NCT Number: NCT06224660
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.
Interested in participating?
Request Info18 year and older
Male
Interventional
Phase 1
The University of Kansas Medical Center, Kansas City, Kansas, United States
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.
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
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
Time frame: From Day 1 to Week 52 and Week 104
Death
Time frame: From Day 1 to Week 52 and Week 104
Adverse events related to the investigational product or the administration procedure
Time frame: From Day 1 to Week 52 and Week 104
Adverse events
Time frame: From Day 1 to Week 52
Cell-mediated immune reaction as assessed by enzyme-linked immunosorbent spot (ELISpot)
Time frame: From Day 1 to Week 52 and Week 104
Change in the heart's electrical activity
Time frame: From Day 1 to Week 52 and Week 104
Hematology, serum chemistries, urinalysis, cardiac enzymes and anti-AAV1 antibodies
Time frame: From baseline to Week 52 and Week 104
Assessed by cardiac magnetic resonance imaging with late gadolinium enhancement
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
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
Time frame: From baseline to Week 52 and Week 104
Assessed by the Duchenne muscular dystrophy quality of life questionnaire
Contact information is provided by the study sponsor or research team.
Sardocor Corp.
Industry
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
Acronym: MUSIC-DMD
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View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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