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

Launched by SARDOCOR CORP. · Jan 24, 2024

Keywords

ClinConnect Summary

This clinical trial is studying an experimental drug called SRD-001, which is a type of gene therapy aimed at helping the hearts of boys with Duchenne muscular dystrophy (DMD). DMD can lead to a serious heart condition called cardiomyopathy, where the heart struggles to pump blood effectively. The researchers hope that by providing extra copies of a specific gene (SERCA2a), the heart muscle cells can work better and improve heart function. Participants in the trial will be divided into two groups: one group will receive the SRD-001 treatment, while the other group will not receive the experimental drug but will continue their current heart medications.

To be eligible for this trial, participants must be male, aged 6 to 74, and have a confirmed diagnosis of DMD along with specific heart problems, such as a weakened heart function. They should also have been on heart medications for at least a year. Over the course of the study, participants will be closely monitored for two years, undergoing heart imaging and assessments of their arm and lung function. After this period, all participants will continue to be followed up long-term. This trial is currently recruiting participants, and it's important for anyone considering joining to discuss it thoroughly with their healthcare provider.

Gender

MALE

Eligibility criteria

• 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