Clinical Research Alert: Phase 1b Study of SRD-001 in Adults with DMD-Related Cardiomyopathy

Researchers at Sardocor Corp are seeking adults with cardiomyopathy (heart disease) secondary to Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial to evaluate the safety and explore the efficacy of the investigational gene therapy SRD-001 (AAV1/SERCA2a). In heart failure, the body does not make enough of the SERCA2a protein, which helps heart muscle cells squeeze and contract by regulating calcium in the heart. SRD-001 is a low-dose, direct administration gene therapy designed to restore normal levels of SERCA2a in the heart, keep calcium levels balanced, and improve heart function.

The study

This is a multicenter, open-label, non-randomized study. Two active treatment cohorts will have three participants each and an observational control group will have six participants. Participants can choose whether they are a part of the treatment group or observational control group. The participants receiving SRD-001 treatment will be required to attend 10 study visits over two years, while the observational control group will have five study visits over two years. For long-term follow-up, participants will have biannual appointments for the next three years, which are completed remotely.

The gene therapy is administered by a one-time infusion into the coronary arteries of the heart. The effects of SRD-001 will be evaluated using a number of tests and procedures including but not limited to: physical examination and vital sign measurement, adverse event reporting, blood draw and analysis of cardiac biomarkers, urinalysis, electrocardiogram (ECG), cardiac MRI, performance of upper limb scale 2.0 (PUL 2.0), pulmonary function tests, and DMD-Quality of Life assessments.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:

• Male at least 18 years of age at time of consent
• Willing and able to provide informed consent to participate in the trial
• Diagnosis of DMD based on clinical and phenotypic manifestations
• Diagnosis of cardiomyopathy with LGE on at least 3 segments of the cardiac MRI
• Left ventricular ejection fraction less than 50% at time of screening
• Receiving standard-of-care cardiac therapy at an experienced, multidisciplinary center
• On a stable dose of systemic glucocorticoid for at least 12 months prior to screening

Individuals are not eligible to participate if they meet any of the following exclusion criteria:

• Have a forced vital capacity <25% of predicted within the past 3 months
• Are unable to undergo cardiac MRI without sedation

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

Travel support is available for eligible participants.

To learn more about the study or inquire about participation, contact the relevant study sites at:

• University of Kansas Medical Center – Yolanda Murr at ymurr@kumc.edu or (913)588-8232
• Cincinnati Children’s Hospital Medical Center – Heart Institute Neuromuscular Intake Line at (513)803-3000
• Nationwide Children’s Hospital – Kelsey Craig at kelsey.craig@nationwidchildrens.org of (614)722-2715
• For general information – patientadvocacy@sardocorcorp.com