Heart failure & cardiomyopathiesRandomised controlled trial
AB-1002 gene therapy expressing active protein phosphatase inhibitor-1 in heart failure: rationale and study design of the GenePHIT trial
- Randomised controlled trial (rationale and study design)
- adaptive
- double-blind
- placebo-controlled
- multicentre
- Phase 2
- 52-week observation period and 4-year long-term follow-up
Authors' conclusion
This Phase 2, placebo-controlled trial will further elucidate the risk-benefit characterization of single-dose AB-1002 in patients with non-ischaemic heart failure.
Study context
- Population
- Adults with non-ischaemic cardiomyopathy, LVEF 15%-35% and NYHA class III symptoms despite standard of carerandomised 1:1:1
- Comparison
- AB-1002 vs placebo
- AB-1002 7.15E13n = 30-50
- AB-1002 1.43E14n = 30-50
- Placebon = 30-50reference
- Primary endpoint
- Hierarchical composite of:
- CV death
- Change in NYHA class
- LVEF improvement ≥5% and 6-minute walk test improvement >30m
- Pooled active doses vs placebo by win ratio (Finkelstein-Schoenfeld)
- Follow-up
- 52 weeks
Original publication
AB-1002 gene therapy expressing active protein phosphatase inhibitor-1 in heart failure: rationale and study design of the GenePHIT trial. Eur J Heart Fail. Published 7 April 2026. doi:10.1093/ejhf/xuag105
Summarised from the published abstract; every number is checked against it