Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Plain-language summary

This study examines SRD-001 gene therapy in patients with cardiomyopathy caused by Duchenne muscular dystrophy (DMD). The treatment targets SERCA2a, which regulates calcium handling in heart cells. Participants have genetically confirmed DMD, a left ventricular ejection fraction below 40% and left ventricular scarring. It is a Phase 1 study and is currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Recruiting

Sponsor

Sardocor Corp.

Intervention

SRD-001

Start date

10/2/2024

Estimated completion

10/1/2030

Countries

United States

Related genes

Locations

• Kansas City, United States
• Cincinnati, United States
• Columbus, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* Abnormal blood pressure
* Non-DMD-related liver function test elevations
* Cystatin C ≥ 1.2 mg/L
* Thrombocytopenia
* Anemia
* Inadequate pulmonary function