Von Willebrand disease, type 3, type 2 (except 2N), or severe type 1 MedDRA version: 20.0 Level: LLT Classification code 10055168 Term: Von Willebrand's factor deficiency System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Wilate 500
Product Name: Wilate
Pharmaceutical Form: Lyophilisate and solvent for solution for injection
INN or Proposed INN: Human Coagulation Factor VIII, Von Willebrand Factor Complex
C
Sponsors
Octapharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients aged =6 years at the time of screening 2. VWD type 1 (baseline von Willebrand factor activity [VWF:Ac], ristocetin-based, =65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: 1. Patients currently on prophylaxis or having received prophylaxis within 6 months before screening (as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeding) 2. History, or current suspicion, of VWF or FVIII inhibitors 3. Medical history of a thromboembolic event within 1 year before enrolment 4. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L) 5. Platelet count 10 mg/day), or similar drugs 7. Pregnant or breast-feeding at the time of enrolment 8. Change in hormonal contraception within 6 months before enrolment 9. Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia) 10. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within 4 weeks before enrolment 11. Other coagulation disorders or bleeding disorders due to anatomical reasons 12. Known hypersensitivity to any of the components of the study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to determine the efficacy of Wilate in the prophylactic treatment of previously treated patients with type 3, type 2 (except 2N), or severe type 1 VWD;Secondary Objective: The secondary objectives of this study are to: - Assess the VWF:Ac and VWF:Ag incremental IVR of Wilate over time (at baseline and at 3, 6, 9, and 12 months of treatment) - Assess the safety and tolerability of Wilate ;Primary end point(s): The primary endpoint of this study is to demonstrate that the total annualised bleeding rate (TABR) during prophylactic treatment with Wilate lowers the patients’ TABR observed during on-demand treatment by more than 50%.;Timepoint(s) of evaluation of this end point: Baseline, 3, 6, 9 and 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Spontaneous annualised bleeding rate (SABR) - Wilate consumption data (VWF/FVIII IU/kg per month per patient) for prophylaxis - Incremental VWF:Ac and VWF:Ag IVR of Wilate over time (at baseline and at 3, 6, 9, and 12 months of treatment) - Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study;Timepoint(s) of evaluation of this end point: Baseline, 3, 6, 9 and 12 months | — |
Countries
Belarus, Bulgaria, Croatia, Czech Republic, Hungary, Lebanon, Russian Federation, Ukraine, United States
Contacts
Public ContactClinical Trial Manager
Octapharma AG
Outcome results
None listed