Biogen Idec and Swedish Orphan Biovitrum announced positive top-line results for the Kids A-LONG Phase 3 clinical study that evaluated the safety and efficacy of Eloctate (recombinant factor VIII Fc fusion protein) in children with severe hemophilia A.
Kids A-LONG was a global, open-label, multicenter study that evaluated the safety, efficacy and pharmacokinetics of Eloctate in previously treated children <12 years old with severe hemophilia A. Seventy one boys with severe hemophilia A (factor VIII activity <1 IU/dL, or 1%) with at least 50 prior exposure days to factor VIII therapies were included. Overall, 67 participants (94%) completed the study (<6 years old (n=33); 6–11 years old (n=34)). The average time participants spent in the study was 25 weeks and 61 participants received Eloctate infusions on at least 50 separate days to assess inhibitor development. The primary endpoint of the study was to evaluate the frequency of inhibitor (neutralizing antibody) development.
Results showed that no inhibitors were detected in these patients. Efficacy analyses also demonstrated that twice-weekly prophylactic dosing with Eloctate maintained low bleeding rates in children. In the study, the relative increase in half-life in children with severe hemophilia A was consistent with the 1.5-fold increase in half-life seen in the A-LONG study of adults and adolescents. Children treated prophylactically with Eloctate had an overall median ABR of 2.0 and a median ABR for spontaneous bleeds of 0.0. Forty-six percent of participants in the study experienced zero bleeding episodes. Overall, ninety three percent of bleeding episodes were controlled by one to two infusions of Eloctate. Additional analyses of the Kids A-LONG study are ongoing.