Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)

Albireo

Status and phase: Completed; Phase 3

Conditions: Alagille Syndrome

Treatments: Drug: Placebo; Drug: Odevixibat

Study type: Interventional

Funder types: Industry

Identifiers

NCT04674761

A4250-012

Details and patient eligibility

About

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

Full description

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

Enrollment: 52 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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52 participants in 2 patient groups, including a placebo group

Odevixibat (A4250)

Experimental group

Description:

Capsules for oral administration once daily for 24 weeks.

Treatment:

Drug: Odevixibat

Placebo

Placebo Comparator group

Description:

Capsules for oral administration (to match active) once daily for 24 weeks.

Treatment:

Drug: Placebo