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Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability of extended dosing with ION-682884 in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).


Clinical Trial Description

This is a multicenter, open-label, Phase 3 study in up to approximately 140 participants. Eligible participants will receive ION-682884 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05071300
Study type Interventional
Source Ionis Pharmaceuticals, Inc.
Contact Ionis Pharmaceuticals
Phone 800-679-4747
Email [email protected]
Status Not yet recruiting
Phase Phase 3
Start date October 2021
Completion date July 2024

See also
  Status Clinical Trial Phase
Active, not recruiting NCT04136184 - NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Phase 3