Results of a phase I/II open-label, safety and efficacy trial of @entity4682 (recombinant), albumin fusion protein in XXXX @entity1 .

@entity1 @entity4777 @entity548 @entity4682
INTRODUCTION: rIX-FP is a @entity4682 (recombinant), albumin fusion protein with more than fivefold half-life prolongation over other standard @entity4682 ( @entity4682 ) products available on the market. AIM: This prospective phase II, open-label study evaluated the safety and efficacy of rIX-FP for the prevention of @entity548 episodes during weekly prophylaxis and assessed the haemostatic efficacy for on-demand treatment of @entity548 episodes in previously treated @entity1 with @entity4777 . METHODS: The study consisted of a 10-14  day evaluation of rIX-FP pharmacokinetics (PK), and an 11  month safety and efficacy evaluation period with subjects receiving weekly prophylaxis treatment. Safety was evaluated by the occurrence of related adverse events, and immunogenic events, including development of inhibitors. Efficacy was evaluated by annualized spontaneous @entity548 rate (AsBR), and the number of injections to achieve haemostasis. RESULTS: Seventeen subjects participated in the study, 13 received weekly prophylaxis and 4 received episodic treatment only. No inhibitors were detected in any subject. The mean and median AsBR were 1.25, and 1.13 respectively in the weekly prophylaxis arm. All @entity548 episodes were treated with 1 or 2 injections of rIX-FP. Three prophylaxis subjects who were treated on demand prior to study entry had >85% reduction in AsBR compared to the @entity548 rate prior to study entry. CONCLUSION: This study demonstrated the efficacy for weekly routine prophylaxis of rIX-FP to prevent spontaneous @entity548 episodes and for the treatment of @entity548 episodes. In addition no safety issues were detected during the study and an improved PK profile was demonstrated.
