Von Willebrand’s disease (VWD) is a common hereditary bleeding disorder. The impaired formation and adhesion of the initial platelet plug is reflected in the prolonged skin bleeding time. In addition, reduced levels of von Willebrand factor:ristocetin cofactor activity, von Willebrand factor antigen, factor VIII coagulation activity, factor VIII antigen, and abnormalities of the multimeric structure of VWF are variably found among the several types and subtypes of VWD. MedDRA version: 7.1 Leve
Conditions
Sponsors
ZLB Behring LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects of any age Clinical and laboratory diagnosis of VWD (VWF:RCo level of =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Known significant hemostatic disorder other than VWD Acquired VWD Known antibodies to FVIII or VWF Known platelet type VWD Emergency surgery or any surgery with a degree of urgency not permitting completion of a pharmacokinetic assessment required by the study protocol History of allergic reaction to Humate-P® Treatment with any other investigational drug in the last four weeks before the entry into the study (with exception of trials concerning anti-HIV agents) Progressive fatal disease/life expectancy of less than 6 months Treatment with DDAVP, cryoprecipitate, whole blood, plasma and plasma derivatives containing substantial quantities of FVIII and/or VWF within 5 days of the pre-surgical pharmacokinetic assessment Subject/family judged unable to comply with study protocol and requirements Currently taking concomitant therapies listed in Section 5.3 of the study protocol Pediatric subjects of insufficient body weight to permit PK sampling Woman in the first 20 weeks of pregnancy Subjects who were previously enrolled and completed this study may not be re-enrolled
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the efficacy and safety of Humate P® in preventing excessive bleeding in pediatric and adult surgical subjects with VWD using individualized dosing based on VWF:RCo and FVIII:C monitoring.;Secondary Objective: To document the pharmacokinetics of Humate-P® in pediatric and adult subjects with various types of VWD. To document the intra- and inter-subject variability in IVR per 1 IU VWF:RCo/kg b.w. over the range of doses (IU/kg) administered. To document the capability of Humate-P® to normalize the coagulation defect in VWD as demonstrated by an increment of the plasma activity of VWF:RCo and FVIII:C. To analyze the actual dosage and duration of treatment in surgical procedures. To analyze the actual dosage and duration of treatment in VWD Type I, II and III subjects. To explore the correlation among VWF:RCo levels, closure time (PFA), FVIII:C levels, CBA and clinical efficacy. ;Primary end point(s): The primary efficacy endpoint is the investigator’s overall hemostatic efficacy assessment based on a four point ordinal scale (excellent, good, moderate/poor, none), to be assessed 24 hours after the last Humate-P® infusion or on Day 14 (whichever is earlier). The primary efficacy analysis will be based on the full analysis dataset. For subjects who withdraw from the study due to lack of efficacy (after pre-surgery loading dose) the efficacy assessment will be assigned as ‘none.’ Subjects receiving desmopressin, cryoprecipitate, or concentrates containing FVIII or VWF other than Humate-P® after the pre-surgical loading dose and before efficacy assessment are considered treatment failures, and an unfavorable response (none) will be assigned unless it is clearly documented that these products were administered for reasons unrelated to efficacy (e.g., pharmacy error). If the assessment at 24 hours after the last infusion is missing, the Day 14 assessment will be used in the analysis instead. If the Day 14 assessment is missing as well, | — |
Countries
Sweden
Outcome results
None listed