Heart Failure, DiastolicHeart Failure With Preserved Ejection Fraction
Investigational drug(s) / intervention(s)
AAV1/SERCA2a
AAV1/SERCA2a: 3E13 viral genomes fixed dose
Study summary
The goal of this clinical trial is to test an experimental gene therapy in participants with heart failure with preserved ejection fraction, also known as diastolic heart failure. The main questions it aims to answer are:
* safety and tolerability of the gene therapy; and
* whether the gene therapy helps the heart ventricles relax during filling. Participants will undergo a one-time infusion of the gene therapy in the cardiac catheterization laboratory and then be followed for safety and effects on left-sided filling pressures while exercising. The first year will have multiple in-person visits followed by 4 years of biannual phone calls.
Eligibility
Sex
ALL
Min age
50 Years
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Change in pulmonary capillary wedge pressure (PCWP) — Week 24 and Week 52 PCWP assessed by right heart catheterization; change in mmHg from baseline
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.