This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
43
Produced from the plasma of human donors, Wilate is presented as a powder or solvent for intravenous injection containing normally 500 IU or 1000 IU human VWF and human FVIII per vial. The ratio between VWF ristocetin co-factor activity (VWF:RCo) and FVIII:C is 1:1. The product contains approximately 100 IU/ml human VWF when reconstituted with 5ml/10mL water for injection with 0.1% polysorbate 80. The specific activity of Wilate is ≥67 IU VWF:RCo/mg protein. The injection or infusion rate should not exceed 2-3mL per minute.
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Republican Research Center for Radiation Medicine and Human Ecology
Homyel, Belarus
Total Annualized Bleeding Rate (TABR)
The TABR was calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds (except menstrual bleeds) occurring in the time period between first dose of the investigational medicinal product (IMP) and the Study Completion Visit, divided by the duration (in years) between first dose of IMP and the Study Completion Visit.
Time frame: 12 months
Comparison of Total Annualized Bleeding Rates (TABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29
Estimated TABR number calculated using a negative binomial counting regression model. Comparison between this number calculated for studies WIL-29 (NCT04053699) and WIL-31. For the comparison of results from WIL-31 to WIL-29 an estimated total annualized bleeding rate was calculated for each cohort, and compared with a negative binomial counting model. As these were estimated rates, there is only one value for each cohort with no measure of spread
Time frame: 12 Months
Spontaneous Annualized Bleeding Rate (SABR)
Spontaneous annualized bleeding rate (SABR) calculated in analogy with TABR
Time frame: 12 months
Comparison of Spontaneous Annualized Bleeding Rates (SABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29.
Estimated SABR number calculated using a negative binomial counting regression model. Comparison between this number calculated for studies WIL-29 (NCT04053699) and WIL-31. For the comparison of results from WIL-31 to WIL-29 an estimated total annualized bleeding rate was calculated for each cohort, and compared with a negative binomial counting model. As these were estimated rates, there is only one value for each cohort with no measure of spread
Time frame: 12 Months
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"Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia
Sofia, Bulgaria
Pediatric Clinic of Haematology and Oncology
Varna, Bulgaria
University Hospital Centre Zagreb
Zagreb, Croatia
Medical Centre Hungarian Defence Forces
Budapest, Hungary
Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ
Debrecen, Hungary
Hotel Dieu de France Hospital
Beirut, Lebanon
American University of Beirut Medical Center
Beirut, Lebanon
Nini Hospital
Tripoli, Lebanon
...and 4 more locations
Wilate Consumption for Prophylaxis (mFAS Population)
Data on the consumption of Wilate (VWF/FVIII IU/kg per month and per week per patient) for prophylactic treatment
Time frame: 12 months
Incremental In Vivo Recovery (IVR) of Von Willebrand Factor Activity (VWF:RCo)
Incremental VWF:RCo IVR of Wilate over time (at baseline and at 1, 2, 3, 6, 9, and 12 months of treatment)
Time frame: From baseline and 12-month visit
Incremental In Vivo Recovery (IVR) of FVIII
FVIII:C of Wilate in pediatric patients (at baseline PK visit) measured by chromogenic assay
Time frame: Baseline and 12-month visit
Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
Treatment efficacy will be assessed at the end of a BE by the patient using predefined criteria of 'Excellent', 'Good', 'Moderate' or 'None'. All effectiveness ratings assessed as either "excellent" or "good" will be considered "successfully treated".
Time frame: 12 months
Wilate Exposure for Prophylaxis (mFAS Population)
Data on the exposure days of Wilate prophylactic treatment
Time frame: 12 months