Von Willebrand Disease
Conditions
Keywords
von Willebrand Disease
Brief summary
The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or Factor VIII (FVIII) levels. The long-term efficacy and safety of a VWF/FVIII concentrate, Biostate, will be investigated in children, adolescents, and adults with 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.
Interventions
BIOLOGICALBiostate
Single bolus doses, administered intravenously. 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.
Sponsors
CSL Behring
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Have completed Study CSLCT-BIO-08-52 (Assessment of Efficacy and Safety of Biostate in Paediatric Subjects with Von Willebrand Disease) or Study CSLCT-BIO-08-54 (Assessment of Efficacy and Safety of Biostate in Adolescent or Adult Subjects with Von Willebrand Disease). * The subject and/or his/her legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.
Exclusion criteria
* Early discontinuation of a subject from the main studies CSLCT-BIO-08-52 or CSLCT-BIO-08-54. * Mental condition rendering the subject (or the subject's legal guardian) unable to understand the nature, scope and possible consequences of the study. * Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study. * Are not willing and/or not able to comply with the study requirements. * Employee at the study site, or spouse/partner or relative of the Investigator or Subinvestigators. * Female subjects of childbearing potential either not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study, or not sexually abstinent for the entire duration of the study, or not surgically sterile. * Intention to become pregnant during the course of the study. * Pregnancy, or nursing mother.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Haemostatic efficacy | Up to 32 months |
Secondary
| Measure | Time frame |
|---|---|
| Development of VWF inhibitors | Up to 32 months |
| Frequency of Adverse events (AEs) per subject | 32 months |
| Severity of AEs per subject | 32 months |
| Development of FVIII inhibitors | Up to 32 months |
| Causality of AEs per subject | 32 months |
| Causality of AEs per infusion | 32 months |
| Frequency of Adverse events (AEs) per infusion | 32 months |
| Severity of AEs per infusion | 32 months |
Countries
Bulgaria, Germany, Poland, Russia, Ukraine
Outcome results
None listed