1993 Volume 4 Issue 3 Pages 179-188
Phase I and II clinical studies were performed to evaluate the safety, efficacy and pharmacokinetics of recombinant factor VIII (BL-160) in hemophilia A patients. Three and seven patients were enrolled in the phase I and phase II study respectively. The half life and recovery observed with recombinant factor VIII were comparable to those observed with plasma-derived factor VIII (Hemofil M). Forty-four bleeding episodes were treated with recombinant factor VIII during the phase II study, hemostatic effects were excellent in 39 and good in 5 bleeding episodes. There is no evidence of neoantigenicity resulting from potential contaminating proteins used in production steps. No adverse reactions were reported. These data indicate that recombinant factor VIII is safe and efficacious for the treatment of bleeding episodes in hemophilia A.
