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NCT Number: NCT07585097

A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.

The main aim of this study is to observe the long-term, everyday 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

Age range

6 month and older

Sex eligibility

All sexes

Study type

Observational

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.
  • Aged 6 months or older at the time of consent.

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.

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)

    An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to 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)

    An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.

Secondary outcomes

  1. Percentage of participants with severe diarrhoea events

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

  2. Percentage of participants with bloody diarrhoea events

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

  3. Percentage of participants experiencing diarrhoea events with concurrent dehydration

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

  4. Percentage of participants experiencing diarrhoea events treated with oral or intravenous rehydration

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

  5. Change from baseline in fat-soluble vitamin (FSV) levels

    Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)

  6. Percentage of participants with fat-soluble vitamin deficiency

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

  7. Percentage of participants with clinical manifestations of fat-soluble vitamin deficiency

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

    For example, bleeding, rickets, or osteopenia.

  8. Percentage of participants with suspected hepatotoxicity requiring interruption of odevixibat treatment

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

  9. Percentage of participants with clinical manifestations related to hepatotoxicity

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

  10. Change from baseline in alanine aminotransferase (ALT)

    Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)

  11. Change from baseline in aspartate aminotransferase (AST)

    Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)

  12. Change from baseline in gamma-glutamyl transferase (GGT)

    Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)

  13. Change from baseline in blood bilirubin

    Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)

  14. Change from baseline in international normalized ratio (INR)

    Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)

  15. Percentage of participants with hospitalisations due to diarrhoea

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

  16. Percentage of participants with hospitalisations due to hepatotoxicity

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

  17. Percentage of participants with hospitalisations due to fat-soluble vitamin deficiency

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

  18. Percentage of participants with treatment discontinuations due to diarrhoea

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

  19. Percentage of participants with treatment discontinuations due to hepatotoxicity

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

  20. Percentage of participants with treatment discontinuations due to fat-soluble vitamin deficiency

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

  21. Percentage of participants with pregnancy and maternal complications

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

  22. Percentage of foetuses, neonates, or infants with adverse effects following exposure to odevixibat during pregnancy and/or lactation

    Time frame: From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)

  23. Percentage of participants with biliary diversion surgery

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

  24. Percentage of participants with liver transplantation

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

  25. Percentage of participants who die from any cause

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

  26. Percentage of participants switching from odevixibat to maralixibat

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

Study contacts

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

Ipsen Clinical Study Enquiries

CONTACT

[email protected]

See e mail

Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Registry information

Official study title

Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)

Important dates

Study start
2026
Primary completion
2031
Study completion
2031
First posted
May 13, 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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