The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD). Pharmacokinetic Component: PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events. Efficacy Component: Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
22
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Study Site
Sofia, Bulgaria
Study Site
Warsaw, Poland
Study Site
Wroclaw, Poland
Study Site
Barnaul, Russia
Study Site
Lviv, Ukraine
Haemostatic efficacy at time of non-surgical bleeding (NSB) event
Time frame: From Day 1 until final study visit
Haemostatic efficacy overall
Time frame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
Number of treatments with blood product transfusions required to resolve any bleeding event
Time frame: From Day 1 until final study visit
vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)
Time frame: From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
Time frame: From Day 1 until final study visit
Number of spontaneous or traumatic NSB events
Time frame: From Day 1 until final study visit
Pharmacokinetic parameters for vWF and FVIII (PK arm only)
Time frame: Up to 72 hours following infusions on Day 1 and approximately Day 180
Development of FVIII inhibitors
Time frame: From Day 1 until final study visit
Development of vWF inhibitors
Time frame: From Day 1 until final study visit
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