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

NCT06224660 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: SRD-001-1004
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

DMD-Associated Dilated Cardiomyopathy

Outcome Measures

Primary Outcomes (4)

• Rate of all-cause mortality

  Death

  From Day 1 to Week 52 and Week 104

• Rate and severity of related treatment-emergent adverse events

  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

  Adverse events

  From Day 1 to Week 52 and Week 104

• Rate of cell-mediated immune reaction

  Cell-mediated immune reaction as assessed by enzyme-linked immunosorbent spot (ELISpot)

  From Day 1 to Week 52

Secondary Outcomes (2)

• Change, including normal/abnormal shifts, in 12-lead electrocardiogram (ECG)

  From Day 1 to Week 52 and Week 104

• Change, including normal/abnormal shifts, in laboratory evaluations

  From Day 1 to Week 52 and Week 104

Other Outcomes (4)

• Change in myocardium and left ventricular structure and function

  From baseline to Week 52 and Week 104

• Change in skeletal muscle function

  From baseline to Week 52 and Week 104

• Change in pulmonary function

  From baseline to Week 52 and Week 104

Study Arms (3)

Low Dose

EXPERIMENTAL

SRD-001

Genetic: SRD-001

High Dose

EXPERIMENTAL

SRD-001

Genetic: SRD-001

Control

NO INTERVENTION

No-Intervention Control

Interventions

SRD-001 GENETIC

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.

Also known as: AAV1/SSERCA2a

High Dose; Low Dose

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Sardocor Corp.: lead

Study Sites (3)

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43215, United States

RECRUITING

MeSH Terms

Conditions

Dmd-Associated Dilated Cardiomyopathy

Central Study Contacts

Sardocor Corp.

CONTACT

+1-617-880-7616; info@sardocorcorp.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 11, 2024
• First Posted: January 25, 2024
• Study Start: October 2, 2024
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2030
• Last Updated: February 27, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)