SRD-001 Gene Therapy for Cardiomyopathy in Duchenne Muscular Dystrophy (MUSIC-DMD)
Study Overview
- Age
- 18 years and older
- Phase
- Phase 1
- Sponsor
- Sardocor
- Therapeutic Approach
- Improving Heart Function
- Variant Requirement
- Diagnosis of DMD with confirmatory genetic testing.
- Eligible Sex
- Male
- Ambulation
- Non-Ambulatory
- Study Start (Actual)
- 2024-10-02
- Primary Completion (Estimated)
- 2027-10
- Study Completion (Estimated)
- 2030-10
- Enrollment (Estimated)
- 12
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment.
Inclusion Criteria
- Cardiomyopathy with left ventricular scar in at least 3 of 16 segments
- Left ventricular ejection fraction < 40%
- 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
Contacts and Locations
Contacts and Locations
Contact
- Name: Sardocor Corp.
- Phone Number: +1-617-880-7616
- Email: [email protected]
Locations
This study has 3 locations
United States
Kansas Locations
Kansas City, Kansas, United States, 66160
Actively Recruiting
The University of Kansas Medical Center
Contact: Jillian Bruenn
Contact Mail: [email protected]
Contact Phone: 913-588-9720
Ohio Locations
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Cincinnati Children's Hospital Medical Center
Contact: Heart Institute Neuromuscular Intake Line
Contact Phone: 513-803-3000
Columbus, Ohio, United States, 43215
Actively Recruiting
Nationwide Children's Hospital
Contact: Kelsey Craig
Contact Mail: [email protected]
Contact Phone: 614-722-2715
Clinical Trial Registry
NCT ID
NCT06224660This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.