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NCT Number: 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

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.

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Key information

Conditions

Progressive Familial Intrahepatic Cholestasis Cholestasis, progressive familial intrahepatic 1

Age range

6 month and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Capital Children's Medical Center, Beijing, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. 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.

    An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment

  2. 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.

    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 outcomes

  1. Event-free survival (EFS)

    Time frame: 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.

  2. Surgical biliary diversion-free survival

    Time frame: 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.

  3. Liver transplant-free survival

    Time frame: 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.

  4. Overall survival

    Time frame: 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.

  5. Pruritus improvement

    Time frame: 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.

  6. Change from baseline in serum bile acid

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Ipsen Clinical Study Enquiries

CONTACT

[email protected]

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Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2031
Study completion
2031
First posted
May 15, 2026
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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