TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Recruiting now · Phase 3
Conditions studied: Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
In brief
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
Key facts
- Study ID
- NCT07223203
- Run by
- Alnylam Pharmaceuticals
- People needed
- 125
- Starts
- 2025-12-12
- Expected to finish
- 2031-06-12
- Last updated by the study team
- 2026-07-23
Who can join
Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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%
You may not qualify if…
- 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
Where it is running
- Clinical Trial Site — Istanbul, Turkey (Türkiye) (enrolling)
- Clinical Trial Site — Chicago, Illinois, United States (enrolling)
- Clinical Trial Site — Baltimore, Maryland, United States (enrolling)
- Clinical Trial Site — Boston, Massachusetts, United States (enrolling)
- Clinical Trial Site — Rochester, Minnesota, United States (enrolling)
- Clinical Trial Site — New York, New York, United States (enrolling)
- Clinical Trial Site — Chapel Hill, North Carolina, United States (enrolling)
- Clinical Trial Site — Dallas, Texas, United States (enrolling)
- Clinical Trial Site — Houston, Texas, United States (enrolling)
- Clinical Trial Site — Darlinghurst, Australia (enrolling)
- Clinical Trial Site — Woolloongabba, Australia (enrolling)
- Clinical Trial Site — Ribeirão Preto, Brazil (enrolling)
- Clinical Trial Site — São Paulo, Brazil (enrolling)
- Clinical Trial Site — Égkomi, Cyprus (enrolling)
- Clinical Trial Site — Créteil, France (enrolling)
- Clinical Trial Site — Le Kremlin-Bicêtre, France (enrolling)
- Clinical Trial Site — Marseille, France (enrolling)
- Clinical Trial Site — Berlin, Germany (enrolling)
- Clinical Trial Site — Taoyuan City, Taiwan (enrolling)
- Clinical Trial Site — Aurora, Colorado, United States (enrolling)
- Clinical Trial Site — Athens, Greece (enrolling)
- Clinical Trial Site — Heraklion, Greece (enrolling)
- Clinical Trial Site — Florence, Italy (enrolling)
- Clinical Trial Site — Milan, Italy (enrolling)
- Clinical Trial Site — Pavia, Italy (enrolling)
Full record on ClinicalTrials.gov
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