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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 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. The trial reports 20 study locations across 14 states.

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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).

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

FieldValue
Enrollment Target 151 participants
Start Date 2022-01-04
Est. Completion 2029-08
Phase Phase 3

Sponsor

Ionis Pharmaceuticals

76 total trials

What the Registry Record Tells You About NCT05071300

The ClinicalTrials.gov registry entry for NCT05071300 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 151 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ionis Pharmaceuticals, which has 76 total studies on file at ClinicalTrials.gov.

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 reports 20 study locations spanning 14 distinct geographic areas - top geographies include 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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.