An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
BOLD-EXT
2 other identifiers
interventional
180
14 countries
30
Brief Summary
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jul 2022
Longer than P75 for phase_3
30 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
June 13, 2022
CompletedFirst Posted
Study publicly available on registry
June 22, 2022
CompletedStudy Start
First participant enrolled
July 5, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 31, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 31, 2028
April 30, 2026
April 1, 2026
6.2 years
June 13, 2022
April 29, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Proportion of patients who are alive and have not undergone liver transplant
From baseline to Week 104
Secondary Outcomes (8)
Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score
From baseline to Week 104
Change in Fibrosis-4 (Fib-4) score
From baseline to Week 104
Change in serum bile acids
From Baseline to Weeks 26, 52, 78, and 104
Change in height
From Baseline to Weeks 26, 52, 78 and 104
Change in weight
From Baseline to Weeks 26, 52, 78 and 104
- +3 more secondary outcomes
Study Arms (1)
Odevixibat (A4250)
EXPERIMENTALCapsules for oral administration once daily for 104 weeks.
Interventions
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).
Eligibility Criteria
You may qualify if:
- Completion of the 104-week Treatment Period of Study A4250-011
- Signed informed consent by caregiver
You may not qualify if:
- Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator's discretion
- Any conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
- Known hypersensitivity to any components of odevixibat
- Patients who are scheduled for a liver transplant or are likely to require a liver transplant in the immediate future based on the investigator's judgment
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (30)
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's Healhcare of Atlanta- Emory University school of medicine
Atlanta, Georgia, 30322, United States
Indiana University school of Medicine
Indianapolis, Indiana, 46202, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64108, United States
Hassenfeld Children's Hospital at NYU Langone
New York, New York, 10016, United States
Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital
New York, New York, 10032, United States
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, 10467, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Royal Children's Hospital
Parkville, 3052, Australia
CHU Sainte-Justine
Montreal, Canada
The Hospital for Sick Children
Toronto, Canada
Guangzhou Women And Childrens Medical Center
Guangdong, China
Children's Hospital of Fudan University
Shanghai, China
Bicêtre Hospital
Le Kremlin-Bicêtre, France
Charité - Universitätsmedizin Berlin
Berlin, Germany
University Children´s Hospital Tuebingen
Tübingen, Germany
ASST Papa Giovanni XXIII
Bergamo, Italy
Azienda Ospedaliera di Padova
Padova, Italy
Hospital Raja Perempuan Zainab II
Kota Bharu, Malaysia
University Malaya Medical Centre
Kuala Lumpur, 59100, Malaysia
University Medical Center Groningen
Groningen, Netherlands
Starship Child Health, Te Whatu Ora - Health New Zealand
Auckland, 1023, New Zealand
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
Seoul National University Childrens Hospital
Seoul, South Korea
Hospital Universitari Vall d'Hebron
Barcelona, Spain
Hacettepe İhsan Doğramacı Children's Hospital
Ankara, Turkey (Türkiye)
Akdeniz University Medical Faculty
Antalya, Turkey (Türkiye)
Istanbul University Istanbul Medical Faculty Hospital
Istanbul, Turkey (Türkiye)
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 13, 2022
First Posted
June 22, 2022
Study Start
July 5, 2022
Primary Completion (Estimated)
August 31, 2028
Study Completion (Estimated)
August 31, 2028
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.