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
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Progressive Familial Intrahepatic Cholestasis. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- South Korea
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay)
Overview
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.
Primary outcome measures
- 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)]
Secondary outcome measures (4)
- Event-free survival (EFS) [Time frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]
- Surgical biliary diversion-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 [Time frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]
- Overall survival (OS) [Time frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
South Korea · 3 centers
- Pusan National University Yangsan Hospital — Gyeongsang
- Seoul National University Hospital — Seoul
- Severance Hospital, Yonsei University Health System — Seoul
Identifiers
NCT: NCT07185919 · CLIN-60240-030