Gene therapy to improve heart function in patients with Duchenne muscular dystrophy
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
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 |
| Enrollment | 12 (estimated) |
| Ages | 18 Years and up |
| Sex | Male |
| Sponsor | Sardocor Corp. Industry-sponsored |
| Locations | 3 sites (Kansas City, Kansas and 2 other locations) |
| Trial ID | NCT06224660 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are individuals diagnosed with Duchenne muscular dystrophy who have cardiomyopathy and a left ventricular ejection fraction of less than 40%.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Kansas City, Kansas and 2 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.