Development of a Cardiac Imaging Supplement for the Duchenne Muscular Dystrophy Therapeutic Area User Guide v1.0


Clinical Trials

Poster Number: M176


Ramona Belfiore-Oshan, PhD, Critical Path Institute, Diane Corey, Critical Path Institute, Rebecca Baker, MS, CDISC, Jon Neville, PSM, CDISC, Jonhatan Soslow, MD, Vanderbilt University Medical Center, Kan Hor, MD, Nationwide Children Hospital, Ohio State University, Chet Villa, MD, Cincinnati Children’s Hospital Medical Center, Karin LaPann, BA, CDISC, Alana St. Clair, MBA, CDISC

DMD-associated cardiomyopathy (DMD-C) is universal, progressive and the primary cause of premature death. Despite its prevalence, cardiac disease in DMD is insufficiently understood, and there is urgent need for improved, cardiac targeted therapeutics. While the number of cardiac DMD studies and the cardiac data ecosystem are growing, standardized protocols for collection, curation, and harmonization of data elements for cardiac imaging data are lacking. The Duchenne Regulatory Science Consortium (D-RSC) at the Critical Path Institute (C-Path) is a non-profit public-private partnership that supports collaborative research and regulatory endorsement of innovative drug development tools for DMD. D-RSC’s dedicated Cardiac Working Group, which includes cardiologists, drug developers, academic and clinical researchers, data managers, patient advocates, and advocacy groups, has established a collaboration with the Clinical Data Interchange Standards Consortium (CDISC) in support of the urgent need to better understand cardiac disease progression in DMD and other dystrophinopathies. Specific goals of the collaboration include the development of new submission and analysis standards examples, to accommodate cardiac imaging concepts and use cases, aligning with the latest Standard Data Tabulation Model (SDTM) Analysis Data Model (ADaM) and Controlled Terminology.
Results will be published as a supplement to the Duchenne Muscular Dystrophy Therapeutic Area User Guide (TAUG-DMD v1.0) and made freely accessible to the clinical and translational research community. Completion of this project will enable more efficient harmonization, integration, and aggregation of cardiac DMD data, and will provide researchers and drug developers with an essential tool to generate quantitative data-driven solutions to optimize clinical trials for cardiac disease in DMD.