Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)

Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.

Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Key Inclusion Criteria:

• Genetically confirmed diagnosis of Alagille syndrome
• History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
• Elevated serum bile acid level

Key Exclusion Criteria:

• History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
• History of liver transplant, or a liver transplant is planned within 6 months of randomization
• ALT >10× upper limit of normal (ULN) at screening
• Total bilirubin >15 × ULN at screening
• Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome