Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Phase 3 Completed 52 enrolled Albireo

RandomizedParallel-groupTriple-blindTreatment

Summary

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

Timeline

Start: 2021-03-19
Primary completion: 2022-09-09
Completion: 2022-09-09

Outcome

Met primary endpoint

registry analysis (superiority test); Odevixibat (A4250) vs Placebo; p = 0.0012; LS mean difference -0.88 (95% CI -1.44 to -0.33); Mixed Models Analysis NCT04674761 ↗

paper which was significantly greater for odevixibat than for placebo (difference in LS mean change from baseline -0·9 [95% CI -1·4 to -0·3]; p=0·0024). PMID 38670135 ↗

Publications

Ovchinsky N, Aumar M, Baker A, Baumann U, Bufler P, Cananzi M, Czubkowski P, Durmaz O, Fischer R, Indolfi G, Karnsakul WW, Lacaille F, Lee WS, Maggiore G, Rosenthal P, Ruiz M, Sokal E, Sturm E, van der Woerd W, Verkade HJ, Wehrman A, Clemson C, Yu Q, Ni Q, Ruvido J, Manganaro S, Mattsson JP. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):632-645. doi: 10.1016/S2468-1253(24)00074-8. Epub 2024 Apr 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Odevixibat	Small molecule	120 ug/kg	Oral