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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07185919

Enrollment

Registered

2025-09-22

Start date

2025-10-30

Completion date

2032-09-30

Last updated

2026-08-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Progressive Familial Intrahepatic Cholestasis

Brief 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.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

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

Exclusion criteria

1. Currently participating in a clinical trial with odevixibat 2. Currently participating in any interventional clinical trial for PFIC 3. Have any contraindication to odevixibat as per the approved label in South Korea.

Design outcomes

Primary

Measure	Time frame	Description
Percentage of participants experiencing Adverse Events (AEs)	From first ICF signature and up to end of data collection (approximately 7 years of data collection)	An Adverse event (AE) is any untoward medical occurrence, temporally associated with the use of study intervention, whether or not related to the study intervention.

Secondary

Measure	Time frame	Description
Event-free survival (EFS)	From first ICF signature and up to end of data collection (approximately 7 years of data collection)	EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death
Surgical biliary diversion-free survival	From first ICF signature and up to end of data collection (approximately 7 years of data collection)	Surgical biliary diversion-free survival is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death
Liver transplant-free survival	From first ICF signature and up to end of data collection (approximately 7 years of data collection)	Liver transplant-free survival is defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death
Overall survival (OS)	From first ICF signature and up to end of data collection (approximately 7 years of data collection)	OS is defined as the time from the start of odevixibat treatment to death

Countries

South Korea

Contacts

CONTACTIpsen Clinical Study Enquiries

clinical.trials@ipsen.comSee e mail

STUDY_DIRECTORIpsen Medical Director

Ipsen

Outcome results