Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

July 1, 2026 updated by: Albireo, an Ipsen Company

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
254

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
      • Parkville, Australia, 3052
        • Royal Children's Hospital
      • Sydney, Australia
        • The Children´s Hospital at Westmead
    • South Australia
      • North Adelaide, South Australia, Australia
        • Women's and Children's Hospital (Women's and Children's Health Network, Incorporated.)
  • Belgium
      • Brussels, Belgium
        • Cliniques universitaires Saint-Luc
      • Ghent, Belgium
        • Uz Gent
  • Canada
      • Montreal, Canada
        • CHU Sainte-Justine
      • Toronto, Canada
        • The Hospital for Sick Children
  • China
      • Shanghai, China
        • Children's Hospital of Fudan University
    • Guangdong
      • Guangzhou, Guangdong, China
        • Guangzhou Women And Children's Medical Center
  • France
      • Bron, France
        • Hôpital Femme Mère Enfant
      • Le Kremlin-Bicêtre, France
        • Bicêtre Hospital
      • Lille, France
        • Jeanne de Flandre Hospital
      • Paris, France
        • Necker University Hospital - Enfants malades
  • Germany
      • Berlin, Germany
        • Charité - Universitätsmedizin Berlin
      • Hamburg, Germany
        • University Medical Center Hamburg-Eppendorf UKE
      • Hanover, Germany
        • Hannover Medical School
      • Munich, Germany
        • Dr von Hauner Children´s Hospital LMU
      • Tübingen, Germany
        • University Children´s Hospital Tuebingen
  • Hungary
      • Budapest, Hungary
        • Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
  • Israel
      • Jerusalem, Israel
        • Shaare Zedek Medical Center
      • Petah Tikva, Israel
        • Schneider Children´s Medical Center of Israel
  • Italy
      • Bergamo, Italy
        • ASST Papa Giovanni XXIII
      • Florence, Italy
        • Meyer Children´s University Hospital
      • Padova, Italy
        • University Hospital Of Padova
      • Palermo, Italy
        • ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
      • Roma, Italy
        • Ospedale Pediatrico Bambino Gesu
      • Turin, Italy
        • Regina Margherita Children´s Hospital
  • Malaysia
      • Kota Bharu, Malaysia
        • Hospital Raja Perempuan Zainab II
      • Kuala Lumpur, Malaysia
        • University of Malaya Medical Centre
  • Netherlands
      • Groningen, Netherlands
        • University Medical Center Groningen
  • New Zealand
      • Auckland, New Zealand
        • Starship Child Health
  • Poland
      • Warsaw, Poland
        • Instytut Pomnik-Centrum Zdrowia Dziecka
  • South Korea
      • Seoul, South Korea
        • Samsung Medical Center
      • Seoul, South Korea
        • Seoul National University Children's Hospital
  • Spain
      • Barcelona, Spain
        • Hospital Universitari Vall d'Hebron
  • Taiwan
      • Taipei, Taiwan
        • National Taiwan University Hospital
      • Taipei, Taiwan
        • Taipei Veterans General Hospital
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye)
        • Hacettepe University Ihsan Dogramaci Childrens Hospital
      • Antalya, Turkey (Türkiye)
        • Akdeniz University Medical Faculty
      • Istanbul, Turkey (Türkiye)
        • Istanbul University, Istanbul Medical Faculty
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Women´s and Children´s Hospital
      • Leeds, United Kingdom
        • Leeds General Infirmary
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford Children's Health
      • San Diego, California, United States, 92123
        • Rady Children's Hospital
      • San Francisco, California, United States, 94158
        • UCSF Benioff Children's Hospital San Francisco
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Nemours/Alfred I. duPont Hospital for Children
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Miami, Florida, United States, 33146
        • University of Miami
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta - Emory University School of Medicine
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University School of Medicine
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins Children's Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Children's Hospital
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital
      • St Louis, Missouri, United States, 63110
        • Washington University in St. Louis
    • New York
      • New York, New York, United States, 10029
        • Icahn School Of Medicine At Mount Sinai
      • New York, New York, United States, 10032
        • NewYork-Presbyterian Morgan Stanley Children's Hospital
      • New York, New York, United States, 10016
        • NYU Grossman School of Medicine
      • The Bronx, New York, United States, 10467
        • The Children's Hospital at Montefiore
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke Central Tower
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children´s Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • UPMC Children's Hospital of Pittsburgh
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • University of Utah
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

No older than 3 years (Child)

Accepts Healthy Volunteers

No

Description

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 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 104 weeks.
Drug: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 104 weeks.
Drug: Placebo
Placebo identical in appearance to experimental drug (odevixibat).

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Time from randomization to first occurrence of liver transplant, or death
Time Frame: From baseline to Week 104
From baseline to Week 104

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Time to onset of any sentinel events
Time Frame: From baseline to Week 104
Time to onset of any sentinel events
From baseline to Week 104
Time to pediatric end-stage liver disease (PELD) score >15
Time Frame: From baseline to Week 104
Time to pediatric end-stage liver disease (PELD) score >15
From baseline to Week 104
Total bilirubin levels
Time Frame: From baseline to Weeks 13, 26, 52 and 104
Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
From baseline to Weeks 13, 26, 52 and 104
Serum bile acid levels
Time Frame: From baseline to Weeks 13, 26, 52 and 104
Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
From baseline to Weeks 13, 26, 52 and 104
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: From baseline to Week 104
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.
From baseline to Week 104
Percentage of participants with clinically significant changes in Physical Examination
Time Frame: From baseline to Week 104
Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.
From baseline to Week 104
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
Time Frame: From baseline to Week 104
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.
From baseline to Week 104
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
Time Frame: From baseline to Week 26 and Week 104
Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.
From baseline to Week 26 and Week 104
Proportion of patients with liver transplant
Time Frame: From baseline to Week 104
Proportion of patients who are alive and have not undergone a liver transplant
From baseline to Week 104

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Albireo, an Ipsen Company

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Ipsen Medical Director, Ipsen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 8, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 22, 2026

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 22, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 27, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 3, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 7, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 2, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 1, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • A4250-011
  • 2019-003807-37 (EudraCT Number)
  • 2024-512086-14-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing Time Frame

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.

IPD Sharing Access Criteria

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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