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Recruiting
NCT07290257
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
Conditions: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Sex: All
Ages: 2 Months – N/A
Healthy volunteers: No
Phase: PHASE4
Enrollment: 230
Sponsor: Mirum Pharmaceuticals, Inc.
Location: Cliniques Universitaires Saint Luc (UCLouvain) Brussels
Summary
In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.
Eligibility Criteria
Inclusion Criteria:
* Understand and execute an Informed consent and assent (as applicable)
* For participants with ALGS ≥2 months of age at Day 1
* For participants with PFIC, ≥3 months of age at Day 1
* A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis
* For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
* For the ALGS supplemental cohort, : Actively using Livmarli prior to study entry
* For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry
Exclusion Criteria:
* History of Liver Transplant
* Any Livmarli contraindications (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.)
* Received another IBAT inhibitor within 7 days before the first dose of Livmarli
* Baseline data before start of treatment of Livmarli are unavailable (\
Source: ClinicalTrials.gov (NCT07290257). StuddyBuddy aggregates publicly available trial information.