Summary

Eligibility
for people ages 18-80 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around
Principal Investigator
by Richard Cheng, MD
Headshot of Richard Cheng
Richard Cheng

Description

Summary

It is believed that targeted SERCA2a enzyme replacement in HFrEF patients will correct defective intracellular Ca2+ hemostasis, resulting in improved cardiac contractile function and energetics which will, in turn, translate to improved clinical outcomes. Additionally, it is hypothesized that correcting SERCA2a dysfunction will also improve coronary blood flow through correction of the impaired endothelium-dependent nitric oxide-mediated vasodilatation observed in heart failure.

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

Details

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.

Keywords

Congestive Heart Failure, Heart Failure, Systolic, Heart Failure, HFrEF - Heart Failure With Reduced Ejection Fraction, Systolic Heart Failure, SRD-001

Eligibility

You can join if…

Open to people ages 18-80

You CAN'T join if...

  • 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

Locations

  • UCSF accepting new patients
    San Francisco California 94143 United States
  • San Diego Cardiac Center accepting new patients
    San Diego California 92123 United States

Lead Scientist at UCSF

  • Richard Cheng, MD
    Dr. Richard Cheng is a cardiologist who specializes in the comprehensive care of patients with heart failure. Having trained in both advanced heart failure and interventional cardiology, his approach combines medical therapy optimization with minimally invasive surgeries and novel devices that can improve heart function.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Sardocor Corp.
ID
NCT04703842
Phase
Phase 1/2 Heart Failure Research Study
Study Type
Interventional
Participants
Expecting 57 study participants
Last Updated