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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRD-001 | Experimental | 3E13 or 4.5E13 vg; one-time intracoronary infusion |
|
| Placebo | Placebo Comparator | One-time intracoronary infusion |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRD-001 | Biological | AAV1/SERCA2a |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in symptomatic parameters | New York Heart Association classification (I, II, III or IV) | Baseline to Month 6 and Month 12 |
| Change from baseline in symptomatic parameters | Quality of life as assessed by Kansas City Cardiomyopathy Questionnaire: 0-24, very poor to poor; 25-49, poor to fair; 50-74, fair to good; and 75-100, good to excellent | Baseline to Month 6 and Month 12 |
| Change from baseline in physical parameter | Distance walked during the 6MWT | Baseline to Month 6 and Month 12 |
| Change from baseline in LV function/remodeling | Left ventricular end systolic volume (LVESV) as assessed by echocardiography | Baseline to Month 6 and Month 12 |
| Rate of recurrent events | HF-related hospitalization, ambulatory worsening heart failure, all-cause death, MCSD and transplant | Baseline to Month 6 and Month 12 |
| Rate of adverse events | Treatment-emergent adverse events | 6 and 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of subjects who complete the trial | Those who complete the trial vs withdraw consent, are lost to follow-up, or withdrawn due to an AE or other reason | 12 months |
| Concomitant medication use |
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Main Inclusion Criteria:
Main Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| COO | Contact | 858-752-2941 | info@sardocorcorp.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| San Diego Cardiac Center | Recruiting | San Diego | California | 92123 | United States |
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| Drug |
SRD-001 matching placebo |
|
Changes in heart failure related medications
| 6 and 12 months |
| Incidence of abnormal laboratory test results | Hematologic, serum chemistries, NT-proBNP and troponin | Baseline to Month 6 and Month 12 |
| Incidence of abnormal ECG results | New arrhythmias | Baseline to Month 6 and Month 12 |
| Incidence of abnormal physical examination findings | Change from baseline in physical examination findings | Baseline to Month 6 and Month 12 |
| Incidence of abnormal findings from interrogation of implantable cardioverter defibrillator | New arrhythmias | Baseline to Month 6 and Month 12 |
| University of California, San Francisco | Recruiting | San Francisco | California | 94143 | United States |
|
| Washington University in Saint Louis | Recruiting | St Louis | Missouri | 63110 | United States |
|
| University of Texas Southwestern Medical Center | Recruiting | Dallas | Texas | 75390 | United States |
|
| University of Washington Medicine | Recruiting | Seattle | Washington | 98195 | United States |
|
| ID | Term |
|---|---|
| D006333 | Heart Failure |
| D054143 | Heart Failure, Systolic |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
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