Von Willebrand Disease Von Willebrand Disease
Conditions
Interventions
This is an open label study to provide VWD patients with Wilate prophylactically.
prophylactic IV Infusion of Wilate
only applicable for patients younger than 17 years
Pharmacokinetic testing
Sponsors
Octapharma AG
Eligibility
Sex/Gender
All
Age
6 Years to 80 Years
Inclusion criteria
Inclusion criteria: Inclusion Criteria: Patients who meet all of the following criteria are eligible for the study: - Aged =6 years at the time of screening - VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (RCo)] <30 IU/dL, 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding - Currently receiving on-demand treatment with a VWF-containing product with at least 1, and an average of =2, documented spontaneous BEs per month in the last 6 months, with at least 2 of these BEs requiring treatment with a VWF-containing product - Availability of records to reliably evaluate type, frequency, and treatment of BEs for at least 6 months of on-demand treatment before screening - Female patients of child-bearing potential must have a negative urine pregnancy test at screening and agree to use adequate birth control measures; in case hormonal contra-ception is used, the medication class should remain unchanged for the duration of the study - All patients to provide voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted
Exclusion criteria
Exclusion criteria: Exclusion Criteria: Patients who meet any of the following criteria are not eligible for the study: - Having received on-demand or prophylactic treatment with a VWF-containing product but having no records available to reliably evaluate the type, frequency, and treatment of BEs over a period of at least 6 months of on-demand treatment - History, or current suspicion, of VWF or FVIII inhibitors - Medical history of a thromboembolic event within 1 year before enrolment - Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate trans-aminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L) - Platelet count 10 mg/day), or similar drugs - Pregnant or breast-feeding at the time of enrolment - Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia) - Treatment with any IMP in another interventional clinical study currently or within 4 weeks before enrolment - Other coagulation disorders or bleeding disorders due to anatomical reasons - Known hypersensitivity to any of the components of the study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Name: total annualised bleeding rate decrease by more than 50%;Timepoints: 12 months after recruitment;Measure: Number of on demand treatment with Von Willebrand Factor Containing product | — |
Secondary
| Measure | Time frame |
|---|---|
| Name: Spontaneous annualised bleeding rate;Timepoints: 1 month, 2 months, 3 months, 6 months, 9 months and 12 months after recruitment;Measure: Number of on demand treatment with Von Willebrand Factor Containing product;Name: Von Willebrand Factor Activity;Timepoints: 1 month, 2 months, 3 months, 6 months, 9 months and 12 months after recruitment;Measure: Blood testing and number of on demand treatment with Von Willebrand containing product;Name: Safety and tolerability of Wilate;Timepoints: From the inclusion of patients till 12 months after recruitment;Measure: Adverse Events experienced by participating patients | — |
Countries
Belarus, Bulgaria, Croatia, Hungary, Lebanon, Russian Federation, Ukraine, United States of America
Contacts
Public ContactHanen Hamid
Ergomed PLC
Outcome results
None listed