Results of a phase I/II open‐label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients. (20th May 2015)

• Record Type: Journal Article
• Authors: Martinowitz, U.; Lissitchkov, T.; Lubetsky, A.; Jotov, G.; Barazani‐Brutman, T.; Voigt, C.; Jacobs, I.; Wuerfel, T.; Santagostino, E.
• Is Part Of: Haemophilia. Volume 21:Number 6(2015:Nov.)
• Page Start: 784
• Page End: 790
• Publication Date: 2015-05-20
• Subjects: Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.12721
• Languages: English
• ISSNs: 1351-8216

Abstract

<abstract abstract-type="main" id="hae12721-abs-0001"> <title> <x xml:space="preserve">Abstract</x> </title> <sec id="hae12721-sec-0001" sec-type="section"> <title>Introduction</title> <p>rIX‐FP is a coagulation factor IX (recombinant), albumin fusion protein with more than fivefold half‐life prolongation over other standard factor IX (FIX) products available on the market.</p> </sec> <sec id="hae12721-sec-0002" sec-type="section"> <title>Aim</title> <p>This prospective phase II, open‐label study evaluated the safety and efficacy of rIX‐FP for the prevention of bleeding episodes during weekly prophylaxis and assessed the haemostatic efficacy for on‐demand treatment of bleeding episodes in previously treated patients with haemophilia B.</p> </sec> <sec id="hae12721-sec-0003" sec-type="section"> <title>Methods</title> <p>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 bleeding rate (AsBR), and the number of injections to achieve haemostasis.</p> </sec> <sec id="hae12721-sec-0004" sec-type="section"> <title>Results</title> <p>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 bleeding 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 bleeding rate prior to study entry.</p> </sec> <sec id="hae12721-sec-0005" sec-type="section"> <title>Conclusion</title> <p>This study demonstrated the efficacy for weekly routine prophylaxis of rIX‐FP to prevent spontaneous bleeding episodes and for the treatment of bleeding episodes. In addition no safety issues were detected during the study and an improved PK profile was demonstrated.</p> </sec> </abstract>