Study of Biostate® in Children With Von Willebrand Disease

CSL Behring

Status and phase

Completed

Phase 3

Conditions

Von Willebrand Disease

Treatments

Biological: Biostate

Study type

Interventional

Funder types

Industry

Identifiers

NCT01213446

CSLCT-BIO-08-52

2009-017753-34 (EudraCT Number)

1494

Details and patient eligibility

About

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.

Enrollment

17 patients

Sex

All

Ages

Under 12 years old

Volunteers

No Healthy Volunteers

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