RecruitingPhase 4NCT07290257
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
Sponsor
Mirum Pharmaceuticals, Inc.
Enrollment
100 participants
Start Date
Sep 25, 2025
Study Type
INTERVENTIONAL
Conditions
Summary
The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.
Eligibility
Min Age: 2 Months
Inclusion Criteria5
- Informed consent and assent (as applicable)
- ≥2 months of age at Day 1
- A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis
- For the primary cohort, prescribed Livmarli at time of study entry
- For the supplemental cohort, prescribed Livmarli prior to study entry
Exclusion Criteria5
- History of Liver Transplant
- Any contraindications against Livmarli (as per SmPC)
- Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
- Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)
- Baseline data before start of treatment of Livmarli are unavailable (\<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter
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Interventions
DRUGLivmarli Oral Product
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Locations(13)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07290257
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