Low Antigenicity Factor VIII

描述（由申请人提供）：Octagen的长期目标是完成其b结构域缺失重组因子VIII （OBI-1）的临床开发并获得FDA批准，这是VIM因子（fVIII）的低抗原性版本。OBI-1是专为治疗患者抑制性，中和抗体人流感病毒。这类“抑制剂”患者包括对人类fVIII难治的先天性血友病患者，以及“获得性血友病”患者。人类fVIII抑制剂的开发是一种严重的、可能危及生命的疾病。目前的治疗方案并不总是有效的，需要改进治疗方法。

DESCRIPTION (provided by applicant): Octagen's broad, long-term objective is to complete the clinical development and secure FDA approval of its b-domain deleted recombinant factor VIII (OBI-1), a low antigenicity version of factor VIM (fVIII). OBI-1 is designed for the treatment of patients with inhibitory, neutralizing antibodies to human fVIII. Such "inhibitor" patients include congenital hemophiliacs who are refractory to human fVIII, as well as patients with "acquired hemophilia." The development of an inhibitor to human fVIII is a serious, potentially life-threatening condition. Current treatment options are not always effective and there is a need for improved therapies. The specific aims of this application are to conduct: 1. an open-label, non-comparative phase II study of the hemostatic efficacy, pharmacokinetics and safety of OBI-1 in non-life and non-limb-threatening bleeding episodes in congenital hemophilia A inhibitor patients, 2. an open-label, non-comparative phase III study of the efficacy and safety of OBI-1 in long-term repeated treatment of hemorrhages congenital hemophilia A inhibitor patients, 3. an open-label, non-comparative phase III study of the efficacy and safety of OBI-1 in the treatment of hemorrhages in patients with acquired hemophilia A, 4. an open-label, non-comparative phase III study of the efficacy and safety of OBI-1 in prophylaxis during surgery in patients with congenital hemophilia A inhibitor patients, 5. an open-label, non-comparative phase III study of the efficacy and safety of OBI-1 in prophylaxis during surgery in patients with acquired hemophilia A, 6. an open-label, non-comparative phase III study of the efficacy and safety of OBI-1 in the treatment of hemorrhages in hemophilia A inhibitor patients and in acquired hemophilia A patients in whom all approved products have failed to control the hemorrhage.