New hope for kids with rare liver disease: long-term drug study launched
NCT ID NCT05035030
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time
Summary
This study looks at the long-term safety and effectiveness of odevixibat in 70 people with Alagille syndrome, a rare genetic condition that affects the liver and other organs. The drug aims to reduce severe itching and improve bile flow. Participants who completed a prior 24-week study can join this extension to see how well the treatment works over time.
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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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AOU Meyer
Florence, Italy
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Antenne pediatrique du CIC-Hopital Jeanne De Flandre
Lille, France
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Atrium Health Carolinas Medical
Durham, North Carolina, 27710, United States
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Azienda Ospedale University
Padova, 35128, Italy
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Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Charité - Universitätsmedizin Berlin
Berlin, 13353, Germany
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
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Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64018, United States
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Childrens Medical Center of Dallas University of Texas Southwestern
Dallas, Texas, 75207, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cliniques Universitaires Saint-Luc Bruxelles
Brussels, 1200, Belgium
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Hassenfeld Children's Hospital at NYU Langone
New York, New York, 10016, United States
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Hopital Necker Enfants Malades
Paris, 75015, France
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Hôpital Femme Mère Enfant de Lyon
Bron, 69677, France
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Instytut Pomnik-Centrum Zdrowia Dzieck
Warsaw, 04-730, Poland
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Istanbul University Istanbul Medical Faculty Hospital
Istanbul, Turkey (Türkiye)
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Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
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King's College Hospital NHS Foundation Trust King's College Hospital Paediatric Research
London, SE5 9RS, United Kingdom
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Medizinische Hochschul
Hanover, 30625, Germany
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Monroe Carell Jr. Childrens Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
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New York-Presbyterian / Columbia University Irving Medical Center
New York, New York, 10032, United States
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Northwell Health System
New Hyde Park, New York, 11042, United States
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Oregon Health Science University School of Medicine
Portland, Oregon, 97239, United States
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Ospedale Pediatrico Bambino Gesu
Rome, 00165, Italy
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Rady Children's Hospital
San Diego, California, 92123, United States
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Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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Texas Liver Institute
San Antonio, Texas, 78215, United States
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The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, 10467, United States
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UCSF
San Francisco, California, 94158, United States
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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Universitatsklinik fur Kinder-und Jugendmedizin Tubingen
Tübingen, 72076, Germany
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University of Malaya Medical Center
Kuala Lumpur, 59100, Malaysia
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Wilhelmina Children's Hospital UMCU Utrecht
Utrecht, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New drug shows promise for babies with rare liver disorders
- New study tracks Real-World safety of odevixibat in rare liver disease
- Can a common cholesterol drug shrink skin lumps in kids with rare liver disease?
- New drug livmarli tracked for Long-Term safety in kids with rare liver disorders
- Liver drug maralixibat tested for Long-Term safety in rare disease patients
- Major study on rare childhood liver diseases halted