Biliary AtresiaPhase 3NCT04336722
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
A Phase 3 study testing Odevixibat in people with Biliary Atresia, run by Albireo, an Ipsen Company at 72 sites in 19 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 111 days
- Conditions the study is for
- Biliary Atresia
- What is being tested?
- Odevixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 254 participants
- Where
- 72 sitesin 19 countries
- Study dates
- 8 Jul 2020to 22 Jun 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 111 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
72 sites in 19 countries.
72 sites in 19 countries
United States30 sites
University of Michigan Children's Hospital
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins Children's Center
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Cleveland Clinic
Cleveland, Ohio, 44195
UT Southwestern Medical Center
Dallas, Texas, 75390
Duke Central Tower
Durham, North Carolina, 27710
Indiana University school of Medicine
Indianapolis, Indiana, 46202
Children's Mercy Hospital
Kansas City, Missouri, 64108
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of Miami
Miami, Florida, 33146
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
NYU Grossman school of Medicine
New York, New York, 10016
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
NewYork-Presbyterian Morgan Stanley Children's Hospital
New York, New York, 10032
Stanford Children's Health
Palo Alto, California, 94304
The Children´s Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health & Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84113
Rady Children's Hospital
San Diego, California, 92123
UCSF Benioff Children's Hospital San Francisco
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University in St. Louis
St Louis, Missouri, 63110
The Children's Hospital at Montefiore
The Bronx, New York, 10467
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803
Italy6 sites
ASST Papa Giovanni XXIII
Bergamo
Meyer Children´s University Hospital
Florence
University Hospital of Padova
Padova
ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
Palermo
Ospedale Pediatrico Bambino Gesù
Roma
Regina Margherita Children´s Hospital
Turin
Germany5 sites
Charité - Universitätsmedizin Berlin
Berlin
University Medical Center Hamburg-Eppendorf UKE
Hamburg
Hannover Medical School
Hanover
Dr von Hauner Children´s Hospital LMU
Munich
University Children´s Hospital Tuebingen
Tübingen
France4 sites
Hôpital Femme Mère Enfant
Bron
Bicêtre Hospital
Le Kremlin-Bicêtre
Jeanne de Flandre Hospital
Lille
Necker University Hospital - Enfants malades
Paris
Australia3 sites
Women's and Children's Hospital (Women's and Children's Health Network, Incorporated.)
North Adelaide, South Australia
Royal Children's Hospital
Parkville, 3052
The Children´s Hospital at Westmead
Sydney
Turkey (Türkiye)3 sites
Hacettepe University Ihsan Dogramaci Childrens Hospital
Ankara
Akdeniz University Medical Faculty
Antalya
Istanbul University, Istanbul Medical Faculty
Istanbul
Canada2 sites
CHU Sainte-Justine
Montreal
The Hospital for Sick Children
Toronto
China2 sites
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong
Children's Hospital of Fudan University
Shanghai
Israel2 sites
Shaare Zedek Medical Center
Jerusalem
Schneider Children´s Medical Center of Israel
Petah Tikva
Malaysia2 sites
Hospital Raja Perempuan Zainab II
Kota Bharu
University of Malaya Medical Centre
Kuala Lumpur
South Korea2 sites
Samsung Medical Center
Seoul
Seoul National University Children's Hospital
Seoul
Taiwan2 sites
National Taiwan University Hospital
Taipei
Taipei Veterans General Hospital
Taipei
United Kingdom2 sites
Birmingham Women´s and Children´s Hospital
Birmingham
Leeds General Infirmary
Leeds
About this study
Official 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
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.
Read the detailed description
Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
- Conditions
- Biliary Atresia
- Being tested
- Odevixibat, Placebo
- Keywords
- Kasai
- Medical subject headings
- Biliary Atresia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OdevixibatDrug
Odevixibat is a small molecule and selective inhibitor of IBAT.
PlaceboDrug
Placebo identical in appearance to experimental drug (odevixibat).
| Study group | Role | What they receive |
|---|---|---|
| Odevixibat (A4250) | Experimental | Capsules for oral administration once daily for 104 weeks. Receives: Drug: Odevixibat |
| Placebo | Placebo comparator | Capsules for oral administration (to match active) once daily for 104 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time from randomization to first occurrence of liver transplant, or death
Measured: From baseline to Week 104
Secondary outcomes (9)
Proportion of patients with liver transplant
Proportion of patients who are alive and have not undergone a liver transplant
Measured: From baseline to Week 104
Time to onset of any sentinel events
Time to onset of any sentinel events
Measured: From baseline to Week 104
Total bilirubin levels
Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
Measured: From baseline to Weeks 13, 26, 52 and 104
Serum bile acid levels
Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
Measured: From baseline to Weeks 13, 26, 52 and 104
Time to pediatric end-stage liver disease (PELD) score >15
Time to pediatric end-stage liver disease (PELD) score >15
Measured: From baseline to Week 104
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
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.
Measured: From baseline to Week 104
Percentage of participants with clinically significant changes in Physical Examination
Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.
Measured: From baseline to Week 104
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
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.
Measured: From baseline to Week 104
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.
Measured: From baseline to Week 26 and Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2020
- Study start8 Jul 2020
- Primary completion22 Jun 2026
- Study completion22 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biliary AtresiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 254 participants (actual)
Sponsor and investigators
- Lead sponsor
- Albireo, an Ipsen Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04336722
- Sponsor's protocol ID
- A4250-011
- Other IDs
- 2019-003807-37, 2024-512086-14-00, 2019
- Status last verified
- Jul 2026
- First submitted
- 27 Mar 2020
- Last updated
- 2 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT04336722
- Acronym / sponsor ID
- A4250-011
- Conditions
- Biliary Atresia
- Keywords
- Kasai
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 254 participants (actual)
- Lead sponsor
- Albireo, an Ipsen CompanyIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2020 (actual)
- Primary completion
- 22 Jun 2026 (actual)
- Study completion
- 22 Jun 2026 (actual)
- First posted
- 7 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Odevixibat (A4250) | Experimental | Drug: OdevixibatCapsules for oral administration once daily for 104 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboCapsules for oral administration (to match active) once daily for 104 weeks. |
Who may qualify
- Ages
- Up to 111 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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 (9 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OdevixibatOdevixibat is a small molecule and selective inhibitor of IBAT. | Drug | Odevixibat (A4250) |
| PlaceboPlacebo identical in appearance to experimental drug (odevixibat). | Drug | Placebo |
Primary outcome
Time from randomization to first occurrence of liver transplant, or death
Time frame: From baseline to Week 104
9 secondary outcomes
Proportion of patients with liver transplant
Time frame: From baseline to Week 104
Time to onset of any sentinel events
Time frame: From baseline to Week 104
Total bilirubin levels
Time frame: From baseline to Weeks 13, 26, 52 and 104
Serum bile acid levels
Time frame: From baseline to Weeks 13, 26, 52 and 104
Time to pediatric end-stage liver disease (PELD) score >15
Time frame: From baseline to Week 104
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 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 Laboratory Parameters (blood chemistry, hematology and coagulation)
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ipsen Medical Director study director
Ipsen
72 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Odevixibat in people with Biliary Atresia, run by Albireo, an Ipsen Company at 72 sites in 19 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 72 sites in United States, Italy, Germany, France, Australia and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time from randomization to first occurrence of liver transplant, or death (time frame: From baseline to Week 104). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2026.