An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia

Biliary Atresia Clinical Trial

— BOLD-EXT  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05426733
• Other study ID #: A4250-016
• Status: Enrolling by invitation
• Phase: Phase 3
• 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
• 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

Related Conditions & MeSH terms

• Biliary Atresia

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