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

NCT07185919 | Recruiting

• 1 other identifier: CLIN-60240-030
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 3

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.

Conditions

Progressive Familial Intrahepatic Cholestasis

Outcome Measures

Primary Outcomes (1)

• Percentage of participants experiencing Adverse Events (AEs)

  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.

  From first ICF signature and up to end of data collection (approximately 7 years of data collection)

Secondary Outcomes (4)

• Event-free survival (EFS)

  From first ICF signature and up to end of data collection (approximately 7 years of data collection)

• Surgical biliary diversion-free survival

  From first ICF signature and up to end of data collection (approximately 7 years of data collection)

• Liver transplant-free survival

  From first ICF signature and up to end of data collection (approximately 7 years of data collection)

• Overall survival (OS)

  From first ICF signature and up to end of data collection (approximately 7 years of data collection)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The registry population will comprise participants with PFIC (all types) enrolled into the Ipsen odevixibat PFIC registry. Participants with PFIC who have been prescribed odevixibat by their treating physician will be eligible.

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Ipsen: lead

Study Sites (3)

Pusan National University Yangsan Hospital

Gyeongsang, South Korea

ACTIVE NOT RECRUITING

Seoul National University Hospital

Seoul, South Korea

NOT YET RECRUITING

Severance Hospital, Yonsei University Health System

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Cholestasis, progressive familial intrahepatic 1

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Central Study Contacts

Ipsen Clinical Study Enquiries

CONTACT

See e mail; clinical.trials@ipsen.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Target Duration: 7 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2025
• First Posted: September 22, 2025
• Study Start: October 30, 2025
• Primary Completion (Estimated): September 30, 2032
• Study Completion (Estimated): September 30, 2032
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
• Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Locations

KR (3)