Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Event-free survival (EFS) | EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death | From first ICF signature and up to end of data collection (approximately 7 years of data collection) |
| Surgical biliary diversion-free survival |
Not provided
Inclusion criteria:
Exclusion criteria:
Not provided
Not provided
Not provided
Not provided
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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ipsen Clinical Study Enquiries | Contact | See e mail | clinical.trials@ipsen.com |
| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pusan National University Yangsan Hospital | Recruiting | Gyeongsang | South Korea | |||
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
Not provided
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.
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Not provided
Not provided
Not provided
Not provided
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 |
| From first ICF signature and up to end of data collection (approximately 7 years of data collection) |
| Liver transplant-free survival | Liver transplant-free survival is defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death | From first ICF signature and up to end of data collection (approximately 7 years of data collection) |
| Overall survival (OS) | OS is defined as the time from the start of odevixibat treatment to death | From first ICF signature and up to end of data collection (approximately 7 years of data collection) |
| Seoul National University Hospital |
| Active, not recruiting |
| Seoul |
| South Korea |
| Severance Hospital, Yonsei University Health System | Recruiting | Seoul | South Korea |
| ID | Term |
|---|---|
| C535933 | Cholestasis, progressive familial intrahepatic 1 |
Not provided
Not provided
Not provided