Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials (NCT07674758)
Evaluating Cardiac Function in Patients With Duchenne Muscular Dystrophy
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: UC Davis — Sacramento, California
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (9 Sites)
- UC Davis — Sacramento, California
- Children's National — Washington D.C., District of Columbia
- Lurie Children's — Chicago, Illinois
- Riley Children's Hospital — Indianapolis, Indiana
- Duke Children's Hospital — Durham, North Carolina
- Nationwide Children's — Columbus, Ohio
- Vanderbilt University Medical Center — Nashville, Tennessee
- Children's Hospital of Richmond at VCU — Richmond, Virginia
- Seattle Children's — Seattle, Washington
📞 Contact the Study Coordinator
Phone: (615) 322-7447
Email: DMDMachineLearning@vumc.org
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Duchenne Muscular Dystrophy (DMD)
- Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping — La Jolla, California
- Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy — Nashville, Tennessee
- The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy — Rochester, New York
- Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) — Hillsborough, North Carolina
- Duchenne Electronic Health Record Study — Little Rock, Arkansas
Explore by condition: All Duchenne Muscular Dystrophy (DMD) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.