The WIL-33 study aims to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20%) under the age of 6 years, over a period of 12 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A
Tulane University
New Orleans, Louisiana, United States
University Hospital Ostrava Department for Pediatric Medicine
Ostrava, Czechia
University Hospital Motol, Department of Paediatric Haematology and Oncology
Prague, Czechia
Total annualised bleeding rate (tABR) during prophylactic treatment with wilate.
Time frame: Up to 12 months of treatment
Area under the curve (AUC) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
AUC normalised for the administered dose (AUCnorm) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
In vivo half-life (T1/2) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
Maximum plasma concentration (Cmax) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
Time to reach maximum plasma concentration (Tmax) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
Mean residence time (MRT) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
Volume of distribution (Vd) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
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Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie
Duisburg, Germany
IMSP Mother and Child Institute
Chisinau, Moldova
PHI University Clinic for Child Diseases
Skopje, North Macedonia
FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology
Moscow, Russia
Morozovskaya Children's Hospital
Moscow, Russia
Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre"
Lviv, Ukraine
Clearance (CL) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate
Incremental in-vivo recovery (IVR) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time
Time frame: At baseline and at 1, 2, 3, 6, 9, and 12 months of treatment
Efficacy of wilate in the prevention and treatment of spontaneous and traumatic breakthrough BEs based on their rate and the proportion of spontaneous and traumatic BEs successfully treated with wilate
Assessed by the use of a 4-point ordinal haemostatic efficacy scale (excellent - good - moderate - none)
Time frame: Up to 12 months of treatment
The overall efficacy of wilate in perioperative prophylaxis against excessive bleeding as assessed at the end of the postoperative period by the responsible treating investigator
Assessed by the use of a 4-point ordinal haemostatic efficacy scale (excellent - good - moderate - none)
Time frame: Up to 12 months of treatment
wilate consumption data for prophylactic treatment, for on-demand treatment and during surgical prophylaxis
Time frame: Up to 12 months of treatment
Incidence of VWF and FVIII inhibitors
Time frame: Up to 12 months of treatment
Incidence of thromboembolic events
Time frame: Up to 12 months of treatment
Joint Health Status determination by the use of the Hemophilia Joint Health Score, given the patient's age and constitutional development allow this assessment
Time frame: At baseline and at 12 months of treatment
Safety and tolerability of wilate assessed by monitoring adverse events (AEs) throughout the study
Time frame: Up to 12 months of treatment