Von Willebrand Disease
Conditions
Keywords
Von Willebrand Disease
Brief summary
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.
Interventions
BIOLOGICALBiostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Sponsors
Parexel
CSL Behring
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosed with VWD * Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available * Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate® * Written informed consent given
Exclusion criteria
(for participation in the PK component): * Actively bleeding immediately prior to initial PK period * Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product * Have Type 2B, 2N or 2M VWD
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharmacokinetic parameters for vWF and FVIII (PK arm only) | Up to 72 hours following infusions on Day 1 and approximately Day 180 |
| Number of spontaneous or traumatic NSB events | From Day 1 until final study visit |
| Haemostatic efficacy overall | 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 | From Day 1 until final study visit |
| Haemostatic efficacy at time of non-surgical bleeding (NSB) event | 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) | From Day 1 until final study visit |
| Assessment of blood loss during any surgical procedure | From Day 1 until final study visit |
Secondary
| Measure | Time frame |
|---|---|
| Development of FVIII inhibitors | From Day 1 until final study visit |
| Development of vWF inhibitors | From Day 1 until final study visit |
Countries
Bulgaria, Poland, Russia, Ukraine
Outcome results
None listed