An extension study to evaluate the long-term efficacy and safety of odevixibat (A4250) in children with biliary atresia (BOLD-EXT)
Phase 1
Recruiting
- Conditions
- Biliary AtresiaMedDRA version: 20.0Level: LLTClassification code: 10004653Term: Biliary atresia Class: 10010331Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-501090-39-00
- Lead Sponsor
- Albireo AB
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 184
Inclusion Criteria
1. Completion of the 104-week Treatment Period of Study A4250-011 22. Signed informed consent by the patient’s parent(s) or legal guardian
Exclusion Criteria
1. Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator’s discretion 2. 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 3. Known hypersensitivity to any components of odevixibat 4. 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Secondary Objective: • To demonstrate a sustained effect of odevixibat on serum bile acids and prevention of liver fibrosis in children with BA • To evaluate the effect of odevixibat on lowering serum bile acids in patients who previously received placebo in Study A4250-011 • To evaluate the long-term safety and tolerability of repeated daily doses of odevixibat • To evaluate the effect of odevixibat on growth • To evaluate the effect of odevixibat on sentinel events (defined as clinically significant ascites, bleeding gastroesophageal varices, thrombocytopenia [platelet count <150,000/µL], liver transplant, or death);Primary end point(s): Time from the date of first dose of study treatment to first occurrence of liver transplant, or death;Main Objective: To demonstrate a sustained effect of odevixibat on native liver survival (NLS) in children with biliary atresia (BA) who have completed study A4250-011 (BOLD)
- Secondary Outcome Measures
Name Time Method Secondary end point(s):• Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score from baseline to Weeks 4, 13, 26, 39, 52, 78, and 104 • Change in fibrosis-4 (Fib-4) score from baseline to Weeks 4, 13, 26, 39, 52, 78 and 104 • Change from baseline in serum bile acids at Weeks 26, 52, 78, and 104;Secondary end point(s):• Change in growth from baseline to Weeks 26, 52, 78, and 104 after initiation of odevixibat treatment, defined as the linear growth deficit (height for age, weight for age, and body mass index [BMI]) compared to a standard growth curve (Z-score, standard deviation [SD] from P50); mid-arm circumference;Secondary end point(s):• Time to onset of first sentinel event during the 104-week treatment period.;Secondary end point(s):• Change in pediatric end-stage liver disease (PELD) score from baseline to Weeks 26, 52, 78, and 104;Secondary end point(s):• Proportion of patients who are alive and have not undergone a liver transplant at Weeks 26, 52, 78 and 104