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

Recruitingn/aAges any - anyAllVanderbilt University Medical Center
NCT07674758 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
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)
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What it tests

  • Not listed
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Where

9 sites
UC Davis
Sacramento, United States
Children's National
Washington D.C., United States
Lurie Children's
Chicago, United States
Riley Children's Hospital
Indianapolis, United States
Duke Children's Hospital
Durham, United States
Nationwide Children's
Columbus, United States
Vanderbilt University Medical Center
Nashville, United States
Children's Hospital of Richmond at VCU
Richmond, United States

…and 1 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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