Long-term Registry Study of Odevixibat Bylvay Treatment in People with Progressive Familial Intrahepatic Cholestasis PFIC in South Korea

What this Trial Is About

This observational study is focused on people with Progressive Familial Intrahepatic Cholestasis PFIC, a rare liver disease that causes impaired liver function, itching, and yellowing of the skin. The study aims to collect long-term information on the safety and effectiveness of odevixibat, a medicine approved to help manage PFIC symptoms in South Korea and other regions. Researchers want to understand how odevixibat works in everyday use for patients with PFIC. Participants in this study will be people diagnosed with any type of PFIC who are prescribed odevixibat. The study will observe participants who are currently taking odevixibat or starting treatment, including those who may have treatment interruptions. The study will collect data over approximately seven years to monitor long-term outcomes and safety, without assigning specific treatments as it is observational. During the study, participants will be followed to record any adverse events and track outcomes such as survival without events, need for surgery, liver transplant, or overall survival. Information will be collected from the time participants agree to join the registry and continue throughout the study period. The study is designed to provide detailed safety and effectiveness data over a long-term period while participants continue their usual care.

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea