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This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.
PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus).
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. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC.
The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of participants experiencing adverse events (AEs) | An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment | From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first. |
| Percentage of participants experiencing serious adverse events (SAEs) | SAEs are collected as part of safety reporting and include events meeting seriousness criteria (e.g., death, life-threatening, hospitalization, etc.) as defined in the protocol | From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first. |
| 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 5 years of data collection |
| Surgical biliary diversion-free survival |
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Inclusion Criteria:
Exclusion Criteria:
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Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) (all types) who have been prescribed odevixibat by their treating physician will be eligible. Participants who started odevixibat treatment before the implementation of the registry may also be enrolled.
| 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 |
|---|---|---|---|---|---|---|
| Capital Children's Medical Center | Active, not recruiting | Beijing | China | |||
| Hunan Children's Hospital |
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.
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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/).
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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 5 years of data collection) |
| Liver transplant-free survival | 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 5 years of data collection) |
| Overall survival | Defined as time from the start of odevixibat treatment to death. | From first ICF signature and up to end of data collection (approximately 5 years of data collection) |
| Pruritus improvement | Pruritus improvement described at each patient visit using a (semi-)objective scoring scale to assess level of pruritus from the start of the odevixibat treatment. | From first ICF signature and up to end of data collection (approximately 5 years of data collection) |
| Change from baseline in serum bile acid | Change from baseline assessed by measuring serum bile acid levels at each patient visit. | From baseline and up to end of data collection (approximately 5 years of data collection |
| Recruiting |
| Hunan |
| China |
| Children's Hospital of Fudan University Endocrinology and Metabolism | Recruiting | Shanghai | China |
| ID | Term |
|---|---|
| C535933 | Cholestasis, progressive familial intrahepatic 1 |
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