ASSERT

A Phase 3 double-blind, randomized, Placebo-controlled study of the safety and efficacy of Odevixibat (A4250) in patients with Alagille syndrome

01.05.2021 bis 01.12.2023

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 evaluate the safety and tolerability of odevixibat in patients with ALGS.
Methodology:
This study includes a screening period of up to 56 days, followed by a 24-week treatment period, and a safety Follow-up Period.
Screening Period: Two Screening visits will occur to establish eligibility and provide electronic diary (eDiary) instructions for recording the observer-reported outcomes (ObsRO) and patient reported outcomes (PRO) for pruritus.
Treatment period: On Day 1, patients will be randomized 2:1 to receive odevixibat 120 μg/kg/day or placebo. The treatment period duration is 24 weeks.
Safety Follow-up Period: A Safety Follow-up visit will take place 28 days (±7) following the Week 24/EOT visit or the date of last dose for patients who prematurely discontinue study drug.
Patients who complete the Week 24/EOT visit in this study will be invited to participate in an open-label extension study in which all patients will receive active treatment. Patients who enroll in the open-label extension study will not participate in the Safety Follow-up Period.