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

Last updated: February 26, 2025
Sponsor: Sardocor Corp.
Overall Status: Active - Recruiting

Phase

1

Condition

Circulation Disorders

Treatment

SRD-001

Clinical Study ID

NCT06224660
SRD-001-1004
  • Ages > 18
  • Male

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 Criteria

Inclusion

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 forat least 12 months prior to enrollment

  • Willing and able to provide informed consent

Exclusion

Exclusion Criteria:

  • Abnormal blood pressure

  • Non-DMD-related liver function test elevations

  • Cystatin C ≥ 1.2 mg/L

  • Thrombocytopenia

  • Anemia

  • Inadequate pulmonary function

Study Design

Total Participants: 12
Treatment Group(s): 1
Primary Treatment: SRD-001
Phase: 1
Study Start date:
October 02, 2024
Estimated Completion Date:
October 31, 2030

Study Description

This phase 1b, multi-center, non-randomized, open-label, ascending dose escalation, no-intervention-control trial will assess the safety and explore the efficacy of SRD-001 administered as a one-time antegrade epicardial coronary artery infusion for the treatment of participants with cardiomyopathy secondary to DMD. SRD-001 is an AAV1 vector expressing the transgene for SERCA2a. Twelve participants will be assigned to either active treatment with SRD-001 or no-intervention based upon their neutralizing antibody status. The objectives of the trial are (1) to evaluate the safety of a one-time intracoronary administration of SRD-001 in participants with cardiomyopathy due to DMD; and (2) to explore the impact of SRD-001 on heart and skeletal muscle function and quality of life. After screening to determine eligibility, participants will be sequentially assigned to low dose SRD-001, high dose SRD-001 or no-intervention. Participants assigned to active treatment with SRD-001 will under cardiac catheterization and angiography just prior to the intracoronary infusion of SRD-001 and spend overnight int he hospital for observation.

Connect with a study center

  • The University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio 43215
    United States

    Active - Recruiting

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