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

Takeda

Status: Not yet enrolling

Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC); Alagille Syndrome (ALGS)

Treatments: Drug: Maralixibat

Study type: Observational

Funder types: Industry

Identifiers

NCT07293897

TAK-625-4002

Details and patient eligibility

About

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).

Enrollment: 50 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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).

Exclusion criteria

- None

Trial design

50 participants in 2 patient groups

Alagille Syndrome (ALGS) Group

Description:

Participants with Alagille Syndrome (ALGS) who received maralixibat in accordance with package insert.

Treatment:

Drug: Maralixibat

Progressive Familial Intrahepatic Cholestasis (PFIC) Group

Description:

Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received maralixibat in accordance with package insert.

Treatment:

Drug: Maralixibat