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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
Not applicable
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)?
Yes
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.
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.
Available to whom?
Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/members/ourmembers/


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