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

Recruiting now

Conditions studied: Duchenne Muscular Dystrophy (DMD), Cardiomyopathy, Becker Muscular Dystrophy, Carrier of Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT07674758

Run by

Vanderbilt University Medical Center

People needed

1000

Starts

2025-01-06

Expected to finish

2029-02-01

Last updated by the study team

2026-07-28

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• 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)

Where it is running

• UC Davis — Sacramento, California, United States (enrolling)
• Children's National — Washington D.C., District of Columbia, United States (enrolling)
• Lurie Children's — Chicago, Illinois, United States (enrolling)
• Riley Children's Hospital — Indianapolis, Indiana, United States (enrolling)
• Duke Children's Hospital — Durham, North Carolina, United States (enrolling)
• Nationwide Children's — Columbus, Ohio, United States (enrolling)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (enrolling)
• Children's Hospital of Richmond at VCU — Richmond, Virginia, United States (enrolling)
• Seattle Children's — Seattle, Washington, United States (enrolling)

Full record on ClinicalTrials.gov

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