NCT05426733

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

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

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 180 (estimated)
• Sponsor: Albireo, an Ipsen Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Biliary Atresia

Interventions

Type	Name	Description
DRUG	Odevixibat	Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).

Timeline

• Start date: 2022-07-05
• Primary completion: 2028-08-31
• Completion: 2028-08-31
• First posted: 2022-06-22
• Last updated: 2026-04-01

Locations

30 sites across 14 countries: United States, Australia, Canada, China, France, Germany, Italy, Malaysia, Netherlands, New Zealand, Poland, South Korea, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study