Heart failure & cardiomyopathiesTrial design
Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy
- Phase 3, randomized, double-blind, placebo-controlled trial
Study context
- Population
- Patients with transthyretin amyloidosis with cardiomyopathy (histological evidence of amyloid deposits or grade 2 to 3 cardiac uptake on cardiac scintigraphy in the absence of plasma cell dyscrasia, New York Heart Association class I–III, end-diastolic interventricular septum thickness >12 millimeters)randomized participants who were dosed with study drug or placebo
- Participants
- 1,432 patients
- Planned comparison
- eplontersen 45 mg or placebo, randomized 1:1, with locally available standard of care including unrestricted use of TTR stabilizers
- Primary endpoint
- Composite of:
- Cardiovascular mortality
- Recurrent clinical cardiovascular events
- Follow-up
- Through 140 weeks
No results yet. This paper sets out the design of a trial; its results are to come.
Original publication
Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. Published 9 May 2026. doi:10.1161/circheartfailure.126.014205
Summarised from the published abstract; every number is checked against it