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Date: 2010-07-12
Phase: I-IIa
Anouncement: Result
Biotech: Biogen Idec (USA) Swedish Orphan Biovitrum (Sweden)
Product: Long-acting recombinant factor IX Fc fusion protein (rFIXFc)
Disease: hemophilia B
Other: Date July 12, 2010
Study SYN-FIXFc-07-001, a Phase 1/2a study of rFIXFc in 14 previously-treated patients with severe hemophilia B (¡Ü 2 U/dL endogenous FIX), was designed as an open-label, multi-center, dose-escalation study to evaluate the safety and pharmacokinetics of a single dose of rFIXFc given as an intravenous injection.
The dose levels investigated ranged from 1 to 100 IU/kg, which were determined based on non-clinical data and data from factor IX products in clinical use. The primary objective of the study was to assess the safety of rFIXFc at different doses; the secondary objective was to estimate the pharmacokinetic parameters of rFIXFc at doses ranging from 12.5 to 100 IU/kg.

rFIXFc was generally well tolerated in this single-dose study and there were no signs of injection site reactions, inhibitor development or anti-rFIXFc drug antibodies. There have been no reports of drug-related serious adverse events. A total of two adverse events, headache and altered taste, were reported as related to rFIXFc dosing.

Additionally, rFIXFc demonstrated an approximately three-fold increase in half-life (52.5¡À9.2 hours) compared to data reported in the literature for existing factor IX therapies. Other pharmacokinetic parameters such as mean residence time and area under the curve were similarly increased. The incremental recovery of rFIXFc appeared to more closely approximate plasma-derived factor IX, in contrast to results for other recombinant DNA-produced factor IX products. These data support the hypothesis that rFIXFc may provide prolonged protection from bleeding. They have been presented at the World Federation of Hemophilia Congress in Buenos Aires, Argentina, on July 11, 2010.