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Promising long-term results for rare liver disease drug in kids

NCT ID NCT03659916

Completed Disease control Sponsor: Albireo, an Ipsen Company Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety and effectiveness of a drug called A4250 (odevixibat) in 116 children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease. The children took the drug for up to 72 weeks. The study measured changes in bile acid levels and itching. Results suggest the drug may help control the disease over time, but it is not a cure.

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Conditions

The condition(s) this trial relates to.

benign recurrent intrahepatic cholestasis cholestasis Cholestasis, progressive familial intrahepatic 1 progressive familial intrahepatic cholestasis progressive familial intrahepatic cholestasis type 1 progressive familial intrahepatic cholestasis type 2

As listed by the trial registrant

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

PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS

Contacts and locations

Locations

  • Akdeniz University

    Antalya, Turkey (Türkiye)

  • Astrid Lindgren Children's Hospital, Karolinska University Hospital

    Solna, Sweden

  • Azienda Ospedaliera Papa Giovanni XXIII

    Bergamo, Italy

  • Baylor College of Medicine - Texas Children's Liver Center

    Houston, Texas, 77030, United States

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

    Birmingham, United Kingdom

  • Children's Hospital Colorado

    Denver, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Cliniques Universitaires Saint-Luc

    Woluwe-Saint-Lambert, Belgium

  • Columbia University Medical Center - Presbyterian Hospital Building

    New York, New York, 10032, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30329, United States

  • Gazi University

    Ankara, Turkey (Türkiye)

  • Hacettepe University Faculty of Medicine

    Ankara, Turkey (Türkiye)

  • Hospital De La Timone

    Marseille, France

  • Hospital Necker-Enfants Maladies

    Paris, France

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Hospital Universitario La Paz

    Madrid, Spain

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Inonu University Medical Faculty

    Malatya, Turkey (Türkiye)

  • Institute of Liver Studies - Kings College Hospital

    London, United Kingdom

  • Instytut Pomnik - Centrum Zarowia Dziecka

    Warsaw, Poland

  • Istanbul University Medical Faculty

    Istanbul, Turkey (Türkiye)

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21287, United States

  • Kinderklinik Tubingen, Universitatsklinikum Tubingen

    Tübingen, Germany

  • King Faisal Specialist Hospital & Research Centre

    Riyadh, Saudi Arabia

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Ospedale Regina Margherita

    Torino, Italy

  • Riley Hospital for Children - Riley Children's Specialists

    Indianapolis, Indiana, 46202, United States

  • Schneider Children's Medical Center Of Israel

    Petah Tikva, Israel

  • Shaare-Zedek Mc

    Jerusalem, Israel

  • The Hospital for Sick Children

    Toronto, Canada

  • The Royal Children's Hospital

    Melbourne, Australia

  • Universitair Medisch Centrum (UMC) Utrecht

    Utrecht, Netherlands

  • Universite Paris SUD - Hpitaux Universitaires Paris-Sud - Hopital Bicetre

    Le Kremlin-Bicêtre, France

  • University Hospital Of Padova

    Padova, Italy

  • University Medical Center Groningen

    Groningen, Netherlands

  • University and Pediatric Hospital of Lyon

    Bron, France

  • Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin

    Tübingen, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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