Long-Term Safety and Outcomes of Livmarli Treatment in Patients with Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the United States

What this Trial Is About

This research aims to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome ALGS or Progressive familial intrahepatic cholestasis PFIC who are treated with Livmarli. The study is a 5-year, prospective, observational cohort trial designed to monitor these patients in the United States. Livmarli is a novel drug that reduces bile acid levels by inhibiting the ileal bile acid transporter and has been approved for treating cholestatic pruritus in ALGS and PFIC patients of certain ages. Participants prescribed Livmarli and diagnosed with ALGS or PFIC will be observed over five years. Two dosing schedules are noted 380 mcgkg once daily and 570 mcgkg twice daily. The study includes regular assessments of liver transplant waitlist status every six months and annual evaluations of growth and development. The treatment is given as prescribed by healthcare providers, and the study collects data without altering the prescribed therapy. During the study, participants will have their long-term clinical outcomes monitored, including events up to 180 days after stopping Livmarli. Data on liver transplant indications, growth measurements like weight and height z-scores, and events related to fat-soluble vitamin deficiencies will be collected at specified intervals. The trial aims to provide comprehensive safety and outcome information over the full 5-year period.

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)