Von Willebrand Disease MedDRA version: 9.1 Level: LLT Classification code 10047715 Term: Von Willebrand's disease MedDRA version: 9.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease
Conditions
Interventions
Product Name: Biostate®
Pharmaceutical Form: Powder and solvent for solution for infusion
Other descriptive name: von Willebrand Factor
Concentration unit: IU international unit(s)
Concentration type:
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female subjects who are at least 12 years of age. 2. Diagnosed with VWD where VWF:RCo is =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: For participants of the PK study: 1. Are actively bleeding immediately prior to initial PK period. 2. Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product. 3. Have Type 2B, 2N or 2M VWD. For all subjects: 4. Requiring a VWF product for a planned surgical procedure at enrolment (treatment with Biostate for surgical procedures subsequent to enrolment in the study is allowed but a planned surgery should not be the reason for inclusion into the study). 5. Have received aspirin or other NSAIDs within 7 days prior to their first dose of study product. 6. Have a known history of, or are suspected to have VWF or FVIII inhibitors. 7. Suffering an acute or chronic medical condition, other than VWD, which may, in the opinion of the Investigator, affect the conduct of the study. 8. Have a known or suspected hypersensitivity or previous evidence of severe side effects to Biostate, VWF/FVIII concentrates or human albumin. 9. Subjects with impaired liver function at screening i.e. bilirubin >1.5 x upper limit of normal (ULN) and/or, AST/ALT >2.5 x ULN (referring to the limits of the laboratory that performs the determination). 10. Subjects having evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit. 11. Have participated in a clinical study or used an investigational compound (e.g. a new chemical entity not registered for clinical use) in the three months preceding the first day of study drug administration, or who are planning to enter such a study during the study period. 12. Female subjects who are pregnant, breast-feeding or who have a positive pregnancy test at screening. The Investigator must confirm that a reliable form of contraception will be used for the duration of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To investigate the initial and repeat pharmacokinetic profile of Biostate in subjects with VWD. 2. To assess the haemostatic efficacy of Biostate in subjects with VWD who require a Von Willebrand Factor product to control a non-surgical bleeding (NSB) event. 3. To assess the effectiveness of a prophylaxis regimen as compared to on-demand therapy with Biostate in preventing NSB events.;Secondary Objective: 1. To assess the safety of Biostate used both as on-demand therapy to treat NSB events and as prophylactic therapy. 2. To assess the haemostatic efficacy of Biostate for subjects who undergo surgical procedures during the study period.;Primary end point(s): • Pharmacokinetic (PK) parameters for each measure of VWF and Factor VIII (FVIII) activity will be derived from plasma concentration values collected following: • an initial single dose of Biostate on Day 1. • a repeat single dose in Type 3 subjects after a minimum of 6 months of study participation (with at least 2 doses of Biostate to have been administered since the Day 1 dose). • Assessment of haemostatic efficacy of Biostate in the treatment of a NSB event according to a 4 point ordinal scale. • Number of NSB events per month during the prophylaxis period as compared to the on-demand period. | — |
Countries
Bulgaria
Outcome results
None listed