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

Condition(s) studied

Investigational drug(s) / intervention(s)

SRD-001: SRD-001 is an adeno-associated virus serotype 1 (AAV1) based gene therapy designed to deliver a copy of the gene encoding the human sarcoplasmic/endoplasmic reticulum Ca(2+) ATPase 2a (SERCA2a). It is administered as a one-time intracoronary infusion.

Study 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

Primary outcome measure(s)

• Rate of all-cause mortality — From Day 1 to Week 52 and Week 104
  Death
• Rate and severity of related treatment-emergent adverse events — From Day 1 to Week 52 and Week 104
  Adverse events related to the investigational product or the administration procedure
• Rate and severity of all treatment-emergent adverse events — From Day 1 to Week 52 and Week 104
  Adverse events
• Rate of cell-mediated immune reaction — From Day 1 to Week 52
  Cell-mediated immune reaction as assessed by enzyme-linked immunosorbent spot (ELISpot)

Trial sites (3)

More Sardocor Corp. trials in the USA