Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Clinical Trial
Official title:
An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
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).
This is a multicenter, open-label, Phase 3 study in up to approximately 140 participants. Eligible participants will receive Eplontersen once every 4 weeks for up to 157 weeks. 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 weeks treatment period, and a 24-week post-treatment evaluation period. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04136184 -
NEURO-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 Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
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Phase 3 |