Open-Label Study Administering One Time Gene Therapy to Patients With Weakened Hearts With Duchenne Muscular Dystrophy
Phase: Recruiting
First Posted: March
Condition(s): Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Other Study ID Number(s): SRD-001-1004
What Is the Purpose of This Study?
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.
Who Can Take Part in This Study?
- 18 years and older
- 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
For a full list of eligibility requirements, visit https://clinicaltrials.gov/study/NCT06224660
What Will Happen During This Study?
- After screening to determine eligibility, participants will be sequentially assigned to low dose SRD-001, high dose SRD-001 or no-intervention.
- Participants assigned to active treatment with SRD-001 will undergo cardiac catheterization and angiography just prior to the intracoronary infusion of SRD-001 and spend overnight in the hospital for observation.
Principal Investigator
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Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare condition defined by worsening muscle weakness and damage. It is the most common inherited neuromuscular disorder that affects all races and ethnicities. DMD only affects males and children with DMD may lose the ability to walk as early as 7 years of age.
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