NCT07185919

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

Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 10 (estimated)
• Sponsor: Ipsen · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives. Odevixibat is a medicine that helps people with PFIC, a type of rare disease that makes their liver not work well and causes itching and yellow skin. Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Obevixibat was approved by the Ministry of Food and Drug Safety (MFDS) in South Korea on 23 August 2024. This study will collect information to see how well and how safe odevixibat is in the long run for participants in South Korea.

Conditions

• Progressive Familial Intrahepatic Cholestasis

Timeline

• Start date: 2025-10-30
• Primary completion: 2032-09-30
• Completion: 2032-09-30
• First posted: 2025-09-22
• Last updated: 2026-04-01

Locations

2 sites across 1 country: South Korea