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)

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)

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.

This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.

Participants will be treated according to standard of care for the duration of the study.

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 (<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter