Endpoint Determination
View article: Supplementary Data1 from Meta-Analysis of the Test–Retest Repeatability of [<sup>18</sup>F]-Fluorodeoxyglucose Standardized Uptake Values: Implications for Assessment of Tumor Response
Supplementary Data1 from Meta-Analysis of the Test–Retest Repeatability of [<sup>18</sup>F]-Fluorodeoxyglucose Standardized Uptake Values: Implications for Assessment of Tumor Response Open
Statistical methodology
View article: Redefining endpoints in heart failure clinical trials: the emerging role of wearable technologies in contemporary trial design
Redefining endpoints in heart failure clinical trials: the emerging role of wearable technologies in contemporary trial design Open
Randomised controlled trials (RCTs) in heart failure (HF) have progressively broadened their primary endpoints over recent decades. Early landmark HF trials demonstrated the life-saving effects of new therapies using all-cause mortality as…
View article: A patient-centric paradigm and tool for clinical research: the DOOR is open
A patient-centric paradigm and tool for clinical research: the DOOR is open Open
Randomized clinical trials are the gold standard for evaluating the benefits and harms of interventions and yet may not provide the evidence needed to inform medical decision-making, an ultimate goal for clinical research. Commonly used de…
View article: Biomarkers of Drug‐Induced Kidney Injury: Use in Clinical Trials and Recent Examples of Impact on Drug Development
Biomarkers of Drug‐Induced Kidney Injury: Use in Clinical Trials and Recent Examples of Impact on Drug Development Open
This manuscript describes the scope of implementation and impact of a regulatory agency‐qualified panel of six urine biomarkers on drug development, the process for which was conducted and funded by the Critical Path Institute, the Foundat…
View article: Comparative Efficacy and Safety of De-escalation, Abbreviation, and Standard Potent P2Y <sub>12</sub> Inhibitor–Based Dual Antiplatelet Therapy Strategies After Acute Coronary Syndrome: A Network Meta-Analysis
Comparative Efficacy and Safety of De-escalation, Abbreviation, and Standard Potent P2Y <sub>12</sub> Inhibitor–Based Dual Antiplatelet Therapy Strategies After Acute Coronary Syndrome: A Network Meta-Analysis Open
Background The efficacy and safety of de-escalation from potent P2Y 12 inhibitor-based dual antiplatelet therapy (DAPT) to clopidogrel-based DAPT, or abbreviation to potent P2Y 12 inhibitor monotherapy, compared with standard 12-month DAPT…
View article: dandi/dandi-archive: v0.19.0
dandi/dandi-archive: v0.19.0 Open
Release Notes Expose allowed schema versions at /info/ endpoint (#2625) The /info/ endpoint now exposes the allowed list of DANDI schema version through allowed_schema_versions key value. 🐛 Bug Fix Expose allowed schema versions at /info/…
View article: Systematic Review of Patient Focused Drug Development Meeting Reports for Conditions Affecting Neurodevelopment
Systematic Review of Patient Focused Drug Development Meeting Reports for Conditions Affecting Neurodevelopment Open
Background: Researchers of rare diseases affecting neurodevelopment struggle with endpoint identification and selection issues that are uniquely associated with developmental concepts but common across conditions. However, the potential to…
View article: Editorial: focus point on advances in radiation therapy
Editorial: focus point on advances in radiation therapy Open
View article: Overall survival as a safety guardrail in cancer drug trials
Overall survival as a safety guardrail in cancer drug trials Open
Surrogate endpoints have accelerated access to oncology drugs but often leave uncertainty about net patient benefit. We propose a pragmatic framework that recentres overall survival (OS) as both the definitive patient-centred efficacy endp…
View article: Optimizing oncology drug development: systematic review of 22 years of myeloma randomized controlled trials
Optimizing oncology drug development: systematic review of 22 years of myeloma randomized controlled trials Open
Introduction Although myeloma represents a key success story in oncology, some drugs have failed to meet primary endpoints in randomized controlled trials (RCTs), despite promising early-phase activity. This analysis aimed to understand fa…
View article: Why to avoid dual primary endpoints in cardiovascular trials
Why to avoid dual primary endpoints in cardiovascular trials Open
Introduction: In an adequately designed randomised controlled trial for confirming the efficacy of a new drug in patients with chronic heart failure (CHF), mortality (all-cause or cardiovascular) should usually be the primary endpoint – ei…
View article: 50 Assessment of three-step blood preservation kit for pharmacodynamic endpoints by flow cytometry for multi-site, global clinical trials
50 Assessment of three-step blood preservation kit for pharmacodynamic endpoints by flow cytometry for multi-site, global clinical trials Open
View article: Causal Inference for First Non‐Fatal Events With the Competing Risk of Death: A Principal Stratification Approach
Causal Inference for First Non‐Fatal Events With the Competing Risk of Death: A Principal Stratification Approach Open
In clinical trials involving both mortality and morbidity, an active treatment can influence the observed risk of the first nonfatal event either directly, through its effect on the underlying nonfatal event process, or indirectly, through…
View article: Corrigendum to “Psychedelic Assisted Therapy as a Complex Intervention: Implications for clinical trial design”
Corrigendum to “Psychedelic Assisted Therapy as a Complex Intervention: Implications for clinical trial design” Open
[This corrects the article DOI: 10.1177/20451253251381074.].
View article: Surrogate Endpoints in Pivotal Clinical Trials for Drug Approval in Japan Compared to the United States
Surrogate Endpoints in Pivotal Clinical Trials for Drug Approval in Japan Compared to the United States Open
Regulatory guidance documents exist on surrogate endpoints in the United States. In Japan, there are no established rules or guidance regarding the use of surrogate endpoints, and various aspects remain unclear. The aim of this study was t…
View article: The Ask Suicide-Screening Questions “Continuous Score”: An Unvalidated Endpoint
The Ask Suicide-Screening Questions “Continuous Score”: An Unvalidated Endpoint Open
View article: КЛІНІЧНА ЦІННІСТЬ МЕТОДУ ВОЛЮМЕТРІЇ, ПОРІВНЯНО З КРИТЕРІЯМИ RECIST 1.1, ПРИ ОЦІНЦІ ВІДПОВІДІ НА ЛІКУВАННЯ МЕТАСТАЗІВ ПЕЧІНКИ У ПАЦІЄНТІВ, ХВОРИХ НА КОЛОРЕКТАЛЬНИЙ РАК
КЛІНІЧНА ЦІННІСТЬ МЕТОДУ ВОЛЮМЕТРІЇ, ПОРІВНЯНО З КРИТЕРІЯМИ RECIST 1.1, ПРИ ОЦІНЦІ ВІДПОВІДІ НА ЛІКУВАННЯ МЕТАСТАЗІВ ПЕЧІНКИ У ПАЦІЄНТІВ, ХВОРИХ НА КОЛОРЕКТАЛЬНИЙ РАК Open
Мета роботи: порівняти інформативність і діагностичну чутливість за методами RECIST 1.1 та волюметрії при оцінці динаміки метастатичного ураження печінки у пацієнтів із колоректальним раком, визначити кореляцію між лінійними та об’ємними п…
View article: Leveraging recent advances in plasma biomarkers to optimize early proof of concept trials in Alzheimer's disease
Leveraging recent advances in plasma biomarkers to optimize early proof of concept trials in Alzheimer's disease Open
INTRODUCTION The importance of biomarkers as a primary outcome or as supportive evidence of clinical effect is rising as the field shifts toward disease‐modifying treatments and earlier intervention, because they have lower variability and…
View article: Intensive glycemic control and kidney disease risk: insights on hierarchical composite endpoint from a randomized clinical trial
Intensive glycemic control and kidney disease risk: insights on hierarchical composite endpoint from a randomized clinical trial Open
Background Clinical trials of intensive glycemic control in patients with type 2 diabetes mellitus (T2DM) and high cardiovascular risk have reported inconsistent findings regarding chronic kidney disease (CKD) outcomes, partly due to heter…
View article: Clinical endpoints in pragmatic heart failure trials: From data collection to clinical endpoint classification
Clinical endpoints in pragmatic heart failure trials: From data collection to clinical endpoint classification Open
Clinical endpoint classification (CEC)—that is, evaluation of clinical events using pre‐defined criteria—is commonly conducted in clinical trial operations to ensure systematic and consistent assessment of endpoints needed to assess the in…
View article: BRIDGE: a multi-stakeholder workstream focused on assessing new clinical endpoints in ophthalmology clinical trials [Building Research Innovations and Developing Global Endpoints]
BRIDGE: a multi-stakeholder workstream focused on assessing new clinical endpoints in ophthalmology clinical trials [Building Research Innovations and Developing Global Endpoints] Open
View article: Type 2 MI a legitimate efficacy endpoint in cardiovascular trials? A critical appraisal
Type 2 MI a legitimate efficacy endpoint in cardiovascular trials? A critical appraisal Open
View article: Utility of plasma GFAP as a secondary endpoint for clinical trials in Alzheimer’s disease
Utility of plasma GFAP as a secondary endpoint for clinical trials in Alzheimer’s disease Open
Plasma GFAP increased in parallel with cognitive decline, making it a candidate for monitoring disease progression in trials aimed at mitigating cognitive deterioration. Although Aβ positivity significantly accelerated GFAP progression, th…
View article: The impact of liver transplantation on endpoint selection in alcohol-associated hepatitis trials
The impact of liver transplantation on endpoint selection in alcohol-associated hepatitis trials Open
Background: Alcohol-associated hepatitis (AH) is a serious liver disease caused by heavy alcohol consumption with severe cases exhibiting a 90-day mortality rate of ~30%. No drugs have been approved for AH, and regulatory approval currentl…
View article: Composite Confirmed Disability Worsening/Progression Is a Useful Clinical Endpoint for Multiple Sclerosis Clinical Trials
Composite Confirmed Disability Worsening/Progression Is a Useful Clinical Endpoint for Multiple Sclerosis Clinical Trials Open
ClinicalTrials.gov Identifiers: NCT01247324 (OPERA I); first submitted November 23, 2010; first patient enrolled: August 31, 2011; available at clinicaltrials.gov/study/NCT01247324. NCT01412333 (OPERA II); first submitted August 8, 2011; f…
View article: Time in range—A new gold standard in type 2 diabetes research?
Time in range—A new gold standard in type 2 diabetes research? Open
Glycated haemoglobin (HbA1c) is currently the gold standard outcome measure for type 2 diabetes trials. Time in range is a continuous glucose monitoring (CGM) metric defined as the proportion of time in euglycemia (3.9–10.0 mmol/L) and may…
View article: Validating candidate endpoints for intermediate age-related macular degeneration trials in a multi-centre setting—lessons from the MACUSTAR study
Validating candidate endpoints for intermediate age-related macular degeneration trials in a multi-centre setting—lessons from the MACUSTAR study Open
View article: Evaluating Minimal Residual Disease Negativity as a Surrogate Endpoint for Treatment Efficacy in Multiple Myeloma: A Meta‐Analysis of Randomized Controlled Trials
Evaluating Minimal Residual Disease Negativity as a Surrogate Endpoint for Treatment Efficacy in Multiple Myeloma: A Meta‐Analysis of Randomized Controlled Trials Open
This meta‐analysis examined the association between minimal residual disease (MRD) negativity and survival outcomes in 15 304 patients with multiple myeloma (MM) enrolled in randomized controlled trials published until June 2, 2024. Overal…
View article: Expanding the use and interpretation of patient-centric cardiovascular clinical trial endpoints
Expanding the use and interpretation of patient-centric cardiovascular clinical trial endpoints Open
Significant improvements have been achieved to enhance the patient-centricity of clinical research, including the development and utilization of novel clinical trial endpoints. These include endpoints that harness outcomes that are importa…
View article: Minimal Residual Disease as a Primary Endpoint in Multiple Myeloma: Implications for Clinical Trials and Everyday Clinical Practice
Minimal Residual Disease as a Primary Endpoint in Multiple Myeloma: Implications for Clinical Trials and Everyday Clinical Practice Open