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.
• 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