NCT05071300

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

An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 151 (actual)
• 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 hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).

Detailed description

This is a multicenter, open-label, Phase 3 study in up to approximately 165 participants. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A. This study will consist of the following periods: less than or equal to (≤) 8-week screening period, a 157-week treatment period, and a 24-week post-treatment evaluation period. Participants may continue to receive treatment in this study for up to 2 years or until ION-682884 becomes commercially available in the patient's country, whichever occurs earlier.

Conditions

• Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Interventions

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

Timeline

• Start date: 2022-01-04
• Primary completion: 2029-08-01
• Completion: 2029-08-01
• First posted: 2021-10-08
• Last updated: 2025-12-04

Locations

33 sites across 13 countries: United States, Argentina, Australia, Brazil, Canada, Cyprus, France, Italy, Portugal, Spain, Sweden, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study