NCT05667493

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1,400 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.

Detailed description

This is a multicenter, open-label extension, Phase 3 study in up to approximately 1400 participants from the 682884-CS2 study. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or until after eplontersen is approved and available in the site's country, whichever occurs first. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A.

Conditions

• Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Interventions

Type	Name	Description
DRUG	Eplontersen	Eplontersen will be administered by SC injection.

Timeline

• Start date: 2022-11-30
• Primary completion: 2029-08-01
• Completion: 2029-08-01
• First posted: 2022-12-28
• Last updated: 2026-04-17

Locations

134 sites across 20 countries: United States, Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia, Denmark, France, Germany, Greece, Israel, Italy, Japan, Poland, Portugal, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study