Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy
3 other identifiers
interventional
254
19 countries
72
Brief Summary
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Jul 2020
Longer than P75 for phase_3
72 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 27, 2020
CompletedFirst Posted
Study publicly available on registry
April 7, 2020
CompletedStudy Start
First participant enrolled
July 8, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 26, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 26, 2026
April 30, 2026
April 1, 2026
6 years
March 27, 2020
April 29, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Time from randomization to first occurrence of liver transplant, or death
From baseline to Week 104
Secondary Outcomes (9)
Proportion of patients with liver transplant
From baseline to Week 104
Time to onset of any sentinel events
From baseline to Week 104
Total bilirubin levels
From baseline to Weeks 13, 26, 52 and 104
Serum bile acid levels
From baseline to Weeks 13, 26, 52 and 104
Time to pediatric end-stage liver disease (PELD) score >15
From baseline to Week 104
- +4 more secondary outcomes
Study Arms (2)
Odevixibat (A4250)
EXPERIMENTALCapsules for oral administration once daily for 104 weeks.
Placebo
PLACEBO COMPARATORCapsules for oral administration (to match active) once daily for 104 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- A male or female patient with a clinical diagnosis of BA
- Age at Kasai HPE ≤90 days
- Eligible to start study treatment within 3 weeks post-Kasai HPE
You may not qualify if:
- Patients with intractable ascites
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (72)
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Stanford Children's Health
Palo Alto, California, 94304, United States
Rady Children's Hospital
San Diego, California, 92123, United States
UCSF Benioff Children's Hospital San Francisco
San Francisco, California, 94158, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Miami
Miami, Florida, 33146, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611, United States
Indiana University school of Medicine
Indianapolis, Indiana, 46202, United States
Johns Hopkins Children's Center
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan Children's Hospital
Ann Arbor, Michigan, 48109, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University in St. Louis
St Louis, Missouri, 63110, United States
NYU Grossman school of Medicine
New York, New York, 10016, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
NewYork-Presbyterian Morgan Stanley Children's Hospital
New York, New York, 10032, United States
The Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
Duke Central Tower
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
The Children´s Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
University of Utah
Salt Lake City, Utah, 84113, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Women's and Children's Hospital (Women's and Children's Health Network, Incorporated.)
North Adelaide, South Australia, Australia
Royal Children's Hospital
Parkville, 3052, Australia
The Children´s Hospital at Westmead
Sydney, Australia
Cliniques Universitaires Saint-Luc
Brussels, Belgium
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
Dr von Hauner Children´s Hospital LMU
Munich, Germany
University Children´s Hospital Tuebingen
Tübingen, Germany
Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
Budapest, Hungary
Shaare Zedek Medical Center
Jerusalem, Israel
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
Padova, 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
Taipei Veterans General 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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Ipsen Medical Director
Ipsen
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 27, 2020
First Posted
April 7, 2020
Study Start
July 8, 2020
Primary Completion (Estimated)
June 26, 2026
Study Completion (Estimated)
June 26, 2026
Last Updated
April 30, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
- Access Criteria
- Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.