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Recruiting Phase 3 NCT07223203

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

Phase 3 – large-scale trial before approval
Conditions: Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy hATTR-PN

Sponsor: Alnylam Pharmaceuticals

trial.available_in: БГ
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
Who can participate
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
Interventions
Nucresiran
DRUG
Vutrisiran
DRUG
Locations 4
South Korea (1)
Clinical Trial Site
Seoul
NOT_YET_RECRUITING
Sweden (1)
Clinical Trial Site
Stockholm
NOT_YET_RECRUITING
United States (2)
Clinical Trial Site
Boston , Massachusetts
Clinical Trial Site
Dallas , Texas
NOT_YET_RECRUITING
Technical details
Status
Recruiting
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
18 Years
Maximum age
85 Years
Healthy volunteers
No
Start date
12.12.2025
Completion date
12.06.2031
Registry ID
NCT07223203
Source
clinicaltrials.gov
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