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Due to recruitment difficulties for pediatric patients. The reason for study cancellation is not related to safety concerns or any adverse events.
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| Name | Class |
|---|---|
| Instituto Grifols, S.A. | INDUSTRY |
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Multicenter, prospective, non-controlled study in a pediatric cohort (<6 years-old) with severe (type 2 or 3) hereditary Von Willebrand Disease (VWD).
This is a multicenter, prospective, open-label, and single-arm study. The study population is planned to include 8 pediatric subjects (<6 years of age) with severe (type 2 or 3) hereditary VWD without inhibitors and with no active bleeding at the time of inclusion. Eligible subjects will receive a single dose of Fanhdi for a PK evaluation and will be followed for 12 months for which the efficacy and safety of Fanhdi will be assessed. In addition, the type 3 VWD subjects, after 6 months of follow-up of the first infusion, will receive the second dose as in the 1st PK evaluation and undergo a 2nd PK evaluation.
The study will consist of 2 phases:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| plasma-derived FVIII/VWF concentrate | Experimental | Pharmacokinetic single dose study with Fanhdi (high-purity Von Willebrand containing FVIII concentrate) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| plasma-derived FVIII/VWF concentrate | Drug | 1 single dose of 80 IU/kg VWF:RCo of Fanhdi will be administered |
|
| Measure | Description | Time Frame |
|---|---|---|
| AUC^0-inf of coagulation factor VIII activity (FVIII:C) | Cumulative area under the concentration time curve extrapolated to infinity of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-inf of von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) | Cumulative area under the concentration time curve extrapolated to infinity of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-inf of von Willebrand factor antigen (VWF:Ag) | Cumulative area under the concentration time curve extrapolated to infinity of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-inf of von Willebrand factor: Collagen binding activity (VWF:CB) | Cumulative area under the concentration time curve extrapolated to infinity of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-T of FVIII:C | Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-T of VWF:RCo | Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-T of VWF:Ag |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Sant Joan de Déu Barcelona | Esplugues de Llobregat | Barcelona | 08950 | Spain | ||
| Hospital Universitario Virgen del RocÃo |
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Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:Ag |
| Prior to the first infusion up to 72 hours postinfusion |
| AUC^0-T of VWF:CB | Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| in vivo recovery of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| in vivo recovery of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| in vivo recovery of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| in vivo recovery of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| Half-life of FVIII:C | Terminal elimination half-life | Prior to the first infusion up to 72 hours postinfusion |
| Half-life of VWF:RCo | Terminal elimination half-life | Prior to the first infusion up to 72 hours postinfusion |
| Half-life of VWF:Ag | Terminal elimination half-life | Prior to the first infusion up to 72 hours postinfusion |
| C^max of FVIII:C | Maximum observed plasma and/or serum concentration of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| C^max of VWF:RCo | Maximum observed plasma and/or serum concentration of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| C^max of VWF:Ag | Maximum observed plasma and/or serum concentration of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| C^max of VWF:CB | Maximum observed plasma and/or serum concentration of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| T^max of FVIII:C | Time of maximum observed plasma and/or serum concentration of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| T^max of VWF:RCo | Time of maximum observed plasma and/or serum concentration of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| T^max of VWF:Ag | Time of maximum observed plasma and/or serum concentration of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| T^max of VWF:CB | Time of maximum observed plasma and/or serum concentration of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| Mean residence time of FVIII:C | Average amount of time that a single molecule of drug stays in the body. | Prior to the first infusion up to 72 hours postinfusion |
| Mean residence time of VWF:RCo | Average amount of time that a single molecule of drug stays in the body of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| Mean residence time of VWF:Ag | Average amount of time that a single molecule of drug stays in the body of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| Mean residence time of VWF:CB | Average amount of time that a single molecule of drug stays in the body of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| Clearance of FVIII:C | Total plasma and/or serum clearance | Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion |
| Clearance of VWF:RCo | Total plasma and/or serum clearance | Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion |
| Clearance of VWF:Ag | Total plasma and/or serum clearance | Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion |
| Clearance of VWF:CB | Total plasma and/or serum clearance | Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion |
| Elimination rate constant of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| Elimination rate constant of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| Elimination rate constant of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| Elimination rate constant of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| Volume of distribution of FVIII:C | Prior to the first infusion up to 72 hours postinfusion |
| Volume of distribution of VWF:RCo | Prior to the first infusion up to 72 hours postinfusion |
| Volume of distribution of VWF:Ag | Prior to the first infusion up to 72 hours postinfusion |
| Volume of distribution of VWF:CB | Prior to the first infusion up to 72 hours postinfusion |
| VWF multimeric pattern | For type 3 VWD subjects | Prior to the first infusion up to 12 hours postinfusion |
| Seville |
| Sevilla |
| 41013 |
| Spain |
| Hospital Universitario La Paz | Madrid | Spain |
| Hospital Universitario Miguel Servet | Zaragoza | Spain |
| 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 |
| D020147 | Coagulation Protein Disorders |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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