The Role of Adaptive and Innovative Trial Designs in Diabetes Research: A Scoping Review.

Abstract

Adaptive and master protocol clinical trials offer significant advantages for diabetes research, including enhanced efficiency and personalized treatment strategies. This scoping review aimed to systematically map the use of adaptive and master protocol designs in interventional trials for type 1 and type 2 diabetes, identify research gaps, and highlight opportunities for broader implementation. A systematic literature search was performed using MEDLINE, Embase, CENTRAL, Emcare, Global Health, Web of Science, and clinical trial registries. Gray literature searches complemented database findings. Studies using adaptive, platform, basket, or umbrella trial designs in people with type 1 or type 2 diabetes were included. Data were charted using a standardized form. Extracted variables included diabetes type, trial design, adaptive features, interventions, end points, and key findings. Of 396 articles screened, 6 published adaptive trials met the inclusion criteria: 3 in type 1 diabetes, 1 in type 2 diabetes, and 2 in diabetes-related neuropathy. Most used adaptive features for dose finding, response-adaptive randomization, and sample size reestimation. No published platform, basket, or umbrella trials were identified. Six ongoing adaptive trials in type 1 diabetes were identified through registry searches, four under an adaptive platform master protocol. Despite a comprehensive search, some gray literature and unpublished studies may have been missed. Risk of bias was not assessed, consistent with scoping review methodology. Adaptive and master protocol trials remain rare in diabetes. Overcoming barriers through targeted training and awareness, robust regulatory frameworks, and strategic incentives could support broader adoption.