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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05426733
Registration number
NCT05426733
Ethics application status
Date submitted
13/06/2022
Date registered
22/06/2022
Date last updated
22/06/2025
Titles & IDs
Public title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
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Scientific title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
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Secondary ID [1]
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2022-501090-39-00
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Secondary ID [2]
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A4250-016
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Universal Trial Number (UTN)
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Trial acronym
BOLD-EXT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Biliary Atresia
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Condition category
Condition code
Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Odevixibat
Experimental: Odevixibat (A4250) - Capsules for oral administration once daily for 104 weeks.
Treatment: Drugs: Odevixibat
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Proportion of patients who are alive and have not undergone liver transplant
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Assessment method [1]
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Timepoint [1]
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From baseline to Week 104
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Secondary outcome [1]
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Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score
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Assessment method [1]
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The APRI (aspartate aminotransferase to platelet ratio index) score estimates the severity of liver fibrosis. A higher score indicates a greater amount of scarring (fibrosis) in the liver. * A score = 0.5 indicates a low likelihood of fibrosis * A score = 1.5 indicates a higher probability of fibrosis
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Timepoint [1]
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From baseline to Week 104
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Secondary outcome [2]
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Change in Fibrosis-4 (Fib-4) score
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Assessment method [2]
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The Fib-4 score provides an estimate of the amount of scarring (fibrosis) in the liver. A higher score indicates a greater amount of scarring in the liver. * A score \< 1.45 indicates a low likelihood of fibrosis * A score \> 3.25 indicates a higher probability of fibrosis
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Timepoint [2]
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From baseline to Week 104
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Secondary outcome [3]
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Change in serum bile acids
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Assessment method [3]
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Timepoint [3]
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From Baseline to Weeks 26, 52, 78, and 104
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Secondary outcome [4]
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Change in height
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Assessment method [4]
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Timepoint [4]
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From Baseline to Weeks 26, 52, 78 and 104
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Secondary outcome [5]
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Change in weight
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Assessment method [5]
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Timepoint [5]
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From Baseline to Weeks 26, 52, 78 and 104
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Secondary outcome [6]
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Change in mid-arm circumference
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Assessment method [6]
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Measure of growth
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Timepoint [6]
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From Baseline to Weeks 26, 52, 78 and 104
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Secondary outcome [7]
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Time to onset of any sentinel events
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Assessment method [7]
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Timepoint [7]
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From Baseline to Week 104
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Secondary outcome [8]
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Change in pediatric end-stage liver disease (PELD) score
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Assessment method [8]
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The PELD (pediatric end-stage liver disease) score is a measure of disease severity. PELD scores can range from a negative value (such as -10) to high numbers (such as 45). A higher score indicates more severe disease.
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Timepoint [8]
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From Baseline to Weeks 26, 52, 78 and 104
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Eligibility
Key inclusion criteria
* Completion of the 104-week Treatment Period of Study A4250-011
* Signed informed consent by caregiver
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
5/07/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
31/08/2028
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Actual
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Sample size
Target
180
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Colorado
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United States of America
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Georgia
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United States of America
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Indiana
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United States of America
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Massachusetts
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Country [5]
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United States of America
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Missouri
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Country [6]
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United States of America
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New York
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Country [7]
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United States of America
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Ohio
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United States of America
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State/province [8]
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Washington
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Country [9]
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Canada
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State/province [9]
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Montréal
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Canada
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Toronto
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China
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Guangdong
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China
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State/province [12]
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Shanghai
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France
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Le Kremlin-Bicêtre
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Germany
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Berlin
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Germany
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Tuebingen
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Italy
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Bergamo
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Italy
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Padova
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Korea, Republic of
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State/province [18]
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Seoul
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Malaysia
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Kota Bharu
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Malaysia
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Kuala Lumpur
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Netherlands
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Groningen
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New Zealand
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Auckland
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Poland
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Warsaw
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Spain
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Barcelona
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Turkey
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Ankara
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Turkey
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Antalya
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Country [27]
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Turkey
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State/province [27]
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Istanbul
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Albireo, an Ipsen Company
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
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Trial website
https://clinicaltrials.gov/study/NCT05426733
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
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When will data be available (start and end dates)?
Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.
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Available to whom?
Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://vivli.org/members/ourmembers/
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05426733
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