Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials
Part of paid clinical trials in Sacramento, California.
- Sponsor
- Vanderbilt University Medical Center
- Study ID
- NCT07674758
- Status
- Recruiting
Conditions
- Becker Muscular Dystrophy
- Cardiomyopathy
- Carrier of Duchenne Muscular Dystrophy
- Duchenne Muscular Dystrophy (DMD)
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Study Details
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 Dates
- Start date
- Jan 6, 2025
- Status verified
- Jun 2026
- Primary completion
- Feb 1, 2029
- Completion
- Feb 1, 2029
Study Design
- Enrollment
- 1,000 participants (estimated)
Arms
- Arm: Duchenne Muscular Dystrophy, Becker muscular dystrophy, and carriers of muscular dystrophyEvaluation of surrogate outcome measures of disease in patients with dystrophinopathy
Primary Outcome Measure
Mortality [ Time Frame: baseline to 10 years ]
Central Contacts
- Jonathan Soslow, MD, MSCI615-322-7447
Locations (9)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| UC Davis | Sacramento | California | 95616 | |
| Children's National | Washington D.C. | District of Columbia | 20010 | |
| Lurie Children's | Chicago | Illinois | 60611 | Study Coordinator 312-227-4100 |
| Riley Children's Hospital | Indianapolis | Indiana | 46202 | |
| Duke Children's Hospital | Durham | North Carolina | 27705 | |
| Nationwide Children's | Columbus | Ohio | 43205 | |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | |
| Children's Hospital of Richmond at VCU | Richmond | Virginia | 23220 | |
| Seattle Children's | Seattle | Washington | 98105 |
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