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NCT07223203
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Conditions: Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
Sex: All
Ages: 18 Years – 85 Years
Healthy volunteers: No
Phase: PHASE3
Enrollment: 125
Sponsor: Alnylam Pharmaceuticals
Location: Clinical Trial Site Aurora Colorado
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
Source: ClinicalTrials.gov (NCT07223203). StuddyBuddy aggregates publicly available trial information.