A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

Phase 3 Completed 131 enrolled argenx

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.

Timeline

Start: 2019-12-16
Primary completion: 2022-02-03
Completion: 2022-02-03

Outcome

Met primary endpoint

paper 22% (17/78) of patients with chronic immune thrombocytopenia receiving efgartigimod reached the primary endpoint compared with 5% (2/40) of those receiving placebo (p=0·032; PMID 37778358 ↗

registry analysis (superiority test); Efgartigimod vs Placebo; p = 0.0316; Odds Ratio (OR) 4.884 (95% CI 1.007 to 43.591); NCT04188379 ↗

release “In adults with primary ITP, ADVANCE met its primary endpoint” zailaboratory.com ↗

Publications

Broome CM, McDonald V, Miyakawa Y, Carpenedo M, Kuter DJ, Al-Samkari H, Bussel JB, Godar M, Ayguasanosa J, De Beuf K, Rodeghiero F, Michel M, Newland A; ADVANCE Investigator Study Group. Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2023 Nov 4;402(10413):1648-1659. doi: 10.1016/S0140-6736(23)01460-5. Epub 2023 Sep 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Efgartigimod Alfa	Protein / enzyme biologic	10 mg/kg	Intravenous