Patients randomized: Patients ≥12 years of age with hemophilia A with inhibitors (N=66) or hemophilia B with inhibitors (N=6) who have experienced at least 5 bleeds during treatment prior to entering the trial.
Study design: A 12-month, international, multicenter, randomized, double-blind, active-controlled, crossover, confirmatory phase 3 trial. Primarily done in the home setting, treatment of bleeding episodes were randomized, either treated with 1 to 3 doses of vatreptacog alfa (340 bleeding episodes) at 80 mcg/kg or 1 to 3 doses of NovoSeven® RT (2227 bleeding episodes) at 90 mcg/kg.
Primary endpoint: Effective bleed control, which was defined as no additional hemostatic treatment needed (other than the original medication) within 12 hours after first dose.
Secondary endpoint: Effective and sustained bleed control at 1 and 2 days after initial dose, number of doses of trial product for each bleed, and changes in pain assessment.4