Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction

NCT ID: NCT04703842

Last Updated: 2024-03-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

57 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-23

Study Completion Date

2028-12-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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SRD-001

3E13 or 4.5E13 vg; one-time intracoronary infusion

Group Type EXPERIMENTAL

SRD-001

Intervention Type BIOLOGICAL

AAV1/SERCA2a

Placebo

One-time intracoronary infusion

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

SRD-001 matching placebo

Interventions

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SRD-001

AAV1/SERCA2a

Intervention Type BIOLOGICAL

Placebo

SRD-001 matching placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Chronic ischemic or non-ischemic cardiomyopathy
* NYHA class III/IV
* LVEF ≤35%
* Guideline-directed medical therapy for heart failure; ICD

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sardocor Corp.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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San Diego Cardiac Center

San Diego, California, United States

Site Status RECRUITING

University of California, San Francisco

San Francisco, California, United States

Site Status RECRUITING

Washington University in Saint Louis

St Louis, Missouri, United States

Site Status RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

University of Washington Medicine

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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COO

Role: CONTACT

858-752-2941

Facility Contacts

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Emma Burkhalter, RN

Role: primary

858-244-6890

Kaye Reambonanza

Role: primary

415-514-6147

Annie Dirks, RN, BSN

Role: primary

314-454-8711

Therese Villina, MPHI, BSN

Role: primary

214-645-7083

Katherine Nassans

Role: primary

206-543-6850

Other Identifiers

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SRD-001-1001

Identifier Type: -

Identifier Source: org_study_id

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