TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
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)
Alnylam Pharmaceuticals
125 participants
Jan 16, 2026
INTERVENTIONAL
Conditions
Summary
The purpose of this study is to: * Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed * Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels
Eligibility
Inclusion Criteria4
- Has documented diagnosis of hATTR-PN
- Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant
- Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive)
- Has a Karnofsky Performance Status (KPS) of ≥60%
Exclusion Criteria7
- Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study
- Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis
- Has a New York Heart Association (NYHA) heart failure classification \>2
- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2.5 upper limit of normal (ULN)
- Has total bilirubin \>1.5 ULN
- Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m\^2
- Has other known causes of sensorimotor or autonomic neuropathy
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Interventions
Nucresiran 300 mg administered SC q6M
Vutrisiran 25 mg administered SC q3M
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07223203