Clinical Trials

Article	Citations
Composite grading algorithm for the National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)	74
Anti-Thrombotic Therapy to Ameliorate Complications of COVID-19 (ATTACC): Study design and methodology for an international, adaptive Bayesian randomized controlled trial	52
Non-adjustment for multiple testing in multi-arm trials of distinct treatments: Rationale and justification	49
Endpoints for randomized controlled clinical trials for COVID-19 treatments	48
Cluster over individual randomization: are study design choices appropriately justified? Review of a random sample of trials	24
The electronic health record as a clinical trials tool: Opportunities and challenges	22
COVID-19 vaccine trials: The use of active controls and non-inferiority studies	22
Use of electronic recruitment methods in a clinical trial of adults with gout	22
The Bayesian Time Machine: Accounting for temporal drift in multi-arm platform trials	20
Effectiveness of social media (Facebook), targeted mailing, and in-person solicitation for the recruitment of young adult in a diabetes self-management clinical trial	20
User-centered design principles in the development of clinical research tools	19
Summarising salient information on historical controls: A structured assessment of validity and comparability across studies	18
Intra-cluster correlations from the CLustered OUtcome Dataset bank to inform the design of longitudinal cluster trials	17
Online monitoring of patient self-reported adverse events in early phase clinical trials: Views from patients, clinicians, and trial staff	16
Meta-analysis of rare adverse events in randomized clinical trials: Bayesian and frequentist methods	16
Characteristics of available studies and dissemination of research using major clinical data sharing platforms	16
Commentary on Parker and Weir	15
Mindfulness, Education, and Exercise for age-related cognitive decline: Study protocol, pilot study results, and description of the baseline sample	14
Design and implementation of an international, multi-arm, multi-stage platform master protocol for trials of novel SARS-CoV-2 antiviral agents: Therapeutics for Inpatients with COVID-19 (TICO/ACTIV-3)	14
The U.S. Food and Drug Administration’s Complex Innovative Trial Design Pilot Meeting Program: Progress to date	13
Retrospective collection of 90-day modified Rankin Scale is accurate	12
An ethics framework for consolidating and prioritizing COVID-19 clinical trials	12
Phase I trial compensation: How much do healthy volunteers actually earn from clinical trial enrollment?	12
US Food and Drug Administration utilization of postmarketing requirements and postmarketing commitments, 2009–2018	12
The DURATIONS randomised trial design: Estimation targets, analysis methods and operating characteristics	12

Methodologies for pragmatic and efficient assessment of benefits and harms: Application to the SOCRATES trial	12
Blinding, sham, and treatment effects in randomized controlled trials for back pain in 2000–2019: A review and meta-analytic approach	12
Randomized comparison of two interventions to enhance understanding during the informed consent process for research	11
Clarifying selection bias in cluster randomized trials	11
Trends and variation in data quality and availability on the European Union Clinical Trials Register: A cross-sectional study	11
Practical challenges in the conduct of pragmatic trials embedded in health plans: Lessons of IMPACT-AFib, an FDA-Catalyst trial	11
Incorporating estimands into clinical trial statistical analysis plans	10
When is it impractical to ask informed consent? A systematic review	10
Clinical trials site recruitment optimisation: Guidance from Clinical Trials: Impact and Quality	10
Effectiveness of participant recruitment strategies for critical care trials: A systematic review and narrative synthesis	10
Strategies for research participant engagement: A synthetic review and conceptual framework	9
Now is the time to fix the evidence generation system	9
Recruitment and retention in randomized controlled trials with urban American Indian/Alaska Native adolescents: Challenges and lessons learned	9
A systematic review of subgroup analyses in randomised clinical trials in cardiovascular disease	9
Strategies for facilitating the delivery of cluster randomized trials in hospitals: A study informed by the CFIR-ERIC matching tool	9
Five analytic challenges in working with electronic health records data to support clinical trials with some solutions	8
COVID-19 impact on multi-site recruitment and enrollment	8
Now is the time to fix the clinical research workforce crisis	8
Research Attitudes Questionnaire scores predict Alzheimer’s disease clinical trial dropout	8
Clinical trial monitoring effectiveness: Remote risk-based monitoring versus on-site monitoring with 100% source data verification	8
Rethinking intercurrent events in defining estimands for tuberculosis trials	7
Lack of harmonization of coronavirus disease ordinal scales	7
Patient accrual and understanding of informed consent in a two-stage consent design	7
Randomized controlled trial of an education-based intervention to improve medication adherence: Design considerations in the medication adherence in glaucoma to improve care study	7
A platform trial design for preventive vaccines against Marburg virus and other emerging infectious disease threats	7
Exploring factors influencing recruitment results of nurses recruiting diabetes patients for a randomized controlled trial	7
Are restricted mean survival time methods especially useful for noninferiority trials?	7
Details of risk–benefit communication in informed consent documents for phase I/II trials	7
Influential methods reports for group-randomized trials and related designs	7
Using modified intention-to-treat as a principal stratum estimator for failure to initiate treatment	7
Recruiting an underserved, difficult to reach population into a cancer trial: Strategies from the Restore-2 Rehabilitation Trial for gay and bisexual prostate cancer patients	7
Experiences of running a stratified medicine adaptive platform trial: Challenges and lessons learned from 10 years of the FOCUS4 trial in metastatic colorectal cancer	7
The representation of Indigenous peoples in chronic disease clinical trials in Australia, Canada, New Zealand, and the United States	7
The PROTEUS-Trials Consortium: Optimizing the use of patient-reported outcomes in clinical trials	6
Timing is everything: The importance of patient-reported outcome assessment frequency when characterizing symptomatic adverse events	6
The triple aim of clinical research	6
Recruiting breast cancer patients for mHealth research: Obstacles to clinic-based recruitment for a mobile phone app intervention study	6
Quantification of hematoma and perihematomal edema volumes in intracerebral hemorrhage study: Design considerations in an artificial intelligence validation (QUANTUM) study	6
Why restricted mean survival time methods are especially useful for non-inferiority trials	6
Dynamic methods for ongoing assessment of site-level risk in risk-based monitoring of clinical trials: A scoping review	6
Adjusting for adherence in randomized trials when adherence is measured as a continuous variable: An application to the Lipid Research Clinics Coronary Primary Prevention Trial	6
Behavioral and social science research to support development of educational materials for clinical trials of broadly neutralizing antibodies for HIV treatment and prevention	6
The ring vaccination trial design for the estimation of vaccine efficacy and effectiveness during infectious disease outbreaks	6
Bayesian methods for pilot studies	6
Improving efficiency in the stepped-wedge trial design via Bayesian modeling with an informative prior for the time effects	6
Clinical trials in a COVID-19 pandemic: Shared infrastructure for continuous learning in a rapidly changing landscape	6
Facilitating clinical research through automation: Combining optical character recognition with natural language processing	5
The maraca plot: A novel visualization of hierarchical composite endpoints	5
Developing an online, searchable database to systematically map and organise current literature on retention research (ORRCA2)	5
Reopening schools safely in the face of COVID-19: Can cluster randomized trials help?	5

Marginal modeling in community randomized trials with rare events: Utilization of the negative binomial regression model	5
Methodological challenges in pragmatic trials in Alzheimer’s disease and related dementias: Opportunities for improvement	5
Exploring mechanisms of action in clinical trials of complex surgical interventions using mediation analysis	5
Data monitoring committee interim reports: We must get there soon!	5
Cost-benefit of outcome adjudication in nine randomised stroke trials	5
Impact of complex, partially nested clustering in a three-arm individually randomized group treatment trial: A case study with the wHOPE trial	5
Double-blinding of an acupuncture randomized controlled trial optimized with clinical translational science award resources	5
A Bayesian response-adaptive dose-finding and comparative effectiveness trial	5
Barriers and enablers to cancer clinical trial participation and initiatives to improve opportunities for rural cancer patients: A scoping review	5
Making a distinction between data cleaning and central monitoring in clinical trials	5
Results publications are inadequately linked to trial registrations: An automated pipeline and evaluation of German university medical centers	5
Reporting of clinical trial safety results in ClinicalTrials.gov for FDA-approved drugs: A cross-sectional analysis	5
Time trends with response-adaptive randomization: The inevitability of inefficiency	5
Current recommendations/practices for anonymising data from clinical trials in order to make it available for sharing: A scoping review	5
The APPROACH trial: Assessing pain, patient-reported outcomes, and complementary and integrative health	5
Joint testing of overall and simple effects for the two-by-two factorial trial design	5
Clinical trials in the time of a pandemic	4
Data management in substance use disorder treatment research: Implications from data harmonization of National Institute on Drug Abuse-funded randomized controlled trials	4
Improving clinical trial transparency at UK universities: Evaluating 3 years of policies and reporting performance on the European Clinical Trial Register	4
Ethics challenges in sharing data from pragmatic clinical trials	4
Covariate adjustment in subgroup analyses of randomized clinical trials: A propensity score approach	4
Barriers to uptake of the hip fracture core outcome set: An international survey of 80 hip fracture trialists	4
Interim data monitoring in cluster randomised trials: Practical issues and a case study	4
Backfilling cohorts in phase I dose-escalation studies	4
Bayesian basket trial design with false-discovery rate control	4
Commentary: Two approaches to analyze platform trials incorporating non-concurrent controls with a common assumption	4
Proceedings of the University of Pennsylvania 12th annual conference on statistical issues in clinical trials: Electronic health records (EHR) in randomized clinical trials—Challenges and opportunitie	4
Choosing and changing the analysis scale in non-inferiority trials with a binary outcome	4
Use of information criteria for selecting a correlation structure for longitudinal cluster randomised trials	4
From screening to ascertainment of the primary outcome using electronic health records: Challenges in the STRIDE trial	4
Measures of fidelity of delivery and engagement in self-management interventions: A systematic review of measures	4
Demographic diversity of US-based participants in GSK-sponsored interventional clinical trials	4
Clinical Trials to authors: Please pre-register your studies!	4
Comparison of survival distributions in clinical trials: A practical guidance	4
Optimal one-stage design and analysis for efficacy expansion in Phase I oncology trials	4
Comparison of weekly and daily recall of pain as an endpoint in a randomized phase 3 trial of cabozantinib for metastatic castration-resistant prostate cancer	4
Analysis of adaptive platform trials using a network approach	4
Intent-to-treat analysis of cluster randomized trials when clusters report unidentifiable outcome proportions	4
Design and analysis of group-sequential clinical trials based on a modestly weighted log-rank test in anticipation of a delayed separation of survival curves: A practical guidance	4
When and how to include vulnerable subjects in clinical trials	4
A modular framework for early-phase seamless oncology trials	4
Navigating the ethics of remote research data collection	4
On the design of early-phase Alzheimer’s disease clinical trials with cerebrospinal fluid tau outcomes	3
Combining factorial and multi-arm multi-stage platform designs to evaluate multiple interventions efficiently	3
Partially clustered designs for clinical trials: Unifying existing designs using consistent terminology	3
Clinical trial site identification practices and the use of electronic health records in feasibility evaluations: An interview study in the Nordic countries	3
Recruitment, retention, and adherence in a clinical trial: The Pediatric Heart Network’s Marfan Trial experience	3
Are self-reported and self-monitored adherence good proxies for reaching relevant plasma concentrations?: Experiences from a study of anti-depressants in healthy volunteers	3
Adverse event load, onset, and maximum grade: A novel method of reporting adverse events in cancer clinical trials	3
Commentary on Bertagnolli et al: Clinical trial designs with routinely collected real-world data—issues of data quality and beyond	3
Methodological standards for using the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE) in cancer clinical trials	3
Power and sample size calculations for cluster randomized trials with binary outcomes when intracluster correlation coefficients vary by treatment arm	3
Addressing the quality of submissions to ClinicalTrials.gov for registration and results posting: The use of a checklist	3
Treatment selection in multi-arm multi-stage designs: With application to a postpartum haemorrhage trial	3
Protocol adherence rates in superiority and noninferiority randomized clinical trials published in high impact medical journals	3
Using simulated infectious disease outbreaks to inform site selection and sample size for individually randomized vaccine trials during an ongoing epidemic	3
A comparison of phase I dose-finding designs in clinical trials with monotonicity assumption violation	3
Microbiota or placebo after antimicrobial therapy for recurrent Clostridioides difficile at home: A clinical trial with novel home-based enrollment	3
Harmonisation of large-scale, heterogeneous individual participant adverse event data from randomised trials of statin therapy	3
Challenges and opportunities for conducting a vaccine trial during the COVID-19 pandemic in the United Kingdom	3
Incentives and payments in pragmatic clinical trials: Scientific, ethical, and policy considerations	3
Drug labeling changes and pediatric hematology/oncology prescribing: Measuring the impact of U.S. legislation	2
Design and analysis of a 2-year parallel follow-up of repeated ivermectin mass drug administrations for control of malaria: Small sample considerations for cluster-randomized trials with count data	2
Gene-targeted therapy for neurofibromatosis and schwannomatosis: The path to clinical trials	2
Accounting for the rarity of the disease when designing clinical trials with a focus on pediatric cancers	2
Mobilizing the clinical trial ecosystem to drive adoption of master protocols	2
Optimal design of cluster randomised trials with continuous recruitment and prospective baseline period	2
Stratified randomization for platform trials with differing experimental arm eligibility	2
Importance of incorporating quantitative imaging biomarker technical performance characteristics when estimating treatment effects	2
Effects of patient-reported outcome assessment order	2
Bringing data monitoring committee charters into the sunlight	2
A hybrid approach to comparing parallel-group and stepped-wedge cluster-randomized trials with a continuous primary outcome when there is uncertainty in the intra-cluster correlation	2
Empirical power comparison of statistical tests in contemporary phase III randomized controlled trials with time-to-event outcomes in oncology	2
COVID-19 vaccine trials: The potential for “hybrid” analyses	2
Alternative models and randomization techniques for Bayesian response-adaptive randomization with binary outcomes	2
Clinician engagement in the ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness) trial	2
Minimization of resource utilization data collected within cost-effectiveness analyses conducted alongside Canadian Cancer Trials Group phase III trials	2
Establishing communication challenges and preferences among clinical trial participants in an under-resourced setting to improve adherence to study visits and participant retention	2
Handling intercurrent events and missing data in non-inferiority trials using the estimand framework: A tuberculosis case study	2
Adjudication of cardiovascular events in patients with chronic obstructive pulmonary disease: SUMMIT trial	2

Landscape of coronavirus disease 2019 clinical trials: New frontiers and challenges	2
A permutation procedure to detect heterogeneous treatment effects in randomized clinical trials while controlling the type I error rate	2
Design of a novel clinical trial of prehospital pediatric airway management	2
Network methods and design of randomized trials: Application to investigation of COVID-19 vaccination boosters	2
Commentary on Bertagnolli et al.: Leveraging electronic health record data for clinical trials—a brave new world	2
Lack of transparent reporting of trial monitoring approaches in randomised controlled trials: A systematic review of contemporary protocol papers	2
Clinical studies sponsored by digital health companies participating in the FDA’s Precertification Pilot Program: A cross-sectional analysis	2
Cost-effective clinical trial design: Application of a Bayesian sequential model to the ProFHER pragmatic trial	2
Assessing the potential for prevention or earlier detection of on-site monitoring findings from randomised controlled trials: Further analyses of findings from the prospective TEMPER triggered monitor	2
Event-specific win ratios and testing with terminal and non-terminal events	2
Partner engagement for planning and development of non-pharmacological care pathways in the AIM-Back trial	1
Design of placebo-controlled randomized trials of anticancer agents: Ethical considerations based on a review of published trials	1
Designing and implementing methodology for double-blind, placebo-controlled clinical trials using blood products within the Department of Veterans Affairs	1
Assessing the use of observational methods and real-world data to emulate ongoing randomized controlled trials	1
Improving data monitoring in Australian clinical trials and research: Free resources and templates	1
Overall average treatment effects from clinical trials, one-variable-at-a-time subgroup analyses and predictive approaches to heterogeneous treatment effects: Toward a more patient-centered evidence-b	1
Importance of patient engagement in the conduct of pragmatic multicenter randomized controlled trials: The ADAPTABLE experience	1
Perspectives of adults with neurofibromatosis regarding the design of psychosocial trials: Results from an anonymous online survey	1
Designing a childhood obesity preventive intervention using the multiphase optimization strategy: The Healthy Bodies Project	1
On what basis did Health Canada approve OxyContin in 1996? A retrospective analysis of regulatory data	1
Practical steps to identifying the research risk of pragmatic trials	1
A hybrid automated event adjudication system for clinical trials	1
Commentary on Chirkova et al.: Some comments on “socialist pharmapolitics”	1
A case study of ascertainment bias for the primary outcome in the Strategies to Reduce Injuries and Develop Confidence in Elders (STRIDE) trial	1
Adapting isotonic dose-finding to a dynamic set of drug combinations with application to a phase I leukemia trial	1
Recommendations for the collection and annotation of biosamples for analysis of biomarkers in neurofibromatosis and schwannomatosis clinical trials	1
A dynamic and collaborative approach to trial recruitment in safetxt, a UK sexual health randomised controlled trial	1
Feasibility of recruiting in prisons during a randomized controlled trial with people with serious mental illness	1
Thirteenth annual UPenn conference on statistical issues in clinical trials: Cluster randomized clinical trials—opportunities and challenges (morning panel session)	1
Experience of conducting clinical trials of investigational medicinal products during a respiratory virus pandemic: Lessons learnt from COVID-19	1
Strategies to facilitate adolescent access to medicines: Improving regulatory guidance	1
Subgroup analyses and pre-specification	1
Risk-proportionate approach to paediatric clinical trials: The legal requirements, challenges, and the way forward under the European Union Clinical Trials Regulation	1
Estimating counterfactual placebo HIV incidence in HIV prevention trials without placebo arms based on markers of HIV exposure	1
Minimisation for the design of parallel cluster-randomised trials: An evaluation of balance in cluster-level covariates and numbers of clusters allocated to each arm	1
Practical issues in pragmatic trials: the implementation of the Diuretic Comparison Project	1
An adaptive clinical trial design to identify the target dose of tenecteplase for treatment of acute pulmonary embolism	1
Methodological aspects of a randomized within-patient concurrent controlled design for clinical trials in spine surgery	1
A Short History of Bernard Fisher’s Contributions to Randomized Clinical Trials	1
Determining the sample size for a cluster-randomised trial using knowledge elicitation: Bayesian hierarchical modelling of the intracluster correlation coefficient	1
Thirteenth annual UPenn conference on statistical issues in clinical trials: Cluster-randomized clinical trials—opportunities and challenges (afternoon panel session)	1
Covariate-constrained randomization with cluster selection and substitution	1
A comparison of different population-level summary measures for randomised trials with time-to-event outcomes, with a focus on non-inferiority trials	1
Cognitive screening considerations for psychosocial clinical trials in HIV, aging, and cognition	1
Ethically designing research to inform multidimensional, rapidly evolving policy decisions: Lessons learned from the PROMISE HIV Perinatal Prevention Trial	1
Assessing the external validity of the VALIDATE-SWEDEHEART trial	1
Constructing and evaluating a validity argument for a performance outcome measure for clinical trials: An example using the Multi-luminance Mobility Test	1
A concern about survival time as an endpoint in coronavirus disease 2019 clinical trials	1
Payments for research participation: Don’t tax the Guinea pig	1
Dynamic use of historical controls in clinical trials for rare disease research: A re-evaluation of the MILES trial	1
Proceedings of the University of Pennsylvania 13th annual conference on statistical issues in clinical trials: Cluster randomized clinical trials—Challenges and opportunities	1
Commentary on Kahrass et al: The sublime inertia of informed consent language in early phase clinical trials involving patients	1
Use of comprehensive recruitment strategies in the glycemia reduction approaches in diabetes: A comparative effectiveness study (GRADE) multi-center clinical trial	1
Inference on subgroups identified based on a heterogeneous treatment effect in a post hoc analysis of a clinical trial	1
Dynamic data-enabled stratified sampling for trial invitations with application in NHS-Galleri	1
Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1	1
Random effect misspecification in stepped wedge designs	1
Contrasting approaches for addressing non-adherence in randomized controlled trials: An illustration from the REFLUX trial	1
The design and conduct of a pragmatic cluster randomized trial of an advance care planning program for nursing home residents with dementia	1
Large-scale prevention trials could provide stronger evidence for decision-makers: Opportunities to design and report with a focus on the benefit–harm balance	1
Is it reasonable to label a trial as pragmatic whose investigators do not? The UK RECOVERY COVID-19 trial case	1
Optimal allocation to treatment sequences in individually randomized stepped-wedge designs with attrition	1
Determining a risk-proportionate approach to the validation of statistical programming for clinical trials	1
Practical considerations in utilizing cluster randomized controlled trials conducted in biopharmaceutical industry	1
An introduction to spillover effects in cluster randomized trials with noncompliance	1
Strengthening the interpretability of clinical trial results by assessing the effect of informative censoring on the primary estimand in PRECISION	1
Finding the (biomarker-defined) subgroup of patients who benefit from a novel therapy: No time for a game of hide and seek	1
Barriers and facilitators to the inclusion of deaf people in clinical trials	1
Algorithmic benchmark modulation: A novel method to develop success rates for clinical studies	1
Can quality management drive evidence generation?	1
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials	1
Highly efficient clinical trial designs for reliable screening of under-performing treatments: Application to the COVID-19 Pandemic	1
Testing Meldonium: Assessing Soviet pragmatic alternatives to the randomized controlled trial	1
Formulary restrictions may impact enrollment in pragmatic trials and limit generalizability of findings to vulnerable populations	1
Adapting international clinical trials during COVID-19 and beyond	1
Registration of clinical trials in Russia: How does it compare with the World Health Organization International Clinical Trials Registry Platform standards?	1
What do we do with our under-enrolled single-center COVID-19 clinical trials?	1