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Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy
Author(s) -
Lake Stephen L.,
Quintana Melanie A.,
Broglio Kristine,
Panagoulias Jennifer,
Berry Scott M.,
Panzara Michael A.
Publication year - 2021
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.9021
Subject(s) - clinical trial , duchenne muscular dystrophy , clinical study design , muscular dystrophy , medicine , placebo , bayesian probability , research design , adaptive design , clinical endpoint , ambulatory , physical therapy , physical medicine and rehabilitation , computer science , alternative medicine , artificial intelligence , statistics , pathology , mathematics
A Bayesian adaptive design is proposed for a clinical trial in Duchenne muscular dystrophy. The trial was designed to demonstrate treatment efficacy on an ambulatory‐based clinical endpoint and to identify early success on a biomarker (dystrophin protein levels) that can serve as a basis for accelerated approval in the United States. The trial incorporates placebo augmentation using placebo data from past clinical trials. A thorough simulation study was conducted to understand the operating characteristics of the trial. This trial design was selected for the US FDA Complex Innovative Trial Design Pilot Meeting Program and the experience in that program is summarized.