Heart failure & cardiomyopathies
AB-1002 gene therapy expressing active protein phosphatase inhibitor-1 in heart failure: rationale and study design of the GenePHIT trial
Study context
- Population
- Adults with non-ischaemic cardiomyopathy, LVEF 15%-35% and NYHA class III symptoms despite standard of carerandomised 1:1:1
- Planned 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%
- 6-minute walk test improvement >30m
- Follow-up
- 52 weeks
Methodological detailsrationale and study design, adaptive, double-blind, placebo-controlled, multicentre, Phase 2, 52-week observation period and 4-year long-term follow-up
- Rationale and study design
- Adaptive
- Double-blind
- Placebo-controlled
- Multicentre
- Phase 2
- 52-week observation period and 4-year long-term follow-up
No results yet. This paper sets out the design of a trial; its results are to come.
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.
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