AB-1002 gene therapy expressing active protein phosphatase inhibitor-1 in heart failure: Rationale and study design of the GenePHIT trial - PubMed
3 hours ago
- #Clinical Trial
- #Gene Therapy
- #Heart Failure
- AB-1002 is an investigational gene therapy using a cardiotropic adeno-associated viral vector to express active protein phosphatase inhibitor-1.
- The therapy aims to restore cardiomyocyte calcium handling and improve cardiac contractility in heart failure patients.
- GenePHIT is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized multicenter trial evaluating AB-1002.
- Participants are adults with non-ischemic cardiomyopathy, left ventricular ejection fraction 15%-35%, and NYHA class III symptoms.
- The trial randomizes participants 1:1:1 to two doses of AB-1002 or placebo via a single antegrade intracoronary infusion.
- The primary endpoint at 52 weeks is a hierarchical composite of cardiovascular death, NYHA class change, LVEF improvement, and six-minute walk test improvement.
- Secondary efficacy, safety, and exploratory endpoints will be assessed at 52 weeks and through 4-year follow-up.
- The trial aims to elucidate the risk-benefit profile of single-dose AB-1002 in non-ischemic heart failure.