Actively Recruiting
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Led by Sardocor Corp. · Updated on 2025-02-27
12
Participants Needed
3
Research Sites
312 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Duchenne muscular dystrophy confirmed by genetic testing
- Cardiomyopathy with left ventricular scar in at least 3 of 16 segments
- Left ventricular ejection fraction less than 40%
- On individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months before enrollment
- Willing and able to provide informed consent
You will not qualify if you...
- Abnormal blood pressure
- Liver function test elevations not related to DMD
- Cystatin C level 1.2 mg/L or higher
- Thrombocytopenia
- Anemia
- Inadequate lung function
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
The University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
2
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
3
Nationwide Children's Hospital
Columbus, Ohio, United States, 43215
Actively Recruiting
Research Team
S
Sardocor Corp.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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