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Taking people in United StatesPhase 1

Tests a gene therapy for weakened heart muscle in Duchenne muscular dystrophy

Tests gene therapy SRD-001 in adults with heart weakness from Duchenne muscular dystrophy.

Key facts

Who runs it
Sardocor Corp.
Where
Cincinnati, Columbus, and Kansas City, United StatesSee the 3 sites
Trial code
Registry title
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Status
Recruiting
Phase
Phase 1
People taking part
12 people, planned
Started
2 Oct 2024
Expected to end
Oct 2030
Type of study
Interventional, 3 groupsOne group gets no study treatment, for comparison.
Treatments tested
  • SRD-001
Record updated
27 February 2025

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for men aged 18 or older who have Duchenne muscular dystrophy with a weakened heart. The trial tests a gene therapy, SRD-001, against no treatment.

Age
18 years or older
Sex
Male only
Disease stage
Duchenne muscular dystrophy (DMD) confirmed by genetic testing, with heart muscle disease: scar in at least 3 of 16 segments of the left ventricle, and left ventricular ejection fraction below 40%.
Earlier treatment
Must have had individual, optimized heart medicine and glucocorticoid treatment for at least 12 months before joining.
Biomarker or test result
DMD diagnosis confirmed by genetic testing.

Main reasons for not taking part

  • Abnormal blood pressure
  • Liver test results raised for reasons not related to DMD
  • Cystatin C of 1.2 mg/L or higher
  • Low platelet count (thrombocytopenia) or anemia
  • Inadequate lung function

The registry criteria, word for word

Show all 11 criteriaShow less

Who can take part

  • 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

Who cannot take part

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

Where in United States

  • Cincinnati Children's Hospital Medical CenterCincinnati, United StatesContact: Heart Institute Neuromuscular Intake LineTaking people
  • Nationwide Children's HospitalColumbus, United StatesContact: Kelsey CraigTaking people
  • The University of Kansas Medical CenterKansas City, United StatesContact: Jillian BruennTaking people

About the trial

What the trial wants to find out

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.

The 3 groups in the trial

  • Low DoseTreatment being tested

    SRD-001

    Treatment: SRD-001

  • High DoseTreatment being tested

    SRD-001

    Treatment: SRD-001

  • ControlNo study treatment, for comparison

    No-Intervention Control

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
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
Plain-language title
Tests a gene therapy for weakened heart muscle in Duchenne muscular dystrophyGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
27 February 2025