A Randomized, Embedded, Multifactorial Adaptive Platform trial for ILD (REMAP-ILD): The conceptual design

Introduction: Fibrotic ILDs (fILDs) encompass a heterogenous group of parenchymal lung disorders with poor outcomes and high unmet need. Conventional clinical trials are limited by lack of generalizability, large sample size requirements and single agent assessment. We present the concept of an international platform trial aiming to increase efficiency in fILD clinical trial delivery.

Methods: REMAP-ILD is a protocol for the global delivery of a Bayesian adaptive platform trial for fILD. Response Adaptive Randomisation following 3 monthly assessments of FVC will be applied. The trial will be embedded into clinical practice to minimize barriers to patient recruitment, promote retention and reduce costs. The trial will be multifactorial, testing multiple interventions within multiple therapeutic domains and patient strata simultaneously. The trial will adapt as data accrue to facilitate rapid implementation of results and generate a global infrastructure to ensure perpetual assessment of novel therapies as new treatments are discovered and standards of care change. Protocol development is ongoing and focused on immunomodulatory, antifibrotic, senolytic, anti-tussive, rehabilitation and vascular therapies. As the trial evolves, further domains may be added.

The primary objective is to determine the efficacy of different interventions alone and/or in combination for patients with fILDs on the slope of FVC. Pre-specified secondary endpoints will assess efficacy in domains which are not anticipated to alter FVC slope.

Conclusions: REMAP-ILD is an innovative trial anticipated to accelerate therapeutic advances and optimize patient care for fILDs.