An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
180
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).
Proportion of patients who are alive and have not undergone liver transplant
Time frame: From baseline to Week 104
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
Time frame: 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
Time frame: From baseline to Week 104
Change in serum bile acids
Time frame: From Baseline to Weeks 26, 52, 78, and 104
Change in height
Time frame: From Baseline to Weeks 26, 52, 78 and 104
Change in weight
Time frame: From Baseline to Weeks 26, 52, 78 and 104
Change in mid-arm circumference
Measure of growth
Time frame: From Baseline to Weeks 26, 52, 78 and 104
Time to onset of any sentinel events
Time frame: From Baseline to Week 104
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Children's Hospital Colorado
Aurora, Colorado, United States
Children's Healhcare of Atlanta- Emory University school of medicine
Atlanta, Georgia, United States
Indiana University school of Medicine
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Mercy Hospital and Clinics
Kansas City, Missouri, United States
Hassenfeld Children's Hospital at NYU Langone
New York, New York, United States
Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital
New York, New York, United States
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Seattle Children's Hospital
Seattle, Washington, United States
...and 20 more locations
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.
Time frame: From Baseline to Weeks 26, 52, 78 and 104