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TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Recruiting
18-85 years
All
Phase 3

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Overview

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 Criteria:

  • 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 Criteria:

  • 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

Study details
    Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy
    hATTR-PN

NCT07223203

Alnylam Pharmaceuticals

31 January 2026

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