CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Summary

To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC).

Timeline

Start

2020-03-13

Primary completion

2026-04

Completion

2026-08

Outcome

Missed primary endpoint

release “the CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet the primary efficacy endpoint” ionis.com ↗

Publications

• Masri A, Cappelli F, Davis MK, Fontana M, Garcia-Pavia P, Gillmore JD, Hanna M, Obici L, Solomon SD, Sperry BW, Tahara N, Waddington-Cruz M, Kristen AV, Falk RH, Shah SJ, Nativi-Nicolau J, Viney NJ, Yang Q, Chen J, Tsimikas S, Maurer MS. Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2026 Jun;19(6):e014205. doi: 10.1161/CIRCHEARTFAILURE.126.014205. Epub 2026 May 9.
• Masri A, Maurer MS, Claggett BL, Kulac I, Waddington Cruz M, Conceicao I, Weiler M, Berk JL, Gertz M, Gillmore JD, Rush S, Chen J, Zhou W, Kwoh J, Duran JM, Tsimikas S, Solomon SD. Effect of Eplontersen on Cardiac Structure and Function in Patients With Hereditary Transthyretin Amyloidosis. J Card Fail. 2024 Aug;30(8):973-980. doi: 10.1016/j.cardfail.2023.11.016. Epub 2023 Dec 7.

Drugs

Indications