FindTrial
Sign In

Eplontersen

DRUG7 trials

Sponsors

Ionis Pharmaceuticals, Inc., AstraZeneca

Conditions

Healthy ParticipantsHereditary Transthyretin-Mediated Amyloid PolyneuropathyTransthyretin Amyloid CardiomyopathyTransthyretin Amyloid Cardiomyopathy (ATTR-CM)Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Phase 1

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants
CompletedNCT04302064
Ionis Pharmaceuticals, Inc.Healthy Participants
Start: 2020-04-23End: 2020-09-10Updated: 2022-03-31

Phase 2

A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM
Active, not recruitingNCT07608354
AstraZenecaTransthyretin Amyloid Cardiomyopathy (ATTR-CM)
Start: 2026-06-11End: 2029-02-06Target: 326Updated: 2026-07-14

Phase 3

CARDIO-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 Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
CompletedNCT04136171
Ionis Pharmaceuticals, Inc.Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Start: 2020-03-13End: 2026-08-12Updated: 2026-09-03
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
CompletedNCT04136184
Ionis Pharmaceuticals, Inc.Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Start: 2019-12-11End: 2023-07-12Updated: 2024-12-13
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
Active, not recruitingNCT05071300
Ionis Pharmaceuticals, Inc.Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Start: 2022-01-04End: 2029-08-31Updated: 2025-12-04
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Enrolling by invitationNCT05667493
Ionis Pharmaceuticals, Inc.Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Start: 2022-11-30End: 2029-08-01Target: 1400Updated: 2026-04-17
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Active, not recruitingNCT06194825
AstraZenecaTransthyretin Amyloid Cardiomyopathy
Start: 2023-12-01End: 2027-01-05Updated: 2026-06-09

Related Papers

Gene Silencer Therapy in Transthyretin Amyloid Cardiomyopathy: A Meta-Analysis of Outcomes Trials.
2026-08-30
Eplontersen for Transthyretin Amyloid Cardiomyopathy
New England Journal of Medicine2026-08-28
Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy
Circulation Heart Failure2026-05-09
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients.
2026-04-06
Effect of Eplontersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy Across Genetic Variants: An Exploratory Analysis From the NEURO-TTRansform Trial.
2026-04-01
Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care.
2025-03-19
Effects of Empagliflozin on Fluid Overload, Weight, and Blood Pressure in CKD
Journal of the American Society of Nephrology2023-12-1240 citations
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy
JAMA2023-09-28169 citations
Evaluation of methodologies for indirect comparison of eplontersen and vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy (P12-4.005)
Neurology2023-04-25
Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen.
2022-12-1625 citations
Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis
British Journal of Clinical Pharmacology2022-07-2323 citations
Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L<sub>Rx</sub> (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.
2021-02-2645 citations
Ligand Conjugated Antisense Oligonucleotide for the Treatment of Transthyretin Amyloidosis: Preclinical and Phase 1 Data
ESC Heart Failure2020-12-0785 citations