Clinical Trial

A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
Protocol Amendment History 24 amendments
This ClinicalTrials.gov record has been amended 24 times since 2021-10-04; most recent amendment 2025-12-02.
Status change: Recruiting → Active, Not Recruiting 2024-09-16
Status change: Not Yet Recruiting → Recruiting 2022-01-07
Trial Details
NCT Number NCT05071300
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Enrollment 151 participants
Start Date 2022-01-04
Primary Completion 2029-08 (estimated)
Study Completion 2029-08 (estimated)
Updated on ClinicalTrials.gov 2025-12-04