Progressive Familial Intrahepatic Cholestasis
Conditions
Brief summary
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.
Interventions
None listed
Sponsors
Ipsen
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Months to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician * On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions) * Signed informed consent and assent, as appropriate
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 China
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants experiencing adverse events (AEs) | 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. | An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment |
| Percentage of participants experiencing serious adverse events (SAEs) | 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. | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Event-free survival (EFS) | From first ICF signature and up to end of data collection (approximately 5 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 5 years of data collection) | 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 5 years of data collection) | Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death. |
| Overall survival | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | Defined as time from the start of odevixibat treatment to death. |
| Pruritus improvement | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | 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. |
| Change from baseline in serum bile acid | From baseline and up to end of data collection (approximately 5 years of data collection | Change from baseline assessed by measuring serum bile acid levels at each patient visit. |
Countries
China
Contacts
CONTACTIpsen Clinical Study Enquiries
STUDY_DIRECTORIpsen Medical Director
Ipsen
Outcome results
None listed