An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 120
- 试验地点
- 51
- 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 rFIXFc in participants with hemophilia B.
The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.
详细描述
Participants will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the patient in the preceding studies, B-LONG 998HB102 (NCT01027364) and Kids B-LONG study 9HB02PED (NCT01440946)
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
- •Ability to understand the purposes & risks of the study and provide signed and dated informed consent.
排除标准
- •High-titer inhibitor (>/=5.00 BU/mL)
- •NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
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), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (\<6 years and 6 to \<12 years old) and treatment regimen for participants from Study 9HB02PED 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)
- Total Number of Exposure Days (EDs)(Approximately 5 years)
- Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale(Approximately 5 years)
- Annualized Bleeding Rate (ABR)(Approximately 5 years)
- Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale(Approximately 5 years)
- Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])(Approximately 5 years)