Tests a gene therapy for weakened heart muscle in Duchenne muscular dystrophy
Tests gene therapy SRD-001 in adults with heart weakness from Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Key facts
- Where
- Cincinnati, Columbus, and Kansas City, United StatesSee the 3 sites
- Phase
- Phase 1
Where in United States
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- The University of Kansas Medical CenterKansas City, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Sardocor Corp.
- Trial code
- NCT06224660
- Record updated
- 27 February 2025
- Original description
This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.Read full original descriptionShow less
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 research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.
Inclusion: 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: Abnormal blood pressure; Non-DMD-related liver function test elevations; Cystatin C ≥ 1.2 mg/L; Thrombocytopenia; Anemia; Inadequate pulmonary function.