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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-017753-34 | EudraCT Number | ||
| 1494 | Other Identifier | CSL Behring |
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| Name | Class |
|---|---|
| Parexel | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Biostate | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Biostate | Biological | PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only. Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition. |
| Measure | Description | Time Frame |
|---|---|---|
| Haemostatic efficacy | From Day 1 until final study visit | |
| Incremental Recovery of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose | |
| Incremental Recovery of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 | |
| Half-life of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose | |
| Half-life of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 | |
| Area under the concentration curve (AUC) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose | |
| AUC of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 | |
| Maximum plasma concentration (Cmax) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose | |
| Maximum plasma concentration (Cmax) of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of adverse events (AEs) per infusion | 13 months | |
| Severity of AEs per infusion | 13 months | |
| Severity of AEs per subject |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Program Director, Clinical R&D | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Study site | Homyel | 246040 | Belarus | |||
| Study site |
| ID | Term |
|---|---|
| D014842 | von Willebrand Diseases |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| Minimum plasma concentration (Cmin) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose |
| Minimum plasma concentration (Cmin) of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 |
| Time to maximum concentration (tmax) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose |
| Time to maximum concentration (tmax) of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 |
| Mean residence time (MRT) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose |
| Mean residence time (MRT) of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 |
| Clearance (CL) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose |
| Clearance (CL) of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 |
| Volume of distribution of steady state (Vss) of VWF | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose |
| Volume of distribution of steady state (Vss) of FVIII | Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 |
| 13 months |
| Relatedness of AEs per infusion | 13 months |
| Relatedness of AEs per subject | 13 months |
| Development of VWF inhibitors | Sample taken at baseline, then every 3 months up to 12 months |
| Development of FVIII inhibitors | Sample taken at baseline, then every 3 months up to 12 months |
| Frequency of adverse events (AEs) per subject | 13 months |
| Minsk |
| 223040 |
| Belarus |
| Study site | Tbilisi | 0179 | Georgia |
| Study site | Bremen | 28177 | Germany |
| Study site | Guatemala City | CP | 01010 | Guatemala |
| Study site | Beirut | Lebanon |
| Study Site | Lviv | Ukraine |
| D020147 | Coagulation Protein Disorders |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |