An Open-label, Single Dose Pharmacokinetic Study Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Male Chinese Subjects With Hemophilia B
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Systemic Clearance (CL)
研究概览
简要总结
The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male Chinese subjects 6 years or older (weight ≥20kg) with moderate to severe hemophilia B (Factor IX activity ≤2%).
- •Subjects should not have received an infusion of any Factor IX products for at least 4 days before the administration of BeneFIX on Day
- •Subjects must be in a non-bleeding state before the administration of BeneFIX on Day 1.
排除标准
- •Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) disease or clinical findings at Screening.
- •Diagnosed with any other bleeding disorder in addition to hemophilia B.
- •Current FIX inhibitor or history of FIX inhibitor (defined as > Upper Limit of Normal (ULN) of the reporting lab).
研究组 & 干预措施
BeneFIX
干预措施: BENEFIX (Drug)
结局指标
主要结局
Systemic Clearance (CL)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
CL is a quantitative measure of the rate at which a drug substance is removed from the body.
Maximum Observed Plasma Concentration (Cmax)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Time to Reach Cmax (Tmax)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Plasma Decay Half-Life (t½)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.
Incremental Recovery
时间窗: Pre-dose, 0.25, 0.5 and 1 hour post-dose
Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight.
Volume of Distribution at Steady State (Vss)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steady-state.
Terminal Phase Rate Constant (Kel)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Linear regression of the log linear concentration time curve. Only those data points judged to describe the terminal log linear decline were used in the regression.
Mean Residence Time (MRT)
时间窗: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method.
次要结局
- Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE)(From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.)
- Number of Participants With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)(Baseline up to 96 hours post-dose (Day 5 or early termination))
- Number of Participants With Inhibitor Development(From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.)
- Number of Participants With Allergic Reactions(From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.)
- Number of Subjects With Thrombogenicity(From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.)
- Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)(Baseline up to 96 hours post-dose (Day 5 or early termination))