Study of Biostate® in Children With Von Willebrand Disease

A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease

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.

Inclusion Criteria:

• Male and female subjects between 0 and <12 years of age
• Diagnosed with VWD Type 1, 2A, or 3
• Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
• von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
• Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization
• Written informed consent given

Exclusion Criteria:

• Active bleeding immediately prior to initial PK period
• Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
• Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
• Known history or suspicion of having VWF or FVIII inhibitors
• Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
• Known or suspected hypersensitivity or previous evidence of severe side effects to other FVIII/VWF concentrates
• Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
• Unwillingness and/or inability to comply with the study requirements