Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

Phase 2/3 Completed 159 enrolled Baxalta now part of Shire

Non-randomizedParallel-groupOpen-labelPrevention

Summary

To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.

Timeline

Start: 2013-01-31
Primary completion: 2014-07-17
Completion: 2014-07-17

Outcome

Met primary endpoint

registry analysis (unspecified test); Prophylaxis vs On-demand; p <0.0001; Ratio of means 0.10 (95% CI 0.06 to 0.19) NCT01736475 ↗

paper The pivotal study met its primary endpoint: PMID 26157075 ↗

Publications

Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ADVATE	Protein / enzyme biologic	—	—
Subject	BAX 855	Protein / enzyme biologic	—	—