Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
NCT ID: NCT04674761
Last Updated: 2023-11-02
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
52 participants
INTERVENTIONAL
2021-03-19
2022-09-09
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Odevixibat (A4250)
Capsules for oral administration once daily for 24 weeks.
Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo
Placebo identical in appearance to experimental drug (odevixibat).
Interventions
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Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo
Placebo identical in appearance to experimental drug (odevixibat).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
* Elevated serum bile acid level
Exclusion Criteria
* History of liver transplant, or a liver transplant is planned within 6 months of randomization
* ALT \>10× upper limit of normal (ULN) at screening
* Total bilirubin \>15 × ULN at screening
* Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome
ALL
No
Sponsors
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Albireo
INDUSTRY
Responsible Party
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Locations
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University of California - San Francisco
San Francisco, California, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Mercy Hospital and Clinics
Kansas City, Missouri, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, United States
Oregon Health Science University School of Medicine
Portland, Oregon, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Cliniques Universitaires Saint-Luc Bruxelles
Brussels, , Belgium
CHU Sainte-Justine
Montreal, Quebec, Canada
Hôpital Femme Mère Enfant de Lyon
Bron, , France
Antenne pédiatrique du CIC - Hopital Jeanne De Flandre
Lille, , France
APHM
Marseille, , France
Hopital Necker Enfants Malades
Paris, , France
Charité-Universitätsmedizin Berlin
Berlin, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Universitätsklinik für Kinder-und Jugendmedizin Tübingen
Tübingen, , Germany
Sharie Zedek
Jerusalem, , Israel
Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
ASST Papa Giovanni XXIII
Bergamo, , Italy
AOU Meyer
Florence, , Italy
Azienda Ospedale Università Padova
Padua, , Italy
Bambino Gesù Children's Hospital
Rome, , Italy
University of Malaya Medical Centre
Kuala Lumpur, , Malaysia
Universitair Medisch Centrum Groningen (UMCG)
Groningen, , Netherlands
University Medical Centre Utrecht, WKZ
Utrecht, , Netherlands
Starship Child Health
Auckland, , New Zealand
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, , Poland
Hacettepe Üniversitesi İhsan Doğramacı Çocuk Hastanesi
Ankara, , Turkey (Türkiye)
Istanbul University Medical Faculty Hospital
Fatih, , Turkey (Türkiye)
King's College Hospital
London, , United Kingdom
Countries
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References
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Ovchinsky N, Aumar M, Baker A, Baumann U, Bufler P, Cananzi M, Czubkowski P, Durmaz O, Fischer R, Indolfi G, Karnsakul WW, Lacaille F, Lee WS, Maggiore G, Rosenthal P, Ruiz M, Sokal E, Sturm E, van der Woerd W, Verkade HJ, Wehrman A, Clemson C, Yu Q, Ni Q, Ruvido J, Manganaro S, Mattsson JP. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):632-645. doi: 10.1016/S2468-1253(24)00074-8. Epub 2024 Apr 23.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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A4250-012
Identifier Type: -
Identifier Source: org_study_id
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