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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04336722




Registration number
NCT04336722
Ethics application status
Date submitted
27/03/2020
Date registered
7/04/2020

Titles & IDs
Public title
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
Scientific title
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
Secondary ID [1] 0 0
2019-003807-37
Secondary ID [2] 0 0
A4250-011
Universal Trial Number (UTN)
Trial acronym
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
Treatment: Drugs - Placebo

Experimental: Odevixibat (A4250) - Capsules for oral administration once daily for 104 weeks.

Placebo comparator: Placebo - Capsules for oral administration (to match active) once daily for 104 weeks.


Treatment: Drugs: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.

Treatment: Drugs: Placebo
Placebo identical in appearance to experimental drug (odevixibat).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time from randomization to first occurrence of liver transplant, or death
Timepoint [1] 0 0
From baseline to Week 104
Secondary outcome [1] 0 0
Proportion of patients with liver transplant
Timepoint [1] 0 0
From baseline to Week 104
Secondary outcome [2] 0 0
Time to onset of any sentinel events
Timepoint [2] 0 0
From baseline to Week 104
Secondary outcome [3] 0 0
Total bilirubin levels
Timepoint [3] 0 0
From baseline to Weeks 13, 26, 52 and 104
Secondary outcome [4] 0 0
Serum bile acid levels
Timepoint [4] 0 0
From baseline to Weeks 13, 26, 52 and 104
Secondary outcome [5] 0 0
Time to pediatric end-stage liver disease (PELD) score >15
Timepoint [5] 0 0
From baseline to Week 104
Secondary outcome [6] 0 0
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [6] 0 0
From baseline to Week 104
Secondary outcome [7] 0 0
Percentage of participants with clinically significant changes in Physical Examination
Timepoint [7] 0 0
From baseline to Week 104
Secondary outcome [8] 0 0
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
Timepoint [8] 0 0
From baseline to Week 104
Secondary outcome [9] 0 0
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
Timepoint [9] 0 0
From baseline to Week 26 and Week 104

Eligibility
Key inclusion criteria
* 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

Key
Minimum age
No limit
Maximum age
111 Days
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/07/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/07/2026
Actual
Sample size
Target
Accrual to date
Final
254
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [2] 0 0
The Children´s Hospital at Westmead - Sydney
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment postcode(s) [2] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Delaware
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
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Georgia
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United States of America
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Illinois
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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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Maryland
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United States of America
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Massachusetts
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United States of America
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Michigan
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Missouri
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New York
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Ohio
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Oregon
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Pennsylvania
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Texas
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Utah
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Washington
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United States of America
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Wisconsin
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Belgium
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Gent
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Canada
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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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Shanghai
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France
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Bron
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France
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Le Kremlin-Bicêtre
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France
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Lille
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France
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Paris
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Germany
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Berlin
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Germany
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Hamburg
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Germany
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Hanover
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Germany
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Tuebingen
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Hungary
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Budapest
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Israel
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Petah tikva
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Italy
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Bergamo
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Italy
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Florence
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Italy
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Padova
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Italy
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Palermo
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Italy
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Roma
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Italy
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Turin
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Korea, Republic of
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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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Taiwan
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Taipei
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Ankara
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Turkey
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Antalya
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Turkey
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Istanbul
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United Kingdom
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Birmingham
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United Kingdom
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Leeds

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
Ipsen Medical Director
Address 0 0
Ipsen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ipsen Clinical Study Enquiries
Address 0 0
Country 0 0
Phone 0 0
See email
Fax 0 0
Email 0 0
clinical.trials@ipsen.com
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/NCT04336722