5 completed, 3 active not recruiting, 1 enrolling by invitation. 5 Phase 3, 3 Phase 1, 2 Phase 2.
By phase and status
Follow eplontersen: new trials by email Literature Review of eplontersen trials Research Report on eplontersen
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix .
Trial Phase Status Sponsor Enrollment Start Updated
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 Eplontersen, ALXN2220, Placebo - Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Phase 2 Active not recruiting 326 2026-06-11 2026-07-14
A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers Eplontersen Solution for Injection - Transthyretin-mediated Amyloidosis Phase 1 Completed 12 2024-08-06 2024-11-21
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 Eplontersen, Placebo - Transthyretin Amyloid Cardiomyopathy Phase 3 Active not recruiting 64 2023-12-01 2026-06-09
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) Eplontersen - Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) Phase 3 Enrolling by invitation 1400 2022-11-30 2026-04-17
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 Eplontersen - Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Phase 3 Active not recruiting 151 2022-01-04 2025-12-04
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 Eplontersen, Placebo - Healthy Participants Phase 1 Completed 24 2020-04-23 2022-03-31
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) Eplontersen, Placebo - Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) Phase 3 Completed 1438 2020-03-13 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 Inotersen, Eplontersen - Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Phase 3 Completed 168 2019-12-11 2024-12-13
Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ). ION-682884, Placebo, Vitamin A - Healthy Volunteers, hATTR Amyloidosis Phase 1 / Phase 2 Completed 47 2018-12-21 2022-12-19
Conditions studied Amyloidosis (3) · Cardiomyopathies (1) · Polyneuropathies (1)
Sponsors Ionis Pharmaceuticals, Inc. (6) · AstraZeneca (3)
Countries Canada (6) · United States (6) · France (5) · Italy (5) · Spain (5) · Sweden (5) · Argentina (4) · Australia (4) · Brazil (4) · Germany (4) · Portugal (4) · China (3) · Greece (3) · Japan (3) · United Kingdom (3)
Top investigators Shuyang Zhang, MD (1) · Yang Lin, Medical PhD (1)
Results publications PMID 35869634 , PMID 38065307 , PMID 33283485 , PMID 33638113 , PMID 36525140 , PMID 37768671 , PMID 41913562 , PMID 41937511 , PMID 42104840 , PMID 42663301 , PMID 42669065 , PMID 42742182
NIH funding 2019 (2)