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

Primary Objective:To demonstrate the efficacy of repeated daily doses of 120 µg/kg/day odevixibat in relieving pruritus in patients with ALGS.Secondary Objectives:To assess the impact of odevixibat on serum bile acid levels in patients with ALGS.To…

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)

This study has been transitioned to CTIS with ID 2024-512086-14-00 check the CTIS register for the current data. Primary Objective:The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in…

The identification of new polycystic liver disease genes.

The identification of a new gene(s) that causes autosomal dominant polycystic liver disease.