Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A
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|ClinicalTrials.gov Identifier: NCT02461992|
Recruitment Status : Completed
First Posted : June 3, 2015
Results First Posted : June 15, 2016
Last Update Posted : September 7, 2016
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A||Drug: Intravenous infusions of Xyntha||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||13 participants|
|Masking:||None (Open Label)|
|Primary Purpose:||Basic Science|
|Official Title:||An Open-label, Single Dose Pharmacokinetic Study Of Xyntha (Moroctocog Alfa (Af-cc), Recombinant Factor Viii) In Male Chinese Subjects With Hemophilia A|
|Study Start Date :||July 2015|
|Actual Primary Completion Date :||August 2015|
|Actual Study Completion Date :||August 2015|
Experimental: Intravenous infusion of Xyntha
Drug: Intravenous infusions of Xyntha
A single dose 50IU/kg dose of Xyntha administered by intravenous infusion within 10 minutes on Day 1
- Maximum Plasma FVIII Activity (Cmax) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]
- Area Under the Plasma FVIII Activity-Time Profile From Time 0 to Time of the Last Quantifiable Concentration (AUClast) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]
- Area Under the Plasma FVIII Activity-Time Profile From Time 0 Extrapolated to Infinite Time (AUCinf) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]
- Time to Reach Maximum Observed Plasma Concentration (Tmax) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]
- Clearance (CL) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.
- Volume of Distribution at Steady-State (Vss) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]Volume of distribution is 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 volume of distribution at steady-state.
- Terminal Phase Rate Constant (Kel) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]Terminal phase rate constant is the absolute value of the slope of a linear regression during the terminal phase of the natural--logarithm transformed concentration--time profile.
- Terminal Elimination Half-Life (t1/2) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]Terminal half-life is the time measured for the plasma concentration to decrease by one half.
- Mean Residence Time (MRT) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]MRT = AUMCinf / AUCinf, where AUMCinf is the area under the first moment curve from zero time to infinity calculated as AUMCinf = AUMCt + ((t x Ct) / kel) + (Ct / kel^2). AUMCt is the area under the first moment curve from zero time to time t calculated using the trapezoidal method.
- Incremental Recovery (INCREC) [ Time Frame: Pre-dose and 0.25, 0.5, 1, 3, 6, 9, 24, 28, 32, 48, and 72 hours post-dose ]Incremental recovery is the increase in circulating FVIII activity for every IU of Xyntha administered per kilogram of body weight.
- Number of Participants With Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Day 28 ]An AE was any untoward medical occurrence in a participant who received study drug. 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; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-SAEs.
- Number of Participants With Laboratory Abnormalities Meeting the Criteria for Potential Clinical Concern [ Time Frame: Baseline up to Day 4 ]The following laboratory parameters were analyzed: hematology (hemoglobin, hematocrit, red blood cell count, RBC morphology, platelet count, white blood cell count, total neutrophils, eosinophils, monocytes, basophils, lymphocytes); blood chemistry (blood urea nitrogen, creatinine, glucose, calcium, sodium, potassium, chloride, total bicarbonate, aspartate aminotransferase, alanine aminotransferase, total bilirubin, alkaline phosphatase, uric acid, albumin, and total protein; urinalysis (pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, urobilinogen, urine bilirubin, microscopy [if urine dipstick was positive for blood, protein, nitrites or leukocyte esterase]); others (urine drug screening, FVIII inhibitor assay, FVIII activity, prothrombin time [PT], activated partial thromboplastin time [APTT], anti-human immunodeficiency virus [HIV] 1, hepatitis C virus antibody [HCVAb], HAVAb, HBsAg, HBsAb, HBcAb). Only parameters which met abnormality criteria are reported.
- Number of Participants With Potentially Clinically Significant Vital Signs Findings [ Time Frame: Baseline up to Day 4 ]Vital signs assessment included pulse rate and blood pressure. Criteria for vital sign values meeting potential clinical concern included: supine pulse rate <50 beats per minute (bpm), >=30 bpm increase from baseline, or >25 bpm decrease from baseline; systolic blood pressure (SBP) <90 milliliters of mercury (mmHg), >=30 mmHg increase from baseline, or >=30 mmHg decrease from baseline; diastolic blood pressure (DBP) <50 mmHg, >=20 mmHg increase from baseline, or >=20 mmHg decrease from baseline.
- Number of Participants With Positive FVIII Inhibitor Activity at Day 4 [ Time Frame: Day 4 ]As with all FVIII products, participants using Xyntha were monitored for the development of FVIII inhibitors. Values >= 0.6 Bethesda Unit (BU) per mL were considered positive results.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02461992
|Phase I Unit, Clinical Pharmacology Research Center, Peking Union Medical College Hospital|
|Beijing, China, 100032|
|Hematology Department,Beijing Children's Hospital, Capital Medical University|
|Beijing, China, 100045|
|Study Director:||Pfizer CT.gov Call Center||Pfizer|