SRD-001 Gene Therapy for Cardiomyopathy in Duchenne Muscular Dystrophy (MUSIC-DMD)
Actively RecruitingPhase 1Improving Heart Function
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
Contact Information
This section provides contact details for people who can answer questions about joining this study
- Name: Sardocor Corp.
- Phone Number: +1-617-880-7616
- Email: [email protected]
Clinical Trial Registry
NCT ID
NCT06224660This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.