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NCT05071300 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study of Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, sponsored by Ionis Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 151
- Enrollment target
- 20
- Study locations
NCT05071300: Active Phase 3 study of Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, sponsored by Ionis Pharmaceuticals.
NCT05071300 is a Phase 3 study of Hereditary Transthyretin-Mediated Amyloid Polyneuropathy that is active but no longer recruiting, run by Ionis Pharmaceuticals. The registered enrollment target is 151 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (80% lower). The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05071300, a Phase 3 study of Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, is active but no longer recruiting, sponsored by Ionis Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 151 participants
- Enrollment target
- 20
- Study locations
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 Studied
Interventions
- DRUG Eplontersen
Study Locations (20)
Other
- Hospital Italiano de Buenos Aires - Buenos Aires
- Instituto Fleni - Buenos Aires
- Hospital El Cruce - San Juan Bautista
- Universidade Estadual de Campinas - Campinas
- Instituto de Neurologia de Curitiba - Curitiba
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - São Paulo
- The Cyprus Institute of Neurology and Genetics - Égkomi
Arizona
- Mayo Clinic - Scottsdale
Indiana
- Indiana University Health University Hospital - Indianapolis
Maryland
- Johns Hopkins University Neurology Research Office - Baltimore
Massachusetts
- Boston University School of Medicine - Boston
New York
- The Neurological Institute of New York - New York
North Carolina
- University of North Carolina Hospitals - Neurology Clinic - Chapel Hill
Oregon
- Oregon Health and Science University - Portland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 151 participants |
| Start Date | 2022-01-04 |
| Est. Completion | 2029-08 |
| Phase | Phase 3 |
What the registry record for NCT05071300 still lists
NCT05071300 is an interventional study that assigns participants to a tested intervention. The registered 151 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (80% lower).
The record links to 1 condition, with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy appearing as the primary indexed condition, and to 1 intervention - of which Eplontersen is the first listed.
NCT05071300 names 20 study sites across 14 states, led by Other, Arizona, Indiana.
Frequently Asked Questions
What is clinical trial NCT05071300 about?
NCT05071300 is a clinical study titled "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". 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).
What is the current status of trial NCT05071300?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 151 participants. The study started on 2022-01-04. Estimated completion is 2029-08.
What conditions does trial NCT05071300 study?
This clinical trial studies the following conditions: Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.
What interventions are being tested in trial NCT05071300?
The interventions under investigation include: Eplontersen (DRUG).
Who is sponsoring clinical trial NCT05071300?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05071300 being conducted?
This trial has 20 study locations across Arizona, Indiana, Maryland, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05071300's enrollment target sits among peer trials
151 1518th of 2000 higher than 482 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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