Gene therapy to improve heart function in patients with Duchenne muscular dystrophy
Official title: A Phase 1b, Open-Label, Controlled Trial Evaluating the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Run by Sardocor Corp. · NCT06224660 · Data updated 2026-11-12
This study is testing a new gene therapy to see if it can help improve heart function in patients with Duchenne muscular dystrophy who have heart problems.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | Men only |
| Enrollment | 12 (estimated) |
| Sponsor | Sardocor Corp. Industry-sponsored |
| Locations | 3 sites (Kansas City, Kansas and 2 other locations) |
| Status | Recruiting |
| Started | October 2, 2024 |
| Main results expected | October 2027 |
| Study ends | October 2030 |
| Registry record updated | February 27, 2025 |
| Trial ID | NCT06224660 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals diagnosed with Duchenne muscular dystrophy who have cardiomyopathy and a left ventricular ejection fraction of less than 40%.
✕Probably not a fit
Patients with non-DMD-related heart issues or those with significant comorbidities affecting their heart or lung function may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial is testing an experimental gene therapy drug called SRD-001, aimed at enhancing heart function in patients with Duchenne muscular dystrophy (DMD) who have developed cardiomyopathy. The therapy involves administering SRD-001 via a one-time infusion to increase the production of the SERCA2a protein in heart muscle cells, potentially improving their ability to contract and pump blood. Participants will be closely monitored for safety and efficacy over a two-year period, with assessments including cardiac imaging and evaluations of muscle and lung function. The study will compare outcomes between those receiving the treatment and those who do not receive any intervention.
Why it matters
Potential benefit: If successful, this therapy could significantly improve heart function and quality of life for patients with DMD-related cardiomyopathy.
How similar studies have gone: While gene therapy approaches for cardiac conditions are emerging, this specific application for DMD-related cardiomyopathy is relatively novel and has not been extensively tested in prior studies.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of DMD with confirmatory genetic testing * Cardiomyopathy with left ventricular scar in at least 3 of 16 segments * Left ventricular ejection fraction \< 40% * Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment * Willing and able to provide informed consent Exclusion Criteria: * Abnormal blood pressure * Non-DMD-related liver function test elevations * Cystatin C ≥ 1.2 mg/L * Thrombocytopenia * Anemia * Inadequate pulmonary function
Where it's running
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The University of Kansas Medical Center
Kansas City, Kansas, United States Recruiting -
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States Recruiting -
Nationwide Children's Hospital
Columbus, Ohio, United States Recruiting
Study contacts
- Study coordinator: Sardocor Corp.
- Email: info@sardocorcorp.com
- Phone: +1-617-880-7616
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Targeting SERCA2a to improve heart function in patients with heart failure
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.