A Phase 3b Open-label, Multicenter, Safety and Efficacy Extension Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 97
- 试验地点
- 79
- 主要终点
- Mean Incremental Recovery of a 50 IU/kg Dose of CSL654 in Previously Untreated Patients (PUPs)
研究概览
简要总结
This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who have not previously been treated with Factor IX products, subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study.
A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery. An additional substudy will examine the safety and PK of subcutaneous (SC) administration of rIX-FP.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 70 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Mean Incremental Recovery of a 50 IU/kg Dose of CSL654 in Previously Untreated Patients (PUPs)
时间窗: Approximately 30 minutes after infusion of CSL654
Incremental Recovery: The increase in plasma concentration per IU/kg of factor administered.
Total Number of Participants Who Developed Inhibitors Against Factor IX (FIX)
时间窗: For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
次要结局
- Total Annualized Bleeding Rate (ABR) by Prophylaxis Regimen in Previously Treated Patients (PTPs)(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Spontaneous ABR by Prophylaxis Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Number of Participants With Investigator's Overall Clinical Assessment of Hemostatic Efficacy for the Treatment of Major Bleeding Events With CSL654 in PUPs(Up to 3 years or the time it takes to achieve 50 EDs)
- Total ABR for On-demand Regimen vs. 14-Day Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Spontaneous ABR for On-demand Regimen vs. 14-Day Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Total ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Average Amount of CSL654 (rIX-FP) Consumed Per Month Per Subject During Routine Prophylaxis Treatment.(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.)
- Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Percentage of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and the Percentage of Participants With at Least One CSL654-related TEAE(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.)
- Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)
- Total ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs(For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).)