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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04336722
Registration number
NCT04336722
Ethics application status
Date submitted
27/03/2020
Date registered
7/04/2020
Date last updated
29/05/2024
Titles & IDs
Public title
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
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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
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Secondary ID [1]
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A4250-011
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Universal Trial Number (UTN)
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Trial acronym
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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
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).
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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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Time from randomization to first occurrence of liver transplant, or death
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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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Proportion of patients with liver transplant
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Assessment method [1]
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Proportion of patients who are alive and have not undergone a liver transplant
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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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Time to onset of any sentinel events
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Assessment method [2]
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Time to onset of any sentinel events
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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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Total bilirubin levels
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Assessment method [3]
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Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
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Timepoint [3]
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From baseline to Weeks 13, 26, 52 and 104
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Secondary outcome [4]
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Serum bile acid levels
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Assessment method [4]
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Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
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Timepoint [4]
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From baseline to Weeks 13, 26, 52 and 104
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Secondary outcome [5]
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Time to pediatric end-stage liver disease (PELD) score >15
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Assessment method [5]
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Time to pediatric end-stage liver disease (PELD) score >15
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Timepoint [5]
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From baseline to Week 104
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Secondary outcome [6]
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Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [6]
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An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
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Timepoint [6]
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From baseline to Week 104
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Secondary outcome [7]
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Percentage of participants with clinically significant changes in Physical Examination
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Assessment method [7]
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Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.
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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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Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
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Assessment method [8]
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Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be decided by the investigator.
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Timepoint [8]
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From baseline to Week 104
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Secondary outcome [9]
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Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
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Assessment method [9]
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Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.
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Timepoint [9]
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From baseline to Week 26 and Week 104
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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
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Minimum age
No limit
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Maximum age
111
Days
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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 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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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
8/07/2020
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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
31/07/2026
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Actual
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Sample size
Target
245
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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 hospital [2]
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The Children´s Hospital at Westmead - Sydney
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment postcode(s) [2]
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- Sydney
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Recruitment outside Australia
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United States of America
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California
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Colorado
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Albireo
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and
safety of odevixibat compared to placebo in children with biliary atresia who have undergone
a Kasai hepatoportoenterostomy.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04336722
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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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Ipsen Medical Director
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Address
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Ipsen
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Contact person for public queries
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Ipsen Clinical Study Enquiries
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Phone
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See email
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04336722
Download to PDF