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
Location: See all (2) locations...
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enrol the participant in this registry) by their treating physician

• On (or starting) active odevixibat treatment Note: Participants can remain in the registry during odevixibat treatment interruptions

Locations
Other Locations
Republic of Korea
Seoul National University Hospital
NOT_YET_RECRUITING
Seoul
Severance Hospital, Yonsei University Health System
RECRUITING
Seoul
Contact Information
Primary
Ipsen Clinical Study Enquiries
clinical.trials@ipsen.com
See e mail
Time Frame
Start Date: 2025-10-30
Estimated Completion Date: 2032-09-30
Participants
Target number of participants: 10
Sponsors
Leads: Ipsen

This content was sourced from clinicaltrials.gov