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Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction
NCT04703842 · Sardocor Corp.
In plain English
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Official title
A Phase 1/2 Trial of the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Heart Failure With Reduced Ejection Fraction
About this study
MUSIC-HFrEF1 is an interventional study of SRD-001, an adeno-associated virus serotype 1 (AAV1) vector expressing the transgene for sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), in anti-AAV1 neutralizing antibody (NAb) negative subjects with ischemic or non-ischemic cardiomyopathy and New York Heart Association (NYHA) class III/IV symptoms of heart failure with reduced ejection fraction (HFrEF).
The Phase 1 trial is an open-label, uncontrolled study investigating two doses of SRD-001 at 3E13 and 4.5E13 viral genomes (vg) in 3-4 participants at each dose level.
The Phase 2 trial is a randomized, double-blind, placebo-controlled trial with prospective assignment to a single dose of either SRD-001 or placebo in a 1:1 ratio and a total sample size of N=50.
Eligibility criteria
Main Inclusion Criteria:
* Chronic ischemic or non-ischemic cardiomyopathy
* NYHA class III/IV
* LVEF ≤35%
* Guideline-directed medical therapy for heart failure; ICD
Main Exclusion Criteria:
* Restrictive cardiomyopathy, hypertrophic cardiomyopathy, acute myocarditis, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease or discrete left ventricular (LV) aneurysm
* Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), mechanical circulatory support device (MCSD) or cardiac shunt
* Likely to receive cardiac resynchronization therapy, cardiomyoplasty, LVRS, conventional revascularization procedure or valvular repair in the 6 months following treatment
* Likely need for an immediate heart transplant or MCSD implant due to hemodynamic instability
* Inadequate hepatic and renal function
* Diagnosis of, or treatment for, any cancer within the last 5 years except for basal cell carcinoma or carcinomas in situ where surgical excision was considered curative
Study design
Enrollment target: 57 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2021-09-23
Estimated completion: 2028-12
Last updated: 2024-03-26
Interventions
Biological: SRD-001Drug: Placebo
Primary outcomes
- • Change from baseline in symptomatic parameters (Baseline to Month 6 and Month 12)
- • Change from baseline in symptomatic parameters (Baseline to Month 6 and Month 12)
- • Change from baseline in physical parameter (Baseline to Month 6 and Month 12)
Sponsor
Sardocor Corp. · industry
Contacts & investigators
ContactCOO · contact · info@sardocorcorp.com · 858-752-2941
All locations (5)
San Diego Cardiac CenterRecruiting
San Diego, California, United States
University of California, San FranciscoRecruiting
San Francisco, California, United States
Washington University in Saint LouisRecruiting
St Louis, Missouri, United States
University of Texas Southwestern Medical CenterRecruiting
Dallas, Texas, United States
University of Washington MedicineRecruiting
Seattle, Washington, United States