Objective: Platform studies are an innovative clinical trial design to evaluate multiple potential therapies simultaneously using a master protocol framework for more rapid discovery of critical new disease-modifying therapies. Initially pioneered in cancer clinical trials, platform studies have more recently been adopted for new vaccine trials and other disease states. This approach has not been used for biomarker(s) validation. Approach: We present the development and successful implementation of a master protocol for a platform study in the National Institute of Diabetes and Digestive and Kidney Diseases Diabetic Foot Consortium (DFC) that concomitantly tests multiple biomarkers that could be used to predict diabetic foot ulcer (DFU) healing outcomes in people with DFUs. Results: This flexible master protocol, allowing the study of multiple biomarkers in a perpetual manner, designed to be inclusive of a broad spectrum of people with DFUs operating under the mantra “No DFU Goes Unstudied” successfully collects participant data and biospecimens for biomarker development and validation in a standardized manner across multiple DFC sites. Innovation: Integrating multiple biomarkers with a rich database of participants’ clinical characteristics, patient-reported outcomes, and standard of care practices at various sites collected longitudinally over 52 weeks following a pragmatic approach and thus relevant to people with DFU as seen in today’s daily practice, enable their subsequent implementation at the point of care to guide timely personalized DFU management decisions. Conclusion: Leveraging this robust platform will help to identify new therapeutic targets and design and conduct effective interventional clinical trials for DFU wound healing, which could inform the standards of care.
Schmidt et al. (Thu,) studied this question.