DMD – BIOMARKERS & OUTCOME MEASURES: P.126 Development of a regulatory-ready clinical trial simulation tool for Duchenne muscular dystrophy

Abstract

Early clinical trials of therapies to treat Duchenne muscular dystrophy were designed based on limited information and understanding of clinical endpoints. This led to suboptimal clinical trial protocols that resulted in uncertainty about the effectiveness of therapies. Over time, increasing data have been collected. The Duchenne Regulatory Science Consortium (D-RSC) has developed the largest available database of Duchenne clinical data, consisting of nearly 5,000 patients in total, with1,137 patients and 23,305 observations used to generate an analysis dataset.