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
- Conditions
- Progressive Familial Intrahepatic Cholestasis
- Registration Number
- NCT07185919
- Lead Sponsor
- Ipsen
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 10
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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