New hope for kids with rare liver disease: drug may delay transplant

NCT ID NCT05426733

First seen Nov 01, 2025 · Last updated May 09, 2026 · Updated 30 times

Summary

This study tests the long-term safety and effectiveness of odevixibat in children with biliary atresia, a rare liver disease. The main goal is to see if the drug helps children stay alive without needing a liver transplant. About 180 children who completed a previous study will take odevixibat and be monitored for liver health and side effects.

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Contacts and locations

Locations

  • ASST Papa Giovanni XXIII

    Bergamo, Italy

  • Akdeniz University Medical Faculty

    Antalya, Turkey (Türkiye)

  • Azienda Ospedaliera di Padova

    Padova, Italy

  • Bicêtre Hospital

    Le Kremlin-Bicêtre, France

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHU Sainte-Justine

    Montreal, Canada

  • Charité - Universitätsmedizin Berlin

    Berlin, Germany

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

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Fudan University

    Shanghai, China

  • Children's Mercy Hospital and Clinics

    Kansas City, Missouri, 64108, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

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

    New York, New York, 10032, United States

  • Guangzhou Women And Childrens Medical Center

    Guangdong, China

  • Hacettepe İhsan Doğramacı Children's Hospital

    Ankara, Turkey (Türkiye)

  • Hassenfeld Children's Hospital at NYU Langone

    New York, New York, 10016, United States

  • Hospital Raja Perempuan Zainab II

    Kota Bharu, Malaysia

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Indiana University school of Medicine

    Indianapolis, Indiana, 46202, United States

  • Instytut Pomnik-Centrum Zdrowia Dziecka

    Warsaw, Poland

  • Istanbul University Istanbul Medical Faculty Hospital

    Istanbul, Turkey (Türkiye)

  • Royal Children's Hospital

    Parkville, 3052, Australia

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Seoul National University Childrens Hospital

    Seoul, South Korea

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

    Auckland, 1023, New Zealand

  • The Childrens Hospital at Montefiore Albert Einstein School of Medicine

    The Bronx, New York, 10467, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • University Children´s Hospital Tuebingen

    Tübingen, Germany

  • University Malaya Medical Centre

    Kuala Lumpur, 59100, Malaysia

  • University Medical Center Groningen

    Groningen, Netherlands

Conditions

Explore the condition pages connected to this study.