Progressive Familial Intrahepatic Cholestasis (PFIC)

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).

This is a cross-sectional, multicenter tissue study with an exploratory aim to estimate the prevalence of genetic mutations that predispose individuals to diseases in the context of cholestatic disorders and hepatobiliary neoplasms. It is intended as a hypothesis-generating study for future empirical investigations.