Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients.

摘要: Introduction rIX-FP is a coagulation factor IX (recombinant), albumin fusion protein with more than fivefold half-life prolongation over other standard (FIX) products available on the market. Aim This prospective phase II, open-label study evaluated safety and efficacy of rIX-FP for prevention bleeding episodes during weekly prophylaxis assessed haemostatic on-demand treatment in previously treated patients haemophilia B. Methods The consisted 10–14 day evaluation pharmacokinetics (PK), an 11 month period subjects receiving treatment. Safety was by occurrence related adverse events, immunogenic including development inhibitors. Efficacy annualized spontaneous rate (AsBR), number injections to achieve haemostasis. Results Seventeen participated study, 13 received 4 episodic only. No inhibitors were detected any subject. The mean median AsBR 1.25, 1.13 respectively arm. All 1 or 2 rIX-FP. Three who demand prior entry had >85% reduction compared entry. Conclusion This demonstrated routine prevent episodes. In addition no issues improved PK profile demonstrated.