Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy
A major trial tests whether a new weekly injection can slow heart damage from a rare protein-buildup disease, with results coming soon.
CARDIO-TTRansform is a Phase 3 RCT testing eplontersen (45mg SC every 4 weeks), a novel antisense oligonucleotide that reduces circulating TTR protein by targeting hepatic TTR mRNA, versus placebo in 1,432 patients with ATTR cardiomyopathy. The trial has completed enrollment and will report cardiovascular mortality and recurrent CV events as its primary endpoint, with echocardiography and CMR substudies.
What the study was
- Study design
- Phase 3 randomized double-blind placebo-controlled trial (design/rationale paper)
- Population
- Adults with ATTR cardiomyopathy (NYHA class I–III, amyloid deposits confirmed, IVS thickness >12mm)
- Sample size
- 1432
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- Circulation: Heart Failure
Why it surfaced
Largest ATTR cardiomyopathy RCT ever conducted (n=1,432); eplontersen is a novel N-GalNAc ASO mechanism for reducing TTR production; Phase 3 with CV mortality endpoint fully enrolled. Design paper (no results yet) caps design_quality at 1 and evidence_maturity at Exploratory, but NOVEL_TREATMENT flag elevates to HIGH per pipeline rules. Results when published will likely be practice-changing.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.