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Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction (MUSIC-HFrEF1)

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ClinicalTrials.gov Identifier: NCT04703842
Recruitment Status : Recruiting
First Posted : January 11, 2021
Last Update Posted : July 28, 2022
Sponsor:
Collaborator:
Covance
Information provided by (Responsible Party):
Sardocor Corp.

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Congestive Heart Failure Heart Failure, Systolic Heart Failure Biological: SRD-001 Drug: Placebo Phase 1 Phase 2

Detailed Description:

The CUPID-3 Trial 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 one dose of SRD-001 at 3E13 viral genomes (vg) in 4 to 8 subjects.

The Phase 2 is a randomized, double-blind, placebo-controlled trial with prospective assignment to a single dose of either SRD-001 at 3E13 vg or placebo in a 1:1 ratio and a total sample size of N=50.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 56 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : December 1, 2021
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2028

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Failure

Arms and Interventions
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Arm Intervention/treatment
Experimental: SRD-001
3E13 vg; one-time intracoronary infusion
 Biological: SRD-001
AAV1/SERCA2a
Other Name: MYDICAR
Placebo Comparator: Placebo
One-time intracoronary infusion
 Drug: Placebo
SRD-001 matching placebo


Outcome Measures
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Primary Outcome Measures :
  1. Change from baseline in symptomatic parameters [ Time Frame: Baseline to Month 6 and Month 12 ]
    New York Heart Association classification (I, II, III or IV)

  2. Change from baseline in symptomatic parameters [ Time Frame: Baseline to Month 6 and Month 12 ]
    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

  3. Change from baseline in physical parameter [ Time Frame: Baseline to Month 6 and Month 12 ]
    Distance walked during the 6MWT

  4. Change from baseline in LV function/remodeling [ Time Frame: Baseline to Month 6 and Month 12 ]
    Left ventricular end systolic volume (LVESV) as assessed by echocardiography

  5. Rate of recurrent events [ Time Frame: Baseline to Month 6 and Month 12 ]
    HF-related hospitalization, ambulatory worsening heart failure, all-cause death, MCSD and transplant

  6. Rate of adverse events [ Time Frame: 6 and 12 months ]
    Treatment-emergent adverse events


Secondary Outcome Measures :
  1. Proportion of subjects who complete the trial [ Time Frame: 12 months ]
    Those who complete the trial vs withdraw consent, are lost to follow-up, or withdrawn due to an AE or other reason

  2. Concomitant medication use [ Time Frame: 6 and 12 months ]
    Changes in heart failure related medications

  3. Incidence of abnormal laboratory test results [ Time Frame: Baseline to Month 6 and Month 12 ]
    Hematologic, serum chemistries, NT-proBNP and troponin

  4. Incidence of abnormal ECG results [ Time Frame: Baseline to Month 6 and Month 12 ]
    New arrhythmias

  5. Incidence of abnormal physical examination findings [ Time Frame: Baseline to Month 6 and Month 12 ]
    Change from baseline in physical examination findings

  6. Incidence of abnormal findings from interrogation of implantable cardioverter defibrillator [ Time Frame: Baseline to Month 6 and Month 12 ]
    New arrhythmias


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Chronic ischemic or non-ischemic cardiomyopathy
  • NYHA class III/IV
  • LVEF ≤35%
  • Maximal, optimized heart failure therapy; 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04703842


Contacts
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Contact: Jonathan Plehn, MD jonathan.plehn@sardocorcorp.com

Locations
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United States, California
San Diego Cardiac Center Recruiting
San Diego, California, United States, 92123
Contact: Lee Vowinkel, RN, MSN    858-244-6890    lvowinkel@sdcardiac.com   
Sponsors and Collaborators
Sardocor Corp.
Covance
More Information
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Responsible Party: Sardocor Corp.
ClinicalTrials.gov Identifier: NCT04703842    
Other Study ID Numbers: SRD-001-1001
First Posted: January 11, 2021    Key Record Dates
Last Update Posted: July 28, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Heart Failure
Heart Failure, Systolic
Heart Diseases
Cardiovascular Diseases