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

NCT07588880 | Recruiting

• 1 other identifier: CLIN-60240-032
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Progressive Familial Intrahepatic Cholestasis

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (6)

• Event-free survival (EFS)

  From first ICF signature and up to end of data collection (approximately 5 years of data collection

• Surgical biliary diversion-free survival

  From first ICF signature and up to end of data collection (approximately 5 years of data collection)

• Liver transplant-free survival

  From first ICF signature and up to end of data collection (approximately 5 years of data collection)

• Overall survival

  From first ICF signature and up to end of data collection (approximately 5 years of data collection)

• Pruritus improvement

  From first ICF signature and up to end of data collection (approximately 5 years of data collection)

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Children's Hospital of Fudan University Endocrinology and Metabolism

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Cholestasis, progressive familial intrahepatic 1

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Central Study Contacts

Ipsen Clinical Study Enquiries

CONTACT

See e-mail; Clinical.trials@ipsen.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2026
• First Posted: May 15, 2026
• Study Start: April 16, 2026
• Primary Completion (Estimated): April 30, 2031
• Study Completion (Estimated): April 30, 2031
• Last Updated: May 15, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• 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
• 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/).

Locations

CN (1)