Observing Long-Term Safety and Effectiveness of Odevixibat Bylvay in Patients With Alagille Syndrome Receiving Ongoing Treatment

What this Trial Is About

This research aims to observe patients with Alagille syndrome ALGS, a rare liver disease causing itching, as they use the medicine odevixibat Bylvay in their daily lives. The study focuses on evaluating the long-term effectiveness and safety of odevixibat in patients who are already receiving this treatment. It seeks to understand how well the medicine performs over time and monitor important health outcomes related to ALGS. Participants in this study are those diagnosed with ALGS who are either currently on odevixibat treatment or starting it. The study collects data over approximately five years, tracking outcomes such as the need for biliary diversion surgery or liver transplantation, survival without these interventions, and overall survival. Researchers will also monitor changes in weight and height, as well as any adverse events linked to vitamin deficiencies, liver problems, bleeding, or other side effects related to the medicine. During the study, participants will have their health status and treatment effects recorded regularly. This includes monitoring surgery-free and transplant-free survival rates alongside overall survival. Safety information covering side effects and vitamin-related issues will be collected from the start of consent through 180 days after the last dose of odevixibat. The study aims to gather comprehensive, long-term data on how patients fare while using this medication in real-world settings.

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment