Development and Evaluation of a Time to Event Endpoint for Clinical Trials in Duchenne Muscular Dystrophy (DMD)
Development and Evaluation of a Time to Event Endpoint for Clinical Trials in Duchenne Muscular Dystrophy (DMD)
Event-driven clinical trials, commonly used in oncology, offer several benefits over continuous outcome measures: i) each patient is assessed individually, ii) after the event has occurred, patients can receive therapy without breaking the blind. This study aimed to develop a time-to-event outcome measure for the North Star Ambulatory Assessment (NSAA) corresponding to clinically meaningful disease progression in DMD.
This poster was presented at WMS 2022 and MDA 2023.
Authors: C. McDonald, F. Muntoni, J. Marden, N. Goemans, A. Gomez-Lievano, A. Zhang, S. Ward, and J. Signorovitch.