ALN-TTRSC04

DRUG3 trials

Sponsors

Alnylam Pharmaceuticals Inc., Alnylam Pharmaceuticals

Conditions

Hereditary transthyretin-mediated amyloidosis with polyneuropathyTransthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)Transthyretin-Mediated Amyloidosis

Phase 1

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects

CompletedNCT05661916

Alnylam PharmaceuticalsTransthyretin-Mediated Amyloidosis

Start: 2023-01-16End: 2025-09-30Updated: 2026-02-17

Phase 3

TRITON-CM: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy (ATTR amyloidosis with cardiomyopathy)

RecruitingCTIS2024-519917-72-00

Alnylam Pharmaceuticals Inc.Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)

Start: 2025-10-29Target: 682Updated: 2026-01-27

TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN)

Not yet recruitingCTIS2025-522544-40-00

Alnylam Pharmaceuticals Inc.Hereditary transthyretin-mediated amyloidosis with polyneuropathy

Target: 7Updated: 2026-02-20

Related Papers

Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules.

2025-12-12

Abstract 4135443: A Phase 1, Single Ascending Dose Study to Evaluate ALN-TTRsc04, a Next-Generation RNA Interference Therapeutic, in Healthy Participants for Potential Treatment of Transthyretin Amyloidosis

Circulation2024-11-121 citations