Идёт набор NCT07293897

A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)

— Наблюдательное Alagille Syndrome (ALGS) Progressive Familial Intrahepatic Cholestasis (PFIC)

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: В протоколе указаны: Maralixibat.
Кому может быть актуально: Состояния в реестре: Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC). Базовые параметры: Без ограничений · 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: Япония
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

A Cohort Study to Investigate the Risk of Liver Disorder of Livmarli Oral Solution in Patients With Japan Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) Registered in the Comprehensive and Informative Registry System for Childhood Liver Disease (CIRCLe)

Обзор

This study is a database study in Japan for maralixibat (TAK-625) used to treat participants with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). The main aim of the study is to evaluate the risk of liver disorder during the use of maralixibat in Japanese patients with ALGS or PFIC. This database study will conduct in use of medical database called Comprehensive and Informative Registry system for Childhood Liver Disease (CIRCLe).

Вмешательства

Препарат Maralixibat
Maralixibat Oral Solution 10 milligrams (mg)/milliliters (mL)

Первичные конечные точки

Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects) [Срок оценки: Up to 6 years as data period]
Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects) [Срок оценки: Up to 6 years as data period]
Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 1 (Side Effects) [Срок оценки: Up to 6 years as data period]
Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 1 (Side Effects) [Срок оценки: Up to 6 years as data period]

Вторичные конечные точки (4)

Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events) [Срок оценки: Up to 6 years as data period]
Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events) [Срок оценки: Up to 6 years as data period]
Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 2 (Adverse Events) [Срок оценки: Up to 6 years as data period]
Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 2 (Adverse Events) [Срок оценки: Up to 6 years as data period]

Критерии участия

Критерии включения

Has a diagnosis of ALGS or PFIC within the enrollment period.
There is a prescription for Livmarli during the enrollment period (Index date: the date of the first prescription within the enrollment period).