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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Single dose of 50 IU/kg of BeneFIX by intravenous infusion within 10 minutes.
Peking Union Medical College Hospital
Beijing, China
Hematology Department,Beijing Children's Hospital, Capital Medical University
Beijing, China
Maximum Observed Plasma Concentration (Cmax)
Time frame: 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)
Time frame: 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)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Time to Reach Cmax (Tmax)
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Volume of Distribution at Steady State (Vss)
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.
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Terminal Phase Rate Constant (Kel)
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.
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Mean Residence Time (MRT)
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.
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Plasma Decay Half-Life (t½)
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Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Systemic Clearance (CL)
CL is a quantitative measure of the rate at which a drug substance is removed from the body.
Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
Incremental Recovery
Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight.
Time frame: Pre-dose, 0.25, 0.5 and 1 hour post-dose
Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE)
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; lifethreatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. TEAE is defined as newly occurring or worsening after first dose.
Time frame: 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)
Clinical laboratory analysis tests included hematology, serium chemistry, prothrombin time and urianalysis. Numbers of subjects with laboratory test abnormalities without regard to baseline abnormality were reported.
Time frame: Baseline up to 96 hours post-dose (Day 5 or early termination)
Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)
Time frame: Baseline up to 96 hours post-dose (Day 5 or early termination)
Number of Participants With Inhibitor Development
Time frame: 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
Time frame: 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
Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.