A Phase II/III 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
- 2 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 63
- 试验地点
- 49
- 主要终点
- Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized)
研究概览
简要总结
This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.
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
- •On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months
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, kidney or liver dysfunction
- •Recent life-threatening bleeding episode
结局指标
主要结局
Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized)
时间窗: Up to 26 weeks for on-demand regimen, and between 1 and 17 months for prophylaxis regimen.
Subjects in the on-demand arm received on-demand dosing with rIX-FP for up to 26 weeks (on-demand regimen), and then received weekly prophylaxis with rIX-FP for the remainder of the study (prophylaxis regimen). The effectiveness of prophylaxis in comparison to on-demand therapy was investigated by comparing the same subject's annualized spontaneous bleeding rate (AsBR) during the on-demand regimen and during the prophylaxis regimen.
Number of Subjects Developing Inhibitors Against Factor IX (FIX)
时间窗: Up to 27.7 months (maximum)
The number of participants developing inhibitors against factor IX (FIX) along with the 95% Clopper-Pearson confidence interval, are summarized for subjects with 50 or more exposure days (EDs) to rIX-FP, and for all participants in the study.
次要结局
- Area Under the Curve (AUC)(336 hours)
- Clearance of a Single Dose of rIX-FP(336 hours)
- Annualized Spontaneous Bleeding Events Compared Between 7 Day Prophylactic and Extended Regimens(During treatment, between median 240 and 386 days per subject.)
- Investigator's Overall Clinical Assessment of Hemostatic Efficacy for Treatment of Bleeding Episodes, Based on a Four Point Ordinal Scales (Excellent, Good, Moderate, Poor/No Response)(For the duration of the study; median 20.27 months)
- Incremental Recovery of rIX-FP(336 hours)
- Half-life (t1/2) of a Single Dose of rIX-FP(336 hours)
- Investigator's (or Surgeon's) Overall Clinical Assessment of Hemostatic Efficacy for Surgical Prophylaxis, Based on a Four Point Ordinal Scale (Excellent, Good, Moderate, Poor/No Response)(Up to 14 days after surgery)
- Proportion of Bleeding Episodes Requiring One or ≤ Two Injections of rIX-FP to Achieve Hemostasis(For the duration of the study; median 20.27 months.)
- rIX-FP Consumed Per Month While Maintaining Assigned Prophylactic Treatment Interval During Routine Prophylaxis.(Median 269, 240, 386 and 316 days, respectively (see Description))
- The Frequency of Related Adverse Events(For the duration of the study; median 20.27 months.)
- Number of Subjects Developing Antibodies Against rIX-FP(For the duration of the study; median 20.27 months.)