RecruitingNot Applicable

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

Japan50 participantsStarted 2026-01-13

Plain-language 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).

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: Up to 6 years as data period

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

Timeframe: Up to 6 years as data period

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

Timeframe: Up to 6 years as data period

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

Timeframe: Up to 6 years as data period

Trial details

NCT IDNCT07293897

SponsorTakeda

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2031-06-30

Contact for this trial

Takeda Contact

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

View on ClinicalTrials.gov More Alagille Syndrome (ALGS) trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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

Timeframe: Up to 6 years as data period

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

Timeframe: Up to 6 years as data period

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

Timeframe: Up to 6 years as data period

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

Timeframe: Up to 6 years as data period