Von Willebrand Disease MedDRA version: 14.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Biostate
Pharmaceutical Form: Powder for injection
CAS Number: N/A
Other descriptive name: HUMAN COAGULATION FACTOR VIII
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 between 0 and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Are actively bleeding immediately prior to initial PK period. 2. Have received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first dose of the IMP. 3. Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period. 4. Have a known history of, or are suspected to have VWF or FVIII inhibitors. 5. Suffering an acute or chronic medical condition, other than VWD, which may in the opinion of the Investigator, affect the conduct of the study. 6. Have a known or suspected hypersensitivity or previous evidence of severe side effects to FVIII/VWF concentrates like Biostate. 7. Have participated in a clinical study or used an investigational compound in another study (eg, a new chemical entity not registered for clinical use) in the 3 months preceding the first day of IMP administration, or are planning to enter such a study during the study period. 8. Are not willing and/or not able to comply with the study requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): • Subjective assessment of haemostatic efficacy of Biostate in its usage with a non surgical bleeding (NSB) event, surgical procedure, or use in a prophylaxis regimen (efficacy component of the study). • PK parameters for each VWF and FVIII will be derived from plasma concentration values after an initial single dose of Biostate on Day 1 (PK component of the study). Parameters will also be determined 6 months after the initial dose in Type 3 VWD subjects (repeat PK, recovery study).;Main Objective: • To assess the efficacy of Biostate in paediatric subjects with VWD. • To investigate the PK profile of Biostate in paediatric subjects with VWD.;Secondary Objective: • To assess the safety of Biostate in paediatric subjects with VWD.;Timepoint(s) of evaluation of this end point: •study duration 12 months, assessment at 3-monthly visits •PK samples will be collected prior to dosing with Biostate and then at 0.5, 4, 8, 24, and 48 h after the infusion. At Month 6, PK samples will be collected for Type 3 VWD subjects at pre-dose and 0.5, 4, 8, 24, and 48 h after end of infusion. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •The nature and incidence of AEs. •The development of FVIII and/or VWF inhibitors. ;Timepoint(s) of evaluation of this end point: •study duration 12 months, assessment at 3-monthly visits •The presence of FVIII and VWD inhibitors will be assessed at Screening, Month 3, 6, 9, and 12, and the Final visit. | — |
Countries
Belarus, European Union, Georgia, Germany, Guatemala, Lebanon, Mexico, Ukraine
Contacts
Public ContactSimone Lang
CSL Behring GmbH
Outcome results
None listed