This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
NCT ID: NCT03566238
Last Updated: 2025-11-28
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
62 participants
INTERVENTIONAL
2018-05-16
2020-07-28
Brief Summary
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Detailed Description
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Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Sweden, Turkey, United Kingdom, United States, and Saudi Arabia
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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A4250 low dose
Capsules for oral administration (40 ug/kg) once daily for 24 weeks
A4250 (odevixibat)
A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).
A4250 high dose
Capsules for oral administration (120 ug/kg) once daily for 24 weeks
A4250 (odevixibat)
A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).
Placebo
Capsules for oral administration (to match active) once daily for 24 weeks
Placebo
Placebo identical in appearance to active drug (A4250).
Interventions
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A4250 (odevixibat)
A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).
Placebo
Placebo identical in appearance to active drug (A4250).
Eligibility Criteria
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Inclusion Criteria
* Participant must have clinical genetic confirmation of PFIC-1 or PFIC-2
* Participant must have elevated serum bile acid (s-BA) concentration
* Participant must have history of significant pruritus and a caregiver reported observed scratching in the eDiary
* Participant and/or legal guardian must sign informed consent (and assent) as appropriate.
* Participants will be expected to have a consistent caregiver(s) for the duration of the study
* Caregivers and age-appropriate participants (≥8 years of age) must be willing and able to use an eDiary device as required by the study
Exclusion Criteria
* Participant with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
1. Biliary atresia of any kind
2. Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs
3. Suspected or proven liver cancer or metastasis to the liver on imaging studies
4. Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis
* Participant with past medical history or ongoing chronic diarrhea
* Any participant with suspected or confirmed cancers except for basal cell carcinoma
* Participant with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m\^2
* Participant with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
* Participant has had a liver transplant or a liver transplant is planned within 6 months of randomization
* Decompensated liver disease
* Participant suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
* Participant who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment
6 Months
18 Years
ALL
No
Sponsors
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Albireo
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Los Angeles
Los Angeles, California, United States
University of California, San Francisco
San Francisco, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
Emory University School of Medicine
Atlanta, Georgia, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
Washington University School of Medicine
St Louis, Missouri, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Columbia University Medical Center - Presbyterian Hospital Building
New York, New York, United States
Cleveland Clinic
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Baylor College of Medicine - Texas Children's Liver Center
Houston, Texas, United States
The Royal Children's Hospital
Melbourne, , Australia
UZ Leuven
Leuven, , Belgium
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, , Belgium
The Hospital for Sick Children
Toronto, , Canada
British Columbia Children's Hospital
Vancouver, , Canada
University and Pediatric Hospital of Lyon
Bron, , France
Universite Paris SUD - Hopitaux Universitaires Paris-Sud - Hopital Bicetre
Le Kremlin-Bicêtre, , France
Hospital de la Timone
Marseille, , France
Hospital Necker-Enfants maladies
Paris, , France
Uniklinikum Essen- Kinderklinik II
Essen, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Kinderklinik Tübingen, Universitätsklinikum Tübingen
Tübingen, , Germany
Rambam Medical Centre
Haifa, , Israel
Shaare-Zedek Mc
Jerusalem, , Israel
Schneider Children's Medical Center Of Israel
Petah Tikva, , Israel
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, , Italy
University Hospital Of Padova
Padua, , Italy
Ospedale Regina Margherita
Torino, , Italy
University Medical Center Groningen
Groningen, , Netherlands
Universitair Medisch Centrum (UMC) Utrecht
Utrecht, , Netherlands
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, , Poland
King Faisal Specialist Hospital & Research Centre
Riyadh, , Saudi Arabia
Hospital Universitari Vall d'Hebron
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Astrid Lindgren Children's Hospital, Karolinska University Hospital
Solna, , Sweden
Gazi University
Ankara, , Turkey (Türkiye)
Hacettepe University Faculty of Medicine
Ankara, , Turkey (Türkiye)
Akdeniz University
Antalya, , Turkey (Türkiye)
Istanbul University Medical Faculty
Istanbul, , Turkey (Türkiye)
Inonu University Medical Faculty
Malatya, , Turkey (Türkiye)
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, , United Kingdom
Leeds General Infirmary
Leeds, , United Kingdom
Institute of Liver Studies - Kings College Hospital
London, , United Kingdom
Countries
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References
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Gwaltney C, Ivanescu C, Karlsson L, Warholic N, Kjems L, Horn P. Validation of the PRUCISION Instruments in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis. Adv Ther. 2022 Nov;39(11):5105-5125. doi: 10.1007/s12325-022-02262-7. Epub 2022 Sep 6.
Thompson RJ, Arnell H, Artan R, Baumann U, Calvo PL, Czubkowski P, Dalgic B, D'Antiga L, Durmaz O, Fischler B, Gonzales E, Grammatikopoulos T, Gupte G, Hardikar W, Houwen RHJ, Kamath BM, Karpen SJ, Kjems L, Lacaille F, Lachaux A, Lainka E, Mack CL, Mattsson JP, McKiernan P, Ozen H, Rajwal SR, Roquelaure B, Shagrani M, Shteyer E, Soufi N, Sturm E, Tessier ME, Verkade HJ, Horn P. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2022 Sep;7(9):830-842. doi: 10.1016/S2468-1253(22)00093-0. Epub 2022 Jul 1.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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A4250-005
Identifier Type: -
Identifier Source: org_study_id
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