A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Maralixibat.
- Who it may be relevant to
- Registry conditions: Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC). Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Japan
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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)
Overview
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).
Interventions
- Drug Maralixibat
Maralixibat Oral Solution 10 milligrams (mg)/milliliters (mL)
Primary outcome measures
- 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 PFIC who Experience Liver Disorder 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 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 Evaluated with Outcome Definition 1 (Side Effects) [Time frame: Up to 6 years as data period]
Secondary outcome measures (4)
- 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 PFIC who Experience Liver Disorder 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 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 Evaluated with Outcome Definition 2 (Adverse Events) [Time frame: Up to 6 years as data period]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Japan · 1 center
- Takeda selected site — Tokyo
Identifiers
NCT: NCT07293897 · TAK-625-4002 · jRCT2031250646