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NCT Number: 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

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.

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Key information

Conditions

Progressive Familial Intrahepatic Cholestasis Cholestasis, progressive familial intrahepatic 1

Sex eligibility

All sexes

Study type

Observational

Primary location

Pusan National University Yangsan Hospital, Gyeongsang, South Korea

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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.

Treatment and study plan

Primary outcomes

  1. Percentage of participants experiencing Adverse Events (AEs)

    Time frame: 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 outcomes

  1. Event-free survival (EFS)

    Time frame: 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

  2. Surgical biliary diversion-free survival

    Time frame: 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

  3. Liver transplant-free survival

    Time frame: 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

  4. Overall survival (OS)

    Time frame: 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

Study contacts

Contact information is provided by the study sponsor or research team.

Ipsen Clinical Study Enquiries

CONTACT

[email protected]

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Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2032
Study completion
2032
First posted
Sep 22, 2025
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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