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

NCT05071300 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 151

Last updated 2025-12-04

No results posted yet for this study

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).

Conditions

  • Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Interventions

DRUG

Eplontersen

Eplontersen will be administered by SC injection.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-04
Primary Completion
2029-08-31
Completion
2029-08-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Brazil
  • Canada
  • Cyprus
  • France
  • Italy
  • Portugal
  • Spain
  • Sweden
  • Taiwan
  • Turkey (Türkiye)

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05071300 on ClinicalTrials.gov