A Phase I/II Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 17
- 试验地点
- 2
- 主要终点
- Number of Subjects Who Developed Antibodies to rIX-FP
研究概览
简要总结
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B. The study consists of a screening period, a pharmacokinetic (PK) period, followed by approximately a 5 month treatment period. Subjects will receive weekly routine prophylactic therapy and on-demand treatment for bleeding episodes. In addition, subjects who are not on routine factor replacement therapy prior to the study will receive only on-demand treatment for bleeding episodes.
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
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects, 12 to 65 years old
- •Severe hemophilia B (FIX activity of ≤ 2%)
- •Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs)
- •No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX
- •Written informed consent for study participation obtained before undergoing any study specific procedures
排除标准
- •Known hypersensitivity to any FIX product or hamster protein
- •Known congenital or acquired coagulation disorder other than congenital FIX deficiency
- •HIV positive subjects with a CD4 count < 200/mm3
- •Low platelet count, abnormal kidney function, or liver disease
- •On-demand subjects experiencing less than 12 or 6 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months, respectively
- •Planned major surgical intervention during the study period
结局指标
主要结局
Number of Subjects Who Developed Antibodies to rIX-FP
时间窗: Pre-dose, Day 10 and Weeks 4, 12, and 20
Antibodies against rIX-FP were detected using a direct binding enzyme-linked immunosorbent assay (ELISA).
Number of Subjects With Treatment-related Adverse Events
时间窗: Approximately 20 weeks
The causal relationship of each adverse event to rIX-FP was assessed by the Investigator.
Number of Subjects With Inhibitors Against Factor IX (FIX)
时间窗: Baseline, Day 10 and Weeks 4, 12 and 20
The presence of inhibitors against FIX was assessed by the central laboratory by a FIX potency assay. To quantify anti-FIX neutralizing antibodies, the Bethesda assay with the Nijmegen modification was used, and the results expressed as Bethesda Units per mL (BU/mL). A positive inhibitor test is \>=0.6 BU/mL.
次要结局
- Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP(Pre-dose and up to 14 days after rIX-FP infusion.)
- Clearance of a Single Dose of rIX-FP(Pre-dose and up to 14 days after rIX-FP infusion)
- Half-life (t1/2) of a Single Dose of rIX-FP(Pre-dose and up to 14 days after infusion)
- Incremental Recovery of rIX-FP at 30 Minutes Following Infusion of rIX-FP(30 minutes after infusion)
- Breakthrough Bleeding Events(Week 9 to approximately Week 20)