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Data availability
The datasets generated and analyzed during the current study are available from the corresponding author on reasonable request.
PMC10141830
Declarations
PMC10141830
Conflict of interest
The authors declare that they have no conflict of interest.
PMC10141830
Ethical approval
The study has been approved by our institutional review board (IRB:21/2022) and has been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments.
PMC10141830
References
PMC10141830
Abstract
PMC10225203
Aim
gastric cancer, breast cancer
GASTRIC CANCER, BREAST CANCER
Pyrotinib (an irreversible pan‐ErbB small‐molecular tyrosine kinase inhibitor) was approved in human epidermal growth factor receptor 2 (HER2)‐positive breast cancer and showed great antitumor activity in preclinical studies of gastric cancer (GC). This study was first designed to prospectively assess pyrotinib in pret...
PMC10225203
Methods
This multicenter, phase I study followed a standard “3 + 3” design and included two parts. In the pyrotinib part, pyrotinib was administered orally, once per day at dose levels of 240, 320, 400, and 480 mg. In the pyrotinib plus docetaxel part, patients received pyrotinib (qd, d1‐21, q3W) combined with docetaxel (60 mg...
PMC10225203
Results
A total of 25 patients were enrolled and received pyrotinib (
PMC10225203
Conclusions
toxicities
Pyrotinib alone and combined with docetaxel showed acceptable toxicities in patients with pretreated HER2‐positive GC.
PMC10225203
Trial Registration
This study was registered with This phase 1 study demonstrate the safety and efficacy of pyrotinib in HER2 positive GC. Pyrotinib alone and plus docetaxel are well‐tolerated and show anti‐tumor activity. But combination with docetaxel cannot improve the efficacy. Dan Liu, Furong Kou, and Jifang Gong contributed equally...
PMC10225203
BACKGROUND
gastric cancer (GC).Pyrotinib, tumor, breast cancer
PROLIFERATION, TUMOR, BREAST CANCER
Human epidermal growth factor receptor 2 (HER2) was a key therapeutic target in gastric cancer (GC).Pyrotinib was a novel oral, irreversible pan‐ErbB small‐molecular TKI blocking HER1, HER2, and HER4, which was approved in HER2‐positive breast cancer (BC). And it has been reported that pyrotinib could inhibit the proli...
PMC10225203
PATIENTS AND METHODS
PMC10225203
Patient eligibility
cancers, autoimmune diseases, hemorrhage, syphilis, chronic disease or, effusion, gastrointestinal obstruction, hypokalemia
HEPATITIS B, CANCERS, VIRUS, HEART DISEASE, BRAIN METASTASES, AUTOIMMUNE DISEASES, HYPOMAGNESEMIA, HUMAN IMMUNODEFICIENCY VIRUS INFECTION, HEMORRHAGE, MALIGNANCIES, SYPHILIS, HEPATITIS C, EFFUSION, ONCOLOGY, GASTROINTESTINAL OBSTRUCTION
Patients with pathologically confirmed advanced HER2‐positive (3+ or 2+ staining intensity by immunohistochemistry or gene amplification by fluorescence in situ hybridization amplification [HER2:CEP17 ratio ≥2]) GC who suffered previous standard treatments (without docetaxel or other anti‐HER2 TKIs) failure or intolera...
PMC10225203
Study design and treatment
This multicenter, open‐label, phase I dose escalation study was conducted in China from September 2014 (ClinicalTrials. gov, NCT02378389). The study was approved by the ethics committee of each study center and performed in accordance with the International Conference on Harmonization Guidelines for Good Clinical Pract...
PMC10225203
Endpoints
SECONDARY
The primary endpoints were to assess the maximum tolerated dose (MTD), safety and the recommended phase II dose (RP2D) of pyrotinib monotherapy and pyrotinib in combination with docetaxel. The secondary endpoints included PK evaluation and preliminary efficacy of pyrotinib monotherapy and pyrotinib combined with doceta...
PMC10225203
Safety assessment
nausea, DLTs, neutropenia, fever, fatigue, diarrhea, thrombocytopenia, vomiting, anemia, heart and renal insufficiency
NEUTROPENIA, ADVERSE EVENTS, THROMBOCYTOPENIA, ANEMIA, BLEEDING TENDENCY
Safety was evaluated throughout the study. All adverse events (AEs) were recorded, the severity was assessed according to NCI‐CTCAE (V.4.0), and the relationship with pyrotinib or docetaxel was judged.DLT criteria included Grade 4 neutropenia with a duration of ≥7 days, Grade 3 or 4 neutropenia with fever, Grade 3 thro...
PMC10225203
The main PK parameters were estimated by a standard noncompartmental method using Phoenix WinNonLin (Pharsight, version 6.3, Pharsight Corp.), including elimination half‐life (In pyrotinib monotherapy part, blood samples were obtained on D1 and D21 of the first cycle at predose; 1, 2, 3, 4, 5, 6, 8, 12, and 24 h after ...
PMC10225203
Efficacy assessment
death, Tumor
DISEASE PROGRESSION, TUMOR
Tumor responses were evaluated based on RECIST 1.1 at screening and every two cycles until disease progression or the start of new antitumor treatment. The complete and partial response would be confirmed after 4 weeks. All the progression‐free survival (PFS) was defined as the time from the start of the first dose to ...
PMC10225203
Statistical analysis
tumor
TUMOR
Statistical analysis was performed by SPSS Version 22.0 (SPSS Inc.). The safety was assessed in patients who received at least one dose of study drugs. Patient characteristics and efficacy were assessed in the full analysis set, including patients who received at least one dose of study drugs. Descriptive statistics we...
PMC10225203
RESULTS
PMC10225203
Patient characteristics
Between September 2014 and February 2017, 25 patients with HER2‐positive GC were finally enrolled in this study. The median age was 58 years (range 39–65). And the majority of patients (Patient demographics and baseline characteristics. Other anti‐HER2 therapies: trastuzumab emtansine (T‐DM1), pertuzumab.All the patien...
PMC10225203
Safety
ADVERSE EVENTS
A total of 25 patients were included in safety analysis (Table Treatment‐related adverse events of all grades in pyrotinib monotherapy part. Treatment‐related adverse events of all grades in pyrotinib combined with docetaxel part.In the combination with docetaxel part, for no DLT observation of pyrotinib alone at dose ...
PMC10225203
PK
Totally, 22 patients provided plasma samples for PK analysis (monotherapy, Mean plasma concentration of pyrotinib following (A) single and (B) multiple doses in the monotherapy part (PK population).
PMC10225203
Antitumor activity
PD, SD, diarrhea
DISEASE PROGRESSION, DISEASE, BEST
In all, from September 2014 to February 2017, 24 patients finished efficacy assessment (monotherapy, Best overall response in evaluable population in monotherapy part and combination therapy part.Abbreviations: CR, complete response; DCR, disease control rate: (CR + PR + SD) %; ORR, overall response rate: (CR + PR) %; ...
PMC10225203
DISCUSSION
toxicity, diarrhea
DISEASE
Pyrotinib is an irreversible pan‐ErbB small‐molecular TKI, which has been approved in HER2‐positive BC. While the safety and efficacy data were limited in HER2‐positive GC, only several small‐sample retrospective studies and case reports involved it.In the monotherapy part, diarrhea was the most common TRAE. And two pa...
PMC10225203
CONCLUSIONS
HER2 POSITIVE GASTRIC CANCER
In brief, pyrotinib alone and combined with docetaxel had shown acceptable safety profile in HER2 positive gastric cancer. Pyrotinib plus docetaxel might not further improve the efficacy.
PMC10225203
AUTHOR CONTRIBUTIONS
PMC10225203
FUNDING INFORMATION
This phase I clinical trial (NCT02378389) was sponsored by Jiangsu Hengrui Medicine Co., Ltd., and supported by the National Natural Science Foundation of China (no. 91959205).
PMC10225203
CONFLICT OF INTEREST STATEMENT
Jianjun Zou and Xiaoyu Zhu are employees of Hengrui. All other authors declare no competing interests.
PMC10225203
ETHICS STATEMENT
Cancer
CANCER
This study was approved by the ethics committee of Beijing Cancer Hospital (2014YW17) and Sun Yat‐sen University Cancer Center (A2014‐029‐02).
PMC10225203
Supporting information
Click here for additional data file.
PMC10225203
ACKNOWLEDGMENTS
We are grateful to all patients and their families and thank the investigators and the research staff of this study.
PMC10225203
DATA AVAILABILITY STATEMENT
data‐access
Reasonable request for data sharing should be submitted to the corresponding author after the indication has been approved in China. The sponsor will review the proposal and consider to share the data providing the requestor signs a data‐access agreement.
PMC10225203
REFERENCES
PMC10225203
INTRODUCTION
DISEASE, CORTEX
UP165, a standardized Current Centers for Disease Control and PreventionWhile there are some psychotropic clinical and anecdotal data available to support the usage of sedative and anxiolytic plants like kava kava, Cortisol, a glucocorticoid released from the adrenal cortex, is more than a stress hormone known to regul...
PMC9889011
MATERIALS AND METHODS
PMC9889011
Receptor binding assay
To test the ability of UP165 to bind to the melatonin binding site of the melatonin receptor 1 (MT1) and 2 (MT2) melatonin receptors, membrane-bound human MT1 and MT2 melatonin receptors were incubated with UP165 in the presence of [Cell membranes from human recombinant Chinese hamster ovary (CHO)-K1 cells expressing t...
PMC9889011
Clinical study design
This study enrolled 45 healthy volunteers (24 female and 21 male subjects) to participate in a 6-week sleep and overall well-being trial. Subjects were randomized to receive UP165 (Supplement) or a nonactive corn starch placebo (Placebo). Subjects (age range 19–73 years) were instructed to consume the supplement or pla...
PMC9889011
Study measuring tools
(1) Objective sleep quality was measured using the Garmin VivosmartThe sum of scores for these seven components yields one global score, with higher scores corresponding to worsening sleep quality and efficiency.
PMC9889011
Inclusion criteria
Healthy adults, able to provide informed consent, and with the ability/desire to participate in a 4-week supplementation regimen and sleep quality study were included in the study.
PMC9889011
Exclusion criteria
sleep disorders
Inability to complete prescribed supplement regimen, current use of incompatible medications or supplements, high use of caffeine (500 mg/day) or other stimulants, off shift and night shift workers, and those diagnosed with severe sleep disorders were excluded from this study.
PMC9889011
Ethics and regulatory approval
MAY
The study was performed in compliance with the requirements of Good Clinical Practice/International Council for Harmonisation (GCP/ICH) Guidelines for clinical trials. The Study gained full board review from Institutional Review Board (IRB) on May 14, 2020, by WCG Aspire IRB. Protocol number Maizinol (UP165) 2020 IRB T...
PMC9889011
Participant informed consent
All subjects provided written informed consent to participate in the study before being screened and a copy was provided for their information. Subjects were also free to withdraw from the study at any time without giving a reason.
PMC9889011
Compliance
ADVERSE EVENT
Adverse event (AE) data were collected by subject reporting of any AE, which were captured in subject charts. This study was conducted in a healthy population, and subjects were contacted weekly by phone call, text, or email. Subjects were given a number to call if there was any issue to report. Compliance was a combin...
PMC9889011
Statistical analysis
Data were analyzed using John's Macintosh Project (JMP) 14.0 data analysis software (SAS Institute, Cary, NC, USA). The results are represented as mean ± standard deviation. Statistical significance between groups was calculated by means of single-factor analysis of variance (ANOVA) followed by a paired
PMC9889011
RESULTS
PMC9889011
Receptor binding assay result
OVARY
UP165 (Lot. No. FP041019-01) was tested in duplicate at 10 concentrations for its MT1 and MT2 binding affinity. UP165 showed a fourfold increase in MT2 receptor binding affinity. UP165 had a dose-responsive curve with an ICPercent inhibition of ligand binding to MT1. Human recombinant CHO-K1 cells expressing the human ...
PMC9889011
Clinical study compliance and completion rate
Participants were compliant for all the procedures and supplementation for the duration of the study. Forty-two (23 female and 19 male) participants completed the study. No AE was reported for test article or placebo. Three participants dropped out of study due to scheduling conflicts (Study Enrollment and DropoutThe s...
PMC9889011
Effect of UP165 on deep sleep
Increase in the deep sleep time was observed for participants who were supplemented with the test article, UP165. Compared to placebo, these increases in the deep sleep time were statistically significant for participants supplemented with the 250 mg/day UP165 at week 2 and 3 with Effect of UP165 on the Average Deep Sl...
PMC9889011
Effect of UP165 on total sleep time
SE
Compared to the baseline, UP165 resulted in a moderate, but statistically significant (The average total sleep time before and after three supplements. The study enrolled 45 volunteers to participate in a 6-week trial. Participants (age range 19–73) consumed the supplement or placebo ∼60 minutes before bedtime daily fo...
PMC9889011
Effect of UP165 supplementation on sleep quality improvement as it was measured by PSQI
SE
The PSQI is a self-rated questionnaire that assesses sleep quality and disturbances over a 4-week time interval. Participants who were supplemented with UP165 showed a statistically significant improvement in self-rated questionnaires, indicating the effect of UP165 in enhancing sleep quality and efficiency in healthy ...
PMC9889011
Effect of UP165 on salivary cortisol level
CORTEX
Cortisol, one of the major glucocorticoid hormones secreted by the adrenal cortex, is among the hormones that regulate human sleep, with its increase being associated with poor sleep quality. In this clinical study, subjects supplemented with UP165 showed a progressive decrease in salivary cortisol level through the co...
PMC9889011
Effect of UP165 on POMS
The POMS is a well-validated psychological rating scale used to assess both overall well-being (Global Mood State) and distinct specific mood states (Subscales). Data were analyzed to determine the presupplementation and postsupplementation effects of the test article, UP165, on the overall well-being of test subjects ...
PMC9889011
DISCUSSION
hypothermia, daytime sleepiness, dizziness, morning grogginess, headache
MUSCLE RELAXATION
Despite significant research efforts for sleep and mental health management, to date, available intervention options from natural sources are very limited. In particular, the sleep aid market is significantly saturated by high doses of synthetic melatonin or cocktails of melatonin and other botanical extracts with mini...
PMC9889011
ACKNOWLEDGMENT
The authors would like to express their best gratitude to Dr. Teresa Horm, who oversaw the receptor binding assay, and participated in data calculation and interpretation; Dr. Ping Jiao, who conducted activity-guided structure elucidations and identification; Dr. Wenwen Ma; Dr. Qi Jia; Mr. Regan Miles; and Unigen's tea...
PMC9889011
AUTHOR DISCLOSURE STATEMENT
Both authors, except S.M.T. and J.A.T. are current Unigen employees; therefore, they have competing financial interests.
PMC9889011
FUNDING INFORMATION
The clinical study was fully sponsored by Unigen. The authors would like to extend their utmost gratitude to Mr. Bill Lee, the owner of Econet/Unigen, Inc., who supported the entire project described in this article.
PMC9889011
REFERENCES
PMC9889011
Background
Children’s conduct and emotional problems increased during the COVID-19 pandemic.
PMC10415938
Objective
We tested whether a smartphone parenting support app,
PMC10415938
Methods
Supporting Parents And Kids Through Lockdown Experiences (SPARKLE), a randomized controlled trial, was embedded in the UK-wide COVID-19: Supporting Parents, Adolescents and Children during Epidemics (Co-SPACE) longitudinal study on families’ mental health during the pandemic. Parents of children aged 4 to 10 years were...
PMC10415938
Results
A total of 320 participants were randomized to
PMC10415938
Trial Registration
ClinicalTrials.gov NCT04786080; https://clinicaltrials.gov/ct2/show/NCT04786080
PMC10415938
Introduction
PMC10415938
Background
SAID
UK COVID-19 mitigation strategies (eg, extended joint confinement, social isolation, and homeschooling) presented families with unprecedented challenges [Providing parents with advice and support can reduce children’s conduct and emotional problems [The focus for each booster and the parenting challenge it was designed...
PMC10415938
Objectives
In Supporting Parents And Kids Through Lockdown Experiences (SPARKLE), using a randomized controlled trial (RCT) comparison of
PMC10415938
Methods
PMC10415938
Patient and Public Involvement
Parents of young children were involved in all aspects of the study, including designing the research, designing the app, participating in the Trial Steering Committee (TSC), and interpreting the results. The SPARKLE Patient and Public Involvement parent panel included 13 members, with 1 parent member who sat on the TS...
PMC10415938
Study Design
SPARKLE was a rapid-deployment 2-arm superiority parallel group RCT of
PMC10415938
Participants
RECRUITMENT
Participants were parents or carers of children aged 4 to 10 years. The inclusion criterion for Co-SPACE was parents aged ≥18 years residing in the United Kingdom with a child aged 4 to 16 years. For SPARKLE, only families with children aged 4 to 10 years and access to a compatible smartphone were included. Recruitment...
PMC10415938
Randomization and Blinding
Participants were allocated to the study arm in a 1:1 ratio by simple randomization automatically at baseline through the
PMC10415938
Ethics Approval
Ethics approval was granted by King’s College London (reference HR-20/21-21,451) and the University of Oxford (references R73153/RE001 for SPARKLE and R69060/RE001 for Co-SPACE).
PMC10415938
Procedures
PMC10415938
Interventions
PMC10415938
Parent Positive
The
PMC10415938
FAU Arm
Parents allocated to FAU received access to
PMC10415938
Data Collection
Measures were administered at baseline (T1) and at1 (T2) and 2 (T3) months after randomization through Qualtrics as part of Co-SPACE data collection.
PMC10415938
Outcomes
PMC10415938
Clinical Outcomes
SECONDARY
The primary outcome was parent-reported child conduct problems using the conduct subscale of the Parent-reported SDQ conduct problems score at 2 months after randomization (T3) was a secondary outcome. Other validated secondary outcomes were T2 and T3 measures of (1) parent-reported child emotional problems measured on...
PMC10415938
Health Economic Measures
Retrospective child-related use of health and social care services, including those provided in schools, was measured at T2 and T3 using a modified version of the
PMC10415938
Intervention Use and Satisfaction
The main
PMC10415938
Other Measures
ADVERSE EVENT, EVENTS
Family characteristics and demographic information were collected at baseline. Data on lockdown-related circumstances (eg, being in lockdown) were collected. Adverse events (AEs) were measured using a questionnaire developed for SPARKLE that asked parents to report negative events related to their child’s and their own...
PMC10415938
Sample Size Calculation
The target sample size of 616 provided 90% power to detect a Cohen
PMC10415938
Analysis
The
PMC10415938
Outcome Analysis
All analyses were carried out using Stata (version 17.0; StataCorp). Baseline and postrandomization app use variables were described by intervention arm and overall, with categorical variables described using frequencies and proportions and continuous variables described using mean and SD or median and IQR as appropria...
PMC10415938
Economic Analysis
Between-arm differences in costs and QALYs were analyzed using generalized linear models with bootstrapped 95% CIs adjusted for prespecified T1 covariates and CHU9D scores. Adjustment for differences in T1 costs was not possible as service use data were not collected in Co-SPACE; the CA-SUS was administered at T2 and T...
PMC10415938
Oversight of the Trial
The independent TSC, consisting of clinicians, statisticians, health economists, policy makers, and Patient and Public Involvement representatives, met every 4 months. No interim analyses were undertaken. The role of the Data Monitoring and Ethics Committee was taken over by the TSC.
PMC10415938
Results
PMC10415938
Recruitment and Retention
MAY
Between May 19, 2021, and July 26, 2021, a total of 646 parents were recruited, of whom 320 (49.5%) were assigned to CONSORT (Consolidated Standards of Reporting Trials) diagram. No longer providing=no Co-SPACE follow-up data. Exclusions: a member of staff signed up to test the randomization system. Early in the study,...
PMC10415938
Service Use, Costs, and Economic Outcomes
Service use and cost data are presented in Tables S5-S7 in Excluding influential outliers, complete case mean costs were lower in the
PMC10415938
Economic Analysis
SECONDARY
The results of the primary, sensitivity, and secondary economic analyses are summarized in Table S9 in
PMC10415938
Discussion
PMC10415938
Limitations
The RCT was conducted during spring 2021 and summer 2021, when pandemic-related restrictions were beginning to ease (ie, children were not homeschooled en masse, and restrictions on social contacts had relaxed). In this sense, we are unable to generalize our findings to more restrictive lockdown periods or to nonrestri...
PMC10415938
Conclusions
ARC, Maudsley, anxiety
POSITIVE
This was the first RCT of a parenting support app designed specifically to support parents in the general population during the COVID-19 pandemic and one of the few examples of a trial within a cohort in our field. It used a rigorous design in a large sample and collected data on a range of outcomes over a 2-month peri...
PMC10415938
Abbreviations
Anxiety
adverse eventChild and Adolescent Service Use ScheduleChild Health Utility–9 DimensionsConsolidated Standards of Reporting TrialsCOVID-19: Supporting Parents, Adolescents and Children during EpidemicsDepression, Anxiety, and Stress ScaleEmpowering Parents Empowering Communitiesfollow-up as usuallinear mixed modelqualit...
PMC10415938
Subject terms
TRD, nausea, somnolence, treatment-resistant depression, depressive disorder, dizziness, treatment-resistant, headache, depression, Depression
ADVERSE EVENTS, REMISSION, NASOPHARYNGITIS
Patients with treatment-resistant depression (TRD) have higher rates of relapse and pronounced decreases in daily functioning and health-related quality of life compared to patients with major depressive disorder who are not treatment-resistant, underscoring the need for treatment choices with sustained efficacy and lo...
PMC10267177
Introduction
depressive disorder, TRD, MDD, treatment-resistant depression
Approximately one-third of patients with major depressive disorder (MDD) do not achieve an antidepressant response despite treatment with multiple antidepressants and are considered to have treatment-resistant depression (TRD) [Esketamine nasal spray (Spravato
PMC10267177
Methods
PMC10267177
Ethical practices
An Institutional Review Board (United States) or Independent Ethics Committee (all other locations) approved the study protocol and its amendments. The study is being conducted in accordance with ethical principles of the Declaration of Helsinki, Good Clinical Practices (GCP), and applicable regulatory requirements. Wr...
PMC10267177