Biliary Atresia Clinical Trial
— BOLD-EXTOfficial title:
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
Verified date | May 2024 |
Source | Ipsen |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
Status | Enrolling by invitation |
Enrollment | 180 |
Est. completion date | December 31, 2026 |
Est. primary completion date | December 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
Australia | Royal Children's Hospital | Parkville | |
Canada | CHU Sainte-Justine | Montréal | |
Canada | The Hospital for Sick Children | Toronto | |
France | Bicêtre Hospital | Le Kremlin-Bicêtre | |
Germany | Charité - Universitätsmedizin Berlin | Berlin | |
Germany | University Children´s Hospital Tuebingen | Tuebingen | |
Italy | ASST Papa Giovanni XXIII | Bergamo | |
Malaysia | University Malaya Medical Centre | Kuala Lumpur | |
Netherlands | University Medical Center Groningen | Groningen | |
New Zealand | Starship Child Health, Te Whatu Ora - Health New Zealand | Auckland | |
Poland | Instytut Pomnik-Centrum Zdrowia Dziecka | Warsaw | |
Spain | Hospital Universitari Vall d'Hebron | Barcelona | |
Turkey | Hacettepe Ihsan Dogramaci Children's Hospital | Ankara | |
Turkey | Akdeniz University Medical Faculty | Antalya | |
Turkey | Istanbul University Istanbul Medical Faculty Hospital | Istanbul | |
United States | Children's Healhcare of Atlanta- Emory University school of medicine | Atlanta | Georgia |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | The Childrens Hospital at Montefiore Albert Einstein School of Medicine | Bronx | New York |
United States | Indiana University school of Medicine | Indianapolis | Indiana |
United States | Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital | New York | New York |
United States | Hassenfeld Children's Hospital at NYU Langone | New York | New York |
United States | UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Seattle Children's Hospital | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
Albireo |
United States, Australia, Canada, France, Germany, Italy, Malaysia, Netherlands, New Zealand, Poland, Spain, Turkey,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Proportion of patients who are alive and have not undergone liver transplant | From baseline to Week 104 | ||
Secondary | 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 |
From baseline to Week 104 | |
Secondary | 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 |
From baseline to Week 104 | |
Secondary | Change in serum bile acids | From Baseline to Weeks 26, 52, 78, and 104 | ||
Secondary | Change in height | From Baseline to Weeks 26, 52, 78 and 104 | ||
Secondary | Change in weight | From Baseline to Weeks 26, 52, 78 and 104 | ||
Secondary | Change in mid-arm circumference | Measure of growth | From Baseline to Weeks 26, 52, 78 and 104 | |
Secondary | Time to onset of any sentinel events | From Baseline to Week 104 | ||
Secondary | 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. | From Baseline to Weeks 26, 52, 78 and 104 |
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