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

NCT07293897 | Recruiting

• 2 other identifiers: TAK-625-4002jRCT2031250646
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

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

Keywords

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

Outcome Measures

Primary Outcomes (4)

• Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects)

  Percentage of participants with ALGS who will experience liver disorder will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment.

  Up to 6 years as data period

• Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects)

  Percentage of participants with PFIC who will experience liver disorder will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment.

  Up to 6 years as data period

• Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 1 (Side Effects)

  Time to onset of liver disorder in participants with ALGS will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment.

  Up to 6 years as data period

• Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 1 (Side Effects)

  Time to onset of liver disorder in participants with PFIC will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment.

  Up to 6 years as data period

Secondary Outcomes (4)

• Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events)

  Up to 6 years as data period

• Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events)

  Up to 6 years as data period

• Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 2 (Adverse Events)

  Up to 6 years as data period

• Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 2 (Adverse Events)

  Up to 6 years as data period

Study Arms (2)

Alagille Syndrome (ALGS) Group

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

Drug: Maralixibat

Progressive Familial Intrahepatic Cholestasis (PFIC) Group

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

Drug: Maralixibat

Interventions

Maralixibat DRUG

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

Also known as: TAK-625, Livmarli Oral Solution

Alagille Syndrome (ALGS) Group; Progressive Familial Intrahepatic Cholestasis (PFIC) Group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The population of this study are all participants who meet the inclusion/exclusion criteria.

You may qualify if:

• 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).

You may not qualify if:

• \- None

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

Takeda selected site

Tokyo, Tokyo, Japan

RECRUITING

Related Links

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MeSH Terms

Conditions

Alagille Syndrome; Cholestasis, progressive familial intrahepatic 1

Interventions

maralixibat

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2025
• First Posted: December 19, 2025
• Study Start: January 13, 2026
• Primary Completion (Estimated): June 30, 2031
• Study Completion (Estimated): June 30, 2031
• Last Updated: February 20, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

JP (1)