NCT07223203 TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
| NCT ID | NCT07223203 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Alnylam Pharmaceuticals |
| Condition | Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy |
| Study Type | INTERVENTIONAL |
| Enrollment | 125 participants |
| Start Date | 2025-12-12 |
| Primary Completion | 2027-12-27 |
Trial Parameters
Eligibility Fast-Check
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Brief 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 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