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

Recruiting now

Conditions studied: Progressive Familial Intrahepatic Cholestasis

In brief

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.

Key facts

Study ID

NCT07588880

Run by

Ipsen

People needed

20

Starts

2026-04-16

Expected to finish

2031-04-30

Last updated by the study team

2026-08-03

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

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

Where it is running

• Hunan Children's Hospital — Hunan, China (enrolling)
• Children's Hospital of Fudan University Endocrinology and Metabolism — Shanghai, China (enrolling)
• Capital Children's Medical Center — Beijing, China
• Nanjing Medical University-Nanjing Children's Hospital — Nanjing, China
• Xiamen Children's Hospital — Xiamen, China

Full record on ClinicalTrials.gov

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