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Clinical Trials in Japan / NCT07293897
Recruiting Observational

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

NCT07293897 · tracked via the Priya Life Science Japan tracker
Sponsor
Takeda
Phase
Observational
Started
2026-01-13
Last updated
2026-02-20

Condition(s) studied

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

Investigational drug(s) / intervention(s)

Maralixibat →

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

Study 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).

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects) — Up to 6 years as data period
    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.
  • Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects) — Up to 6 years as data period
    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.
  • Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 1 (Side Effects) — Up to 6 years as data period
    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.
  • Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 1 (Side Effects) — Up to 6 years as data period
    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.

Trial sites (1)

FacilityCityRegionStatus
Takeda selected site Tokyo Tokyo Recruiting

More Takeda trials in Japan

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A Study of TAK-360 in People With Narcolepsy or Idiopathic Hypersomnia Recruiting
Survey of Brigatinib Used To Treat People With Non-Small Cell Lung Cancer Recruiting
A Study of TAK-279 in Participants With Moderate-to-Severe Plaque Psoriasis Active, not recruiting
Continuation Study of Zasocitinib in Adults With Psoriatic Arthritis Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07293897 on ClinicalTrials.gov ↗ ← All trials in Japan