Biliary AtresiaPhase 3BOLD-EXTNCT05426733
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
A Phase 3 study testing Odevixibat in people with Biliary Atresia, run by Albireo, an Ipsen Company at 30 sites in 14 countries.
Trial summary
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Biliary Atresia
- What is being tested?
- Odevixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 180 participants
- Where
- 30 sitesin 14 countries
- Study dates
- 5 Jul 2022to 28 Oct 2026
- Phase
- Phase 3
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of the 104-week Treatment Period of Study A4250-011
- Signed informed consent by caregiver
Who cannot join
- 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
Trial locations and contacts
30 sites in 14 countries.
30 sites in 14 countries
United States10 sites
Children's Healhcare of Atlanta- Emory University school of medicine
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Indiana University school of Medicine
Indianapolis, Indiana, 46202
Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64108
Hassenfeld Children's Hospital at NYU Langone
New York, New York, 10016
Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital
New York, New York, 10032
Seattle Children's Hospital
Seattle, Washington, 98105
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, 10467
Turkey (Türkiye)3 sites
Hacettepe İhsan Doğramacı Children's Hospital
Ankara
Akdeniz University Medical Faculty
Antalya
Istanbul University Istanbul Medical Faculty Hospital
Istanbul
Canada2 sites
CHU Sainte-Justine
Montreal
The Hospital for Sick Children
Toronto
China2 sites
Guangzhou Women And Childrens Medical Center
Guangdong
Children's Hospital of Fudan University
Shanghai
Germany2 sites
Charité - Universitätsmedizin Berlin
Berlin
University Children´s Hospital Tuebingen
Tübingen
Italy2 sites
ASST Papa Giovanni XXIII
Bergamo
Azienda Ospedaliera di Padova
Padova
Malaysia2 sites
Hospital Raja Perempuan Zainab II
Kota Bharu
University Malaya Medical Centre
Kuala Lumpur, 59100
New Zealand1 site
Starship Child Health, Te Whatu Ora - Health New Zealand
Auckland, 1023
About this study
Official title: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
Read the detailed description
This is a Phase 3, multi-center, open-label extension study to evaluate the long-term efficacy and safety of odevixibat in patients with Biliary Atresia (BA). Patients who completed treatment in the A4250-011 BOLD study and meet eligibility criteria for Study A4250-016 (BOLD-EXT) can participate. The duration of the treatment period is 104 weeks, followed by a 4-week Safety Follow-up Period. Patients who wish to continue receiving odevixibat after 104 weeks can remain on treatment in the optional extension period (OEP).
Up to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the Middle East and Asia-Pacific.
- Conditions
- Biliary Atresia
- Being tested
- Odevixibat
- 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
Also called A4250
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).
| Study group | Role | What they receive |
|---|---|---|
| Odevixibat (A4250) | Experimental | Capsules for oral administration once daily for 104 weeks. Receives: Drug: Odevixibat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients who are alive and have not undergone liver transplant
Measured: From baseline to Week 104
Secondary outcomes (8)
Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score
The APRI (aspartate aminotransferase to platelet ratio index) score estimates the severity of liver fibrosis. A higher score indicates a greater amount of scarring (fibrosis) in the liver.
- A score ≤ 0.5 indicates a low likelihood of fibrosis
- A score ≥ 1.5 indicates a higher probability of fibrosis
Measured: From baseline to Week 104
Change in Fibrosis-4 (Fib-4) score
The Fib-4 score provides an estimate of the amount of scarring (fibrosis) in the liver. A higher score indicates a greater amount of scarring in the liver.
- A score < 1.45 indicates a low likelihood of fibrosis
- A score > 3.25 indicates a higher probability of fibrosis
Measured: From baseline to Week 104
Change in serum bile acids
Measured: From Baseline to Weeks 26, 52, 78, and 104
Change in height
Measured: From Baseline to Weeks 26, 52, 78 and 104
Change in weight
Measured: From Baseline to Weeks 26, 52, 78 and 104
Change in mid-arm circumference
Measure of growth
Measured: From Baseline to Weeks 26, 52, 78 and 104
Time to onset of any sentinel events
Measured: From Baseline to Week 104
Change in pediatric end-stage liver disease (PELD) score
The PELD (pediatric end-stage liver disease) score is a measure of disease severity. PELD scores can range from a negative value (such as -10) to high numbers (such as 45). A higher score indicates more severe disease.
Measured: From Baseline to Weeks 26, 52, 78 and 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2022
- Study start5 Jul 2022
- Primary completion28 Oct 2026 (estimated)
- Study completion28 Oct 2026 (estimated)
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 180 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Albireo, an Ipsen Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05426733
- Sponsor's protocol ID
- A4250-016
- Other IDs
- 2022-501090-39-00
- Acronym
- BOLD-EXT
- Status last verified
- Oct 2026
- First submitted
- 13 Jun 2022
- Last updated
- 23 Oct 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.