BT) to assess adherence to manuals in both treatment conditions. | PMC10288821 | ||
Background measures | Anxiety, anxiety, traumatic, PTSD, depression, Depression, Post-Traumatic Stress Disorder | Age, sex, and years since last completed dental treatment were registered at enrolment along with symptoms of anxiety, depression and Post-Traumatic Stress Disorder (PTSD). Self-report scales were employed. All the scales are extensively used worldwide and have been shown to be valid and reliable (see details in the de... | PMC10288821 | |
Outcome measures | Anxiety, anxiety | The change in dental anxiety from pre- to one-year after-treatment was used as a measure of treatment efficiency. Dental anxiety was measured before, after and one year after treatment with the Modified Dental Anxiety Scale (MDAS) and the Index of Dental Anxiety and Fear (IDAF-4C). MDAS mentions 5 potentially frighteni... | PMC10288821 | |
Missing data | Fourteen patients did not complete the treatment. In addition, 5 patient records were lost at the storage facility (see details in Additional file [Additional file | PMC10288821 | ||
Statistical analysis | The data was not normally distributed (see additional file for normality tests [Additional file All analyses were done using the Stata/SE 16.0 statistical software. | PMC10288821 | ||
Discussion | phobia, anxiety, traumatic, PTSD, depression | EVENT, DISORDERS | In the present study comorbid symptoms of anxiety, depression or PTSD did not inhibit the treatment efficiency of two dentist-administered dental anxiety treatment methods. Nor did the treatment delivered coincide with an increase in symptoms of anxiety, depression, or PTSD in neither method. On the contrary, comorbid ... | PMC10288821 |
Conclusions | depression, anxiety | ADVERSE EFFECTS | In conclusion, efficient dental anxiety treatment could be administered by a trained dentist by at least two methods available in a general dental practice. The treatments investigated were not associated with adverse effects and were adequate for patients with severe dental anxiety and for patients with elevated sympt... | PMC10288821 |
Acknowledgements | We would like to thank professor Asle Hoffart and professor Olav Vassend for important contributions to study design, as well as Kari Laudal for her invaluable assistance throughout the course of the study and Anne Birgit Vintermyr for her important influence in ensuring that the study continued undisturbed through adm... | PMC10288821 | ||
Authors’ contributions | MSH contributed to conception, design, data acquisition and interpretation, statistical analysis, drafted and critically revised the manuscript. TW contributed to conception, design, data interpretation, drafted and critically revised the manuscript. BS contributed to conception, design, data interpretation, drafted an... | PMC10288821 | ||
Funding | Open access funding provided by University of Oslo (incl Oslo University Hospital). The study was funded by the Norwegian Directorate of Health. | PMC10288821 | ||
Availability of data and materials | The data that support the findings of this study are available from the Dental Faculty at the University of Oslo, but restrictions apply to the availability of these data, which were used under license for the current study, and so are not publicly available. Data are however available from Mariann Saanum Hauge or prof... | PMC10288821 | ||
Declarations | PMC10288821 | |||
Ethics approval and consent to participate | The trial was approved by REC (Norwegian regional committee for medical and health research ethics) with ID number 2017/97. A written informed consent was obtained from all participants. All methods were carried out in accordance with relevant guidelines and regulations in the Declaration of Helsinki. | PMC10288821 | ||
Consent for publication | Not applicable. | PMC10288821 | ||
Competing interests | The authors declare that they have no competing interests. | PMC10288821 | ||
References | PMC10288821 | |||
Background | SRS | BRAIN METASTASES, SOLID TUMORS | Comparative prospective data regarding different radiosurgery (SRS) modalities for treating brain metastases (BMs) from solid tumors are not available. To investigate with a single institute phase III randomized trial whether SRS executed with linac (Arm-B) is superior to a dedicated multi-source gamma-ray stereotactic... | PMC9906937 |
Methods | brain failure, radionecrosis | SOLID TUMORS | Adults patients with 1–4 BMs from solid tumors up to 30 mm in maximum diameter were randomly assigned to arms A and B. The primary endpoint was cumulative incidence of symptomatic (grade 2–3) radionecrosis (CIRN). Secondary endpoints were local progression cumulative incidence (CILP), distant brain failure, disease-fre... | PMC9906937 |
Results | A total of 251 patients were randomly assigned to Arm-A (121) or Arm-B (130). The 1-year RN cumulative incidence was 6.7% in whole cohort, 3.8% (95% CI 1.9–7.4%) in Arm-B, and 9.3% (95% CI 6.2–13.8%) in the Arm-A ( | PMC9906937 | ||
Supplementary Information | The online version contains supplementary material available at 10.1186/s13014-023-02216-5. | PMC9906937 | ||
Keywords | PMC9906937 | |||
Introduction | cancer | BRAIN METASTASES, CANCER | The occurrence of brain metastases (BMs) is a challenging issue affecting about 20–40% of cancer patients [ | PMC9906937 |
Methods and materials | PMC9906937 | |||
Study design and patients | SCLC, active extra-cranial disease, brain failure, SRS | ADVERSE EVENT, SCLC, DISEASE, HEMATOLOGIC MALIGNANCY, SOLID TUMORS, SMALL CELL LUNG CANCER | The present is a single institute prospective randomized, double-arms phase 3 trial approved by our institutional review board, registered at the ClinicalTrials.gov site with the number NCT02355613. The trial was approved by the Institutional Review Board, and it was monitored by the institutional Data and Safety Monit... | PMC9906937 |
Study design and randomization | The primary endpoint was the cumulative incidence of patients experiencing symptomatic RN (grade 2–4). A superiority design was assumed. Symptomatic RN was expected at around 10% at one year for Arm-A. Considering a rate of ~ 1% for Arm-B, 250 patients were required for the study to have a power of 80% to demonstrate t... | PMC9906937 | ||
Treatment regimens | tumor, isodose, T1MRI, OSMS, SRS | SEPARATION, TUMOR, METASTASES | Concerning patient immobilization and treatment planning imaging, in Arm-A, a Leksell frame system was applied, and a volumetric MRI was performed. The target volume was defined as the contrast-enhancing tumor on the volumetric T1MRI. Prescription doses were 24 Gy for BMs ≤ 20 mm, and 20 Gy for lesions 21–30 mm, delive... | PMC9906937 |
Follow-up and outcomes | toxicities, SRS | RECURRENCES, DISEASE PROGRESSION, ADVERSE EVENT, BRAIN | Clinical and radiological evaluations have been performed at baseline, one month after treatment, and every 3 months after that, or until disease progression; they included history and physical examination, blood tests, and KPS. Hematologic and non-hematologic toxicities were graded according to Common Terminology Crit... | PMC9906937 |
Statistical analysis | primary tumor, death, SRS | EVENT, DISEASE, LUNG METASTASES, PRIMARY TUMOR, METASTASES | Time to RN was defined as starting from the day of SRS up to the date of the MRI evaluation. Time to progression was defined as the interval from the date of SRS to the documentation of progression and evaluated for CNS progression (DBF), local progression and DFS, according to the different kinds of failures. Local pr... | PMC9906937 |
Results | PMC9906937 | |||
Patients and tumor characteristics | NSCLC, Renal carcinoma cancer, tumor, HCC, Hepatocellular carcinoma, RCC, SRS | TUMOR, BRAIN METASTASES, NON SMALL CELL LUNG CANCER, NSCLC, METASTASES, RCC | Between October 2014 and June 2020, 251 patients for 449 treated BMs were enrolled and randomly assigned to Arm-A (121) or Arm-B (130), as shown in Fig. CONSORT flow diagram of patients enrolled in the randomized trial comparing Arm-A and Arm-BThe total number of eligible patients was 312, and 19.5% were excluded for t... | PMC9906937 |
Discussion | SRS | MINOR, METASTASES | The results of a randomized phase 3 trial comparing two SRS treatment approaches using different platforms, each characterized by specific features, were reported. The main differing characteristics among the two arms are: (i) the use of a rigid frame system in one and a frameless in the other; (ii) the employ of margi... | PMC9906937 |
Conclusion | BRAIN METASTASES | Given the technical differences between the treatment platforms investigated in this single-institution study, linac-based SRS (Arm-B) did not lead to significantly lower rates of grade 2–3 RN versus the multi-source gamma-ray system (Arm-A) in a population of patients with limited brain metastases of limited volume. N... | PMC9906937 | |
Author contributions | Study concept and design: MS, ST, PN. Acquisition, analysis, or interpretation of data: EC, LB, AF, FP, ST, LC, PN. Statistical analysis: VT. Administrative, technical, or material support: LA, CF, MM, LR, DF, GR, FL. Drafting of the manuscript: PN, ST. Critical revision of the manuscript for important intellectual con... | PMC9906937 | ||
Funding | Research reported in this publication was partially supported by Varian Medical Systems, Palo Alto, USA. Role of funder/sponsor: This manuscript’s contents are solely the responsibility of the authors and do not necessarily represent the official views of Varian Medical systems. The funder had no role in the design, co... | PMC9906937 | ||
Availability of data and materials | Research data are store in an institutional repository and will be shared upon request to the corrisponding author. | PMC9906937 | ||
Declarations | PMC9906937 | |||
Ethics approval and consent to participate | The present is a single institute prospective randomized, double-arms phase 3 trial approved by our institutional review board, registered at the ClinicalTrials.gov site with the number NCT02355613. The trial was approved by the Institutional Review Board, and it was monitored by the institutional Data and Safety Monit... | PMC9906937 | ||
Consent for publication | Post my preprint. | PMC9906937 | ||
Competing interests | Cancer | CANCER | L.C. acts as Scientific Advisor to Varian Medical Systems and is Clinical Research Scientist at Humanitas Cancer Center. The other authors declare no competing interests. | PMC9906937 |
References | PMC9906937 | |||
Background | Sanfilippo type B, neuronopathic | MUCOPOLYSACCHARIDOSIS (MPS) I | Sanfilippo type B is a mucopolysaccharidosis (MPS) with a major neuronopathic component characterized by heparan sulfate (HS) accumulation due to mutations in the | PMC9843052 |
Methods | In this phase I/II open-label study, patients with MPS type IIIB ( | PMC9843052 | ||
Results | hepatomegaly | RESOLUTION | In the Part 1 dose escalation (30, 100, and 300 mg) phase, a 300 mg dose of tralesinidase alfa was necessary to achieve normalization of HS and HS-NRE levels in the CSF and plasma. In Part 2, 300 mg tralesinidase alfa sustained HS and HS-NRE normalization in the CSF and stabilized cortical gray matter volume (CGMV) ove... | PMC9843052 |
Conclusion | Administration of tralesinidase alfa i.c.v. effectively normalized HS and HS-NRE levels as a prerequisite for clinical efficacy. Peripheral drug exposure data suggest a role for the glymphatic system in altering tralesinidase alfa efficacy. | PMC9843052 | ||
Trial registration | Clinicaltrials.gov NCT02754076. | PMC9843052 | ||
FUNDING | BioMarin Pharmaceutical Inc. and Allievex Corporation. | PMC9843052 | ||
Introduction | LSDs, PD, MPS IIIB | LYSOSOMAL STORAGE DISEASES, GENETIC DISORDERS, SANFILIPPO SYNDROME, SANFILIPPO SYNDROME TYPE B | Lysosomal storage diseases (LSDs) are a family of genetic disorders affecting the breakdown and recycling of complex molecules within the lysosomes (Sanfilippo syndrome, or MPS type III, is categorized into 4 subtypes, A–D, according to defects in genes coding for 4 different enzymes. Sanfilippo syndrome type B (MPS II... | PMC9843052 |
Results | PMC9843052 | |||
Patients’ characteristics and adverse events. | anaphylactic reaction, neurodegenerative disease, choking, vomiting, pyrexia, TEAEs, vomiting, rash, SAEs, pyrexia, infection, angioedema, headache, deaths, hypersensitivity, vomiting, listlessness | ANAPHYLACTIC REACTION, NEURODEGENERATIVE DISEASE, ADVERSE EVENTS, CSF PLEOCYTOSIS, EVENT, UPPER RESPIRATORY TRACT INFECTION, WOUND INFECTION, COMPLICATIONS, EVENTS, CHOKING, BLOOD, INFECTION, ADVERSE EVENT, ANGIOEDEMA, CSF LEAKAGE, HYPERSENSITIVITY, SUBDURAL HYGROMA | Interventional Study 250-201 was divided into Part 1 and Part 2 (Patient 9001 in Study 250-201 Part 1 received 28 weekly doses at 30 mg of tralesinidase alfa, followed by 10 doses at 100 mg, and then 11 doses at 300 mg before being recruited to Study 250-201 Part 2. Patient 9002 received 8, 10, and 19 doses of 30, 100,... | PMC9843052 |
Immunogenicity. | The ADA response was monitored in both CSF and serum during the course of Study 250-201 Part 2 (The only 3 patients with ADA in serum at week 1 were those who had already received tralesinidase alfa treatment in Study 250-201 Part 1 (The largest drop in plasma drug exposure occurred between week 1 and week 5 of Study 2... | PMC9843052 | ||
Tralesinidase alfa 300 mg normalizes HS and HS-NRE. | Total HS and HS-NRE concentrations in CSF were measured weekly over the course of Study 250-201 Part 1 (During the course of Study 250-201 Part 2, HS-NRE levels in the CSF were normalized after the first 4 weeks of treatment to levels below the 10 ng/mL higher-end-of-normal cutoff (We observed significant correlations ... | PMC9843052 | ||
Weekly administration of tralesinidase alfa 300 mg resolves organomegaly. | MPS IIIB | Changes in liver and spleen volumes at weeks 1, 24, and 48 of Study 250-201 Part 2 are depicted in At Study 250-201 Part 1 or Part 2 baseline, spleen volumes in children with MPS IIIB ranged from 0.15 to 0.44 L/m | PMC9843052 | |
Tralesinidase alfa stabilizes brain atrophy in patients with MPS IIIB. | atrophy, MPS IIIB | ATROPHY | CGMV is a measure of the region of the brain most affected by atrophy in patients with MPS IIIB within the age range included in Study 250-201 (Participants enrolled in Study 250-201 lost, on average, 58 mL (median 68 mL) of CGMV from baseline to week 24, but lost only 2 mL from week 24 to week 48, for a total loss of ... | PMC9843052 |
Correlations between tralesinidase alfa exposure and cognitive decline trajectory and plasma HS-NRE levels. | MPS IIIB | DISEASE | A treatment duration of longer than 48 weeks will be needed to establish whether there is a clinically meaningful benefit for cognition related to treatment with tralesinidase alfa. However, to elucidate whether there is early evidence of an effect of tralesinidase alfa on disease trajectory in patients with MPS IIIB, ... | PMC9843052 |
Correlations of cognitive decline trajectory compared with plasma HS-NRE and cortical gray matter volume. | We also observed an inverse correlation between cumulative plasma HS-NRE concentrations and a change in cognitive AEq score from week 1 to week 48 of Study 250-201 Part 2 (We observed a correlation between a change in the cognitive AEq score over the course of Study 250-201 Part 2 and a change in CGMV during the same p... | PMC9843052 | ||
Discussion | sleep behavior, hepatomegaly, MPS, MPS IIIB, hepatosplenomegaly, PD, cognitive and communication skills, LSDs, cognitive decline | BRAIN ATROPHY, DISEASE | In the present study, we show that, within weeks of weekly i.c.v. administration, 300 mg tralesinidase alfa could effectively compensate for NAGLU enzymatic activity as demonstrated by the normalization of HS and HS-NRE levels in both the CSF and plasma. Consequently, we observed resolution of hepatomegaly in most of t... | PMC9843052 |
Methods | PMC9843052 | |||
Study designs. | MPS, MPS IIIB | Interventional study NCT02754076, i.e., Study 250-201, was a phase I/II open-label, dose-escalation (Part 1), and stable-dose (Part 2) study involving males and females between 1 and less than 11 years of age (i.e., 12 months and 132 months) with a confirmed diagnosis of MPS IIIB. Three patients, i.e., patients 9001, 9... | PMC9843052 | |
Inclusion and exclusion criteria. | All study participants had deficient NAGLU enzyme activity and | PMC9843052 | ||
Cognition. | Cognitive function was measured using the nonverbal scales of the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) (The BSID-III is a validated and standardized developmental tool comprising 5 domains (cognitive, language, motor, social-emotional, and adaptive functioning) intended to assess deve... | PMC9843052 | ||
MRI assessments. | LIVER, BRAIN | Head and abdominal MRIs were used to assess regional brain, liver, and spleen volumes at baseline, week 24, and week 48 of Study 250-201. Brain volumetric analysis was performed on 3D T1-weighted images using the FreeSurfer Image Analysis Suite, version 5.3 (Martinos Center, Harvard University) (Brain volumes are noted... | PMC9843052 | |
Laboratory testing. | Tralesinidase alfa quantification in CSF and K2EDTA plasma was based on an electrochemiluminescence immunoassay (ECLA) technique using standard-bind, 96-well Meso Scale Discovery (MSD) streptavidin-coated plates. Samples were collected before dosing and 0.5, 4, 10, 24, 48, 72, 96, and 168 hours after drug administratio... | PMC9843052 | ||
Statistical analysis. | No corrections or carry-forwards were made for missing values. A 2-tailed, paired | PMC9843052 | ||
Study approval. | Written informed consent from a parent or legal guardian and assent from the participant, if required, was obtained prior to conducting any study-specific assessments. This study was approved by the IRB affiliated with each individual clinical site: Children’s Hospital Oakland, Oakland, California, USA; Mackay Memorial... | PMC9843052 | ||
Author contributions | AJS and SMM designed the clinical protocol. NM, AK, KVC, IO, FE, PH, MJDCL, MLC, SPL, SB, MC, and MS recruited participants and performed and supervised clinical practices. IN designed and performed the brain MRI analysis. B Kuca supervised the clinical study execution and was responsible for regulatory submissions. EZ... | PMC9843052 | ||
Supplementary Material | PMC9843052 | |||
Supplemental data | PMC9843052 | |||
Trial reporting checklists | PMC9843052 |
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