This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
17
PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only. Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition.
Study site
Homyel, Belarus
Study site
Minsk, Belarus
Study site
Tbilisi, Georgia
Study site
Bremen, Germany
Study site
Haemostatic efficacy
Time frame: From Day 1 until final study visit
Incremental Recovery of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Incremental Recovery of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Half-life of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Half-life of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Area under the concentration curve (AUC) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
AUC of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Maximum plasma concentration (Cmax) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Maximum plasma concentration (Cmax) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Minimum plasma concentration (Cmin) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Guatemala City, CP, Guatemala
Study site
Beirut, Lebanon
Study Site
Lviv, Ukraine
Minimum plasma concentration (Cmin) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Time to maximum concentration (tmax) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Time to maximum concentration (tmax) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Mean residence time (MRT) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Mean residence time (MRT) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Clearance (CL) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Clearance (CL) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Volume of distribution of steady state (Vss) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Volume of distribution of steady state (Vss) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Frequency of adverse events (AEs) per infusion
Time frame: 13 months
Severity of AEs per infusion
Time frame: 13 months
Severity of AEs per subject
Time frame: 13 months
Relatedness of AEs per infusion
Time frame: 13 months
Relatedness of AEs per subject
Time frame: 13 months
Development of VWF inhibitors
Time frame: Sample taken at baseline, then every 3 months up to 12 months
Development of FVIII inhibitors
Time frame: Sample taken at baseline, then every 3 months up to 12 months
Frequency of adverse events (AEs) per subject
Time frame: 13 months