An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
- Registration Number
- NCT05426733
- Lead Sponsor
- Albireo, an Ipsen Company
- 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 180
- Completion of the 104-week Treatment Period of Study A4250-011
- Signed informed consent by caregiver
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Odevixibat (A4250) Odevixibat Capsules for oral administration once daily for 104 weeks.
- Primary Outcome Measures
Name Time Method Proportion of patients who are alive and have not undergone liver transplant From baseline to Week 104
- Secondary Outcome Measures
Name Time Method 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 fibrosisChange 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 serum bile acids 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
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.
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 fibrosisTime to onset of any sentinel events From Baseline to Week 104
Trial Locations
- Locations (30)
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Indiana University school of Medicine
🇺🇸Indianapolis, Indiana, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Guangzhou Women And Childrens Medical Center
🇨🇳Guangdong, China
Children's Mercy Hospital and Clinics
🇺🇸Kansas City, Missouri, United States
Royal Children's Hospital
🇦🇺Parkville, Australia
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Children's Hospital of Fudan University
🇨🇳Shanghai, China
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
🇺🇸Bronx, New York, United States
Children's Healhcare of Atlanta- Emory University school of medicine
🇺🇸Atlanta, Georgia, 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
Instytut Pomnik-Centrum Zdrowia Dziecka
🇵🇱Warsaw, Poland
University Malaya Medical Centre
🇲🇾Kuala Lumpur, Malaysia
Starship Child Health, Te Whatu Ora - Health New Zealand
🇳🇿Auckland, New Zealand
University Children´s Hospital Tuebingen
🇩🇪Tuebingen, Germany
Hospital Raja Perempuan Zainab II
🇲🇾Kota Bharu, Malaysia
University Medical Center Groningen
🇳🇱Groningen, Netherlands
Akdeniz University Medical Faculty
🇹🇷Antalya, Turkey
Seoul National University Childrens Hospital
🇰🇷Seoul, Korea, Republic of
The Hospital for Sick Children
🇨🇦Toronto, Canada
Bicêtre Hospital
🇫🇷Le Kremlin-Bicêtre, France
Hacettepe İhsan Doğramacı Children's Hospital
🇹🇷Ankara, Turkey
Istanbul University Istanbul Medical Faculty Hospital
🇹🇷Istanbul, Turkey
CHU Sainte-Justine
🇨🇦Montréal, Canada
ASST Papa Giovanni XXIII
🇮🇹Bergamo, Italy
Azienda Ospedaliera di Padova
🇮🇹Padova, Italy
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany