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Recruiting NCT07588880

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

— Observational Progressive Familial Intrahepatic Cholestasis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Progressive Familial Intrahepatic Cholestasis. Basic parameters: from 6 months · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Primary outcome measures

  • Percentage of participants experiencing adverse events (AEs) [Time frame: 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) [Time frame: 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 (6)
  • Event-free survival (EFS) [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection]
  • Surgical biliary diversion-free survival [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Liver transplant-free survival [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Overall survival [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Pruritus improvement [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Change from baseline in serum bile acid [Time frame: From baseline and up to end of data collection (approximately 5 years of data collection]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

China · 5 centers
  • Capital Children's Medical Center — Beijing
  • Hunan Children's Hospital — Hunan
  • Nanjing Medical University-Nanjing Children's Hospital — Nanjing
  • Children's Hospital of Fudan University Endocrinology and Metabolism — Shanghai
  • Xiamen Children's Hospital — Xiamen

Identifiers

NCT: NCT07588880 · CLIN-60240-032

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗