Brief Summary
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
Brief Title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat 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.
Up to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the Middle East and Asia-Pacific.
Categories
Completion Date
Completion Date Type
Estimated
Conditions
Biliary Atresia
Eligibility Criteria
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
* 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
Inclusion Criteria
Inclusion Criteria:
* Completion of the 104-week Treatment Period of Study A4250-011
* Signed informed consent by caregiver
* Completion of the 104-week Treatment Period of Study A4250-011
* Signed informed consent by caregiver
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
NCT Id
NCT05426733
Org Class
Industry
Org Full Name
Ipsen
Org Study Id
A4250-016
Overall Status
Enrolling by invitation
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
Primary Outcomes
Outcome Measure
Proportion of patients who are alive and have not undergone liver transplant
Outcome Time Frame
From baseline to Week 104
Secondary Ids
Secondary Id
2022-501090-39-00
Secondary Outcomes
Outcome Description
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
* A score ≤ 0.5 indicates a low likelihood of fibrosis
* A score ≥ 1.5 indicates a higher probability of fibrosis
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score
Outcome Description
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
* A score \< 1.45 indicates a low likelihood of fibrosis
* A score \> 3.25 indicates a higher probability of fibrosis
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Change in Fibrosis-4 (Fib-4) score
Outcome Time Frame
From Baseline to Weeks 26, 52, 78, and 104
Outcome Measure
Change in serum bile acids
Outcome Time Frame
From Baseline to Weeks 26, 52, 78 and 104
Outcome Measure
Change in height
Outcome Time Frame
From Baseline to Weeks 26, 52, 78 and 104
Outcome Measure
Change in weight
Outcome Description
Measure of growth
Outcome Time Frame
From Baseline to Weeks 26, 52, 78 and 104
Outcome Measure
Change in mid-arm circumference
Outcome Time Frame
From Baseline to Week 104
Outcome Measure
Time to onset of any sentinel events
Outcome Description
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.
Outcome Time Frame
From Baseline to Weeks 26, 52, 78 and 104
Outcome Measure
Change in pediatric end-stage liver disease (PELD) score
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
Nadia Ovchinsky
Investigator Email
NOVCHINS@montefiore.org
Investigator Phone