Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (MUSIC-DMD)

February 26, 2025 updated by: Sardocor Corp.

A Phase 1b, Open-Label, Controlled Trial Evaluating the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • The University of Kansas Medical Center
        • Principal Investigator:
          • Pradeep Mammen, MD
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Principal Investigator:
          • Chet Villa, MD
        • Contact:
          • Heart Institute Neuromuscular Intake Line
          • Phone Number: 513-803-3000
      • Columbus, Ohio, United States, 43215
        • Recruiting
        • Nationwide Children's Hospital
        • Contact:
        • Principal Investigator:
          • Deipanjan Nandi, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low Dose
SRD-001
Genetic: 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.
Other Names:
  • AAV1/SSERCA2a
Experimental: High Dose
SRD-001
Genetic: 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.
Other Names:
  • AAV1/SSERCA2a
No Intervention: Control
No-Intervention Control

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change, including normal/abnormal shifts, in 12-lead electrocardiogram (ECG)
Time Frame: From Day 1 to Week 52 and Week 104
Change in the heart's electrical activity
From Day 1 to Week 52 and Week 104
Change, including normal/abnormal shifts, in laboratory evaluations
Time Frame: From Day 1 to Week 52 and Week 104
Hematology, serum chemistries, urinalysis, cardiac enzymes and anti-AAV1 antibodies
From Day 1 to Week 52 and Week 104

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in myocardium and left ventricular structure and function
Time Frame: From baseline to Week 52 and Week 104
Assessed by cardiac magnetic resonance imaging with late gadolinium enhancement
From baseline to Week 52 and Week 104
Change in skeletal muscle function
Time Frame: From baseline to Week 52 and Week 104
Assessed by PUL 2.0, grip strength, key and tip-to-tip pinch strength and elbow flexion strength
From baseline to Week 52 and Week 104
Change in pulmonary function
Time Frame: From baseline to Week 52 and Week 104
Assessed by slow vital capacity, forced expiratory volume in 1 second, forced vital capacity, peak expiratory flow, maximum inspiratory pressure, maximum expiratory pressure, peak cough flow and inspiratory flow reserve
From baseline to Week 52 and Week 104
Change in quality of life
Time Frame: From baseline to Week 52 and Week 104
Assessed by the Duchenne muscular dystrophy quality of life questionnaire
From baseline to Week 52 and Week 104

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sardocor Corp.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 2, 2024

Primary Completion (Estimated)

October 1, 2027

Study Completion (Estimated)

October 1, 2030

Study Registration Dates

First Submitted

January 11, 2024

First Submitted That Met QC Criteria

January 24, 2024

First Posted (Actual)

January 25, 2024

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 26, 2025

Last Verified

February 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRD-001-1004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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