NCT04136171

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)

A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1,438 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a multicenter, double-blind study in 1438 participants, who will be randomized to receive subcutaneous (SC) injections of either eplontersen or placebo once every 4 weeks. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.

Conditions

• Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Interventions

Type	Name	Description
DRUG	Eplontersen	Eplontersen by subcutaneous injection
DRUG	Placebo	Eplontersen-matching placebo by subcutaneous injection

Timeline

• Start date: 2020-03-13
• Primary completion: 2026-04-01
• Completion: 2026-08-01
• First posted: 2019-10-23
• Last updated: 2025-08-08

Locations

142 sites across 21 countries: United States, Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia, Denmark, France, Germany, Greece, Israel, Italy, Japan, Poland, Portugal, Puerto Rico, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study