Идёт набор NCT07588880

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China

— Наблюдательное Progressive Familial Intrahepatic Cholestasis

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
Кому может быть актуально: Состояния в реестре: Progressive Familial Intrahepatic Cholestasis. Базовые параметры: от 6 мес. · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Китай
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).

Обзор

This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China. PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus). Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC. The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.

Первичные конечные точки

Percentage of participants experiencing adverse events (AEs) [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.]
Percentage of participants experiencing serious adverse events (SAEs) [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.]

Вторичные конечные точки (6)

Event-free survival (EFS) [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection]
Surgical biliary diversion-free survival [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
Liver transplant-free survival [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
Overall survival [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
Pruritus improvement [Срок оценки: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
Change from baseline in serum bile acid [Срок оценки: From baseline and up to end of data collection (approximately 5 years of data collection]

Критерии участия

Критерии включения

Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician
On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions)
Signed informed consent and assent, as appropriate