Low Antigenicity Factor VIII

低抗原性因子VIII

• 批准号: 6990357
• 负责人: JOEL MORGANROTH
• 金额: $ 100.42万
• 依托单位: OCTAGEN CORPORATION
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-07-01 至 2008-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6990357
• 关键词: Macaca fascicularis; antigen antibody reaction; antigens; biotherapeutic agent; coagulation factor VIII; drug design /synthesis /production; drug screening /evaluation; hemophilia As; human subject; immunologic substance development /preparation; laboratory mouse; neutralizing antibody; patient oriented research; protein engineering; recombinant proteins

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.

描述（由申请人提供）：Octagen的广泛长期目标是完成其B型域删除的重组因子VIII（OBI-1）的临床开发并获得安全的批准，这是Factor VIM（FVIII）的低抗原性版本。 OBI-1的设计用于治疗抑制性，中和对人FVIII的抗体。这种“抑制剂”患者包括对人FVIII难治性的先天性血友病，以及“获得性血友病”的患者。抑制剂向人类FVIII的发展是一种严重的，潜在的威胁生命的疾病。当前的治疗选择并不总是有效的，需要改善疗法。 本申请的具体目的是进行： 1。对OBI-1在非生命生命和非LIMB威胁性的出血发作中的止血疗效，药代动力学和安全性的开放标签，非舒张期II期研究 2。对OBI-1在长期重复治疗先天性血友病的疗效和安全性的开放标签，非舒张期III期研究 3。对OBI-1的疗效和安全性治疗出血患者的疗效和安全性的开放标签，非舒张期III期研究 4。对OBI-1对先天性血友病患者A抑制剂患者的疗效和安全性的开放标签，非舒张期III期研究 5。对OBI-1在手术期间预防性和安全性在手术中的疗效和安全性的开放标签，非舒张期III期研究 6。对OBI-1在血友病A抑制剂患者和获得性血友病的患者中，对OBI-1治疗OBI-1的疗效和安全性的开放标签，非舒张期III期研究。