Modeling heart failure in Duchenne and Becker muscular dystrophy to find surrogate heart measures

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study will build a large registry of patients with DMD, BMD, and MDC, focusing on dystrophin-related cardiomyopathy. It will use cardiac MRI data and advanced image analysis, including deep learning, to identify surrogate outcome measures that predict mortality or significant cardiac events. A risk toolkit will be developed to help clinicians tailor monitoring and therapy. Enrollment aims for about 950 patients with more than 4,000 MRI studies across multiple high-volume centers. The goal is to enable earlier intervention and improve quality of life through better understanding of disease progression.

Why it matters

Who can join: the full rules

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)

Where it's running

• UC Davis
  Sacramento, California, United States Recruiting
• Children's National
  Washington D.C., District of Columbia, United States Recruiting
• Lurie Children's
  Chicago, Illinois, United States Recruiting
• Riley Children's Hospital
  Indianapolis, Indiana, United States Recruiting
• Duke Children's Hospital
  Durham, North Carolina, United States Recruiting
• Nationwide Children's
  Columbus, Ohio, United States Recruiting

Study contacts

• Study coordinator: Jonathan Soslow, MD, MSCI
• Email: DMDMachineLearning@vumc.org
• Phone: 615-322-7447

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Respiratory progression in adults with Duchenne muscular dystrophy: a long-term observational study

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.