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A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All patients | Experimental | All patients will receive Wilate for prophylactic treatment. Patients will also receive Wilate for treatment of breakthrough bleeding events as required |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Wilate | Drug | von Willebrand factor / Factor VIII (plasma derived) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate AUC is hours (h) x international units (IU)/decilitre (dL). | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The mean area under the curve normalised for the administered dose (AUCnorm) was calculated for Wilate. The units of measure used were AUC divided by dose. | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate half-life is hours. | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C | PK assessments of FVIII:C were determined using the one-stage (OS) assays. The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study. Units of measure for maximum plasma concentration are international units (IU)/ decilitre (dL) | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Tmax is hours (h). |
| Measure | Description | Time Frame |
|---|---|---|
| Total Annualized Bleeding Rate (TABR) | The total number of bleeding events (BEs) in the time period between the first dose of IMP and the study completion visit, divided by the duration (in years) between the first dose of IMP and the study completion visit. Surgery periods, and BEs occurring within these periods, will be excluded from the calculation of TABR. | 6 months |
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Inclusion Criteria:
The interval between the Screening Visit and the PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/μL for patients to be enrolled (i.e., inclusion criterion no. 4).
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Cristina Solomon, MD | Octapharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kirov SSC Hematology and Transfusiology | Kirov | Russia | ||||
| "National Children's Specialized Clinic "OKHMATDYT" |
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| ID | Title | Description |
|---|---|---|
| FG000 | Wilate | A total of 11 patients were enrolled in this study. For the pharmacokinetic (PK) assessment a single dose of Wilate (50±5 IU/kg BW) was administered to 10 patients. Prophylactic treatment: Wilate (20-40 IU/kg BW) was administered every 2-3 days for 6 months. In case of unacceptably frequent spontaneous breakthrough bleeding episodes (BEs) the dose of Wilate was to be increased by approximately 5 IU/kg. The dose (and duration) of treatment for breakthrough BEs was dependent on the location and extent of bleeding and on the clinical condition of the patient; range: 10-50 IU/kg every 12-24 hours or 8-24 hours until resolved. Two patients underwent surgery treated with Wilate (SURG population). Minor surgeries received 15-30 IU/kg of Wilate every 24 hours until healing was achieved. Major surgeries were treated with 40-50 IU/kg, repeat injection every 8-24 hours until adequate wound healing, then therapy for at least another 7 days to maintain a FVIII activity of 30% to 60%. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10).
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| ID | Title | Description |
|---|---|---|
| BG000 | Wilate | The safety (SAF) population includes all patients who received at least one injection of Wilate during the study (n=10). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Age at screening (years) | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients aged 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate AUC is hours (h) x international units (IU)/decilitre (dL). | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | h*IU/dL | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
|
Between the screening visit and the follow up contact visit (30 days after the 6-month study completion visit)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Wilate | The safety (SAF) population includes all patients who received at least one injection of Wilate during the study (n=10). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cryptochidism | Congenital, familial and genetic disorders | Systematic Assessment | One patient had a treatment emergent adverse event (TEAE) that was classified as serious because it required hospitalisation for surgery. The adverse event was judged as moderate and not related to Wilate. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Respiratory tract infection | Infections and infestations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sylvia Werner | Octapharma AG | 604-1149 | +1 (201) | sylvia.werner@octapharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 19, 2017 | Dec 21, 2020 | Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 8, 2019 | Dec 21, 2020 | SAP_003.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D014841 | von Willebrand Factor |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
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| 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate MRT is hours. | 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Vd is decilitre (dL)/ kilograms (kg). | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
| Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate clearance are decilitre (dL)/ hours (h)/ kilograms (kg). | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
| Incremental In Vivo Recovery (IVR) of FVIII:C | The incremental IVR was determined from all patients at baseline was determined using the one-stage (OS) assay (standardised to 50 IU/kg). The units of measure to calculate IVR is kilograms (kg) / deciliter (dL) | 48 h following a single dose of Wilate |
| Spontaneous Annualized Bleeding Rate (SABR) | The SABR was calculated in analogy to the TABR. The total number of spontaneous bleeding events (BEs) in the time period between the first dose of IMP and the study completion visit, divided by the duration (in years) between the first dose of IMP and the study completion visit. Surgery periods, and BEs occurring within these periods, will be excluded from the calculation of the SABR. | 6 months |
| Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs) | The proportion of BEs successfully treated with Wilate was assessed by the patient (together with the investigator in case of on-site treatment) in a patient diary. The treatment efficacy for all BEs was assessed using a pre-defined four-point scale: 'excellent', 'good', 'moderate', 'none'. '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); 'good 'was defined as "definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection, requiring up to 2 injections for complete resolution". All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated'. 'Moderate' was defined as "probable or slight beneficial effect within approximately 12 hours after the first injection" and 'none' defined as "no improvement within 12 hours, or worsening of symptoms". | 6 months |
| Wilate Consumption Data: Average Dose of Wilate Per Week of Study | The average consumption of Wilate per week of the study (IU/kg) for all patients receiving prophylaxis | 6 months |
| Incremental in Vivo Recovery (IVR) of Wilate Over Time | The rise in FVIII:C activity in IU/dl per unit dose administered in IU/kg was determined for all patients at baseline, 3 and 6 months, using the one-stage (OS) assay. | Baseline, and 3 and 6 months of treatment |
| 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 a possible association between the ABO blood type and the FVIII:C half-life of Wilate. This was analysed by calculating the mean square in a one-stage (OS) assay. | 6 months |
| Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate | Analysis of variance (ANOVA) was used in an exploratory sense to assess a possible association between VWF:Ag with the FVIII:C half-life of Wilate. This was analysed by calculating the mean square in a one-stage (OS) assay. | 6 months |
| Safety and Tolerability of Wilate by Monitoring The Number of Adverse Events (AEs) Throughout the Study | At each visit (whether scheduled or unscheduled) , AEs will be documented by the investigator throughout the study. In addition, the investigator will check the patient diaries for any documented event. | 6 months |
| Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months | FVIII inhibitor activity was determined at each study visit: screening, PK, Day 14 visit, Day 30 visit, 3 Months visit and 6 Months visit before injection. | 6 months |
| Virus Safety Measured by the Number With 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 at the PK visit. All patients negative at screening were tested again at the Study Completion visit. The number of Parvovirus B19 seroconversions between BL and end of study was recorded | 6 months |
| Kyiv |
| Ukraine |
| "Western Ukrainian Specialized Children's Medical Center" | Lviv | Ukraine |
| Mean |
| Standard Deviation |
| years |
|
| Sex/Gender, Customized | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Count of Participants | Participants |
|
| Race (NIH/OMB) | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Count of Participants | Participants |
|
| Weight | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Mean | Standard Deviation | kg |
|
| Body Mass Index (BMI) | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Mean | Standard Deviation | kg/m^2 |
|
| Blood groups | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Count of Participants | Participants |
|
| Previous Factor (F)VIII treatment | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Count of Participants | Participants |
|
| Previous annualised bleeding rate (ABR) | Previous ABR is based on the bleeding rate in the 6 months prior to entry into the study. | The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Mean | Standard Deviation | Bleeding events per year (ABR) |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Primary | Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The mean area under the curve normalised for the administered dose (AUCnorm) was calculated for Wilate. The units of measure used were AUC divided by dose. | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | h*kg/dL | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
|
|
|
| Primary | Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate half-life is hours. | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | hours | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
|
|
|
| Primary | Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C | PK assessments of FVIII:C were determined using the one-stage (OS) assays. The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study. Units of measure for maximum plasma concentration are international units (IU)/ decilitre (dL) | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | IU/dL | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
|
|
|
| Primary | Pharmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Tmax is hours (h). | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | hours | 48 h following a single dose of Wilate |
|
|
|
| Primary | Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate MRT is hours. | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | hours | 48 h following a single dose of Wilate |
|
|
|
| Primary | Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Vd is decilitre (dL)/ kilograms (kg). | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | dL/kg | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
|
|
|
| Primary | Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C | PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate clearance are decilitre (dL)/ hours (h)/ kilograms (kg). | The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | dL/h/kg | 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate |
|
|
|
| Primary | Incremental In Vivo Recovery (IVR) of FVIII:C | The incremental IVR was determined from all patients at baseline was determined using the one-stage (OS) assay (standardised to 50 IU/kg). The units of measure to calculate IVR is kilograms (kg) / deciliter (dL) | This analysis was performed for the full-analysis (FAS) population which included all patients who has at least one injection of Wilate (n=10). The FAS population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | kg/dL | 48 h following a single dose of Wilate |
|
|
|
| Secondary | Total Annualized Bleeding Rate (TABR) | The total number of bleeding events (BEs) in the time period between the first dose of IMP and the study completion visit, divided by the duration (in years) between the first dose of IMP and the study completion visit. Surgery periods, and BEs occurring within these periods, will be excluded from the calculation of TABR. | This analysis was performed for the full-analysis (FAS) population which included all patients who has at least one injection of Wilate (n=10). The FAS population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | Bleeding events per year (TABR) | 6 months |
|
|
|
| Secondary | Spontaneous Annualized Bleeding Rate (SABR) | The SABR was calculated in analogy to the TABR. The total number of spontaneous bleeding events (BEs) in the time period between the first dose of IMP and the study completion visit, divided by the duration (in years) between the first dose of IMP and the study completion visit. Surgery periods, and BEs occurring within these periods, will be excluded from the calculation of the SABR. | This analysis was performed for the full-analysis (FAS) population which included all patients who has at least one injection of Wilate (n=10). The FAS population comprised 5 patients aged 1-<6 years and 5 patients aged 6-<12 years. | Posted | Mean | Standard Deviation | Spontaneous bleeding events per year | 6 months |
|
|
|
| Secondary | Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs) | The proportion of BEs successfully treated with Wilate was assessed by the patient (together with the investigator in case of on-site treatment) in a patient diary. The treatment efficacy for all BEs was assessed using a pre-defined four-point scale: 'excellent', 'good', 'moderate', 'none'. '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); 'good 'was defined as "definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection, requiring up to 2 injections for complete resolution". All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated'. 'Moderate' was defined as "probable or slight beneficial effect within approximately 12 hours after the first injection" and 'none' defined as "no improvement within 12 hours, or worsening of symptoms". | Analysis was performed in the per-protocol (PP) population. Of the patients in the PP population, only 8 patients had evaluable bleeding events. Five of these patients were aged 1-<6 years, and 3 aged 6-<12 years. | Posted | Count of Units | Bleeding Events (BEs) | 6 months | Bleeding Events (BEs) | Bleeding Events (BEs) |
|
|
|
| Secondary | Wilate Consumption Data: Average Dose of Wilate Per Week of Study | The average consumption of Wilate per week of the study (IU/kg) for all patients receiving prophylaxis | The analysis was performed in the full analysis (FAS) population (total: n=10). The FAS comprised 5 patients were aged 1-<6 years and 5 were aged 6-<12 years. | Posted | Mean | Standard Deviation | IU/kg per week | 6 months |
|
|
|
| Secondary | Incremental in Vivo Recovery (IVR) of Wilate Over Time | The rise in FVIII:C activity in IU/dl per unit dose administered in IU/kg was determined for all patients at baseline, 3 and 6 months, using the one-stage (OS) assay. | The analysis was performed in the full analysis (FAS) population (total: n=10). The FAS comprised 5 patients were aged 1-<6 years and 5 were aged 6-<12 years. | Posted | Mean | Standard Deviation | kg/dL | Baseline, and 3 and 6 months of treatment |
|
|
|
| Secondary | 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 a possible association between the ABO blood type and the FVIII:C half-life of Wilate. This was analysed by calculating the mean square in a one-stage (OS) assay. | The analysis was performed for the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-<6 years and 5 aged 6-<12 years. | Posted | Number | Correlation coefficient | 6 months |
|
|
|
|
| Secondary | Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate | Analysis of variance (ANOVA) was used in an exploratory sense to assess a possible association between VWF:Ag with the FVIII:C half-life of Wilate. This was analysed by calculating the mean square in a one-stage (OS) assay. | The analysis was performed for the PK population which included all patients who underwent PK assessment during the study (total: n=10). | Posted | Number | Correlation coefficient | 6 months |
|
|
|
|
| Secondary | Safety and Tolerability of Wilate by Monitoring The Number of Adverse Events (AEs) Throughout the Study | At each visit (whether scheduled or unscheduled) , AEs will be documented by the investigator throughout the study. In addition, the investigator will check the patient diaries for any documented event. | The safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). | Posted | Number | Adverse events | 6 months |
|
|
|
| Secondary | Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months | FVIII inhibitor activity was determined at each study visit: screening, PK, Day 14 visit, Day 30 visit, 3 Months visit and 6 Months visit before injection. | The analysis was performed in the overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed. | Posted | Count of Participants | Participants | 6 months |
|
|
|
|
| Secondary | Virus Safety Measured by the Number With 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 at the PK visit. All patients negative at screening were tested again at the Study Completion visit. The number of Parvovirus B19 seroconversions between BL and end of study was recorded | Analysis was performed in all patients who underwent full analysis (FAS population) (n=10). | Posted | Number | Participants with seroconversions | 6 months |
|
|
|
| 0 |
| 10 |
| 1 |
| 10 |
| 3 |
| 10 |
|
| Varicella | Infections and infestations | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Parvovirus B19 test positive | Investigations | Systematic Assessment |
|
Not provided
Not provided
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001685 |
| Biological Factors |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| B |
|
| B |
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
|
| Total |
|
|
| Title | Measurements |
|---|---|
|
| Moderate |
|
| None |
|
|
| 6 months |
|
|
| Total |
|
|
P-Value for patients aged 6-<12 years (N=5)
| Other |
| ANOVA | 0.8273 | P-Value for total PK population (N=10) | Other |
|
| Total |
|
|
P-Value for patients aged 6-<12 years (N=5)
| Other |
| ANOVA | 0.9791 | P-Value for total PK population (N=10) | Other |