A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

Phase 2/3 Completed 63 enrolled CSL Behring

Non-randomizedParallel-groupOpen-labelTreatment

Summary

This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.

Timeline

Start: 2012-02
Primary completion: 2014-07
Completion: —

Outcome

Met primary endpoint

paper 100% reduction in median annualized spontaneous bleeding rate (AsBR) and 100% resolution of target joints when subjects switched from on-demand to prophylaxis PMID 26755710 ↗

registry p <0.0001; Wilcoxon signed-rank test NCT01496274 ↗

Publications

Santagostino E, Martinowitz U, Lissitchkov T, Pan-Petesch B, Hanabusa H, Oldenburg J, Boggio L, Negrier C, Pabinger I, von Depka Prondzinski M, Altisent C, Castaman G, Yamamoto K, Alvarez-Roman MT, Voigt C, Blackman N, Jacobs I; PROLONG-9FP Investigators Study Group. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016 Apr 7;127(14):1761-9. doi: 10.1182/blood-2015-09-669234. Epub 2016 Jan 11.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	rIX-FP	Protein / enzyme biologic	40 iu/kg	Intravenous
Subject	rIX-FP	Protein / enzyme biologic	75 iu/kg	Intravenous