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.

NCT Number	NCT07290257
Lead Sponsor	Mirum Pharmaceuticals, Inc.
Conditions	Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Enrollment	230 participants
Start Date	2025-09-25
Primary Completion	2030-09-22 (estimated)
Study Completion	2030-12-22 (estimated)
Updated on ClinicalTrials.gov	2026-06-23