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Heart MRI to study heart problems in Duchenne and Becker muscular dystrophy

Uses heart MRI to track heart function in people with dystrophin-related muscle disease.

Plain-language title generated from the registry record
Registry title: Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials

Key facts

Where
Chicago, Columbus, Durham, Indianapolis, Nashville, Richmond, Sacramento, Seattle, and Washington D.C., United StatesSee the 9 sites

Where in United States

  • Lurie Children'sChicago, United StatesTaking people
  • Nationwide Children'sColumbus, United StatesTaking people
  • Duke Children's HospitalDurham, United StatesTaking people
  • Riley Children's HospitalIndianapolis, United StatesTaking people
  • Vanderbilt University Medical CenterNashville, United StatesTaking people
  • Children's Hospital of Richmond at VCURichmond, United StatesTaking people
  • UC DavisSacramento, United StatesTaking people
  • Seattle Children'sSeattle, United StatesTaking people
  • Children's NationalWashington D.C., United StatesTaking people

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Trial details

Who runs it
Vanderbilt University Medical Center
Trial code
NCT07674758
Record updated
28 July 2026
Original description
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.Read full original descriptionShow less

Evaluating Cardiac Function in Patients With Duchenne Muscular Dystrophy

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.

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

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

ClinicalTrials.gov NCT07674758