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    Clinical Trial Results:
    Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Pediatric Patients with Severe Hemophilia A

    Summary
    EudraCT number
    		 2017-001531-40
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    02 Nov 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Nov 2020
    First version publication date
    30 Nov 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WIL-30
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Octapharma AG
    Sponsor organisation address
    Seidenstrasse 2, Lachen, Switzerland, CH-8853
    Public contact
    VP, Clinical R&D Haematology, Octapharma AG, +41 554512189, cristina.solomon@octapharma.com
    Scientific contact
    VP, Clinical R&D Haematology, Octapharma AG, +41 554512189, cristina.solomon@octapharma.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    16 Oct 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Nov 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to determine the FVIII:C pharmacokinetics (PK) for Wilate in previously treated patients (PTP) with severe hemophilia A aged 1 to <12 years.
    Protection of trial subjects
    This trial was conducted in accordance to the principles of ICH- GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the Declaration of Helsinki and national regulatory requirements. Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and risk factors associated with the investigational medicinal product. Throughout the study safety was assessed, such as of monitoring of AEs, SAEs and concomitant medications.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Nov 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Russian Federation: 3
    Country: Number of subjects enrolled
    Ukraine: 7
    Worldwide total number of subjects
    10
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    10
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Male patients with documented diagnosis of Severe Haemophilia A were screened according to predefined in- and exclusion criteria.

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Wilate
    Arm description
    		 The dosage and frequency of Wilate treatment during the study was determined by the individual patient’s clinical situation. 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.Two patients underwent surgeries treated with Wilate.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Wilate
    Investigational medicinal product code
    Other name
    HUMAN COAGULATION FACTOR VIII, VON WILLEBRAND FACTOR COMPLEX
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    The dosage and frequency of Wilate treatment during the study was determined by the individual patient’s clinical situation. In the 2-day PK Assessment Phase performed before the start of the Prophylactic Treatment, Wilate was administered to all patients as a single dose of 50 ± 5 IU kg BW. In the Prophylactic Treatment Phase, Wilate was to be administered every 2 to 3 days at a dose of 20–40 IU/kg Wilate/kg BW for 6 months (+2 weeks) and at least 50 EDs. In case of unacceptably frequent spontaneous breakthrough BEs the dose of Wilate was to be increased by approximately 5 IU/kg.

    Number of subjects in period 1
    		Wilate
    Started
    10
    Completed
    9
    Not completed
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall Trial Total
    Number of subjects
    		 10 10
    Age categorical
    Units: Subjects
        1 - <6 years
    		 5 5
        6 -<12 years
    		 5 5
    Age continuous
    Units: years
        median (full range (min-max))
    		 7 (2 to 11) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 10 10

    End points

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    End points reporting groups
    Reporting group title
    Wilate
    Reporting group description
    		 The dosage and frequency of Wilate treatment during the study was determined by the individual patient’s clinical situation. 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.Two patients underwent surgeries treated with Wilate.

    Subject analysis set title
    SAF Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The safety (SAF) set included all patients who received at least one infusion of IMP

    Subject analysis set title
    PK Set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK set includes all patients for whom a valid Wilate PK profile was obtained.

    Subject analysis set title
    PP Set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    The PP set, i.e., a subset of the FAS, excludes patients with major protocol deviations that may have an impact on the evaluation of the efficacy outcome parameters. 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.

    Subject analysis set title
    SURG Set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    The surgery (SURG) set is a subset of the FAS, containing all patients who underwent a surgical procedure treated with Wilate during their Prophylactic Treatment Phase.

    Subject analysis set title
    PK Set 1-<6 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    patients in PK Set aged 1-<6 years

    Subject analysis set title
    PK Set 6-<12 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    patients in PK Set aged 6-<12 years

    Subject analysis set title
    FAS Set 1 to <6 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    FAS Set 1 to <6 years

    Subject analysis set title
    FAS Set 6 to <12 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    FAS Set 6 to <12 years

    Subject analysis set title
    FAS Set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The full analysis set (FAS) defined according to the intention-to-treat (ITT) principle will include all enrolled subjects who received at least one infusion of IMP (after the initial PK visit).

    Subject analysis set title
    PP Set 1-<6 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All patients in the PP population aged 1-<6 years. 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

    Subject analysis set title
    PP Set 6-<12 Years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All patients in the PP population aged 6-<12 years. 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

    Primary: Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC) of FVIII:C

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    End point title
    		 Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC) of FVIII:C [1]
    End point description
    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).
    End point type
    Primary
    End point timeframe
    0h, 0.25h, 1h, 6h, 24h and 48 h after the end of injection
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: h*IU/dL
        arithmetic mean (standard deviation)
    720.3 ( 277.3 )
    768.8 ( 288.5 )
    671.9 ( 289.7 )
    No statistical analyses for this end point

    Primary: Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate

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    End point title
    		 Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate [2]
    End point description
    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.
    End point type
    Primary
    End point timeframe
    0h, 0.25h, 1h, 6h, 24h and 48 h after end of injection
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: h*kg*IU/dL/IU
        arithmetic mean (standard deviation)
    14.41 ( 5.55 )
    15.38 ( 5.77 )
    13.44 ( 5.79 )
    No statistical analyses for this end point

    Primary: Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C

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    End point title
    		 Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C [3]
    End point description
    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)
    End point type
    Primary
    End point timeframe
    0h, 0.25h, 1h, 6h, 24h and 48 h after the end of injection
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: IU/dL
        arithmetic mean (standard deviation)
    80.99 ( 20.94 )
    83.0 ( 16.5 )
    78.94 ( 26.56 )
    No statistical analyses for this end point

    Primary: Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C

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    End point title
    		 Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C [4]
    End point description
    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.
    End point type
    Primary
    End point timeframe
    0h, 0.25h, 1h, 6h, 24h and 48 h after the end of injection
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: hours
        arithmetic mean (standard deviation)
    8.82 ( 1.97 )
    8.28 ( 1.51 )
    9.35 ( 2.40 )
    No statistical analyses for this end point

    Primary: harmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C

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    End point title
    		 harmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C [5]
    End point description
    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).
    End point type
    Primary
    End point timeframe
    48 h after the end of injection
    Notes
    [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: hours
        arithmetic mean (standard deviation)
    0.25 ( 0.00 )
    0.25 ( 0.00 )
    0.25 ( 0.00 )
    No statistical analyses for this end point

    Primary: Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C

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    End point title
    		 Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C [6]
    End point description
    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.
    End point type
    Primary
    End point timeframe
    48 h after the end of injection
    Notes
    [6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: hour
        arithmetic mean (standard deviation)
    12.01 ( 2.88 )
    11.47 ( 2.34 )
    12.56 ( 3.53 )
    No statistical analyses for this end point

    Primary: Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C

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    End point title
    		 Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C [7]
    End point description
    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).
    End point type
    Primary
    End point timeframe
    0h, 0.25h, 1h, 6h, 24h and 48 h after the end of injection
    Notes
    [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: dL/kg
        arithmetic mean (standard deviation)
    0.933 ( 0.384 )
    0.784 ( 0.177 )
    1.081 ( 0.494 )
    No statistical analyses for this end point

    Primary: Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C

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    End point title
    		 Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C [8]
    End point description
    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).
    End point type
    Primary
    End point timeframe
    0h, 0.25h, 1h, 6h, 24h and 48 h after the end of injection
    Notes
    [8] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: dL/h/kg
        arithmetic mean (standard deviation)
    0.084 ( 0.053 )
    0.071 ( 0.019 )
    0.098 ( 0.074 )
    No statistical analyses for this end point

    Primary: Incremental In Vivo Recovery (IVR) of FVIII:C

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    End point title
    		 Incremental In Vivo Recovery (IVR) of FVIII:C [9]
    End point description
    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).
    End point type
    Primary
    End point timeframe
    48 h after the end of injection
    Notes
    [9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system does not permit reporting of statistical analyses for studies with only 1 arm/reporting group. Therefore, only results for this endpoint are provided.
    End point values
    		 FAS Set 1 to <6 years FAS Set 6 to <12 years FAS Set
    Number of subjects analysed
    5
    5
    10
    Units: kg/dL
        arithmetic mean (standard deviation)
    1.65 ( 0.33 )
    1.57 ( 0.53 )
    1.61 ( 0.42 )
    No statistical analyses for this end point

    Secondary: Efficacy of prophylactic treatment with Wilate based on Total Annualized Bleeding Rate (TABR)

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    End point title
    		 Efficacy of prophylactic treatment with Wilate based on Total Annualized Bleeding Rate (TABR)
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 FAS Set 1 to <6 years FAS Set 6 to <12 years FAS Set
    Number of subjects analysed
    5
    5
    10
    Units: Bleeding events per year
        arithmetic mean (standard deviation)
    6.47 ( 6.61 )
    10.62 ( 9.06 )
    8.54 ( 7.79 )
    No statistical analyses for this end point

    Secondary: Efficacy of prophylactic treatment with Wilate based on Spontaneous Annualized Bleeding Rate (SABR)

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    End point title
    		 Efficacy of prophylactic treatment with Wilate based on Spontaneous Annualized Bleeding Rate (SABR)
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 FAS Set 1 to <6 years FAS Set 6 to <12 years FAS Set
    Number of subjects analysed
    5
    5
    10
    Units: Spontaneous bleeding events per year
        arithmetic mean (standard deviation)
    2.70 ( 2.93 )
    1.20 ( 2.68 )
    1.95 ( 2.76 )
    No statistical analyses for this end point

    Secondary: Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)

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    End point title
    		 Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
    End point description
    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".
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 PP Set PP Set 1-<6 years PP Set 6-<12 Years
    Number of subjects analysed
    8
    5
    3
    Units: Bleeding Events
    number (not applicable)
        Excellent
    16
    7
    9
        Good
    17
    9
    8
        Moderate
    2
    1
    1
        None
    0
    0
    0
        Total Number of BE
    35
    17
    18
    No statistical analyses for this end point

    Secondary: Wilate Consumption Data: Average Dose of Wilate Per Week of Study

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    End point title
    		 Wilate Consumption Data: Average Dose of Wilate Per Week of Study
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 FAS Set 1 to <6 years FAS Set 6 to <12 years FAS Set
    Number of subjects analysed
    5
    5
    10
    Units: IU/kg per week
        arithmetic mean (standard deviation)
    58.52 ( 14.85 )
    68.08 ( 19.02 )
    63.30 ( 16.86 )
    No statistical analyses for this end point

    Secondary: Incremental in Vivo Recovery (IVR) of Wilate Over Time

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    End point title
    		 Incremental in Vivo Recovery (IVR) of Wilate Over Time
    End point description
    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. (Standardised to 50 IU/kg). 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.
    End point type
    Secondary
    End point timeframe
    Baseline, and 3 and 6 months of treatment
    End point values
    		 FAS Set 1 to <6 years FAS Set 6 to <12 years FAS Set
    Number of subjects analysed
    5
    5
    10
    Units: kg/dL
    arithmetic mean (standard deviation)
        Baseline
    1.65 ( 0.33 )
    1.57 ( 0.53 )
    1.61 ( 0.42 )
        3 months
    1.55 ( 0.14 )
    1.56 ( 0.57 )
    1.56 ( 0.39 )
        6 months
    1.63 ( 0.24 )
    1.32 ( 0.45 )
    1.48 ( 0.38 )
    No statistical analyses for this end point

    Secondary: Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)

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    End point title
    		 Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: p-value (ANOVA)
    number (not applicable)
        Mean square
    0.922
    0.185
    6.100
        p-value
    0.8273
    0.9593
    0.3752
    No statistical analyses for this end point

    Secondary: Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate

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    End point title
    		 Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 PK Set PK Set 1-<6 years PK Set 6-<12 years
    Number of subjects analysed
    10
    5
    5
    Units: p-value (ANOVA)
    number (not applicable)
        Mean Square
    0.003
    0.121
    0.006
        P-Value
    0.9791
    0.8536
    0.9791
    No statistical analyses for this end point

    Secondary: Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months

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    End point title
    		 Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months
    End point description
    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 (estimated along with 95% Pearson-Clopper CIs)
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 SAF Set
    Number of subjects analysed
    10
    Units: number of subjects
        number (confidence interval 95%)
    0 (0 to 30.85)
    No statistical analyses for this end point

    Secondary: Virus Safety Measured by the Number With Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study

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    End point title
    		 Virus Safety Measured by the Number With Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
    End point description
    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
    End point type
    Secondary
    End point timeframe
    6 month
    End point values
    		 FAS Set
    Number of subjects analysed
    10
    Units: number of subjects
        number (not applicable)
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Throughout the Study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    Safety Population (SAF)
    Reporting group description
    		 -

    Serious adverse events
    		 Safety Population (SAF)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 10 (10.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Congenital, familial and genetic disorders
    Cryptorchism
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Safety Population (SAF)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 10 (30.00%)
    Investigations
    Parvovirus B19 test positive
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Infections and infestations
    Respiratory tract infection
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Varicella
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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