New drug shows promise for rare childhood liver disease
NCT ID NCT03566238
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called odevixibat (A4250) in 62 children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2, a rare liver disease that causes severe itching and bile buildup. The drug works by blocking the reabsorption of bile acids in the gut. The goal was to see if it could lower bile acid levels and reduce itching compared to a placebo. Results could lead to a new treatment option for these children.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
odevixibat (A4250)
What this could lead to
If successful, odevixibat could become a new treatment to reduce bile acid buildup and severe itching in children with PFIC, potentially delaying or avoiding the need for liver transplant.
What could go wrong
This is a relatively small Phase 3 trial (62 children), and the drug may not work for all types of PFIC. Long-term safety and effectiveness are still being studied.
Disclaimer
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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.
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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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
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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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British Columbia Children's Hospital
Vancouver, Canada
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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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Cleveland Clinic
Cleveland, Ohio, 44195, 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 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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Hospital de la Timone
Marseille, France
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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 Zdrowia 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 Tübingen, Universitätsklinikum Tübingen
Tübingen, Germany
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King Faisal Specialist Hospital & Research Centre
Riyadh, 11211, 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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Rambam Medical Centre
Haifa, Israel
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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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UZ Leuven
Leuven, Belgium
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Uniklinikum Essen- Kinderklinik II
Essen, Germany
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Universitair Medisch Centrum (UMC) Utrecht
Utrecht, Netherlands
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Universite Paris SUD - Hopitaux 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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University of California, San Francisco
San Francisco, California, 94143, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States