Maralixibat (Livmarli) use in Alagille syndrome and PFIC — Japanese database analysis
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 project will look at Japanese patients with Alagille syndrome or PFIC who received maralixibat (Livmarli) to see if taking the drug is linked to liver problems.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Sex | All |
| Sponsor | Takeda Industry-sponsored |
| Locations | 1 site (Tokyo, Tokyo) |
| Trial ID | NCT07293897 on ClinicalTrials.gov |
What this trial studies
This observational database analysis uses the Comprehensive and Informative Registry system for Childhood Liver Disease (CIRCLe) in Japan to identify patients diagnosed with ALGS or PFIC who were prescribed maralixibat during the enrollment period. The index date is the first prescription of Livmarli, and existing medical records will be reviewed to document occurrences of liver disorders during treatment. No exclusion criteria are specified and the work involves retrospective data analysis rather than new interventions or visits. The project is sponsored by Takeda and aims to estimate the risk profile of maralixibat in a Japanese patient cohort.
Who should consider this trial
Good fit: Ideal candidates are Japanese patients with a recorded diagnosis of ALGS or PFIC who have at least one prescription for Livmarli during the enrollment window.
Not a fit: Patients without a diagnosis of ALGS or PFIC, those who never received Livmarli, or individuals whose care is not captured in the CIRCLe database would not be included and are unlikely to benefit directly.
Why it matters
Potential benefit: If successful, the study could clarify the risk of liver disorders with maralixibat in Japanese patients and help doctors make safer prescribing decisions.
How similar studies have performed: Clinical trials of maralixibat and other IBAT inhibitors have shown improvements in symptoms like pruritus and bile acid levels in pediatric cholestatic liver disease, but targeted database safety analyses in Japanese ALGS/PFIC patients are relatively limited.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Tokyo, Tokyo
- Takeda selected site — Tokyo, Tokyo, Japan (Recruiting)
Study contacts
- Study coordinator: Takeda Contact
- Email: medinfoUS@takeda.com
- Phone: +1-877-825-3327
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.