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

Haemophilia - Tập 21 Số 6 - Trang 784-790 - 2015
Uri Martinowitz1, Toshko Lissitchkov2, Aaron Lubetsky1, Gantcho Jotov2, Tami Brutman‐Barazani1, C. Voigt3, Iris Jacobs3, T. Wuerfel4, Elena Santagostino5
1The Israeli National Hemophilia Center, Chaim Sheba Medical Center, Tel Hashomer, Israel
2Department of Coagulation Disorders and Anemia, SHAT Joan Pavel, Sofia, Bulgaria
3CRD, CSL Behring, King of Prussia, PA, USA
4PRD, CSL Behring GmbH, Marburg, Germany
5Maggiore Hospital Policlinico IRCCS Cà Granda Foundation Milano Italy

Tóm tắt

IntroductionrIX‐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.AimThis 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.MethodsThe 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.ResultsSeventeen 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.ConclusionThis 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.

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Haemophilia

Tập 21 Số 6

784-790

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