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    Clinical Trial Results:
    An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects with Von Willebrand Disease

    Summary
    EudraCT number
    		 2013-003305-25
    Trial protocol
    		 GB   DE   AT   IE   PL   GR  
    Global end of trial date
    15 Feb 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    29 Aug 2018
    First version publication date
    29 Aug 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CSLCT-BIO-12-83
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02552576
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    CSL Behring GmbH
    Sponsor organisation address
    Emil-von-Behring-Str.76, Marburg, Germany, 35041
    Public contact
    Clinical Study Manager, CSL Behring GmbH , clinicaltrials@cslbehring.com
    Scientific contact
    Clinical Study Manager, CSL Behring GmbH , clinicaltrials@cslbehring.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
    07 Mar 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Feb 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to collect long-term data on the haemostatic efficacy of Voncento in subjects with VWD who require a VWF product to control an NSB event or as prophylaxis therapy.
    Protection of trial subjects
    This study was carried out in accordance with the International Council on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) principles of Good Clinical Practice, the Declaration of Helsinki, and with standard operating procedures for clinical research and development at CSL Behring and at the Contract Research Organization involved. The design of the study was discussed and agreed with the Pharmacovigilance Risk Assessment Committee (PRAC), the CHMP, and the Blood Products Working Party (BPWP). The study was planned to comply with the requirements of the EMA CHMP Guideline on the Clinical Investigation of Human Plasma Derived VWF Products (CPMP/BPWG/220/02), specifically in relation to the directives regarding post-marketing studies.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Oct 2015
    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
    Poland: 11
    Country: Number of subjects enrolled
    United Kingdom: 10
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Greece: 1
    Worldwide total number of subjects
    26
    EEA total number of subjects
    26
    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)
    4
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    20
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was conducted at 10 to15 sites in the United Kingdom, Germany, Greece, and Poland

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Voncento (on-demand)
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Voncento
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Voncento was administered as bolus IV infusion with a rate not to exceed 6 mL/min. For on-demand treatment, for both adult and paediatric subjects, usually 40 to 80 IU/kg of Voncento corresponding to 20 to 40 IU FVIII:C/kg of body weight were recommended to achieve haemostasis. An initial dose of 80 IU/kg Voncento was to be required, especially in patients with Type 3 VWD.

    Arm title
    		 Voncento (prophylaxis)
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Voncento
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Voncento was administered as bolus IV infusion with a rate not to exceed 6 mL/min. For long-term prophylaxis in subjects 12 years and older, 25 to 40 IU of Voncento per kg body weight was to be considered at a frequency of 1 to 3 times per week; in subjects under 12 years, a prophylactic dose range of 40 to 80 IU Voncento/kg body weight was administered 1 to 3 times a week.

    Number of subjects in period 1 [1]
    		Voncento (on-demand) Voncento (prophylaxis)
    Started
    11
    14
    Completed
    11
    10
    Not completed
    0
    4
         Consent withdrawn by subject
    -
    2
         Pregnancy
    -
    1
         Lost to follow-up
    -
    1
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: Analysis was done on the Safety Population which comprised all subjects who received at least 1 dose of Voncento. Twenty-six (26) subjects were enrolled in the study, but 1 subject that did not meet inclusion criteria was enrolled by mistake and was terminated by the sponsor before receiving any study drug, therefore n=25.

    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
    		 25 25
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 4 4
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 19 19
        From 65-84 years
    		 2 2
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 35.8 ( 19.13 ) -
    Gender categorical
    Units: Subjects
        Female
    		 12 12
        Male
    		 13 13

    End points

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    End points reporting groups
    Reporting group title
    Voncento (on-demand)
    Reporting group description
    		 -

    Reporting group title
    Voncento (prophylaxis)
    Reporting group description
    		 -

    Subject analysis set title
    Safety Population (SP)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The Safety population comprised all subjects who received at least 1 dose of Voncento.

    Subject analysis set title
    Efficacy Population (EP)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Efficacy population: The Efficacy population included all subjects of the safety population who had at least 1 post-baseline haemostatic efficacy assessment (“excellent”, “good”, “moderate”, or “none”) of Voncento for a non-surgical bleeding (NSB) event or a surgical procedure, or had at least 1 post-baseline assessment (“excellent”, “good”, “moderate”, or “none”) for a prophylaxis treatment.

    Primary: Investigator’s Assessment of Haemostatic Efficacy on Non Surgical Bleeding (NSB) Events (EP)

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    End point title
    		 Investigator’s Assessment of Haemostatic Efficacy on Non Surgical Bleeding (NSB) Events (EP) [1]
    End point description
    Efficacy Grading Scale: Excellent = Haemostasis achieved / cessation of bleeding. Good = Slight oozing / partial but adequate control of bleeding; did not require additional product for unplanned treatment. Moderate = Moderate bleeding / moderate control of bleeding; required additional product for unplanned treatment. None = Severe uncontrolled bleeding.
    End point type
    Primary
    End point timeframe
    Up to 12 months
    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: Only descriptive statistics were used
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: Number of NSB events
    number (not applicable)
        Excellent
    22
    59
        Good
    36
    5
        Moderate
    11
    8
        None
    0
    0
    No statistical analyses for this end point

    Primary: Subjects’ Assessment of Haemostatic Efficacy per Bleeding Day on NSB events (EP)

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    End point title
    		 Subjects’ Assessment of Haemostatic Efficacy per Bleeding Day on NSB events (EP) [2]
    End point description
    Efficacy Grading Scale: Excellent = Haemostasis achieved / cessation of bleeding. Good = Slight oozing / partial but adequate control of bleeding; did not require additional product for unplanned treatment. Moderate = Moderate bleeding / moderate control of bleeding; required additional product for unplanned treatment. None = Severe uncontrolled bleeding.
    End point type
    Primary
    End point timeframe
    Up to 12 months
    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: Only descriptive statistics were used
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: Number of NSB events
    number (not applicable)
        Excellent
    14
    52
        Good
    77
    45
        Moderate
    49
    14
        None
    0
    106
    No statistical analyses for this end point

    Primary: Number of NSB events per 3-month interval (EP)

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    End point title
    		 Number of NSB events per 3-month interval (EP) [3]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 12 months
    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: Only descriptive statistics were used
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: Number of NSB events
    number (not applicable)
        Month 1-3
    17
    21
        Month 4-6
    13
    19
        Month 7-9
    18
    28
        Month 10-12
    34
    10
    No statistical analyses for this end point

    Primary: Total Annual Bleeding Rate for Treated NSB Events(EP)

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    End point title
    		 Total Annual Bleeding Rate for Treated NSB Events(EP) [4]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 12 months
    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: Only descriptive statistics were used
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: bleeds per year per subject
        arithmetic mean (standard deviation)
    6.2 ( 7.65 )
    6.2 ( 6.80 )
    No statistical analyses for this end point

    Primary: Number of infusions per NSB event (EP)

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    End point title
    		 Number of infusions per NSB event (EP) [5]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 12 months
    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: Only descriptive statistics were used
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: infusions per event
        arithmetic mean (standard deviation)
    2.4 ( 2.85 )
    2.5 ( 4.37 )
    No statistical analyses for this end point

    Primary: Total dose of Voncento per NSB event (EP)

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    End point title
    		 Total dose of Voncento per NSB event (EP) [6]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 12 months
    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: Only descriptive statistics were used
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: IU VWF:RCo/kg
        arithmetic mean (standard deviation)
    147.6 ( 171.83 )
    167.2 ( 161.98 )
    No statistical analyses for this end point

    Secondary: Investigator's Assessment of Hemostatic Efficacy on Prophylaxis Treatment (EP)

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    End point title
    		 Investigator's Assessment of Hemostatic Efficacy on Prophylaxis Treatment (EP) [7]
    End point description
    Efficacy Grading Scale: Excellent = Haemostasis achieved / cessation of bleeding. Good = Slight oozing / partial but adequate control of bleeding; did not require additional product for unplanned treatment. Moderate = Moderate bleeding / moderate control of bleeding; required additional product for unplanned treatment. None = Severe uncontrolled bleeding.
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    Notes
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data only collected on the Prophylaxis arm not the On-demand arm
    End point values
    		 Voncento (prophylaxis)
    Number of subjects analysed
    14
    Units: Number of events
    number (not applicable)
        Excellent
    12
        Good
    2
        Moderate
    1
        None
    0
    No statistical analyses for this end point

    Secondary: Subject's Assessment of Hemostatic Efficacy on Prophylaxis Treatment (EP)

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    End point title
    		 Subject's Assessment of Hemostatic Efficacy on Prophylaxis Treatment (EP) [8]
    End point description
    Efficacy Grading Scale: Excellent = Haemostasis achieved / cessation of bleeding. Good = Slight oozing / partial but adequate control of bleeding; did not require additional product for unplanned treatment. Moderate = Moderate bleeding / moderate control of bleeding; required additional product for unplanned treatment. None = Severe uncontrolled bleeding.
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data only collected on the Prophylaxis arm not the On-demand arm
    End point values
    		 Voncento (prophylaxis)
    Number of subjects analysed
    14
    Units: Number of events
    number (not applicable)
        Excellent
    11
        Good
    4
        Moderate
    1
        None
    2
    No statistical analyses for this end point

    Secondary: Investigator's Assessment of Hemostatic Efficacy for Surgery (EP)

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    End point title
    		 Investigator's Assessment of Hemostatic Efficacy for Surgery (EP)
    End point description
    Efficacy Grading Scale: Excellent = Haemostasis achieved / cessation of bleeding. Good = Slight oozing / partial but adequate control of bleeding; did not require additional product for unplanned treatment. Moderate = Moderate bleeding / moderate control of bleeding; required additional product for unplanned treatment. None = Severe uncontrolled bleeding.
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: Number of surgical events
    number (not applicable)
        Excellent
    4
    3
        Good
    5
    0
        Moderate
    0
    0
        None
    0
    0
        Missing
    0
    1
    No statistical analyses for this end point

    Secondary: Investigator's Assessment of Blood Loss for Surgery (EP)

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    End point title
    		 Investigator's Assessment of Blood Loss for Surgery (EP)
    End point description
    The blood loss was judged to be “less”, “equivalent”, or “more” compared with the expected blood loss from a subject without a bleeding disorder undergoing the same procedure.
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: Number of surgical events
    number (not applicable)
        Less
    1
    1
        Equivalent
    8
    2
        More
    0
    0
        Missing
    0
    1
    No statistical analyses for this end point

    Secondary: Number of infusions required to treat a surgical bleeding event (EP)

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    End point title
    		 Number of infusions required to treat a surgical bleeding event (EP)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    4
    4
    Units: Number of infusions
        arithmetic mean (standard deviation)
    15.8 ( 9.6 )
    3.3 ( 2.63 )
    No statistical analyses for this end point

    Secondary: Dose per infusion of Voncento required to treat a surgical bleeding event (EP)

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    End point title
    		 Dose per infusion of Voncento required to treat a surgical bleeding event (EP)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    4
    4
    Units: IU VWF:RCo/kg
        arithmetic mean (standard deviation)
    58.48 ( 37.670 )
    104.53 ( 87.105 )
    No statistical analyses for this end point

    Secondary: Summary of Inhibitors (SP)

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    End point title
    		 Summary of Inhibitors (SP)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: BU/mL
    number (not applicable)
        von Willebrand Inhibitor
    0
    0
        Factor VIII Inhibitor
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects with Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Subjects with Treatment Emergent Adverse Events (TEAEs)
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 months
    End point values
    		 Voncento (on-demand) Voncento (prophylaxis)
    Number of subjects analysed
    11
    14
    Units: Number of subjects
    number (not applicable)
        TEAEs
    7
    12
        Serious TEAEs
    1
    1
        TEAEs of special interest
    0
    2
        Treatment-related TEAEs
    0
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    12 months per subject
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    Voncento (on-demand)
    Reporting group description
    		 -

    Reporting group title
    Voncento (prophylaxis)
    Reporting group description
    		 -

    Serious adverse events
    		 Voncento (on-demand) Voncento (prophylaxis)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 14 (7.14%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Voncento (on-demand) Voncento (prophylaxis)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 11 (63.64%)
    12 / 14 (85.71%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Injection site pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    6
    Pyrexia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 14 (7.14%)
         occurrences all number
    1
    1
    Psychiatric disorders
    Compulsive lip biting
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Depression
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Sleep disorder
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Blood iron decreased
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Face injury
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    0
    11
    Dizziness
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Seizure
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    11
    Tongue biting
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Microcytosis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Dry eye
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    0
    2
    Nausea
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 14 (7.14%)
         occurrences all number
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Constipation
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Dry mouth
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Faeces discoloured
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Swelling face
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    0
    13
    Pain in extremity
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 14 (7.14%)
         occurrences all number
    1
    2
    Arthritis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Back pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Osteitis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 14 (14.29%)
         occurrences all number
    1
    7
    Acute sinusitis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Bronchitis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Ear infection
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Otitis media
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Post procedural infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Iron deficiency
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Aug 2015
    •Prophylaxis treatment was added as treatment option in the study •The removal of the age restriction for participating subjects •Overall study duration was expected to be 2.5 years instead of 4

    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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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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