Promising new drug may ease severe itching in kids with rare liver disorder
NCT ID NCT03566238
First seen Jan 08, 2026 · Last updated May 21, 2026 · Updated 21 times
Summary
This study tested a medicine called 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 goal was to see if the drug could lower bile acid levels and reduce scratching. Children took either a low dose, high dose, or placebo for 24 weeks. The results help determine if A4250 can control the disease, though lifelong management is still needed.
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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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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
Conditions
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