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    Clinical Trial Results:
    CLINICAL STUDY TO INVESTIGATE THE EFFICACY, PHARMACOKINETICS, IMMUNOGENICITY AND SAFETY OF WILATE IN SEVERE VON WILLEBRAND DISEASE PATIENTS UNDER THE AGE OF 6 YEARS

    Summary
    EudraCT number
    		 2020-004344-28
    Trial protocol
    		 CZ   DE  
    Global end of trial date
    16 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Aug 2025
    First version publication date
    07 Aug 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WIL-33
    Additional study identifiers
    ISRCTN number
    		 ISRCTN11217735
    US NCT number
    		 NCT04953884
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND Number: 011303
    Sponsors
    Sponsor organisation name
    Octapharma Pharmazeutika Produktionsges.m.b.H
    Sponsor organisation address
    Oberlaaerstr. 235, Vienna, Austria, 1100
    Public contact
    Tarja-Elina Weisz, Octapharma Pharmazeutika Produktionsges.m.b.H., tarja-elina.weisz@octapharma.com
    Scientific contact
    Dr. Cristina Solomon, Octapharma AG, 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
    13 Jun 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    16 Dec 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to determine the efficacy of wilate in the prophylactic treatment of up to 12 paediatric patients (eight evaluable) with severe VWD (defined as screening von Willebrand factor ristocetin cofactor activity [VWF:RCo] <20%) under the age of 6 years, for a period of 12 months.
    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 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 monitoring of adverse events, vital signs, bleeding episodes and concomitant medication.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Sep 2021
    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
    Czechia: 3
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Moldova, Republic of: 1
    Country: Number of subjects enrolled
    North Macedonia: 1
    Country: Number of subjects enrolled
    Russian Federation: 2
    Country: Number of subjects enrolled
    Ukraine: 2
    Country: Number of subjects enrolled
    United States: 1
    Worldwide total number of subjects
    12
    EEA total number of subjects
    5
    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)
    2
    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
    		 Children under the age of 6 years with severe von Willebrand disease (VWD) requiring substitution therapy with a VWF-containing product were screened according to predefined in- and exclusion criteria.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Wilate
    Arm description
    		 The VWF/FVIII concentrate wilate, produced from the plasma of human donors, is presented as a powder and solvent for intravenous injection containing nominally 500 IU (international units) or 1000 IU human VWF and human FVIII per vial. Dosing was chosen based on the European Summary of Product Characteristics [16] and adjusted in line with the well described lower IVR and shorter half-life of coagulation factor concentrates in young children.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Wilate
    Investigational medicinal product code
    Other name
    Wilate 500
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    For the PK assessment, a single dose of 80 IU/kg BW wilate was administered, based on the BW measured at the beginning of the visit. The exact dose was calculated according to the nominal potency, with 70–85 IU/kg BW as the acceptable range. IVR assessments were performed following administration of a prophylactic dose, except for baseline IVR, which was done following administration of a single dose of 80 (70–85) IU/kg BW for the PK assessment. In case of unacceptably frequent breakthrough bleeding episodes (BEs) (i.e. two or more BEs within a 30-day period or one major BE), the dose of wilate was to be increased by ~5 IU/kg BW (depending on the vial size of the additional vial(s) that needed to be injected) and/or the treatment frequency increased.

    Number of subjects in period 1
    		Wilate
    Started
    12
    Completed
    11
    Not completed
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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

    Reporting group values
    		 Overall Period Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
        1 year
    		 2 2
        2-3 years
    		 8 8
        4-5 years
    		 2 2
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 6 6

    End points

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    End points reporting groups
    Reporting group title
    Wilate
    Reporting group description
    		 The VWF/FVIII concentrate wilate, produced from the plasma of human donors, is presented as a powder and solvent for intravenous injection containing nominally 500 IU (international units) or 1000 IU human VWF and human FVIII per vial. Dosing was chosen based on the European Summary of Product Characteristics [16] and adjusted in line with the well described lower IVR and shorter half-life of coagulation factor concentrates in young children.

    Subject analysis set title
    Safety Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The safety set (SAF) that included all patients who received at least one dose of wilate.

    Subject analysis set title
    Full Analysis 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, which included all enrolled patients who received at least one dose of wilate after the PK visit.

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    The per-protocol (PP) set, i.e., a subset of the FAS, which excluded patients with major protocol deviations that may have an impacted the evaluation of the primary study outcome parameter(s)

    Subject analysis set title
    BEs (all BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All Bleeding episodes in patients during prophylaxe. (FAS)

    Subject analysis set title
    TABR -mean (all BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Total annualized bleeding rate (FAS)

    Subject analysis set title
    BEs (treated BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All Bleeding episodes in patients treated with wilate (FAS)

    Subject analysis set title
    TABR - mean (treated BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Total annualized bleeding rate in patients treated with wilate (FAS)

    Subject analysis set title
    BEs (all BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All Bleeding episodes in patients during prophylaxis. (PP)

    Subject analysis set title
    TABR - mean (all BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Total Annual bleeding Rate (PP)

    Subject analysis set title
    BEs (treated BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All Bleeding episodes in patients treated with wilate (PP)

    Subject analysis set title
    TABR - mean (treated BEs)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All Bleeding episodes in patients treated with wilate (PP)

    Subject analysis set title
    PK-PP set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    The PK-PP set was a subset of the PK set excluding subjects with a major protocol deviation regarding PK dosing.

    Subject analysis set title
    Surgery Set (SURG)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    The surgery set (SURG) was 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
    VWF:RCo, N=10
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    VWF:RCo

    Subject analysis set title
    FVIII OS, N=8
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    FVIII OS

    Subject analysis set title
    VWF:RCo
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    VWF ristocetin cofactor assay.

    Subject analysis set title
    FVIII (OS)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    FVIII one stage

    Subject analysis set title
    FAS (N=12) [47 BEs]
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Efficacy Assessment of Treatment of BEs with wilate in FAS (N=12)[47 BEs]

    Subject analysis set title
    PP (N=8) [18 BEs]
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Efficacy Assessment of Treatment of BEs with wilate in PP (N=8) [18 BEs]

    Subject analysis set title
    FAS (N=10), 47 BEs
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Consumption of wilate for the Treatment of BEs FAS (N=10), 47 BEs

    Subject analysis set title
    PP (N=7), 18 BEs
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Consumption of wilate for the Treatment of BEs; PP (N=7), 18 BEs

    Subject analysis set title
    Dose per Inj. (IU/kg)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Dose per Inj. (IU/kg)

    Subject analysis set title
    Dose per ED (IU/kg)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Dose per ED (IU/kg)

    Subject analysis set title
    15 min after PK infusion
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    15 min after PK infusion

    Subject analysis set title
    24 h after PK infusion
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    24 h after PK infusion

    Primary: Total annualized bleeding rate (TABR) - FAS

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    End point title
    		 Total annualized bleeding rate (TABR) - FAS [1]
    End point description
    The primary endpoint of this study is to determine the total annualised bleeding rate (TABR) during prophylactic treatment with IMP. The TABR was calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds occurring in the time period between first prophylactic dose of wilate and the study completion visit, divided by the duration (in years) between first prophylactic dose of wilate and the study completion visit.
    End point type
    Primary
    End point timeframe
    Time period between first prophylactic dose of wilate and the study completion visit
    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
    		 BEs (all BEs) TABR -mean (all BEs) BEs (treated BEs) TABR - mean (treated BEs)
    Number of subjects analysed
    12
    12
    12
    12
    Units: number
        number (not applicable)
    56
    4.6
    45
    3.7
    No statistical analyses for this end point

    Primary: Total annualized bleeding rate (TABR) - PP

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    End point title
    		 Total annualized bleeding rate (TABR) - PP [2]
    End point description
    The primary endpoint of this study is to determine the total annualized bleeding rate (TABR) during prophylactic treatment with IMP. The TABR was calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds occurring in the time period between first prophylactic dose of wilate and the study completion visit, divided by the duration (in years) between first prophylactic dose of wilate and the study completion visit.
    End point type
    Primary
    End point timeframe
    time period between first prophylactic dose of wilate and the study completion visit
    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
    		 BEs (all BEs) TABR - mean (all BEs) BEs (treated BEs) TABR - mean (treated BEs)
    Number of subjects analysed
    8
    8
    8
    8
    Units: number
        number (not applicable)
    24
    3.0
    17
    2.1
    No statistical analyses for this end point

    Secondary: PK profile characteristics of wilate (PK-PP)

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    End point title
    		 PK profile characteristics of wilate (PK-PP)
    End point description
    For the PK assessment, a single dose of 80 (70–85) IU/kg BW wilate was to be adminis-tered. All PK assessments were performed according to schedule with respect to time points and dosage in the patients were included in the PK-PP set (N=10).
    End point type
    Secondary
    End point timeframe
    Pre-dose (baseline), 15 min, 3, 9, 24, 48 and 72 h after dosing of 80 IU/kg BW wilate
    End point values
    		 VWF:RCo, N=10 FVIII OS, N=8
    Number of subjects analysed
    10
    8
    Units: value
    arithmetic mean (standard deviation)
        AUC, h*IU/dL
    1100 ( 532 )
    2410 ( 1122 )
        AUCnorm, h*kg*IU/dL/IU
    13.7 ( 6.7 )
    30.1 ( 14.2 )
        CL, dL/h/kg
    0.09 ( 0.04 )
    0.04 ( 0.03 )
        Cmax, IU/dL
    105.0 ( 19.7 )
    114.1 ( 27.1 )
        MRT, h
    15.6 ( 9.5 )
    22.5 ( 9.4 )
        T1/2, h
    11.7 ( 9.6 )
    15.0 ( 7.5 )
        Tmax, h
    0.27 ( 0.04 )
    0.26 ( 0.05 )
        Vd, dL/kg
    1.12 ( 0.24 )
    0.82 ( 0.28 )
    No statistical analyses for this end point

    Secondary: IVR of wilate VWF:RCo over time

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    End point title
    		 IVR of wilate VWF:RCo over time
    End point description
    End point type
    Secondary
    End point timeframe
    In -Vivo Recovery for von Willebrand Factor Activity VWF:RCo Assay over time (at baseline and at 1, 2, 3, 6, 9, and 12 months of treatment)
    End point values
    		 VWF:RCo
    Number of subjects analysed
    12 [3]
    Units: kg/dL
    arithmetic mean (confidence interval 95%)
        Baseline PK (n=12)
    1.261 (1.00 to 1.53)
        1-Month (n=10)
    1.705 (1.42 to 1.99)
        2-Months (n=10)
    1.616 (1.33 to 1.90)
        3-Months (n=11)
    1.627 (1.45 to 1.80)
        6-Months (n=11)
    1.585 (1.45 to 1.72)
        9-Months (n=11)
    1.647 (1.46 to 1.84)
        12-Months/Completion (n=11)
    1.754 (1.58 to 1.92)
    Notes
    [3] - FAS (n=12)
    No statistical analyses for this end point

    Secondary: IVR of Wilate FVIII:C[OS]

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    End point title
    		 IVR of Wilate FVIII:C[OS]
    End point description
    End point type
    Secondary
    End point timeframe
    In -Vivo Recovery for von Willebrand Factor Factor VIII Activity One-Stage Assay over time (at baseline and at 1, 2, 3, 6, 9, and 12 months of treatment)
    End point values
    		 FVIII (OS)
    Number of subjects analysed
    12 [4]
    Units: kg/dL
    arithmetic mean (confidence interval 95%)
        Baseline PK (n=9)
    1.479 (1.23 to 1.73)
        1-Month (n=11)
    1.671 (1.41 to 1.93)
        2-Months (n=9)
    1.795 (1.40 to 2.19)
        3-Months (n=11)
    1.677 (1.39 to 1.97)
        6-Months (n=10)
    1.670 (1.50 to 1.85)
        9-Months (n=9)
    1.789 (1.59 to 1.99)
        12-Months/Completion (n=8)
    1.691 (1.33 to 2.05)
    Notes
    [4] - FAS (n=12)
    No statistical analyses for this end point

    Secondary: Efficacy of wilate in the treatment of BEs

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    End point title
    		 Efficacy of wilate in the treatment of BEs
    End point description
    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 was assessed by a 4-point ordinal haemostatic efficacy scale (excellent – good – moderate – none)
    End point type
    Secondary
    End point timeframe
    Assessed for all treated BEs
    End point values
    		 FAS (N=12) [47 BEs] PP (N=8) [18 BEs]
    Number of subjects analysed
    12
    8
    Units: BEs
    number (not applicable)
        Excellent
    46
    17
        Excellent %
    97.9
    94.4
        Good
    1
    1
        Good %
    2.1
    5.6
        Moderate
    0
    0
        None
    0
    0
        Success
    47
    18
        Success %
    100
    100
        Non-success
    0
    0
    No statistical analyses for this end point

    Secondary: Efficacy of wilate as surgical prophylaxis

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    End point title
    		 Efficacy of wilate as surgical prophylaxis
    End point description
    End point type
    Secondary
    End point timeframe
    Assessed for all treated BEs
    End point values
    		 Surgery Set (SURG)
    Number of subjects analysed
    1
    Units: BEs
    number (not applicable)
        Excellent
    1
        Good
    0
        Moderate
    0
        None
    0
        Sucess %
    100
    No statistical analyses for this end point

    Secondary: Time on prophylaxis (consumption of wilate for prophylaxis)

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    End point title
    		 Time on prophylaxis (consumption of wilate for prophylaxis)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 Full Analysis Set Per Protocol Set
    Number of subjects analysed
    12
    8
    Units: month
        arithmetic mean (standard deviation)
    11.4 ( 3.5 )
    12.2 ( 0.2 )
    No statistical analyses for this end point

    Secondary: Number of EDs (consumption of wilate for prophylaxis)

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    End point title
    		 Number of EDs (consumption of wilate for prophylaxis)
    End point description
    ED = exposure day
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 Full Analysis Set Per Protocol Set
    Number of subjects analysed
    12
    8
    Units: ED
        arithmetic mean (standard deviation)
    109.0 ( 36.1 )
    118.4 ( 15.4 )
    No statistical analyses for this end point

    Secondary: Number of injections (consumption of wilate for prophylaxis)

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    End point title
    		 Number of injections (consumption of wilate for prophylaxis)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 Full Analysis Set Per Protocol Set
    Number of subjects analysed
    12
    8
    Units: number
        arithmetic mean (standard deviation)
    109.0 ( 36.1 )
    118.4 ( 15.4 )
    No statistical analyses for this end point

    Secondary: Dose (consumption of wilate for prophylaxis)

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    End point title
    		 Dose (consumption of wilate for prophylaxis)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 Full Analysis Set Per Protocol Set
    Number of subjects analysed
    12
    8
    Units: IU/kg
    arithmetic mean (standard deviation)
        Dose per injection
    54.0 ( 27.5 )
    42.9 ( 7.0 )
        Dose per week
    120.1 ( 65.0 )
    95.8 ( 19.4 )
    No statistical analyses for this end point

    Secondary: Number of injections per BE (consumption of wilate for on demand treatment)

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    End point title
    		 Number of injections per BE (consumption of wilate for on demand treatment)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 FAS (N=10), 47 BEs PP (N=7), 18 BEs
    Number of subjects analysed
    10
    7
    Units: number
        arithmetic mean (standard deviation)
    1.1 ( 0.2 )
    1.1 ( 0.3 )
    No statistical analyses for this end point

    Secondary: Number of EDs per BE (consumption of wilate for on demand treatment)

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    End point title
    		 Number of EDs per BE (consumption of wilate for on demand treatment)
    End point description
    Number of EDs per BE (ED = exposure day)
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 FAS (N=10), 47 BEs PP (N=7), 18 BEs
    Number of subjects analysed
    10
    7
    Units: ED
        arithmetic mean (standard deviation)
    1.0 ( 0.2 )
    1.1 ( 0.2 )
    No statistical analyses for this end point

    Secondary: Dose (consumption of wilate for on demand treatment)

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    End point title
    		 Dose (consumption of wilate for on demand treatment)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months of treatment
    End point values
    		 FAS (N=10), 47 BEs PP (N=7), 18 BEs
    Number of subjects analysed
    10
    7
    Units: IU/kg
    arithmetic mean (standard deviation)
        Dose per BE
    58.7 ( 27.6 )
    54.2 ( 30.1 )
        Dose per injection
    55.0 ( 22.3 )
    46.8 ( 12.2 )
    No statistical analyses for this end point

    Secondary: Number of injections (consumption of wilate during surgical procedures)

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    End point title
    		 Number of injections (consumption of wilate during surgical procedures)
    End point description
    End point type
    Secondary
    End point timeframe
    during surgical procedure
    End point values
    		 Surgery Set (SURG)
    Number of subjects analysed
    1
    Units: number
    number (not applicable)
        Preoperative (Within 3 h before Start)
    1
        Intraoperative
    0
        Postoperative
    4
        Perioperative
    5
    No statistical analyses for this end point

    Secondary: Number of EDs (consumption of wilate during surgical procedures)

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    End point title
    		 Number of EDs (consumption of wilate during surgical procedures)
    End point description
    End point type
    Secondary
    End point timeframe
    during surgery
    End point values
    		 Surgery Set (SURG)
    Number of subjects analysed
    1
    Units: number
    number (not applicable)
        Preoperative (Within 3 h before Start)
    1
        Intraoperative
    0
        Postoperative
    4
        Perioperative
    4
    No statistical analyses for this end point

    Secondary: Dose (consumption of wilate during surgical procedures)

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    End point title
    		 Dose (consumption of wilate during surgical procedures)
    End point description
    End point type
    Secondary
    End point timeframe
    during surgery
    End point values
    		 Dose per Inj. (IU/kg) Dose per ED (IU/kg)
    Number of subjects analysed
    1
    1
    Units: IU/kg
    number (not applicable)
        Preoperative (Within 3 h before Start)
    62.11
    62.11
        Intraoperative
    0
    0
        Postoperative
    31.06
    31.06
        Perioperative
    37.27
    46.58
    No statistical analyses for this end point

    Secondary: Total Haemophilia Joint Health Score

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    End point title
    		 Total Haemophilia Joint Health Score
    End point description
    Joint health status determination with the HJHS, given that the patient’s age and constitutional development allow this assessment
    End point type
    Secondary
    End point timeframe
    Change from baseline to 12-months
    End point values
    		 Full Analysis Set Per Protocol Set
    Number of subjects analysed
    11 [5]
    7
    Units: number
    arithmetic mean (standard deviation)
        Baseline
    1.55 ( 3.01 )
    2.43 ( 3.55 )
        12-months
    0.80 ( 1.93 )
    1.14 ( 2.27 )
        Change from baseline
    -0.90 ( 1.37 )
    -1.29 ( 1.50 )
    Notes
    [5] - Baseline (N=11) 12-months (N=10) Change from baseline (N=10)
    No statistical analyses for this end point

    Secondary: Incidence of VWF and FVIII inhibitors

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    End point title
    		 Incidence of VWF and FVIII inhibitors
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 12-Months
    End point values
    		 Full Analysis Set
    Number of subjects analysed
    12
    Units: Reference range indicator
    number (not applicable)
        Factor VIII (FVIII) inhibitor positive
    0
    No statistical analyses for this end point

    Secondary: Incidence of thromboembolic events

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    End point title
    		 Incidence of thromboembolic events
    End point description
    End point type
    Secondary
    End point timeframe
    (Baseline up to Study Completion Visit)
    End point values
    		 Full Analysis Set
    Number of subjects analysed
    12
    Units: number
    number (not applicable)
        TEE
    0
    No statistical analyses for this end point

    Secondary: Exploratory endpoint (percentage of Small, Intermediate and Large Multimers)

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    End point title
    		 Exploratory endpoint (percentage of Small, Intermediate and Large Multimers)
    End point description
    The exploratory endpoint of this study, which was solely investigated in the hereditary Type 3 VWD patient group with ≥14.5 kg BW, was a VWF multimer analysis from the PK samples taken at 15 min, and 24 h after wilate injection, by using multimer analysis using low- and high-resolution electrophoresis gels.
    End point type
    Secondary
    End point timeframe
    after wilate Infusion at the PK Assessment
    End point values
    		 15 min after PK infusion 24 h after PK infusion
    Number of subjects analysed
    3
    3
    Units: proportion
    arithmetic mean (standard deviation)
        Small (1–5)
    39.4 ( 6.3 )
    49.2 ( 9.8 )
        Intermediate (6–10)
    45.8 ( 2.3 )
    41.5 ( 7.3 )
        Large (>10)
    14.8 ( 4.1 )
    9.2 ( 2.6 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs have been elicited at each visit (Baseline up to Study Completion Visit) , whether scheduled or unscheduled.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Safety Set ( SAF)
    Reporting group description
    		 The safety set (SAF) included all patients who received at least one dose of wilate.

    Serious adverse events
    		 Safety Set ( SAF)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 12 (33.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Soft tissue infection
         subjects affected / exposed
    1 / 12 (8.33%)
         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 Set ( SAF)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 12 (83.33%)
    Injury, poisoning and procedural complications
    Limb injury
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Lip injury
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Skin laceration
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    4
    General disorders and administration site conditions
    Pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    6
    Gastrointestinal disorders
    Dental caries
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Mouth swelling
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    3
    Toothache
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    6
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Psychiatric disorders
    Attention deficit hyperactivity disorder
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Ear infection
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Pharyngitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Pneumonia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Pulpitis dental
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Respiratory tract infection viral
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Rhinitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Scarlet fever
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Soft tissue infection
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Vitamin D deficiency
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 Aug 2021
    CSP Version 02 (amended protocol): The original study protocol for this study set a minimum weight of 12.5 kg for all patients at the time of screening to meet the blood volume restriction requirements for paediatric patients (based on an allowable blood draw of 1 % of total blood volume per day and a maximum of 3% per month). Given the rare disease in the underlying study, the challenge of identifying patients, and in order to meet the established recruitment timelines, it was decided to lower the minimum weight criterion to 11.0 kg, which allowed inclusion of patients who miss the initial 12.5 kg criterion by the end of the recruitment period. This could only be accompanied by a saving in blood collection volumes, achieved by shifting the protocol-specified time for the retention sample for later possible virus testing to a later time before first treatment and by omitting one test in a specific pharmacokinetic sample for patients with a body weight below 12.5 kg.
    05 Dec 2022
    CSP Version 03 (amended protocol) Further sites have been identified and new countries submitted and so the expected end date of the clinical trial had to be pushed back and was be reset to Q2/2024.

    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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