New hope for kids with rare liver disease: long-term drug study launched

NCT ID NCT05035030

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This study looks at the long-term safety and effectiveness of odevixibat in 70 people with Alagille syndrome, a rare genetic condition that affects the liver and other organs. The drug aims to reduce severe itching and improve bile flow. Participants who completed a prior 24-week study can join this extension to see how well the treatment works over time.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • AOU Meyer

    Florence, Italy

  • Antenne pediatrique du CIC-Hopital Jeanne De Flandre

    Lille, France

  • Atrium Health Carolinas Medical

    Durham, North Carolina, 27710, United States

  • Azienda Ospedale University

    Padova, 35128, Italy

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, United Kingdom

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353, Germany

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329, United States

  • Children's Mercy Hospital and Clinics

    Kansas City, Missouri, 64018, United States

  • Childrens Medical Center of Dallas University of Texas Southwestern

    Dallas, Texas, 75207, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cliniques Universitaires Saint-Luc Bruxelles

    Brussels, 1200, Belgium

  • Hassenfeld Children's Hospital at NYU Langone

    New York, New York, 10016, United States

  • Hopital Necker Enfants Malades

    Paris, 75015, France

  • Hôpital Femme Mère Enfant de Lyon

    Bron, 69677, France

  • Instytut Pomnik-Centrum Zdrowia Dzieck

    Warsaw, 04-730, Poland

  • Istanbul University Istanbul Medical Faculty Hospital

    Istanbul, Turkey (Türkiye)

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287, United States

  • King's College Hospital NHS Foundation Trust King's College Hospital Paediatric Research

    London, SE5 9RS, United Kingdom

  • Medizinische Hochschul

    Hanover, 30625, Germany

  • Monroe Carell Jr. Childrens Hospital at Vanderbilt

    Nashville, Tennessee, 37232, United States

  • New York-Presbyterian / Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Northwell Health System

    New Hyde Park, New York, 11042, United States

  • Oregon Health Science University School of Medicine

    Portland, Oregon, 97239, United States

  • Ospedale Pediatrico Bambino Gesu

    Rome, 00165, Italy

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Riley Hospital for Children at IU Health

    Indianapolis, Indiana, 46202, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • Texas Liver Institute

    San Antonio, Texas, 78215, United States

  • The Childrens Hospital at Montefiore Albert Einstein School of Medicine

    The Bronx, New York, 10467, United States

  • UCSF

    San Francisco, California, 94158, United States

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ, Netherlands

  • Universitatsklinik fur Kinder-und Jugendmedizin Tubingen

    Tübingen, 72076, Germany

  • University of Malaya Medical Center

    Kuala Lumpur, 59100, Malaysia

  • Wilhelmina Children's Hospital UMCU Utrecht

    Utrecht, Netherlands

More trials for these conditions

Other studies related to the condition(s) this trial covers.