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
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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
Padua, 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.