New hope for kids with rare liver disease: long-term drug shows promise
NCT ID NCT03659916
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 31 times
Summary
This study looked at the long-term safety and effectiveness of a drug called A4250 (odevixibat) in 116 children with a rare liver disease called progressive familial intrahepatic cholestasis (PFIC). The goal was to see if the drug could lower bile acid levels and reduce itching over many weeks. Children who had already been in a previous study were included, and all received the drug.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Akdeniz University
Antalya, Turkey (Türkiye)
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Astrid Lindgren Children's Hospital, Karolinska University Hospital
Solna, Sweden
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Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
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Baylor College of Medicine - Texas Children's Liver Center
Houston, Texas, 77030, United States
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Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
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Children's Hospital Colorado
Denver, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
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Columbia University Medical Center - Presbyterian Hospital Building
New York, New York, 10032, United States
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Emory University School of Medicine
Atlanta, Georgia, 30329, United States
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Gazi University
Ankara, Turkey (Türkiye)
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Hacettepe University Faculty of Medicine
Ankara, Turkey (Türkiye)
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Hospital De La Timone
Marseille, France
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Hospital Necker-Enfants Maladies
Paris, France
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Hospital Universitari Vall d'Hebron
Barcelona, Spain
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Hospital Universitario La Paz
Madrid, Spain
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Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Inonu University Medical Faculty
Malatya, Turkey (Türkiye)
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Institute of Liver Studies - Kings College Hospital
London, United Kingdom
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Instytut Pomnik - Centrum Zarowia Dziecka
Warsaw, Poland
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Istanbul University Medical Faculty
Istanbul, Turkey (Türkiye)
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Johns Hopkins School of Medicine
Baltimore, Maryland, 21287, United States
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Kinderklinik Tubingen, Universitatsklinikum Tubingen
Tübingen, Germany
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King Faisal Specialist Hospital & Research Centre
Riyadh, Saudi Arabia
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Leeds General Infirmary
Leeds, United Kingdom
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Medizinische Hochschule Hannover
Hanover, Germany
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Ospedale Regina Margherita
Torino, Italy
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Riley Hospital for Children - Riley Children's Specialists
Indianapolis, Indiana, 46202, United States
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Schneider Children's Medical Center Of Israel
Petah Tikva, Israel
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Shaare-Zedek Mc
Jerusalem, Israel
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The Hospital for Sick Children
Toronto, Canada
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The Royal Children's Hospital
Melbourne, Australia
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Universitair Medisch Centrum (UMC) Utrecht
Utrecht, Netherlands
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Universite Paris SUD - Hpitaux Universitaires Paris-Sud - Hopital Bicetre
Le Kremlin-Bicêtre, France
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University Hospital Of Padova
Padova, Italy
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University Medical Center Groningen
Groningen, Netherlands
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University and Pediatric Hospital of Lyon
Bron, France
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Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin
Tübingen, Germany
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.