Extended trial offers hope for kids battling rare liver disease

NCT ID NCT03659916

Summary

This study followed children with a rare, inherited liver disease called PFIC to see if a drug called odevixibat (A4250) remained safe and effective over a long period. The 116 participants, who had previously been in a shorter trial, took the drug for up to 72 weeks. Researchers measured its effects on severe itching and liver health markers to understand its long-term benefits.

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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

    Padua, 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

Conditions

Explore the condition pages connected to this study.