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NCT Number: NCT06850038

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.

Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching.

The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.

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Key information

Conditions

Alagille Syndrome Abnormalities, Multiple Bile Duct Diseases Biliary Tract Diseases Cardiovascular Abnormalities Cardiovascular Diseases Cholestasis Cholestasis, Intrahepatic Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Heart Defects, Congenital Liver Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's Hospital Los Angeles, Los Angeles, California, United States

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 ALGS.
  • On (or starting) active odevixibat treatment.
  • Signed informed consent and assent, as appropriate. Consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves.

Exclusion criteria

  • Currently participating in a clinical trial with odevixibat.
  • Currently participating in any interventional clinical trial for ALGS.
  • Have any contraindication to odevixibat as per the locally approved label.
  • Had liver transplant before enrolment

Treatment and study plan

Primary outcomes

  1. Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.

    Time frame: From first dose to end of study (approximately 5 years data collection)

  2. Surgical biliary diversion-free survival

    Time frame: From first dose to end of study (approximately 5 years 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 dose to end of study (approximately 5 years 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 dose to end of study (approximately 5 years data collection)

    Defined as time from the start of odevixibat treatment to death.

Secondary outcomes

  1. Change from Baseline in Weight

    Time frame: From first dose to end of study (approximately 5 years data collection)

    This endpoint will measure the change in participants' weight from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.

  2. Change from Baseline in Height

    Time frame: From first dose to end of study (approximately 5 years data collection)

    This endpoint will measure the change in participants' height from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.

  3. Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.

    Time frame: From signing of the Informed Consent Form (ICF) to 180 days after the last dose intake of odevixibat

    An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

  4. Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat

    Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat

    An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

  5. Percentage of participants with bleeding AEs

    Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat

    An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

  6. Percentage of participants with AEs

    Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat

    An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

Study contacts

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

Ipsen Recruitment Enquiries

CONTACT

[email protected]

See email

Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Registry information

Official study title

Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
First posted
Feb 27, 2025
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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