TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Фаза 3 – широко изпитване преди одобрение
Заболявания:
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy
hATTR-PN
Спонсор: Alnylam Pharmaceuticals
Налично на:
БГ
Обобщение
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
Кой може да участва
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