The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
57
Specialized Hospital for Active Treatment "Joan Pavel"
Sofia, Bulgaria
National Haemophilia Centre
Budapest, Hungary
Krakowskie Centrum Medyczne
Krakow, Poland
Korczowski Bartosz Gabinet Lekarski
Rzeszów, Poland
Total Annualized Bleeding Rate (TABR)
The total number of bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
Time frame: 6 months
Spontaneous Annualized Bleeding Rate (SABR)
The number of spontaneous bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
Time frame: 6 months
Efficacy of Wilate in the Treatment of Breakthrough BEs
The proportion of BEs successfully treated with Wilate were documented by the patient (together with the investigator in case of on-site treatments) in the patient diary for all BEs according to a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none', where 'excellent' was defined as "Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection" (best outcome) and 'none' was defined as "No improvement within 12 hours, or worsening of symptoms, requiring more than two injections for complete resolution" (worst outcome). All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated.'
Time frame: 6 months
Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis
The average consumption of Wilate per month of study (IU/kg) for all patients receiving prophylaxis.
Time frame: 6 months
Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C
PK assessments of FVIII:C were conducted using the one-stage (OS) assay. The value of the AUCnorm of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Centrul Medical Unirea -Policlinica Enescu
Bucharest, Romania
Barnaul Branch of RAMS hematology center
Barnaul, Russia
Federal Scientific Hematology Center
Moscow, Russia
Time frame: Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C
PK assessments of FVIII:C were conducted using the one-stage (OS) assay. The in vivo half-life of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
Time frame: Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C
PK assessments of FVIII:C were conducted using the one-stage (OS) assay. The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
Time frame: Initial PK assessment (Day -1) and 6 months
Incremental in Vivo Recovery (IVR) of Wilate Over Time
The rise in FVIII activity in IU/dl per unit dose administered in IU/kg was determined from all patients at baseline, 3 and 6 months, using the OS assay.
Time frame: Baseline, 3 and 6 months
Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
Analysis of variance (ANOVA) was used in an exploratory sense to assess an association between ABO blood type and the FVIII:C half-life of Wilate. This was analyzed by calculating the mean square in a one-stage assay.
Time frame: 6 months
Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
ANOVA was used in an exploratory sense to assess an association between VWF:Ag with the FVIII:C half-life of Wilate. This was analyzed by calculating the mean square in a one-stage assay.
Time frame: 6 months
Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study
At each (scheduled or unscheduled) study visit, AEs were documented by the investigator throughout the study.
Time frame: 6 months
Immunogenicity of Wilate by Testing for FVIII Inhibitors
FVIII inhibitor activity was determined at each study visit before the injection of Wilate using the modified Bethesda assay (Nijmegen modification).
Time frame: 6 months
Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
Virus safety was evaluated by taking a plasma sample for parvovirus B19 antibody testing before the first injection of Wilate. All patients negative at screening were tested again at the study completion visit. The number with Parvovirus B19 seroconversions between BL and end of study was recorded.
Time frame: 6 months