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

Status: Recruiting
Location: See all (3) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
United States
Kansas
The University of Kansas Medical Center
RECRUITING
Kansas City
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Nationwide Children's Hospital
RECRUITING
Columbus
Contact Information
Primary
Sardocor Corp.
info@sardocorcorp.com
+1-617-880-7616
Time Frame
Start Date: 2024-10-02
Estimated Completion Date: 2030-10
Participants
Target number of participants: 12
Treatments
Experimental: Low Dose
SRD-001
Experimental: High Dose
SRD-001
No_intervention: Control
No-Intervention Control
Sponsors
Leads: Sardocor Corp.

This content was sourced from clinicaltrials.gov