BAX 855 Pediatric Study

Phase 3 Completed 75 enrolled Baxalta now part of Shire

Non-randomizedParallel-groupOpen-labelTreatment

Summary

The study purpose is: * To assess the incidence of FVIII inhibitory antibodies during 6 months of twice weekly prophylactic treatment with BAX 855 or 50 exposure days (EDs), whichever occurs last. * To compare pharmacokinetic (PK) parameters to ADVATE. * To assess hemostatic efficacy in prophylaxis and the treatment of bleeding episodes. * To evaluate safety and immunogenicity.

Timeline

Start: 2014-10-31
Primary completion: 2015-10-23
Completion: 2015-10-23

Outcome

Met primary endpoint

release “Results from the study showed that ADYNOVATE met its primary endpoint with no previously treated children having developed inhibitory antibodies to ADYNOVATE.” takeda.com ↗

Publications

Results Mullins ES, Stasyshyn O, Alvarez-Roman MT, Osman D, Liesner R, Engl W, Sharkhawy M, Abbuehl BE. Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A. Haemophilia. 2017 Mar;23(2):238-246. doi: 10.1111/hae.13119. Epub 2016 Nov 27.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT	Protein / enzyme biologic	50 iu/kg	—
Subject	ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT	Protein / enzyme biologic	60 iu/kg	—
Subject	Antihemophilic Factor (Recombinant)	Protein / enzyme biologic	60 iu/kg	—