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)	73
Anti-Thrombotic Therapy to Ameliorate Complications of COVID-19 (ATTACC): Study design and methodology for an international, adaptive Bayesian randomized controlled trial	52
Endpoints for randomized controlled clinical trials for COVID-19 treatments	47
Non-adjustment for multiple testing in multi-arm trials of distinct treatments: Rationale and justification	45
Cluster over individual randomization: are study design choices appropriately justified? Review of a random sample of trials	24
Use of electronic recruitment methods in a clinical trial of adults with gout	22
COVID-19 vaccine trials: The use of active controls and non-inferiority studies	21
The electronic health record as a clinical trials tool: Opportunities and challenges	21
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
The Bayesian Time Machine: Accounting for temporal drift in multi-arm platform trials	18
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
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
Online monitoring of patient self-reported adverse events in early phase clinical trials: Views from patients, clinicians, and trial staff	16
Commentary on Parker and Weir	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)	13
The U.S. Food and Drug Administration’s Complex Innovative Trial Design Pilot Meeting Program: Progress to date	13
Mindfulness, Education, and Exercise for age-related cognitive decline: Study protocol, pilot study results, and description of the baseline sample	13
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
Phase I trial compensation: How much do healthy volunteers actually earn from clinical trial enrollment?	12
Blinding, sham, and treatment effects in randomized controlled trials for back pain in 2000–2019: A review and meta-analytic approach	12

Practical challenges in the conduct of pragmatic trials embedded in health plans: Lessons of IMPACT-AFib, an FDA-Catalyst trial	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
An ethics framework for consolidating and prioritizing COVID-19 clinical trials	11
Randomized comparison of two interventions to enhance understanding during the informed consent process for research	11
Retrospective collection of 90-day modified Rankin Scale is accurate	11
When is it impractical to ask informed consent? A systematic review	10
Effectiveness of participant recruitment strategies for critical care trials: A systematic review and narrative synthesis	10
Recruitment and retention in randomized controlled trials with urban American Indian/Alaska Native adolescents: Challenges and lessons learned	9
Clinical trials site recruitment optimisation: Guidance from Clinical Trials: Impact and Quality	9
Strategies for facilitating the delivery of cluster randomized trials in hospitals: A study informed by the CFIR-ERIC matching tool	9
Incorporating estimands into clinical trial statistical analysis plans	9
Now is the time to fix the evidence generation system	9
Strategies for research participant engagement: A synthetic review and conceptual framework	8
Now is the time to fix the clinical research workforce crisis	8
Towards more credible shams for physical interventions: A Delphi survey	8
COVID-19 impact on multi-site recruitment and enrollment	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
Exploration of baseline patient-reported side effect bother from cancer therapy	8
Five analytic challenges in working with electronic health records data to support clinical trials with some solutions	8
A systematic review of subgroup analyses in randomised clinical trials in cardiovascular disease	8
A platform trial design for preventive vaccines against Marburg virus and other emerging infectious disease threats	7
Lack of harmonization of coronavirus disease ordinal scales	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
Exploring factors influencing recruitment results of nurses recruiting diabetes patients for a randomized controlled trial	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
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
Patient accrual and understanding of informed consent in a two-stage consent design	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 ring vaccination trial design for the estimation of vaccine efficacy and effectiveness during infectious disease outbreaks	6
Bayesian methods for pilot studies	6
The representation of Indigenous peoples in chronic disease clinical trials in Australia, Canada, New Zealand, and the United States	6
Recruiting breast cancer patients for mHealth research: Obstacles to clinic-based recruitment for a mobile phone app intervention study	6
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	6
The PROTEUS-Trials Consortium: Optimizing the use of patient-reported outcomes in clinical trials	6
Dynamic methods for ongoing assessment of site-level risk in risk-based monitoring of clinical trials: A scoping review	6
The triple aim of clinical research	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
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
Improving efficiency in the stepped-wedge trial design via Bayesian modeling with an informative prior for the time effects	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
Making a distinction between data cleaning and central monitoring in clinical trials	5
The maraca plot: A novel visualization of hierarchical composite endpoints	5
Timing is everything: The importance of patient-reported outcome assessment frequency when characterizing symptomatic adverse events	5
A Bayesian response-adaptive dose-finding and comparative effectiveness trial	5
Impact of complex, partially nested clustering in a three-arm individually randomized group treatment trial: A case study with the wHOPE trial	5

The cluster randomized crossover trial: The effects of attrition in the AB/BA design and how to account for it in sample size calculations	5
Clinical trials in a COVID-19 pandemic: Shared infrastructure for continuous learning in a rapidly changing landscape	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
Facilitating clinical research through automation: Combining optical character recognition with natural language processing	5
Cost-benefit of outcome adjudication in nine randomised stroke trials	5
Reporting of clinical trial safety results in ClinicalTrials.gov for FDA-approved drugs: A cross-sectional analysis	5
Marginal modeling in community randomized trials with rare events: Utilization of the negative binomial regression model	5
Rethinking intercurrent events in defining estimands for tuberculosis trials	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
Results publications are inadequately linked to trial registrations: An automated pipeline and evaluation of German university medical centers	5
Enrollment and transition challenges in the International Maternal Pediatric and Adolescent AIDS Clinical Trials (IMPAACT) network’s PROMISE trial for resource-limited regions	5
Developing an online, searchable database to systematically map and organise current literature on retention research (ORRCA2)	5
Current recommendations/practices for anonymising data from clinical trials in order to make it available for sharing: A scoping review	5
Double-blinding of an acupuncture randomized controlled trial optimized with clinical translational science award resources	5
Reopening schools safely in the face of COVID-19: Can cluster randomized trials help?	5
Barriers and enablers to cancer clinical trial participation and initiatives to improve opportunities for rural cancer patients: A scoping review	5
Time trends with response-adaptive randomization: The inevitability of inefficiency	4
Data management in substance use disorder treatment research: Implications from data harmonization of National Institute on Drug Abuse-funded randomized controlled trials	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
Barriers to uptake of the hip fracture core outcome set: An international survey of 80 hip fracture trialists	4
Backfilling cohorts in phase I dose-escalation studies	4
Bayesian basket trial design with false-discovery rate control	4
Clinical trials in the time of a pandemic	4
Improving clinical trial transparency at UK universities: Evaluating 3 years of policies and reporting performance on the European Clinical Trial Register	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
Ethics challenges in sharing data from pragmatic clinical trials	4
Covariate adjustment in subgroup analyses of randomized clinical trials: A propensity score approach	4
Interim data monitoring in cluster randomised trials: Practical issues and a case study	4
Clinical Trials to authors: Please pre-register your studies!	4
Comparison of survival distributions in clinical trials: A practical guidance	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
Optimal one-stage design and analysis for efficacy expansion in Phase I oncology trials	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
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
A modular framework for early-phase seamless oncology trials	4
Navigating the ethics of remote research data collection	4
Data monitoring committee interim reports: We must get there soon!	4
Using simulated infectious disease outbreaks to inform site selection and sample size for individually randomized vaccine trials during an ongoing epidemic	3
When and how to include vulnerable subjects in clinical trials	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
Addressing the quality of submissions to ClinicalTrials.gov for registration and results posting: The use of a checklist	3
Challenges and opportunities for conducting a vaccine trial during the COVID-19 pandemic in the United Kingdom	3
Adverse event load, onset, and maximum grade: A novel method of reporting adverse events in cancer clinical trials	3
From screening to ascertainment of the primary outcome using electronic health records: Challenges in the STRIDE trial	3
Measures of fidelity of delivery and engagement in self-management interventions: A systematic review of measures	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
Demographic diversity of US-based participants in GSK-sponsored interventional clinical trials	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
On the design of early-phase Alzheimer’s disease clinical trials with cerebrospinal fluid tau outcomes	3
Commentary on Bertagnolli et al: Clinical trial designs with routinely collected real-world data—issues of data quality and beyond	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
Power and sample size calculations for cluster randomized trials with binary outcomes when intracluster correlation coefficients vary by treatment arm	3
Commentary: Two approaches to analyze platform trials incorporating non-concurrent controls with a common assumption	3
Treatment selection in multi-arm multi-stage designs: With application to a postpartum haemorrhage trial	3
Incentives and payments in pragmatic clinical trials: Scientific, ethical, and policy considerations	3
Protocol adherence rates in superiority and noninferiority randomized clinical trials published in high impact medical journals	3
A permutation procedure to detect heterogeneous treatment effects in randomized clinical trials while controlling the type I error rate	2
Network methods and design of randomized trials: Application to investigation of COVID-19 vaccination boosters	2
COVID-19 vaccine trials: The potential for “hybrid” analyses	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
Adjudication of cardiovascular events in patients with chronic obstructive pulmonary disease: SUMMIT trial	2
Effects of patient-reported outcome assessment order	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
Design of a novel clinical trial of prehospital pediatric airway management	2
Accounting for the rarity of the disease when designing clinical trials with a focus on pediatric cancers	2
Clinician engagement in the ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness) trial	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

Harmonisation of large-scale, heterogeneous individual participant adverse event data from randomised trials of statin therapy	2
Landscape of coronavirus disease 2019 clinical trials: New frontiers and challenges	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
Gene-targeted therapy for neurofibromatosis and schwannomatosis: The path to clinical trials	2
Alternative models and randomization techniques for Bayesian response-adaptive randomization with binary outcomes	2
Mobilizing the clinical trial ecosystem to drive adoption of master protocols	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
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
Registration of clinical trials in Russia: How does it compare with the World Health Organization International Clinical Trials Registry Platform standards?	1
Contrasting approaches for addressing non-adherence in randomized controlled trials: An illustration from the REFLUX trial	1
Design of placebo-controlled randomized trials of anticancer agents: Ethical considerations based on a review of published trials	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
A Short History of Bernard Fisher’s Contributions to Randomized Clinical Trials	1
Optimal allocation to treatment sequences in individually randomized stepped-wedge designs with attrition	1
Determining the sample size for a cluster-randomised trial using knowledge elicitation: Bayesian hierarchical modelling of the intracluster correlation coefficient	1
Commentary on Chirkova et al.: Some comments on “socialist pharmapolitics”	1
Covariate-constrained randomization with cluster selection and substitution	1
Recommendations for the collection and annotation of biosamples for analysis of biomarkers in neurofibromatosis and schwannomatosis clinical trials	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
Ethically designing research to inform multidimensional, rapidly evolving policy decisions: Lessons learned from the PROMISE HIV Perinatal Prevention Trial	1
A concern about survival time as an endpoint in coronavirus disease 2019 clinical trials	1
Bringing data monitoring committee charters into the sunlight	1
Proceedings of the University of Pennsylvania 13th annual conference on statistical issues in clinical trials: Cluster randomized clinical trials—Challenges and opportunities	1
Thirteenth annual UPenn conference on statistical issues in clinical trials: Cluster randomized clinical trials—opportunities and challenges (morning panel session)	1
Adapting isotonic dose-finding to a dynamic set of drug combinations with application to a phase I leukemia trial	1
Inference on subgroups identified based on a heterogeneous treatment effect in a post hoc analysis of a clinical trial	1
Using predictive analytics to improve pragmatic trial design	1
Algorithmic benchmark modulation: A novel method to develop success rates for clinical studies	1
Feasibility of recruiting in prisons during a randomized controlled trial with people with serious mental illness	1
Estimating counterfactual placebo HIV incidence in HIV prevention trials without placebo arms based on markers of HIV exposure	1
Random effect misspecification in stepped wedge designs	1
Subgroup analyses and pre-specification	1
Designing and implementing methodology for double-blind, placebo-controlled clinical trials using blood products within the Department of Veterans Affairs	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
Improving data monitoring in Australian clinical trials and research: Free resources and templates	1
Determining a risk-proportionate approach to the validation of statistical programming for clinical trials	1
Importance of patient engagement in the conduct of pragmatic multicenter randomized controlled trials: The ADAPTABLE experience	1
Practical considerations in utilizing cluster randomized controlled trials conducted in biopharmaceutical industry	1
Constructing and evaluating a validity argument for a performance outcome measure for clinical trials: An example using the Multi-luminance Mobility Test	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
Barriers and facilitators to the inclusion of deaf people in clinical trials	1
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials	1
Dynamic use of historical controls in clinical trials for rare disease research: A re-evaluation of the MILES trial	1
Formulary restrictions may impact enrollment in pragmatic trials and limit generalizability of findings to vulnerable populations	1
Handling intercurrent events and missing data in non-inferiority trials using the estimand framework: A tuberculosis case study	1
Drug labeling changes and pediatric hematology/oncology prescribing: Measuring the impact of U.S. legislation	1
Adapting international clinical trials during COVID-19 and beyond	1
What do we do with our under-enrolled single-center COVID-19 clinical trials?	1
Payments for research participation: Don’t tax the Guinea pig	1
Commentary on Kahrass et al: The sublime inertia of informed consent language in early phase clinical trials involving patients	1
Partner engagement for planning and development of non-pharmacological care pathways in the AIM-Back trial	1
Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1	1
Practical issues in pragmatic trials: the implementation of the Diuretic Comparison Project	1
The design and conduct of a pragmatic cluster randomized trial of an advance care planning program for nursing home residents with dementia	1
Methodological aspects of a randomized within-patient concurrent controlled design for clinical trials in spine surgery	1
Perspectives of adults with neurofibromatosis regarding the design of psychosocial trials: Results from an anonymous online survey	1
Thirteenth annual UPenn conference on statistical issues in clinical trials: Cluster-randomized clinical trials—opportunities and challenges (afternoon panel session)	1
Designing a childhood obesity preventive intervention using the multiphase optimization strategy: The Healthy Bodies Project	1
Can quality management drive evidence generation?	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 case study of ascertainment bias for the primary outcome in the Strategies to Reduce Injuries and Develop Confidence in Elders (STRIDE) trial	1
A dynamic and collaborative approach to trial recruitment in safetxt, a UK sexual health randomised controlled trial	1
Testing Meldonium: Assessing Soviet pragmatic alternatives to the randomized controlled trial	1
Finding the (biomarker-defined) subgroup of patients who benefit from a novel therapy: No time for a game of hide and seek	1
Experience of conducting clinical trials of investigational medicinal products during a respiratory virus pandemic: Lessons learnt from COVID-19	1
Use of comprehensive recruitment strategies in the glycemia reduction approaches in diabetes: A comparative effectiveness study (GRADE) multi-center clinical trial	1
Assessing the external validity of the VALIDATE-SWEDEHEART trial	1
Highly efficient clinical trial designs for reliable screening of under-performing treatments: Application to the COVID-19 Pandemic	1
Strategies to facilitate adolescent access to medicines: Improving regulatory guidance	1
Risk-proportionate approach to paediatric clinical trials: The legal requirements, challenges, and the way forward under the European Union Clinical Trials Regulation	1