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Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials

Evaluating Cardiac Function in Patients With Duchenne Muscular Dystrophy

Who can join

All ages · All sexes

Inclusion Criteria:

* Clinical phenotype of Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), or muscular dystrophy carrier (MDC) confirmed with muscle biopsy or genotype

Exclusion Criteria:

* Additional genetic or congenital abnormality that may affect cardiovascular function or progression
* Current investigational therapy that may affect cardiovascular function (would preclude ongoing data collection but prior data would still be used)

About the study

Dystrophin associated heart dysfunction is a leading cause of death in patients with Duchenne and Becker Muscular dystrophy (DMD/BMD) and Duchenne and Becker muscular dystrophy carriers (MDC); however, the evolution of heart dysfunction is not well-understood. The central objectives of this proposal are to elucidate this evolution of heart dysfunction and identify measures from cardiac MRI images that can predict death or significant heart disease in patients with DMD/BMD/MDC. This study will create a large clinical and cardiac MRI registry of dystrophin associated heart dysfunction, will utilize advanced image analysis techniques, including deep learning neural networks, to comprehensively evaluate every patient, and will create a risk toolkit accessible to clinicians around the world; this proposal has the potential to improve the quality of life in patients with dystrophin associated heart dysfunction by allowing for earlier and more intensive therapy in patients with severe disease and by identifying surrogate outcome measures for use in therapeutic trials.

Sponsor: Vanderbilt University Medical Center · Participants: 1,000 · Started: Jan 6, 2025

Contact the study team

• Jonathan Soslow, MD, MSCI · Phone: 615-322-7447

Official record on ClinicalTrials.gov — NCT07674758

Locations in the U.S.

Conditions

• Duchenne Muscular Dystrophy
• Cardiomyopathy

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.