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.

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.

Contemporary clinical trials communications. 2024 Oct 18*** epublish ***

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

MRC Clinical Trials Unit at UCL, Institute of Clinical Trials & Methodology, 2nd Floor 90 High Holborn, London, WC1V 6LJ, UK., Royal Marsden Hospital, Royal Marsden Hospital, 203 Fulham Rd, Chelsea, London, SW3 6JJ, UK., St Bartholomew's Hospital, W Smithfield, London, EC1A 7B, UK., University of Cambridge, Department of Surgery, University of Cambridge, Cambridge Biomedical Campus, Cambridge, CB2 0QQ, UK., Institut Gustave Roussy, 114 Rue Edouard Vaillant 94805, Villejuif, France., Royal Free London NHS Foundation Trust UCL Division of Surgery and Interventional Science, Pond Street, London, NW3 2QG, UK., Dana-Farber Cancer Institute, 450 Brookline Ave, Boston, MA, 02215, United States., Monash University Eastern Health Clinical School, Level 2, 5 Arnold Street, Box Hill, Victoria, 3128, Australia., Department of Oncology, Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital, Cambridge Biomedical Campus, Hill's Road Cambridge, CB2 0QQ, UK., Faculty of Health and Medicine, The University of Newcastle, Australia., University of St Andrews, North Haugh, St Andrews, KY16 9TF, UK., Mount Vernon Cancer Centre, Rickmansworth Rd, Northwood, HA6 2RN, UK., NHMRC Clinical Trials Centre, University of Sydney, Camperdown, NSW, 2006, Australia., Vall d'Hebron Institute of Oncology, Vall d'Hebron University Hospital, 08035, Barcelona, Spain., School of Cancer Sciences, Beatson Institute, University of Glasgow, G61 1BD, UK.