Biliary Atresia
Conditions
Brief summary
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
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.
Interventions
DRUGOdevixibat
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).
Sponsors
Albireo, an Ipsen Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Completion of the 104-week Treatment Period of Study A4250-011 * Signed informed consent by caregiver
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of patients who are alive and have not undergone liver transplant | From baseline to Week 104 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score | From baseline to Week 104 | 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 |
| Change in Fibrosis-4 (Fib-4) score | From baseline to Week 104 | 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 |
| Change in serum bile acids | From Baseline to Weeks 26, 52, 78, and 104 | — |
| Change in height | From Baseline to Weeks 26, 52, 78 and 104 | — |
| Change in weight | From Baseline to Weeks 26, 52, 78 and 104 | — |
| Change in mid-arm circumference | From Baseline to Weeks 26, 52, 78 and 104 | Measure of growth |
| Time to onset of any sentinel events | From Baseline to Week 104 | — |
| Change in pediatric end-stage liver disease (PELD) score | From Baseline to Weeks 26, 52, 78 and 104 | 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. |
Countries
Australia, Canada, China, France, Germany, Italy, Malaysia, Netherlands, New Zealand, Poland, South Korea, Spain, Turkey (Türkiye), United States
Outcome results
None listed