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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT05426733
Other study ID # A4250-016
Secondary ID
Status Enrolling by invitation
Phase Phase 3
First received
Last updated
Start date July 5, 2022
Est. completion date December 31, 2026

Study information

Verified date May 2024
Source Ipsen
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia


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 information / eligibility

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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Odevixibat
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Albireo

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany,  Italy,  Malaysia,  Netherlands,  New Zealand,  Poland,  Spain,  Turkey, 

Outcome

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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