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    Clinical Trial Results:
    A single center, open label clinical trial investigating the long-term efficacy of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) treatment in subjects with alpha-Mannosidosis who previously participated in Lamazym trials.

    Summary
    EudraCT number
    2014-003950-15
    Trial protocol
    DK  
    Global end of trial date
    13 Jun 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    29 Jul 2016
    First version publication date
    29 Jul 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    rhLAMAN-10
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02478840
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Zymenex A/S
    Sponsor organisation address
    Roskildevej 12C, Hilleroed, Denmark, 3400
    Public contact
    Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., +39 0521 2791, clinicalTrials_info@chiesi.com
    Scientific contact
    Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., +39 0521 2791, clinicaltrials_info@chiesi.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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Jun 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    13 Jun 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jun 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The overall objective of the trial was the evaluation of the long-term efficacy of Lamazym treatment in patients with alpha-mannosidosis who were previously enrolled in trials with Lamazym and were currently receiving the treatment according to the AfterCare Program agreed with the National Authorities. Primary objective: The primary objective of the trial was to evaluate the impact of the long-term treatment with Lamazym in patients with alpha-mannosidosis upon the level of biomarker oligosaccharides in serum and upon the endurance as measured by the change from Baseline in the number of steps climbed in 3 minutes (three minute stair climb test, 3MSCT). To address the need for long-term data collection, the data collected during the present study was to be integrated with that from four previous studies (rhLAMAN-02, rhLAMAN-03, rhLAMAN-04 and rhLAMAN-05) and two ongoing studies (rhLAMAN-07 and rhLAMAN-09).
    Protection of trial subjects
    The study was conducted in accordance with the Declaration of Helsinki, Good Clinical Practice (GCP) guidelines and local law requirements. Other than routine care, no specific measures for protection of trial subjects were implemented.
    Background therapy
    There was no restriction on the use of concomitant medications/therapies during the trial. There are currently no known interactions of Lamazym with other medications.
    Evidence for comparator
    -
    Actual start date of recruitment
    16 Feb 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
    Denmark: 33
    Worldwide total number of subjects
    33
    EEA total number of subjects
    33
    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)
    9
    Adolescents (12-17 years)
    10
    Adults (18-64 years)
    14
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    A total of 34 patients (pts) were recruited with 35 pts identifiers; 1 patient participated in LAMAN-02/03 and later in LAMAN-05 and was counted once. Of the 34 pts, one patient received placebo in LAMAN 05 and then Lamazym in the AfterCare program. This patient discontinued shortly after starting the program and was excluded from all analyses.

    Pre-assignment
    Screening details
    Of the 34 patients recruited, 1 (from the LAMAN05 study) discontinued the treatment shortly after starting the AfterCare program. As such, no data for this patient was collected during the active treatment, and so this patient was excluded from all analyses. Hence, 33 patients in total were actually analysed.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded
    Blinding implementation details
    This was an open-label study.

    Arms
    Arm title
    Lamazym 1mg/kg body weight
    Arm description
    Data displays were generally for patients overall. Each patient received the weekly dose of Lamazym of 1mg/kg.
    Arm type
    Experimental

    Investigational medicinal product name
    Lamazym
    Investigational medicinal product code
    Other name
    recombinant human lysosomal alpha-mannosidase
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravascular use
    Dosage and administration details
    Lamazym, 1mg/kg body weight as intravenous infusion. Each patient received the weekly dose of Lamazym (as scheduled by their AfterCare program) in connection with an evaluation visit.

    Number of subjects in period 1
    Lamazym 1mg/kg body weight
    Started
    33
    Completed
    33

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Lamazym 1mg/kg body weight
    Reporting group description
    Data displays were generally for patients overall. Each patient received the weekly dose of Lamazym of 1mg/kg.

    Reporting group values
    Lamazym 1mg/kg body weight Total
    Number of subjects
    33 33
    Age categorical
    Units: Subjects
        Children (2-11 years)
    9 9
        Adolescents (12-17 years)
    10 10
        Adults (18-64 years)
    14 14
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    17.1 ± 7.8 -
    Gender categorical
    Units: Subjects
        Female
    13 13
        Male
    20 20
    Subject analysis sets

    Subject analysis set title
    Overall - FAS
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Full Analysis Set (FAS): consisted of all patients who were dosed and whose efficacy was evaluated post-baseline. The end-point values for this Subject Analysis set reported are the changes from baseline to last observation.

    Subject analysis set title
    Overall - safety population
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The safety analysis set (SAS) consisted of all patients who received at least one dose of trial medication.

    Subject analysis set title
    Baseline population
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Baseline values were defined as the last non-missing value before the first dose of Lamazym (derived from parental trials rhLAMAN-02 and rhLAMAN-05, respectively).

    Subject analysis set title
    Last observation population
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Last observation values were defined as the last available value at the end of rhLAMAN trials (derived from the last trial the patient participated in).

    Subject analysis sets values
    Overall - FAS Overall - safety population Baseline population Last observation population
    Number of subjects
    33
    33
    33
    33
    Age categorical
    Units: Subjects
        Children (2-11 years)
    9
    9
    9
    9
        Adolescents (12-17 years)
    10
    10
    10
    10
        Adults (18-64 years)
    14
    14
    14
    14
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    17.1 ± 7.8
    17.1 ± 7.8
    17.1 ± 7.8
    17.1 ± 7.8
    Gender categorical
    Units: Subjects
        Female
    13
    13
    13
    13
        Male
    20
    20
    20
    20

    End points

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    End points reporting groups
    Reporting group title
    Lamazym 1mg/kg body weight
    Reporting group description
    Data displays were generally for patients overall. Each patient received the weekly dose of Lamazym of 1mg/kg.

    Subject analysis set title
    Overall - FAS
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Full Analysis Set (FAS): consisted of all patients who were dosed and whose efficacy was evaluated post-baseline. The end-point values for this Subject Analysis set reported are the changes from baseline to last observation.

    Subject analysis set title
    Overall - safety population
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The safety analysis set (SAS) consisted of all patients who received at least one dose of trial medication.

    Subject analysis set title
    Baseline population
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Baseline values were defined as the last non-missing value before the first dose of Lamazym (derived from parental trials rhLAMAN-02 and rhLAMAN-05, respectively).

    Subject analysis set title
    Last observation population
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Last observation values were defined as the last available value at the end of rhLAMAN trials (derived from the last trial the patient participated in).

    Primary: Absolute change from baseline to last observation in serum oligosaccharides

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    End point title
    Absolute change from baseline to last observation in serum oligosaccharides
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Primary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation.
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: μmol/L
        arithmetic mean (standard deviation)
    -4.59 ± 3.23
    6.9 ± 2.3
    2.31 ± 2.19
    Attachments
    Untitled (Filename: SERUM OLIGOSACCARIDES.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.74
         upper limit
    -3.45

    Primary: % change from baseline to last observation in serum oligosaccharides

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    End point title
    % change from baseline to last observation in serum oligosaccharides
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Primary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: Percent
        arithmetic mean (standard deviation)
    -62.8 ± 33.61
    6.9 ± 2.3
    2.31 ± 2.19
    Attachments
    Untitled (Filename: SERUM OLIGOSACCARIDES.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -74.7
         upper limit
    -50.8

    Primary: Absolute change from baseline to last observation in the 3MSCT

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    End point title
    Absolute change from baseline to last observation in the 3MSCT
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Primary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: steps/min
        arithmetic mean (standard deviation)
    6.384 ± 10.54
    53.6 ± 12.53
    59.98 ± 16.29
    Attachments
    Untitled (Filename: 3MSCT.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    2.645
         upper limit
    10.12

    Primary: % change from baseline to last observation in the 3MSCT

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    End point title
    % change from baseline to last observation in the 3MSCT
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Primary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: steps/min
        arithmetic mean (standard deviation)
    13.77 ± 25.83
    53.6 ± 12.53
    59.98 ± 16.29
    Attachments
    Untitled (Filename: 3MSCT.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.004
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    4.609
         upper limit
    22.92

    Secondary: Absolute change from baseline to last observation in the 6MSCT

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    End point title
    Absolute change from baseline to last observation in the 6MSCT
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: meters
        arithmetic mean (standard deviation)
    22.4 ± 63.2
    466.6 ± 90.1
    489 ± 85.7
    Attachments
    Untitled (Filename: 6MWT.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.05
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0
         upper limit
    44.8

    Secondary: % change from baseline to last observation in the 6MSCT

    Close Top of page
    End point title
    % change from baseline to last observation in the 6MSCT
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: meters
        arithmetic mean (standard deviation)
    7.1 ± 22
    466.6 ± 90.1
    489 ± 85.7
    Attachments
    Untitled (Filename: 6MWT.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.071
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.7
         upper limit
    14.9

    Secondary: Absolute change from baseline to last observation in FVC (%)

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    End point title
    Absolute change from baseline to last observation in FVC (%)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [1]
    29 [2]
    31 [3]
    Units: liters
        arithmetic mean (standard deviation)
    8.1 ± 14.8
    84.9 ± 18.6
    93.1 ± 21.7
    Attachments
    Untitled (Filename: FVC %.pdf)
    Notes
    [1] - This is the actual number of subjects on which the analysis was made
    [2] - This is the actual number of subjects on which the analysis was made
    [3] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.007
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    2.4
         upper limit
    13.7

    Secondary: % change from baseline to last observation in FVC (%)

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    End point title
    % change from baseline to last observation in FVC (%)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [4]
    29 [5]
    31 [6]
    Units: percent
        arithmetic mean (standard deviation)
    10.5 ± 20.9
    84.9 ± 18.6
    93.1 ± 21.7
    Attachments
    Untitled (Filename: FVC %.pdf)
    Notes
    [4] - This is the actual number of subjects on which the analysis was made
    [5] - This is the actual number of subjects on which the analysis was made
    [6] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.011
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    2.6
         upper limit
    18.5

    Secondary: Absolute change from baseline to last observation in FVC (L)

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    End point title
    Absolute change from baseline to last observation in FVC (L)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [7]
    29 [8]
    31 [9]
    Units: liters
        arithmetic mean (standard deviation)
    0.578 ± 0.704
    2.651 ± 1.083
    3.233 ± 1.153
    Attachments
    Untitled (Filename: FVC L.pdf)
    Notes
    [7] - This is the actual number of subjects on which the analysis was made
    [8] - This is the actual number of subjects on which the analysis was made
    [9] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.31
         upper limit
    0.845

    Secondary: % change from baseline to last observation in FVC (L)

    Close Top of page
    End point title
    % change from baseline to last observation in FVC (L)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [10]
    29 [11]
    31 [12]
    Units: liters
        arithmetic mean (standard deviation)
    28.38 ± 37.79
    2.651 ± 1.083
    3.233 ± 1.153
    Attachments
    Untitled (Filename: FVC L.pdf)
    Notes
    [10] - This is the actual number of subjects on which the analysis was made
    [11] - This is the actual number of subjects on which the analysis was made
    [12] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    14.04
         upper limit
    42.76

    Secondary: Absolute change from baseline to last observation in predicted FEV1

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    End point title
    Absolute change from baseline to last observation in predicted FEV1
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [13]
    29 [14]
    31 [15]
    Units: percent
        arithmetic mean (standard deviation)
    3.7 ± 16.2
    83.8 ± 17.6
    88.3 ± 22.6
    Attachments
    Untitled (Filename: FEV %.pdf)
    Notes
    [13] - This is the actual number of subjects on which the analysis was made
    [14] - This is the actual number of subjects on which the analysis was made
    [15] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.226
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.4
         upper limit
    9.9

    Secondary: % change from baseline to last observation in predicted FEV1

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    End point title
    % change from baseline to last observation in predicted FEV1
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [16]
    29 [17]
    31 [18]
    Units: percent
        arithmetic mean (standard deviation)
    5.1 ± 22.9
    83.8 ± 17.6
    88.3 ± 22.6
    Attachments
    Untitled (Filename: FEV %.pdf)
    Notes
    [16] - This is the actual number of subjects on which the analysis was made
    [17] - This is the actual number of subjects on which the analysis was made
    [18] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.241
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.6
         upper limit
    13.8

    Secondary: Absolute change from baseline to last observation in FEV1

    Close Top of page
    End point title
    Absolute change from baseline to last observation in FEV1
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [19]
    29 [20]
    31 [21]
    Units: Liters
        arithmetic mean (standard deviation)
    0.399 ± 0.615
    2.441 ± 1
    2.864 ± 1.044
    Attachments
    Untitled (Filename: FEV1 L.pdf)
    Notes
    [19] - This is the actual number of subjects on which the analysis was made
    [20] - This is the actual number of subjects on which the analysis was made
    [21] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.002
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.165
         upper limit
    0.633

    Secondary: % change from baseline to last observation in FEV1

    Close Top of page
    End point title
    % change from baseline to last observation in FEV1
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [22]
    29 [23]
    31 [24]
    Units: liters
        arithmetic mean (standard deviation)
    21.31 ± 35.71
    2.441 ± 1
    2.864 ± 1.044
    Attachments
    Untitled (Filename: FEV1 L.pdf)
    Notes
    [22] - This is the actual number of subjects on which the analysis was made
    [23] - This is the actual number of subjects on which the analysis was made
    [24] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.003
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    7.724
         upper limit
    34.889

    Secondary: Absolute change from baseline to last observation in PEF

    Close Top of page
    End point title
    Absolute change from baseline to last observation in PEF
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [25]
    29 [26]
    31 [27]
    Units: L/s
        arithmetic mean (standard deviation)
    1.16 ± 1.733
    4.852 ± 2.044
    6.005 ± 2.371
    Attachments
    Untitled (Filename: PEF.pdf)
    Notes
    [25] - This is the actual number of subjects on which the analysis was made
    [26] - This is the actual number of subjects on which the analysis was made
    [27] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.501
         upper limit
    1.819

    Secondary: % change from baseline to last observation in PEF

    Close Top of page
    End point title
    % change from baseline to last observation in PEF
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    29 [28]
    29 [29]
    31 [30]
    Units: L/s
        arithmetic mean (standard deviation)
    32.245 ± 56.081
    4.852 ± 2.044
    6.005 ± 2.371
    Attachments
    Untitled (Filename: PEF.pdf)
    Notes
    [28] - This is the actual number of subjects on which the analysis was made
    [29] - This is the actual number of subjects on which the analysis was made
    [30] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.004
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    10.912
         upper limit
    53.577

    Secondary: Absolute change from baseline to last observation in BOT-2

    Close Top of page
    End point title
    Absolute change from baseline to last observation in BOT-2
    End point description
    BOT-2 total score is expressed as "point score". Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: score
        arithmetic mean (standard deviation)
    5.1 ± 23.9
    107 ± 47.6
    112.1 ± 46
    Attachments
    Untitled (Filename: BOT-2.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.23
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.4
         upper limit
    13.6

    Secondary: % change from baseline to last observation in BOT-2

    Close Top of page
    End point title
    % change from baseline to last observation in BOT-2
    End point description
    BOT-2 total score is expressed as "point score". Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: score
        arithmetic mean (standard deviation)
    13 ± 33.9
    107 ± 47.6
    112.1 ± 46
    Attachments
    Untitled (Filename: BOT-2.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.035
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1
         upper limit
    25

    Secondary: Absolute change from baseline to last observation in CSF oligosaccharides

    Close Top of page
    End point title
    Absolute change from baseline to last observation in CSF oligosaccharides
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: μMol/L
        arithmetic mean (standard deviation)
    -0.59 ± 1.97
    10.64 ± 3.53
    10.05 ± 3.31
    Attachments
    Untitled (Filename: CSF_OLIGOSACCHARIDES.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.094
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.29
         upper limit
    0.11

    Secondary: % from baseline to last observation in CSF oligosaccharides

    Close Top of page
    End point title
    % from baseline to last observation in CSF oligosaccharides
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation.
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: μMol/L
        arithmetic mean (standard deviation)
    -3.54 ± 20.82
    10.64 ± 3.53
    10.05 ± 3.31
    Attachments
    Untitled (Filename: CSF_OLIGOSACCHARIDES.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.336
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -10.9
         upper limit
    3.84

    Secondary: Absolute change from baseline to last observation in TAU protein

    Close Top of page
    End point title
    Absolute change from baseline to last observation in TAU protein
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: ng/L
        arithmetic mean (standard deviation)
    20.8 ± 162.6
    655.6 ± 318.4
    676.4 ± 293.6
    Attachments
    Untitled (Filename: CSF-Tau.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.468
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -36.9
         upper limit
    78.4

    Secondary: % change from baseline to last observation in TAU protein

    Close Top of page
    End point title
    % change from baseline to last observation in TAU protein
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: ng/L
        arithmetic mean (standard deviation)
    7.7 ± 26.7
    655.6 ± 318.4
    676.4 ± 293.6
    Attachments
    Untitled (Filename: CSF-Tau.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.106
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.7
         upper limit
    17.2

    Secondary: Absolute change from baseline to last observation in CSF-NFL

    Close Top of page
    End point title
    Absolute change from baseline to last observation in CSF-NFL
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: ng/L
        arithmetic mean (standard deviation)
    -26.2 ± 209.4
    430.3 ± 256.2
    -404.1 ± 136.7
    Attachments
    Untitled (Filename: CSF-NFL.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.477
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -100
         upper limit
    48

    Secondary: % change from baseline to last observation in CSF-NFL

    Close Top of page
    End point title
    % change from baseline to last observation in CSF-NFL
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: ng/L
        arithmetic mean (standard deviation)
    11.6 ± 46.1
    430.3 ± 256.2
    404.1 ± 136.7
    Attachments
    Untitled (Filename: CSF-NFL.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.158
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.8
         upper limit
    28

    Secondary: Absolute change from baseline to last observation in CSF-GFA-P

    Close Top of page
    End point title
    Absolute change from baseline to last observation in CSF-GFA-P
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: ng/L
        arithmetic mean (standard deviation)
    174.8 ± 498.8
    520.9 ± 379.1
    695.8 ± 420.9
    Attachments
    Untitled (Filename: CSF-GFA-P.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.053
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2
         upper limit
    351.7

    Secondary: % change from baseline to last observation in CSF-GFA-P

    Close Top of page
    End point title
    % change from baseline to last observation in CSF-GFA-P
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: ng/L
        arithmetic mean (standard deviation)
    101.9 ± 147.6
    520.9 ± 379.1
    695.8 ± 420.9
    Attachments
    Untitled (Filename: CSF-GFA-P.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    49.6
         upper limit
    154.3

    Secondary: Absolute change from baseline to last observation in serum immunoglobulin G

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    End point title
    Absolute change from baseline to last observation in serum immunoglobulin G
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    24 [31]
    24 [32]
    24 [33]
    Units: g/L
        arithmetic mean (standard deviation)
    3.05 ± 1.56
    8.37 ± 4.2
    11.42 ± 4.59
    Attachments
    Untitled (Filename: IGg.pdf)
    Notes
    [31] - This is the actual number of subjects on which the analysis was made
    [32] - This is the actual number of subjects on which the analysis was made
    [33] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    2.39
         upper limit
    3.71

    Secondary: % change from baseline to last observation in serum Immunoglobulin G

    Close Top of page
    End point title
    % change from baseline to last observation in serum Immunoglobulin G
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    24 [34]
    24 [35]
    24 [36]
    Units: g/L
        arithmetic mean (standard deviation)
    44.07 ± 27.23
    8.37 ± 4.2
    11.42 ± 4.52
    Attachments
    Untitled (Filename: IGg.pdf)
    Notes
    [34] - This is the actual number of subjects on which the analysis
    [35] - This is the actual number of subjects on which the analysis
    [36] - This is the actual number of subjects on which the analysis
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    32.58
         upper limit
    55.57

    Secondary: Absolute change from baseline to last observation in Leiter R Total Equivalent Age VR

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    End point title
    Absolute change from baseline to last observation in Leiter R Total Equivalent Age VR
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: years
        arithmetic mean (standard deviation)
    0.265 ± 0.637
    5.879 ± 1.565
    6.144 ± 1.612
    Attachments
    Untitled (Filename: Leiter Scale VR.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.023
    Method
    paired 2-sided T test
    Confidence interval
         sides
    2-sided
         lower limit
    0.039
         upper limit
    0.491

    Secondary: % change from baseline to last observation in Leiter R Total Equivalent Age VR

    Close Top of page
    End point title
    % change from baseline to last observation in Leiter R Total Equivalent Age VR
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: years
        arithmetic mean (standard deviation)
    5.338 ± 10.45
    5.879 ± 1.565
    6.144 ± 1.612
    Attachments
    Untitled (Filename: Leiter Scale VR.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.006
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.633
         upper limit
    9.043

    Secondary: Absolute change from baseline to last observation in Leiter R Total Equivalent Age AM

    Close Top of page
    End point title
    Absolute change from baseline to last observation in Leiter R Total Equivalent Age AM
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    24 [37]
    24 [38]
    24 [39]
    Units: years
        arithmetic mean (standard deviation)
    0.156 ± 1.519
    6.514 ± 2.176
    6.67 ± 1.757
    Attachments
    Untitled (Filename: Leiter Scale AM.pdf)
    Notes
    [37] - This is the actual number of subjects on which the analysis was made
    [38] - This is the actual number of subjects on which the analysis was made
    [39] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.619
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.485
         upper limit
    0.798

    Secondary: % change from baseline to last observation in Leiter R Total Equivalent Age AM

    Close Top of page
    End point title
    % change from baseline to last observation in Leiter R Total Equivalent Age AM
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    24 [40]
    24 [41]
    24 [42]
    Units: years
        arithmetic mean (standard deviation)
    9.345 ± 32.48
    6.514 ± 2.176
    6.67 ± 1.757
    Attachments
    Untitled (Filename: Leiter Scale AM.pdf)
    Notes
    [40] - This is the actual number of subjects on which the analysis was made
    [41] - This is the actual number of subjects on which the analysis was made
    [42] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.172
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.372
         upper limit
    23.063

    Secondary: Absolute change from baseline to last observation in bone conduction (best ear)

    Close Top of page
    End point title
    Absolute change from baseline to last observation in bone conduction (best ear)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    32 [43]
    32 [44]
    33 [45]
    Units: dbHL
        arithmetic mean (standard deviation)
    -0.49 ± 6.58
    52.57 ± 12.36
    52.16 ± 13.13
    Attachments
    Untitled (Filename: Bone conduction best ear.pdf)
    Notes
    [43] - This is the actual number of subjects on which the analysis was made
    [44] - This is the actual number of subjects on which the analysis was made
    [45] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.674
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.86
         upper limit
    1.88

    Secondary: % change from baseline to last observation in bone conduction (best ear)

    Close Top of page
    End point title
    % change from baseline to last observation in bone conduction (best ear)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    32 [46]
    32 [47]
    33 [48]
    Units: dbHL
        arithmetic mean (standard deviation)
    -0.72 ± 14.54
    52.57 ± 12.36
    52.16 ± 13.13
    Attachments
    Untitled (Filename: Bone conduction best ear.pdf)
    Notes
    [46] - This is the actual number of subjects on which the analysis was made
    [47] - This is the actual number of subjects on which the analysis was made
    [48] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.782
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.96
         upper limit
    4.52

    Secondary: Absolute change from baseline to last observation in bone conduction (left ear)

    Close Top of page
    End point title
    Absolute change from baseline to last observation in bone conduction (left ear)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: dbHL
        arithmetic mean (standard deviation)
    -2.83 ± 7.14
    62.34 ± 16.51
    59.51 ± 15.01
    Attachments
    Untitled (Filename: Air conduction left ear.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.03
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.36
         upper limit
    -0.29

    Secondary: % change from baseline to last observation in bone conduction (left ear)

    Close Top of page
    End point title
    % change from baseline to last observation in bone conduction (left ear)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: dbHL
        arithmetic mean (standard deviation)
    -3.79 ± 10.7
    62.34 ± 16.51
    59.51 ± 15.01
    Attachments
    Untitled (Filename: Air conduction left ear.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.05
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.58
         upper limit
    0

    Secondary: Absolute change from baseline to last observation in bone conduction (right ear)

    Close Top of page
    End point title
    Absolute change from baseline to last observation in bone conduction (right ear)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: dbHL
        arithmetic mean (standard deviation)
    -1.41 ± 10.31
    61.85 ± 17.9
    60.44 ± 15.7
    Attachments
    Untitled (Filename: Air conduction right ear.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.438
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.06
         upper limit
    2.25

    Secondary: % change from baseline to last observation in bone conduction (right ear)

    Close Top of page
    End point title
    % change from baseline to last observation in bone conduction (right ear)
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: dbHL
        arithmetic mean (standard deviation)
    0.54 ± 20.19
    61.85 ± 17.9
    60.44 ± 15.7
    Attachments
    Untitled (Filename: Air conduction right ear.pdf)
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.878
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.62
         upper limit
    7.7

    Secondary: Absolute change from baseline to last observation in CHAQ Disability Index

    Close Top of page
    End point title
    Absolute change from baseline to last observation in CHAQ Disability Index
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    33
    33
    33
    Units: integer
        arithmetic mean (standard deviation)
    -0.13 ± 0.44
    1.36 ± 0.77
    1.23 ± 0.66
    Attachments
    Untitled (Filename: CHAQ Disability Index.pdf)
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.095
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.29
         upper limit
    0.02

    Secondary: % change from baseline to last observation in CHAQ Disability Index

    Close Top of page
    End point title
    % change from baseline to last observation in CHAQ Disability Index
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    31 [49]
    33 [50]
    33 [51]
    Units: integer
        arithmetic mean (standard deviation)
    -2.41 ± 45.03
    1.36 ± 0.77
    1.23 ± 0.66
    Attachments
    Untitled (Filename: CHAQ Disability Index.pdf)
    Notes
    [49] - This is the actual number of subjects on which the analysis was made
    [50] - This is the actual number of subjects on which the analysis was made
    [51] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.768
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -18.9
         upper limit
    14.11

    Secondary: Absolute change from baseline to last observation in CHAQ VAS pain

    Close Top of page
    End point title
    Absolute change from baseline to last observation in CHAQ VAS pain
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    32 [52]
    32
    33
    Units: integer
        arithmetic mean (standard deviation)
    -0.173 ± 0.647
    0.618 ± 0.731
    0.431 ± 0.616
    Attachments
    Untitled (Filename: CHAQ VAS PAIN.pdf)
    Notes
    [52] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.139
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.407
         upper limit
    0.06

    Secondary: % change from baseline to last observation in CHAQ VAS pain

    Close Top of page
    End point title
    % change from baseline to last observation in CHAQ VAS pain
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    21 [53]
    32 [54]
    33 [55]
    Units: integer
        arithmetic mean (standard deviation)
    -17 ± 109.8
    0.618 ± 0.731
    0.431 ± 0.616
    Attachments
    Untitled (Filename: CHAQ VAS PAIN.pdf)
    Notes
    [53] - This is the actual number of subjects on which the analysis was made
    [54] - This is the actual number of subjects on which the analysis was made
    [55] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.485
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -67
         upper limit
    32.94

    Secondary: Absolute change from baseline to last observation in CHAQ VAS general

    Close Top of page
    End point title
    Absolute change from baseline to last observation in CHAQ VAS general
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    32 [56]
    32 [57]
    33 [58]
    Units: integer
        arithmetic mean (standard deviation)
    -0.068 ± 0.621
    1.049 ± 0.77
    0.952 ± 0.723
    Attachments
    Untitled (Filename: CHAQ General VAS.pdf)
    Notes
    [56] - This is the actual number of subjects on which the analysis was made
    [57] - This is the actual number of subjects on which the analysis was made
    [58] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.543
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.291
         upper limit
    0.156

    Secondary: % change from baseline to last observation in CHAQ VAS general

    Close Top of page
    End point title
    % change from baseline to last observation in CHAQ VAS general
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    27 [59]
    32 [60]
    33 [61]
    Units: integer
        arithmetic mean (standard deviation)
    10.36 ± 73.63
    1.049 ± 0.77
    0.952 ± 0.723
    Attachments
    Untitled (Filename: CHAQ General VAS.pdf)
    Notes
    [59] - This is the actual number of subjects on which the analysis was made
    [60] - This is the actual number of subjects on which the analysis was made
    [61] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.471
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -18.8
         upper limit
    39.48

    Secondary: Absolute change from baseline to last observation in EQ-5D-5L Health Index

    Close Top of page
    End point title
    Absolute change from baseline to last observation in EQ-5D-5L Health Index
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    24 [62]
    24 [63]
    24 [64]
    Units: integer
        arithmetic mean (standard deviation)
    0.05 ± 0.135
    0.622 ± 0.17
    0.672 ± 0.167
    Attachments
    Untitled (Filename: EQ-5D-L.pdf)
    Notes
    [62] - This is the actual number of subjects on which the analysis was made
    [63] - This is the actual number of subjects on which the analysis was made
    [64] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    Last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.08
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.0066
         upper limit
    0.1075

    Secondary: % change from baseline to last observation in EQ-5D-5L Health Index

    Close Top of page
    End point title
    % change from baseline to last observation in EQ-5D-5L Health Index
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    24 [65]
    24 [66]
    24 [67]
    Units: integer
        arithmetic mean (standard deviation)
    11.23 ± 24.72
    0.622 ± 0.17
    0.672 ± 0.167
    Attachments
    Untitled (Filename: EQ-5D-L.pdf)
    Notes
    [65] - This is the actual number of subjects on which the analysis was made
    [66] - This is the actual number of subjects on which the analysis was made
    [67] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.0036
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.79
         upper limit
    21.6682

    Secondary: absolute change from baseline to last observation in MRI ADC grey

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRI ADC grey
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [68]
    9 [69]
    9 [70]
    Units: 10^-6 mm2/s
        arithmetic mean (standard deviation)
    160.2 ± 68.4
    796.9 ± 37.1
    957.1 ± 65.8
    Attachments
    Untitled (Filename: MRI ADC Grey.pdf)
    Notes
    [68] - This is the actual number of subjects on which the analysis was made
    [69] - This is the actual number of subjects on which the analysis was made
    [70] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    107.7
         upper limit
    212.8

    Secondary: % change from baseline to last observation in MRI ADC grey

    Close Top of page
    End point title
    % change from baseline to last observation in MRI ADC grey
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [71]
    9 [72]
    9 [73]
    Units: 10^-6 mm2/s
        arithmetic mean (standard deviation)
    20.3 ± 8.8
    796.8 ± 37.1
    957.1 ± 65.8
    Attachments
    Untitled (Filename: MRI ADC Grey.pdf)
    Notes
    [71] - This is the actual number of subjects on which the analysis was made
    [72] - This is the actual number of subjects on which the analysis was made
    [73] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    < 0.001
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    13.5
         upper limit
    27

    Secondary: absolute change from baseline to last observation in MRI ADC standard

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRI ADC standard
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [74]
    9 [75]
    9 [76]
    Units: 10^-6mm2/s
        arithmetic mean (standard deviation)
    -5.7 ± 42.4
    856.1 ± 91.7
    850.4 ± 63.4
    Attachments
    Untitled (Filename: MRI ADC Standard.pdf)
    Notes
    [74] - This is the actual number of subjects on which the analysis was made
    [75] - This is the actual number of subjects on which the analysis was made
    [76] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.699
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -38.3
         upper limit
    27

    Secondary: % change from baseline to last observation in MRI ADC standard

    Close Top of page
    End point title
    % change from baseline to last observation in MRI ADC standard
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [77]
    9 [78]
    9 [79]
    Units: 10^-6mm2/s
        arithmetic mean (standard deviation)
    -0.3 ± 4.5
    856.1 ± 91.7
    850.4 ± 63.4
    Attachments
    Untitled (Filename: MRI ADC Standard.pdf)
    Notes
    [77] - This is the actual number of subjects on which the analysis was made
    [78] - This is the actual number of subjects on which the analysis was made
    [79] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.83
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.8
         upper limit
    3.2

    Secondary: absolute change from baseline to last observation in MRI ADC white

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRI ADC white
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [80]
    9 [81]
    9 [82]
    Units: 10^-6mm2/s
        arithmetic mean (standard deviation)
    -113 ± 93.5
    913 ± 119.5
    800.3 ± 47.3
    Attachments
    Untitled (Filename: MRI ADC White.pdf)
    Notes
    [80] - This is the actual number of subjects on which the analysis was made
    [81] - This is the actual number of subjects on which the analysis was made
    [82] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.007
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -185
         upper limit
    -40.8

    Secondary: % change from baseline to last observation in MRI ADC white

    Close Top of page
    End point title
    % change from baseline to last observation in MRI ADC white
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [83]
    9 [84]
    9 [85]
    Units: 10^-6mm2/s
        arithmetic mean (standard deviation)
    -11.5 ± 8.3
    913 ± 119.5
    800.3 ± 47.3
    Attachments
    Untitled (Filename: MRI ADC White.pdf)
    Notes
    [83] - This is the actual number of subjects on which the analysis was made
    [84] - This is the actual number of subjects on which the analysis was made
    [85] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.003
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -17.8
         upper limit
    -5.1

    Secondary: absolute change from baseline to last observation in MRS mannose complex visual Grey

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRS mannose complex visual Grey
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [86]
    9 [87]
    9 [88]
    Units: integer
        arithmetic mean (standard deviation)
    -0.4 ± 0.5
    1.9 ± 0.8
    1.4 ± 1.1
    Attachments
    Untitled (Filename: Mannose Complex Visual Grey.pdf)
    Notes
    [86] - This is the actual number of subjects on which the analysis was made
    [87] - This is the actual number of subjects on which the analysis was made
    [88] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.035
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.8
         upper limit
    0

    Secondary: % change from baseline to last observation in MRS mannose complex visual Grey

    Close Top of page
    End point title
    % change from baseline to last observation in MRS mannose complex visual Grey
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [89]
    9 [90]
    9 [91]
    Units: integer
        arithmetic mean (standard deviation)
    -33.3 ± 43.3
    1.9 ± 0.8
    1.4 ± 1.1
    Attachments
    Untitled (Filename: Mannose Complex Visual Grey.pdf)
    Notes
    [89] - This is the actual number of subjects on which the analysis was made
    [90] - This is the actual number of subjects on which the analysis was made
    [91] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.05
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -66.6
         upper limit
    0

    Secondary: absolute change from baseline to last observation in MRS mannose complex visual Standard

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRS mannose complex visual Standard
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    8 [92]
    8 [93]
    9 [94]
    Units: integer
        arithmetic mean (standard deviation)
    -0.1 ± 0.6
    1.6 ± 1.2
    1.3 ± 1.3
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index Standard.pdf)
    Notes
    [92] - This is the actual number of subjects on which the analysis was made
    [93] - This is the actual number of subjects on which the analysis was made
    [94] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.598
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.7
         upper limit
    0.4

    Secondary: % change from baseline to last observation in MRS mannose complex visual Standard

    Close Top of page
    End point title
    % change from baseline to last observation in MRS mannose complex visual Standard
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    6 [95]
    8 [96]
    9 [97]
    Units: integer
        arithmetic mean (standard deviation)
    -8.3 ± 37.6
    1.6 ± 1.2
    1.3 ± 1.3
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index Standard.pdf)
    Notes
    [95] - This is the actual number of subjects on which the analysis was made
    [96] - This is the actual number of subjects on which the analysis was made
    [97] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.611
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -47.8
         upper limit
    31.2

    Secondary: absolute change from baseline to last observation in MRS mannose complex visual White

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRS mannose complex visual White
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [98]
    9 [99]
    9 [100]
    Units: integer
        arithmetic mean (standard deviation)
    0 ± 0.9
    1.8 ± 1.2
    1.8 ± 0.8
    Attachments
    Untitled (Filename: MRI ADC White.pdf)
    Notes
    [98] - This is the actual number of subjects on which the analysis was made
    [99] - This is the actual number of subjects on which the analysis was made
    [100] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Last observation population v Baseline population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 1
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.7
         upper limit
    0.7

    Secondary: % change from baseline to last observation in MRS mannose complex visual White

    Close Top of page
    End point title
    % change from baseline to last observation in MRS mannose complex visual White
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    7 [101]
    9 [102]
    9 [103]
    Units: integer
        arithmetic mean (standard deviation)
    -4.8 ± 51.6
    1.8 ± 1.2
    1.8 ± 0.8
    Attachments
    Untitled (Filename: MRI ADC White.pdf)
    Notes
    [101] - This is the actual number of subjects on which the analysis was made
    [102] - This is the actual number of subjects on which the analysis was made
    [103] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Last observation population v Baseline population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.815
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -52.4
         upper limit
    42.9

    Secondary: absolute change from baseline to last observation in MRS numerical mannose complex index grey

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRS numerical mannose complex index grey
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [104]
    9 [105]
    9 [106]
    Units: integer
        arithmetic mean (standard deviation)
    -0.2 ± 0.4
    1 ± 0.5
    0.9 ± 0.3
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index. Grey.pdf)
    Notes
    [104] - This is the actual number of subjects on which the analysis was made
    [105] - This is the actual number of subjects on which the analysis was made
    [106] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.329
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.5
         upper limit
    0.2

    Secondary: % change from baseline to last observation in MRS numerical mannose complex index grey

    Close Top of page
    End point title
    % change from baseline to last observation in MRS numerical mannose complex index grey
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    9 [107]
    9 [108]
    9 [109]
    Units: integer
        arithmetic mean (standard deviation)
    -4.5 ± 34.1
    1 ± 0.5
    0.9 ± 0.3
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index. Grey.pdf)
    Notes
    [107] - This is the actual number of subjects on which the analysis was made
    [108] - This is the actual number of subjects on which the analysis was made
    [109] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    18
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.705
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -30.7
         upper limit
    21.7

    Secondary: absolute change from baseline to last observation in MRS numerical mannose complex index standard

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRS numerical mannose complex index standard
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    7 [110]
    7 [111]
    9 [112]
    Units: integer
        arithmetic mean (standard deviation)
    -0.1 ± 0.4
    1 ± 0.3
    0.9 ± 0.4
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index Standard.pdf)
    Notes
    [110] - This is the actual number of subjects on which the analysis was made
    [111] - This is the actual number of subjects on which the analysis was made
    [112] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=16 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.704
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.4
         upper limit
    0.3

    Secondary: % change from baseline to last observation in MRS numerical mannose complex index standard

    Close Top of page
    End point title
    % change from baseline to last observation in MRS numerical mannose complex index standard
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    7 [113]
    7 [114]
    9 [115]
    Units: integer
        arithmetic mean (standard deviation)
    -7.7 ± 28.6
    1 ± 0.3
    0.9 ± 0.4
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index Standard.pdf)
    Notes
    [113] - This is the actual number of subjects on which the analysis was made
    [114] - This is the actual number of subjects on which the analysis was made
    [115] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=16 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.504
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -34.2
         upper limit
    18.8

    Secondary: absolute change from baseline to last observation in MRS numerical mannose complex index white

    Close Top of page
    End point title
    absolute change from baseline to last observation in MRS numerical mannose complex index white
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    8 [116]
    8 [117]
    9 [118]
    Units: integer
        arithmetic mean (standard deviation)
    -0.1 ± 0.3
    1 ± 0.4
    0.8 ± 0.2
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index White.pdf)
    Notes
    [116] - This is the actual number of subjects on which the analysis was made
    [117] - This is the actual number of subjects on which the analysis was made
    [118] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.287
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.4
         upper limit
    0.1

    Secondary: % change from baseline to last observation in MRS numerical mannose complex index white

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    End point title
    % change from baseline to last observation in MRS numerical mannose complex index white
    End point description
    Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
    End point type
    Secondary
    End point timeframe
    Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
    End point values
    Overall - FAS Baseline population Last observation population
    Number of subjects analysed
    8 [119]
    8 [120]
    9 [121]
    Units: integer
        arithmetic mean (standard deviation)
    -2.9 ± 33.7
    1 ± 0.4
    0.8 ± 0.2
    Attachments
    Untitled (Filename: Numerical Mannose Complex Index White.pdf)
    Notes
    [119] - This is the actual number of subjects on which the analysis was made
    [120] - This is the actual number of subjects on which the analysis was made
    [121] - This is the actual number of subjects on which the analysis was made
    Statistical analysis title
    last observation vs baseline
    Statistical analysis description
    In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
    Comparison groups
    Baseline population v Last observation population
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.813
    Method
    paired 2-sided T test
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -31.1
         upper limit
    25.3

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first dose to last observation.
    Adverse event reporting additional description
    A total of 546 TEAEs were reported in 29 patients. Approxi. one third of patients (12 patients) experienced serious TEAEs. Only 3 patients experienced TEAEs that were severe in intensity. There were no deaths, and no TEAEs led to treatment discontinuation.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.0
    Reporting groups
    Reporting group title
    Overall population-safety
    Reporting group description
    The safety analysis set (SAS) consisted of all patients who received at least one dose of trial medication.

    Serious adverse events
    Overall population-safety
    Total subjects affected by serious adverse events
         subjects affected / exposed
    12 / 33 (36.36%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    Craniocerebral injury
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Knee deformity
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sjogren-Larsson syndrome
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Syncope
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Loss of consciousness
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Somnolence
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Malabsorption from administration site
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Renal failure acute
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthritis
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Joint swelling
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Device related infection
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Ear infection
         subjects affected / exposed
    1 / 33 (3.03%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 33 (3.03%)
         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
    Overall population-safety
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    29 / 33 (87.88%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Surgical and medical procedures
    Catheter removal
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Ear tube insertion
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Tooth extraction
         subjects affected / exposed
    4 / 33 (12.12%)
         occurrences all number
    4
    General disorders and administration site conditions
    Chills
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    9
    Fatigue
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    4
    Malaise
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    3
    Oedema peripheral
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    3
    Pyrexia
         subjects affected / exposed
    11 / 33 (33.33%)
         occurrences all number
    26
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    4 / 33 (12.12%)
         occurrences all number
    9
    Respiratory, thoracic and mediastinal disorders
    Bronchitis
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Cough
         subjects affected / exposed
    9 / 33 (27.27%)
         occurrences all number
    12
    Rhinorrhoea
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    4
    Investigations
    Weight increased
         subjects affected / exposed
    6 / 33 (18.18%)
         occurrences all number
    7
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    4
    Contusion
         subjects affected / exposed
    6 / 33 (18.18%)
         occurrences all number
    10
    Excoriation
         subjects affected / exposed
    5 / 33 (15.15%)
         occurrences all number
    18
    Ligament sprain
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Post lumbar puncture syndrome
         subjects affected / exposed
    4 / 33 (12.12%)
         occurrences all number
    4
    Wound
         subjects affected / exposed
    7 / 33 (21.21%)
         occurrences all number
    10
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    4
    Headache
         subjects affected / exposed
    13 / 33 (39.39%)
         occurrences all number
    27
    Loss of consciousness
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Syncope
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Eye disorders
    Conjunctival hyperaemia
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Eye infection
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Eye pruritus
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    5
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    3
    Abdominal pain upper
         subjects affected / exposed
    4 / 33 (12.12%)
         occurrences all number
    4
    Diarrhoea
         subjects affected / exposed
    9 / 33 (27.27%)
         occurrences all number
    11
    Nausea
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    3
    Reflux gastritis
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Toothache
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    3
    Vomiting
         subjects affected / exposed
    10 / 33 (30.30%)
         occurrences all number
    14
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Erythema
         subjects affected / exposed
    4 / 33 (12.12%)
         occurrences all number
    5
    Rash
         subjects affected / exposed
    5 / 33 (15.15%)
         occurrences all number
    5
    Scar pain
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    7 / 33 (21.21%)
         occurrences all number
    14
    Back pain
         subjects affected / exposed
    5 / 33 (15.15%)
         occurrences all number
    5
    Myalgia
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    3
    Pain in extremity
         subjects affected / exposed
    6 / 33 (18.18%)
         occurrences all number
    14
    Infections and infestations
    Acute tonsillitis
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Ear infection
         subjects affected / exposed
    6 / 33 (18.18%)
         occurrences all number
    7
    Gastroenteritis
         subjects affected / exposed
    6 / 33 (18.18%)
         occurrences all number
    7
    Influenza
         subjects affected / exposed
    3 / 33 (9.09%)
         occurrences all number
    3
    Laryngitis
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Nasopharyngitis
         subjects affected / exposed
    23 / 33 (69.70%)
         occurrences all number
    89
    Urinary tract infection
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Otitis media
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    2 / 33 (6.06%)
         occurrences all number
    2

    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.
    There are no limitations or caveats to this summary of results
    For support, Contact us.
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    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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