Long-Term Safety and Clinical Outcomes Study of Livmarli in Children and Adults with Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in Europe

What this Trial Is About

Researchers are evaluating the long-term safety, tolerability, and effectiveness of Livmarli in patients with Alagille Syndrome ALGS and Progressive Familial Intrahepatic Cholestasis PFIC. The study focuses on monitoring potential liver toxicity and the impact of chronic exposure to propylene glycol in PFIC patients, as well as assessing growth and development over time. This is a phase 4, open-label, low-intervention clinical study conducted across multiple centers in the European Union. Participants diagnosed with ALGS or PFIC will be treated with Livmarli according to standard care throughout the study. The study includes a primary cohort of at least 45 previously untreated ALGS patients and supplemental cohorts of ALGS patients already on Livmarli, along with at least 130 PFIC patients receiving Livmarli. Treatment will continue for the study duration with no placebo group, and patient care follows usual clinical practice. During the study, participants will undergo regular assessments every 3 to 4 months for up to 5 years. These assessments include monitoring adverse events, liver function tests, fat-soluble vitamin levels, international normalized ratio, clinical outcomes, and propylene glycol toxicity for PFIC patients. The study aims to collect long-term data on safety and clinical outcomes while patients remain on Livmarli treatment, with participation potentially lasting several years.

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)