NCT03659916

Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 116 (actual)
• Sponsor: Albireo, an Ipsen Company · Industry
• Sex: All
• Age: 0 Months – 100 Years
• Healthy volunteers: Not accepted

Summary

Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.

Conditions

• Progressive Familial Intrahepatic Cholestasis

Interventions

Type	Name	Description
DRUG	A4250 (odevixibat)	A4250 is a small molecule and selective inhibitor of IBAT

Timeline

• Start date: 2018-09-28
• Primary completion: 2024-02-15
• Completion: 2025-12-02
• First posted: 2018-09-06
• Last updated: 2026-04-02
• Results posted: 2025-07-10

Locations

41 sites across 15 countries: United States, Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Saudi Arabia, Spain, Sweden, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study