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

February 18, 2026 updated by: Takeda

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)

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Japan
    • Tokyo
      • Tokyo, Tokyo, Japan
        • Recruiting
        • Takeda Selected Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Alagille Syndrome (ALGS) Group
Participants with Alagille Syndrome (ALGS) who received maralixibat in accordance with package insert.
Drug: Maralixibat
Maralixibat Oral Solution 10 milligrams (mg)/milliliters (mL)
Other Names:
  • TAK-625
  • Livmarli Oral Solution
Progressive Familial Intrahepatic Cholestasis (PFIC) Group
Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received maralixibat in accordance with package insert.
Drug: Maralixibat
Maralixibat Oral Solution 10 milligrams (mg)/milliliters (mL)
Other Names:
  • TAK-625
  • Livmarli Oral Solution

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects)
Time Frame: 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.
Up to 6 years as data period
Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects)
Time Frame: 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.
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 Frame: 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.
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 Frame: 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.
Up to 6 years as data period

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events)
Time Frame: Up to 6 years as data period
Percentage of participants with ALGS who will experience liver disorder will be reported. Outcome Definition 2 (Adverse Events) will be used to estimate this outcome measure. Outcome Definition 2 (Adverse Events) 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.
Up to 6 years as data period
Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events)
Time Frame: Up to 6 years as data period
Percentage of participants with PFIC who will experience liver disorder will be reported. Outcome Definition 2 (Adverse Events) will be used to estimate this outcome measure. Outcome Definition 2 (Adverse Events) 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.
Up to 6 years as data period
Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 2 (Adverse Events)
Time Frame: Up to 6 years as data period
Time to onset of liver disorder in participants with ALGS will be reported. Outcome Definition 2 (adverse events) will be used to estimate this outcome measure. Outcome Definition 2 (adverse events) 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.
Up to 6 years as data period
Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 2 (Adverse Events)
Time Frame: Up to 6 years as data period
Time to onset of liver disorder in participants with PFIC will be reported. Outcome Definition 2 (adverse events) will be used to estimate this outcome measure. Outcome Definition 2 (adverse events) 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.
Up to 6 years as data period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Takeda

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Study Director, Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 13, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 30, 2031

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 30, 2031

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 17, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 17, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 19, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 20, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 18, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • TAK-625-4002
  • jRCT2031250646 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

IPD Sharing 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.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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