Adapting the design of the ongoing RAMPART trial in response to external evidence: An example for trials which take many years to run and report
Year: 2024
Type: article
Abstract: Clinical trials to establish the efficacy of new agents in the adjuvant cancer setting typically take many years to complete. During that time, external factors can impact recruitment and reporting plans. An example is a new standard of care becoming available during the recruitment period. In this paper we describe how we modified the design of the RAMPART trial (NCT03288532) which was set up to investigate immune checkpoint inhibitor therapy in the adjuvant renal cancer setting. The trial had been initiated when no globally accepted adjuvant strategy after nephrectomy existed. A subsequent change in the standard of care for many patients with early renal cancer meant it was no longer feasible to continue to recruit. We needed to find a way to maximise the contribution that RAMPART participants could make to the evidence base for immune checkpoint inhibitor therapy without introducing bias or detriment to the integrity of the trial results. We describe how we agreed and incorporated all design and timeline changes while remaining blinded to accumulating data within the trial, thus protecting the reliability of the future results. We share details of our design modifications to guide others who may have similar experiences, particularly as more agents and combinations of agents are developed and investigated in similar adjuvant settings.
Authors: Angela Meade, Elena Frangou, Babak Choodari-Oskooei, James Larkin, Tom Powles, Grant D. Stewart, Laurence Albiges, Axel Bex, Toni K. Choueiri, Ian D. Davis, Tim Eisen, Alison Fielding, Craig Gedye, David J. Harrison, Rick Kaplan, Salena Mulhere, Paul Nathan, Grisma Patel, Jay Patel, Hannah Plant, Alastair Ritchie, Hannah Rush, Clare Shakeshaft, Martin R. Stockler, Cristina Suarez, Jemima Thompson, Nat Thorogood, Balaji Venugopal, Mahesh K.B. Parmar
Institutions: MRC Clinical Trials Unit at UCL, University College London, Royal Marsden Hospital, St Bartholomew's Hospital, University of Cambridge, Institut Gustave Roussy, Royal Free London NHS Foundation Trust, The Netherlands Cancer Institute, Dana-Farber Cancer Institute, Eastern Health, Monash University, Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital, University of Newcastle Australia, University of St Andrews, Mount Vernon Cancer Centre, The University of Sydney, Australian and New Zealand Urogenital and Prostate Cancer Trials Group, National Health and Medical Research Council, Vall d'Hebron Hospital Universitari, Vall d'Hebron Institute of Oncology, Cancer Research UK Scotland Institute, University of Glasgow
Language: English
Subfield: Statistics and Probability
Field: Mathematics
Domain: Physical Sciences
Open Access status: gold
Estimated APC paid: $2,350
OpenAlex ID: W4403521667