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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

Titles & IDs
Public title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
Scientific title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
Secondary ID [1] 0 0
2022-501090-39-00
Secondary ID [2] 0 0
A4250-016
Universal Trial Number (UTN)
Trial acronym
BOLD-EXT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Biliary Atresia 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients who are alive and have not undergone liver transplant
Timepoint [1] 0 0
From baseline to Week 104
Secondary outcome [1] 0 0
Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score
Timepoint [1] 0 0
From baseline to Week 104
Secondary outcome [2] 0 0
Change in Fibrosis-4 (Fib-4) score
Timepoint [2] 0 0
From baseline to Week 104
Secondary outcome [3] 0 0
Change in serum bile acids
Timepoint [3] 0 0
From Baseline to Weeks 26, 52, 78, and 104
Secondary outcome [4] 0 0
Change in height
Timepoint [4] 0 0
From Baseline to Weeks 26, 52, 78 and 104
Secondary outcome [5] 0 0
Change in weight
Timepoint [5] 0 0
From Baseline to Weeks 26, 52, 78 and 104
Secondary outcome [6] 0 0
Change in mid-arm circumference
Timepoint [6] 0 0
From Baseline to Weeks 26, 52, 78 and 104
Secondary outcome [7] 0 0
Time to onset of any sentinel events
Timepoint [7] 0 0
From Baseline to Week 104
Secondary outcome [8] 0 0
Change in pediatric end-stage liver disease (PELD) score
Timepoint [8] 0 0
From Baseline to Weeks 26, 52, 78 and 104

Eligibility
Key inclusion criteria
* Completion of the 104-week Treatment Period of Study A4250-011
* Signed informed consent by caregiver
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/08/2028
Actual
Sample size
Target
180
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Canada
State/province [9] 0 0
Montréal
Country [10] 0 0
Canada
State/province [10] 0 0
Toronto
Country [11] 0 0
China
State/province [11] 0 0
Guangdong
Country [12] 0 0
China
State/province [12] 0 0
Shanghai
Country [13] 0 0
France
State/province [13] 0 0
Le Kremlin-Bicêtre
Country [14] 0 0
Germany
State/province [14] 0 0
Berlin
Country [15] 0 0
Germany
State/province [15] 0 0
Tuebingen
Country [16] 0 0
Italy
State/province [16] 0 0
Bergamo
Country [17] 0 0
Italy
State/province [17] 0 0
Padova
Country [18] 0 0
Korea, Republic of
State/province [18] 0 0
Seoul
Country [19] 0 0
Malaysia
State/province [19] 0 0
Kota Bharu
Country [20] 0 0
Malaysia
State/province [20] 0 0
Kuala Lumpur
Country [21] 0 0
Netherlands
State/province [21] 0 0
Groningen
Country [22] 0 0
New Zealand
State/province [22] 0 0
Auckland
Country [23] 0 0
Poland
State/province [23] 0 0
Warsaw
Country [24] 0 0
Spain
State/province [24] 0 0
Barcelona
Country [25] 0 0
Turkey
State/province [25] 0 0
Ankara
Country [26] 0 0
Turkey
State/province [26] 0 0
Antalya
Country [27] 0 0
Turkey
State/province [27] 0 0
Istanbul

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Albireo, an Ipsen Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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