Gene therapy for heart failure with preserved ejection fraction
A Phase 1b, Pilot Trial Evaluating the Safety and Pharmacodynamic Effects of SRD-001 (AAV1-SERCA2a) in Subjects With Heart Failure With Preserved Ejection Fraction
PHASE1 · Sardocor Corp. · NCT06061549
This study is testing a new gene therapy to see if it can help people with heart failure feel better by improving how their heart relaxes and fills with blood.
Quick facts
| Phase | PHASE1 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 50 Years and up |
| Sex | All |
| Sponsor | Sardocor Corp. (industry) |
| Locations | 2 sites (Durham, North Carolina and 1 other locations) |
| Trial ID | NCT06061549 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and effectiveness of an experimental gene therapy targeting SERCA2a in patients with heart failure with preserved ejection fraction. Participants will receive a one-time infusion of the gene therapy directly into the heart and will be monitored for safety and changes in heart function during exercise. The study includes multiple follow-up visits in the first year and biannual assessments over the next four years to track overall health and heart performance. The trial seeks to determine if the therapy can improve the heart's ability to relax and fill with blood.
Who should consider this trial
Good fit: Ideal candidates include individuals with NYHA class II or III heart failure, a left ventricular ejection fraction of 50% or greater, and evidence of elevated filling pressures.
Not a fit: Patients with advanced heart failure (NYHA class IV), recent hospitalizations for heart failure, or significant comorbid conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could significantly improve heart function and quality of life for patients with diastolic heart failure.
How similar studies have performed: While gene therapy approaches for heart failure are still emerging, similar studies have shown promise in improving heart function, making this a potentially impactful area of research.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Willing and able to provide informed consent * Negative for anti-AAV1 neutralizing antibodies * NYHA class II or III * Left ventricular ejection fraction ≥ 50% * Evidence of resting or exercise-induced left ventricle filling pressure * On oral diuretic therapy * Adequate birth control Exclusion Criteria: * NYHA class IV * Heart failure requiring hospitalization in the past 3 months * Manifested or provocable ischemic heart disease * Atrial fibrillation * History of congenital heart disease, restrictive or infiltrative cardiomyopathy, hypertrophic cardiomyopathy, acute myocarditis, pericardial disease, uncorrected thyroid disease or discrete left ventricular (LV) aneurysm * History of amyloidosis * Untreated left-sided valvular disease * Severe COPD * BMI \> 50 kg/m\^2 * Severe liver, kidney or hematologic dysfunction * Cancer within the past 5 years * Unstable concurrent conditions
Where this trial is running
Durham, North Carolina and 1 other locations
- Duke University — Durham, North Carolina, United States (RECRUITING)
- University of Texas Southwestern Medical Center — Dallas, Texas, United States (NOT_YET_RECRUITING)
Study contacts
- Study coordinator: Sardocor
- Email: info@sardocorcorp.com
- Phone: 858-752-2941
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Heart Failure, Diastolic, Heart Failure With Preserved Ejection Fraction