A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

Phase 3 Completed 207 enrolled argenx

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.

Timeline

Start: 2020-12-16
Primary completion: 2023-10-09
Completion: 2023-10-09

Outcome

Missed primary endpoint

paper Superior efficacy of efgartigimod PH20 SC versus placebo was not demonstrated for the primary or secondary endpoints PMID 41943179 ↗

registry p = 0.5081; Difference in percentage -3.5 (95% CI -14.7 to 7.0) NCT04687072 ↗

Publications

Cooper N, Broome CM, Miyakawa Y, McDonald V, Al-Samkari H, Khelif A, Cataland SR, Barcellini W, Yang R, Mei H, Matthijssens F, Hultberg A, Ciurlia G, Gandini D, Ayguasanosa J, De Beuf K, Pastouret S, Ghanima W, Rodeghiero F, Bussel JB; ADVANCE SC Investigator Trial Group. Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial. Am J Hematol. 2026 Jul;101(7):1494-1506. doi: 10.1002/ajh.70303. Epub 2026 Apr 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Efgartigimod Alfa	Protein / enzyme biologic	1000 mg	Subcutaneous