Hope for kids with rare liver disease: new drug targets debilitating itching and liver damage

NCT ID NCT03566238

Completed Disease control Sponsor: Albireo Source: ClinicalTrials.gov ↗

Summary

This study tested whether a drug called A4250 could help children with rare, progressive liver diseases known as PFIC1 and PFIC2. The main goals were to see if the drug could lower harmful bile acids in the blood and reduce the severe, life-altering itching these children experience. It was a Phase 3 trial, meaning it was a large, late-stage test comparing the drug to a placebo in 62 children across 15 countries.

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Contacts and locations

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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
British Columbia Children's Hospital

Vancouver, Canada
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
Cleveland Clinic

Cleveland, Ohio, 44195, 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 Necker-Enfants maladies

Paris, France
Hospital Universitari Vall d'Hebron

Barcelona, Spain
Hospital Universitario La Paz

Madrid, Spain
Hospital de la Timone

Marseille, France
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 Zdrowia Dziecka

Warsaw, Poland
Istanbul University Medical Faculty

Istanbul, Turkey (Türkiye)
Johns Hopkins School of Medicine

Baltimore, Maryland, 21287, United States
Kinderklinik Tübingen, Universitätsklinikum Tübingen

Tübingen, Germany
King Faisal Specialist Hospital & Research Centre

Riyadh, 11211, Saudi Arabia
Leeds General Infirmary

Leeds, United Kingdom
Medizinische Hochschule Hannover

Hanover, Germany
Ospedale Regina Margherita

Torino, Italy
Rambam Medical Centre

Haifa, Israel
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
UZ Leuven

Leuven, Belgium
Uniklinikum Essen- Kinderklinik II

Essen, Germany
Universitair Medisch Centrum (UMC) Utrecht

Utrecht, Netherlands
Universite Paris SUD - Hopitaux 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
University of California, San Francisco

San Francisco, California, 94143, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.

PFIC1 PFIC2