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Completed Phase 3 Interventional Results available

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

ClinicalTrials.gov ID: NCT04136184

Public ClinicalTrials.gov record NCT04136184. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 22, 2026, 1:39 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN). For more information, please visit http://www.neuro-ttransform.com/.

Study identification

NCT ID
NCT04136184
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Enrollment
168 participants

Conditions and interventions

Eligibility (public fields only)

Age range
18 Years to 82 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Dec 11, 2019 (Actual)
Primary completion
Apr 11, 2023 (Actual)
Completion
Jul 12, 2023 (Actual)
Last update posted
Dec 13, 2024

2019 – 2023

United States locations

U.S. sites
12
U.S. states
12
U.S. cities
12
Facility City State ZIP Site status
Mayo Clinic - Arizona Scottsdale Arizona 85259 —
Mayo Clinic - Jacksonville Jacksonville Florida 32224 —
Indiana University School of Medicine - Indianapolis Indianapolis Indiana 46202 —
University of Kansas Medical Center Kansas City Kansas 66160 —
Johns Hopkins University Neurology Research Office Baltimore Maryland 21224 —
Boston University School of Medicine Boston Massachusetts 02118 —
Mayo Clinic - Rochester Rochester Minnesota 55905 —
The Neurological Institute of New York New York New York 10032 —
University of North Carolina Hospitals - Neurology Clinic Chapel Hill North Carolina 27599 —
Oregon Health and Science University Portland Oregon 97239 —
Penn Presbyterian Medical Center Philadelphia Pennsylvania 19104 —
University of Washington Medical Center Seattle Washington 98195 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 33 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04136184, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Dec 13, 2024 · Synced Oct 22, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT04136184 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →