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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)

Approved WMORecruiting

Primary Objective (Cohort 1)To demonstrate a sustained effect of A4250 on serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2.Primary Objective (Cohort 2)To evaluate the effect of A4250…

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

Approved WMOPending

This study has been transitioned to CTIS with ID 2023-509028-17-00 check the CTIS register for the current data. To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)