An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 240
- 试验地点
- 4
- 主要终点
- Number of Participants With Any Positive Inhibitor Development
研究概览
简要总结
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
详细描述
Participant will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the participant in the preceding studies A-LONG - 997HA301 (NCT01181128), pediatric study 8HA02PED (NCT01458106), 997HA307 (NCT02083965) and 997HA309 (NCT02502149).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
- •Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).
排除标准
- •Confirmed positive high-titer inhibitor (≥5.00 BU/mL).
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
研究组 & 干预措施
On-Demand
The individual dose of rFVIIIFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor VIII (FVIII) levels.
干预措施: rFVIIIFc (Drug)
Prophylaxis
Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.
干预措施: rFVIIIFc (Drug)
结局指标
主要结局
Number of Participants With Any Positive Inhibitor Development
时间窗: Approximately 5 years
An inhibitor test result greater than or equal to (\>=) 0.6 Bethesda units per milliliter (BU/mL), identified and confirmed by re-testing of a second sample obtained within 2 to 4 weeks, was considered positive. Both tests were to be performed using the Nijmegen-modified Bethesda Assay by the central laboratory. Data was summarized by treatment regimen for participants from 997HA301/997HA307/997HA309 combined and by age cohort (\<6 years and 6 to \<12 years old) and treatment regimen for participants from 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
次要结局
- Annualized Spontaneous Joint Bleeding Episodes(Approximately 5 years)
- Annualized Bleeding Rate (ABR)(Approximately 5 years)
- Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])(Approximately 5 years)
- Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale(Approximately 5 years)
- Total Number of Exposure Days (EDs)(Approximately 5 years)
- Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale(Approximately 5 years)