A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China

Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).

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.

Study Overview

• Status: Recruiting
• Conditions: Progressive Familial Intrahepatic Cholestasis

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Ipsen Clinical Study Enquiries; Phone Number: See e-mail; Email: clinical.trials@ipsen.com

Study Locations

• China
  • Shanghai, China
    • Recruiting
    • Children's Hospital of Fudan University Endocrinology and Metabolism

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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.

Secondary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Ipsen
• Investigators: Study Director: Ipsen Medical Director, Ipsen

Study record dates

Study Major Dates

• Study Start (Actual): April 16, 2026
• Primary Completion (Estimated): April 30, 2031
• Study Completion (Estimated): April 30, 2031

Study Registration Dates

• First Submitted: May 8, 2026
• First Submitted That Met QC Criteria: May 14, 2026
• First Posted (Actual): May 15, 2026

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 14, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cholestasis, progressive familial intrahepatic 1
• Other Study ID Numbers: CLIN-60240-032

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

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.

• IPD Sharing Time Frame: 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
• IPD Sharing Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No