An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia

Last updated: May 27, 2026
Sponsor: Albireo, an Ipsen Company
Overall Status: Active - Enrolling

Phase

3

Condition

Gall Bladder Disorders

Treatment

Odevixibat

Clinical Study ID

NCT05426733
A4250-016
2022-501090-39-00
  • All Genders

Study Summary

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Completion of the 104-week Treatment Period of Study A4250-011

  • Signed informed consent by caregiver

Exclusion

Exclusion Criteria:

  • Patients who were not compliant with study drug treatment or procedures in StudyA4250-011 as per the investigator's discretion

  • Any conditions or abnormalities which, in the opinion of the investigator, maycompromise the safety of the patient, or interfere with the patient participating inor completing the study

  • Known hypersensitivity to any components of odevixibat

  • Patients who are scheduled for a liver transplant or are likely to require a livertransplant in the immediate future based on the investigator's judgment

Study Design

Total Participants: 180
Treatment Group(s): 1
Primary Treatment: Odevixibat
Phase: 3
Study Start date:
July 05, 2022
Estimated Completion Date:
June 30, 2028

Study Description

This is a Phase 3, multi-center, open-label extension study to evaluate the long-term efficacy and safety of odevixibat in patients with Biliary Atresia (BA). Patients who completed treatment in the A4250-011 BOLD study and meet eligibility criteria for Study A4250-016 (BOLD-EXT) can participate. The duration of the treatment period is 104 weeks, followed by a 4-week Safety Follow-up Period. Patients who wish to continue receiving odevixibat after 104 weeks can remain on treatment in the optional extension period (OEP).

Up to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the Middle East and Asia-Pacific.

Connect with a study center

  • Royal Children's Hospital

    Parkville, 3052
    Australia

    Site Not Available

  • CHU Sainte-Justine

    Montreal,
    Canada

    Site Not Available

  • CHU Sainte-Justine

    Montréal,
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto,
    Canada

    Site Not Available

  • Guangzhou Women And Childrens Medical Center

    Guangdong,
    China

    Site Not Available

  • Children's Hospital of Fudan University

    Shanghai,
    China

    Site Not Available

  • Bicêtre Hospital

    Le Kremlin-Bicêtre,
    France

    Site Not Available

  • Charité - Universitätsmedizin Berlin

    Berlin,
    Germany

    Site Not Available

  • University Children´s Hospital Tuebingen

    Tuebingen,
    Germany

    Site Not Available

  • University Children´s Hospital Tuebingen

    Tübingen,
    Germany

    Site Not Available

  • ASST Papa Giovanni XXIII

    Bergamo,
    Italy

    Site Not Available

  • Azienda Ospedaliera di Padova

    Padova,
    Italy

    Site Not Available

  • Hospital Raja Perempuan Zainab II

    Kota Bharu,
    Malaysia

    Site Not Available

  • University Malaya Medical Centre

    Kuala Lumpur, 59100
    Malaysia

    Site Not Available

  • University Medical Center Groningen

    Groningen,
    Netherlands

    Site Not Available

  • Starship Child Health, Te Whatu Ora - Health New Zealand

    Auckland, 1023
    New Zealand

    Site Not Available

  • Instytut Pomnik-Centrum Zdrowia Dziecka

    Warsaw,
    Poland

    Site Not Available

  • Seoul National University Childrens Hospital

    Seoul,
    South Korea

    Site Not Available

  • Hospital Universitari Vall d'Hebron

    Barcelona,
    Spain

    Site Not Available

  • Hacettepe İhsan Doğramacı Children's Hospital

    Ankara,
    Turkey (Türkiye)

    Site Not Available

  • Akdeniz University Medical Faculty

    Antalya,
    Turkey (Türkiye)

    Site Not Available

  • Istanbul University Istanbul Medical Faculty Hospital

    Istanbul,
    Turkey (Türkiye)

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Children's Healhcare of Atlanta- Emory University school of medicine

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Indiana University school of Medicine

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Children's Mercy Hospital and Clinics

    Kansas City, Missouri 64108
    United States

    Site Not Available

  • The Childrens Hospital at Montefiore Albert Einstein School of Medicine

    Bronx, New York 10467
    United States

    Site Not Available

  • Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital

    New York, New York 10032
    United States

    Site Not Available

  • Hassenfeld Children's Hospital at NYU Langone

    New York, New York 10016
    United States

    Site Not Available

  • The Childrens Hospital at Montefiore Albert Einstein School of Medicine

    The Bronx, New York 10467
    United States

    Site Not Available

  • Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital

    New York 5128581, New York 5128638 10032
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania 15224
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Site Not Available

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