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Набира участници Фаза 3 NCT07223203

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
Интервенции
Nucresiran
DRUG
Vutrisiran
DRUG
Места на провеждане 4
Южна Корея (1)
Clinical Trial Site
Seoul
NOT_YET_RECRUITING
Швеция (1)
Clinical Trial Site
Stockholm
NOT_YET_RECRUITING
САЩ (2)
Clinical Trial Site
Boston , Massachusetts
Clinical Trial Site
Dallas , Texas
NOT_YET_RECRUITING
Технически детайли
Статус
Набира участници
Фаза
Фаза 3
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Минимална възраст
18 Years
Максимална възраст
85 Years
Здрави доброволци
Не
Начална дата
12.12.2025
Крайна дата
12.06.2031
Регистрационен номер
NCT07223203
Източник
clinicaltrials.gov
Запитване за медицински туризъм

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