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)

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

CT.gov Identifier
NCT07223203
EudraCT Identifier
N/A
CTIS:
2025-522544-40-00
Sponsor
Alnylam Pharmaceuticals
Collaborator
N/A
Study Contact Information
N/A
Recruiting
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Study Details

Diseases
Alport syndrome
Study Drug
Vutrisiran
See More
ALN-TTRSC02
ALN-TTRSC04
AMVUTTRA
Nucresiran
Genes
COL4A4
COL4A3
Study Dates
Dec 2025 - Dec 2027
Sex
Ignored
Age
18 - 85 Years
Inclusion and Exclusion Criteria
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

Protocol 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

Study Locations

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