Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
NCT ID: NCT04336722
Last Updated: 2025-12-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
254 participants
INTERVENTIONAL
2020-07-08
2026-06-26
Brief Summary
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Detailed Description
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Conditions
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Keywords
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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 104 weeks.
Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo
Capsules for oral administration (to match active) once daily for 104 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).
Eligibility Criteria
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Inclusion Criteria
* Age at Kasai HPE ≤90 days
* Eligible to start study treatment within 3 weeks post-Kasai HPE
Exclusion Criteria
* Ileal resection surgery
* ALT ≥10× upper limit of normal (ULN) at screening
* Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization
* Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
* Choledochal cystic disease
* INR \>1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
* Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
* Weight \<3.5kg at randomization
111 Days
ALL
No
Sponsors
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Albireo, an Ipsen Company
INDUSTRY
Responsible Party
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Principal Investigators
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Ipsen Medical Director
Role: STUDY_DIRECTOR
Ipsen
Locations
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Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford Children's Health
Palo Alto, California, United States
Rady Children's Hospital
San Diego, California, United States
UCSF Benioff Children's Hospital San Francisco
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Miami
Miami, Florida, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
Indiana University school of Medicine
Indianapolis, Indiana, United States
Johns Hopkins Children's Center
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan Children's Hospital
Ann Arbor, Michigan, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Washington University in St. Louis
St Louis, Missouri, United States
NYU Grossman school of Medicine
New York, New York, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
NewYork-Presbyterian Morgan Stanley Children's Hospital
New York, New York, United States
The Children's Hospital at Montefiore
The Bronx, New York, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
The Children´s Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
UT Southwestern Medical Center
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Royal Children's Hospital
Parkville, , Australia
The Children´s Hospital at Westmead
Sydney, , Australia
UZ Gent
Ghent, , Belgium
CHU Sainte-Justine
Montreal, , Canada
The Hospital for Sick Children
Toronto, , Canada
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China
Children's Hospital of Fudan University
Shanghai, , China
Hôpital Femme Mère Enfant
Bron, , France
Bicêtre Hospital
Le Kremlin-Bicêtre, , France
Jeanne de Flandre Hospital
Lille, , France
Necker University Hospital - Enfants malades
Paris, , France
Charité - Universitätsmedizin Berlin
Berlin, , Germany
University Medical Center Hamburg-Eppendorf UKE
Hamburg, , Germany
Hannover Medical School
Hanover, , Germany
University Children´s Hospital Tuebingen
Tübingen, , Germany
Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
Budapest, , Hungary
Schneider Children´s Medical Center of Israel
Petah Tikva, , Israel
ASST Papa Giovanni XXIII
Bergamo, , Italy
Meyer Children´s University Hospital
Florence, , Italy
University Hospital of Padova
Padua, , Italy
ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
Palermo, , Italy
Ospedale Pediatrico Bambino Gesù
Roma, , Italy
Regina Margherita Children´s Hospital
Turin, , Italy
Hospital Raja Perempuan Zainab II
Kota Bharu, , Malaysia
University of Malaya Medical Centre
Kuala Lumpur, , Malaysia
University Medical Center Groningen
Groningen, , Netherlands
Starship Child Health
Auckland, , New Zealand
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, , Poland
Samsung Medical Center
Seoul, , South Korea
Seoul National University Children's Hospital
Seoul, , South Korea
Hospital Universitari Vall d'Hebron
Barcelona, , Spain
National Taiwan University Hospital
Taipei, , Taiwan
Hacettepe University Ihsan Dogramaci Childrens Hospital
Ankara, , Turkey (Türkiye)
Akdeniz University Medical Faculty
Antalya, , Turkey (Türkiye)
Istanbul University, Istanbul Medical Faculty
Istanbul, , Turkey (Türkiye)
Birmingham Women´s and Children´s Hospital
Birmingham, , United Kingdom
Leeds General Infirmary
Leeds, , United Kingdom
Countries
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Other Identifiers
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2019-003807-37
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-512086-14-00
Identifier Type: CTIS
Identifier Source: secondary_id
A4250-011
Identifier Type: -
Identifier Source: org_study_id