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

NCT04703842 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: SRD-001-1001
• Study Type: interventional
• Target: 57
• Locations: 1 country
• Sites: 5

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.

Conditions

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

Outcome Measures

Primary Outcomes (6)

• 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

Secondary Outcomes (6)

• Proportion of subjects who complete the trial

  12 months

• Concomitant medication use

  6 and 12 months

• Incidence of abnormal laboratory test results

  Baseline to Month 6 and Month 12

• Incidence of abnormal ECG results

  Baseline to Month 6 and Month 12

• Incidence of abnormal physical examination findings

  Baseline to Month 6 and Month 12

Study Arms (2)

SRD-001

EXPERIMENTAL

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

Biological: SRD-001

Placebo

PLACEBO COMPARATOR

One-time intracoronary infusion

Drug: Placebo

Interventions

SRD-001 BIOLOGICAL

AAV1/SERCA2a

SRD-001

Placebo DRUG

SRD-001 matching placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify 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

Sponsors & Collaborators

• Sardocor Corp.: lead

Study Sites (5)

San Diego Cardiac Center

San Diego, California, 92123, United States

RECRUITING

University of California, San Francisco

San Francisco, California, 94143, United States

RECRUITING

Washington University in Saint Louis

St Louis, Missouri, 63110, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

University of Washington Medicine

Seattle, Washington, 98195, United States

RECRUITING

MeSH Terms

Conditions

Heart Failure; Heart Failure, Systolic

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Central Study Contacts

COO

CONTACT

858-752-2941; info@sardocorcorp.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2021
• First Posted: January 11, 2021
• Study Start: September 23, 2021
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 1, 2028
• Last Updated: March 26, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)