NCT01488994已完成2 期
BAX 326 (Recombinant Factor IX): A Phase 2/3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity in Previously Treated Pediatric Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level 1-2%) Hemophilia B
Baxalta now part of Shire22 个研究点 分布在 6 个国家目标入组 23 人开始时间: 2011年12月20日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 23
- 试验地点
- 22
- 主要终点
- Adverse Events (AEs) Possibly or Probably Related to BAX326
研究概览
简要总结
The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
详细描述
The secondary outcome measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours (h) Post-infusion analysis was not done due to the different time-points for the last PK blood sample, AUC0-72 h was redundant and only total AUC was included in the PK analysis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Main Inclusion Criteria:
- •Participant and/or legal representative has/have voluntarily provided signed informed consent
- •Participant has severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B
- •Participant is < 12 years old at the time of screening
- •Participant has no evidence of a history of FIX inhibitors (based on the participant's medical records)
- •Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm^3
排除标准
- •Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 Bethesda Unit (BU)
- •Participant has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
- •Participant has evidence of an ongoing or recent thrombotic disease
- •Participant has an inherited or acquired hemostatic defect other than hemophilia B
结局指标
主要结局
Adverse Events (AEs) Possibly or Probably Related to BAX326
时间窗: Throughout study period (approximately 17 months)
次要结局
- Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose)(Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.)
- Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL)(Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.)
- Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR)(Throughout study period (approximately 17 months))
- Pharmacokinetics (PK): Mean Residence Time (MRT)(Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.)
- Pharmacokinetics (PK): Incremental Recovery (IR)(Within 30 mins pre-infusion and 30 mins post-infusion)
- Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2)(Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.)
- Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized)(Throughout study period (approximately 17 months))
- Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score(Baseline and 6 months)
- Pharmacokinetics (PK): Incremental Recovery (IR) Over Time(Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26.)
- Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis(Throughout study period (approximately 17 months))
- Safety: Number of Participants With Thrombotic Events(Throughout study period (approximately 17 months))
- Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin)(Throughout study period (approximately 17 months))
- Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose)(Within 30 mins pre-infusion and 4 post-infusion timepoints)
- Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed(Throughout study period (approximately 17 months))
- Consumption of BAX326: Weight-adjusted Consumption Per Month(Throughout study period (approximately 17 months))
- Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX)(Throughout study period (approximately 17 months))
- Health Resource Use: Emergency Room Visits(Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26)
- Consumption of BAX326: Weight-adjusted Consumption Per Event(Throughout study period (approximately 17 months))
- Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss)(Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.)
- Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode(Throughout study period (approximately 17 months))
- Consumption of BAX326: Number of Infusions Per Month(Throughout study period (approximately 17 months))
- Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs(Throughout study period (approximately 17 months))
- Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX)(Throughout study period (approximately 17 months))
- Consumption of BAX326: Number of Infusions Per Year(Throughout study period (approximately 17 months))
- Health Resource Use: Number of Hospitalizations(Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26)
- Health Resource Use: Length of Hospitalization(Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26)
- Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score(Baseline and 6 months)
- Health Resource Use: Unscheduled Doctor's Office Visits(Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26)
- Health Resource Use: Days Lost From School(Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26)
研究者
研究点 (22)
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