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    Clinical Trial Results:
    A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study

    Summary
    EudraCT number
    		 2011-001805-28
    Trial protocol
    		 GB   DE  
    Global end of trial date
    09 Sep 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Sep 2017
    First version publication date
    28 Sep 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    POM-002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01435772
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    BioMarin Pharmaceutical Inc.
    Sponsor organisation address
    105 Digital Drive, Novato, United States, 94949
    Public contact
    BMN701 Clinical Program Management, BioMarin Europe Ltd., +44 0782455 2081, POMPE@bmrn.com
    Scientific contact
    BMN701 Clinical Program Management, BioMarin Europe Ltd., +44 0782455 2081, POMPE@bmrn.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
    28 Jul 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Sep 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Sep 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objectives of the study are: • To provide long-term, ongoing, treatment to patients who have participated in a BMN 701 clinical trial. • To evaluate the long-term safety and tolerability both during and following BMN 701 administration; • To determine the anti-BMN 701 antibody response to BMN 701 • To determine the anti-IGF-I and anti-IGF-II antibody response to BMN 701.
    Protection of trial subjects
    The DMC reviewed the study data on a schedule defined in the DMC Charter, and offered advice on whether or not to proceed with, modify, or terminate study enrollment on the basis of toxicity. Meetings of the DMC were convened at the discretion of the DMC Chair or the Study Medical Officer.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    10 Aug 2011
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    United States: 7
    Country: Number of subjects enrolled
    Australia: 6
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Germany: 1
    Worldwide total number of subjects
    21
    EEA total number of subjects
    8
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    21
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Individuals eligible to participate in this study met all of the following criteria: Have participated in a prior BMN 701 clinical development study; Have been diagnosed with late-onset Pompe disease, based on the entry criteria of a prior BMN 701 study. There were no screening failures

    Period 1
    Period 1 title
    0-144 weeks (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 5 mg/kg
    Arm description
    		 5 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMN 701
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    BMN 701 at doses of 5 mg/kg, 10 mg/kg, or 20 mg/kg for IV administration over approximately 1.5 to 4 hours qow over repeatable 24-week treatment cycles.

    Arm title
    		 10 mg/kg
    Arm description
    		 10 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMN 701
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    BMN 701 at doses of 5 mg/kg, 10 mg/kg, or 20 mg/kg for IV administration over approximately 1.5 to 4 hours qow over repeatable 24-week treatment cycles.

    Arm title
    		 20 mg/kg
    Arm description
    		 20 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMN 701
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    BMN 701 at doses of 5 mg/kg, 10 mg/kg, or 20 mg/kg for IV administration over approximately 1.5 to 4 hours qow over repeatable 24-week treatment cycles.

    Number of subjects in period 1
    		5 mg/kg 10 mg/kg 20 mg/kg
    Started
    3
    3
    15
    Completed
    2
    2
    10
    Not completed
    1
    1
    5
         Consent withdrawn by subject
    -
    1
    3
         Adverse event
    1
    -
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    5 mg/kg
    Reporting group description
    		 5 mg/kg

    Reporting group title
    10 mg/kg
    Reporting group description
    		 10 mg/kg

    Reporting group title
    20 mg/kg
    Reporting group description
    		 20 mg/kg

    Reporting group values
    		 5 mg/kg 10 mg/kg 20 mg/kg Total
    Number of subjects
    		 3 3 15 21
    Age categorical
    Units: Subjects
        18-65
    		 3 3 15 21
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 52.3 ± 6.66 42.7 ± 13.05 50.1 ± 5.28 -
    Gender categorical
    Units: Subjects
        Female
    		 0 2 6 8
        Male
    		 3 1 9 13

    End points

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    End points reporting groups
    Reporting group title
    5 mg/kg
    Reporting group description
    		 5 mg/kg

    Reporting group title
    10 mg/kg
    Reporting group description
    		 10 mg/kg

    Reporting group title
    20 mg/kg
    Reporting group description
    		 20 mg/kg

    Subject analysis set title
    Full Analysis Set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The Full Analysis Set (FAS) includes all 21 subjects enrolled in POM-002 who received at least 1 dose (or a partial dose) of BMN 701.

    Primary: Anti-BMN 701 Antibody Response(Positive) to BMN 701 at Day 1

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    End point title
    		 Anti-BMN 701 Antibody Response(Positive) to BMN 701 at Day 1 [1]
    End point description
    End point type
    Primary
    End point timeframe
    0-144 weeks
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    2
    1
    12
    15
    Units: number
    2
    1
    11
    14
    No statistical analyses for this end point

    Primary: Anti-BMN 701 Antibody Response(Positive) to BMN 701 at Week 144

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    End point title
    		 Anti-BMN 701 Antibody Response(Positive) to BMN 701 at Week 144 [2]
    End point description
    End point type
    Primary
    End point timeframe
    0-144 weeks
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    2
    2
    10
    14
    Units: Number
    2
    2
    10
    14
    No statistical analyses for this end point

    Primary: Anti-IGF-I Antibody Response(Positive) to BMN 701 at Baseline

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    End point title
    		 Anti-IGF-I Antibody Response(Positive) to BMN 701 at Baseline [3]
    End point description
    End point type
    Primary
    End point timeframe
    0-144 weeks
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    3
    15
    21
    Units: Number
    0
    2
    0
    2
    No statistical analyses for this end point

    Primary: Anti-IGF-I Antibody Response(Positive) to BMN 701 at Week 144

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    End point title
    		 Anti-IGF-I Antibody Response(Positive) to BMN 701 at Week 144 [4]
    End point description
    End point type
    Primary
    End point timeframe
    0-144 weeks
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    2
    2
    10
    14
    Units: Number
    0
    1
    5
    6
    No statistical analyses for this end point

    Primary: Anti-IGF-II Antibody Response(Positive) to BMN 701 at Baseline

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    End point title
    		 Anti-IGF-II Antibody Response(Positive) to BMN 701 at Baseline [5]
    End point description
    End point type
    Primary
    End point timeframe
    0-144 weeks
    Notes
    [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    3
    15
    21
    Units: Number
    1
    2
    2
    5
    No statistical analyses for this end point

    Primary: Anti-IGF-II Antibody Response(Positive) to BMN 701 at Week 144

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    End point title
    		 Anti-IGF-II Antibody Response(Positive) to BMN 701 at Week 144 [6]
    End point description
    End point type
    Primary
    End point timeframe
    0-144 weeks
    Notes
    [6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    2
    2
    10
    14
    Units: Number
    0
    2
    7
    9
    No statistical analyses for this end point

    Secondary: Baseline - Percent Predicted MIP

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    End point title
    		 Baseline - Percent Predicted MIP
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [7]
    3
    15
    18
    Units: percent
        arithmetic mean (standard deviation)
    ±
    39.53 ± 21.868
    40.2 ± 25.863
    40.09 ± 24.641
    Notes
    [7] - NA
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 144 - Percent Predicted MIP

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    End point title
    		 Change from Baseline to Week 144 - Percent Predicted MIP
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [8]
    2
    11
    13
    Units: percent
        arithmetic mean (standard deviation)
    ±
    21.21 ± 14.537
    18.48 ± 18.599
    18.89 ± 17.518
    Notes
    [8] - NA
    No statistical analyses for this end point

    Secondary: Baseline - Percent Predicted MEP

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    End point title
    		 Baseline - Percent Predicted MEP
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [9]
    3
    15
    18
    Units: percent
        arithmetic mean (standard deviation)
    ±
    31.07 ± 6.641
    36.7 ± 15.939
    35.76 ± 14.801
    Notes
    [9] - NA
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 144 - Percent Predicted MEP

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    End point title
    		 Change from Baseline to Week 144 - Percent Predicted MEP
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [10]
    2
    11
    13
    Units: percent
        arithmetic mean (standard deviation)
    ±
    -0.28 ± 5.841
    5.11 ± 16.395
    4.28 ± 15.197
    Notes
    [10] - NA
    No statistical analyses for this end point

    Secondary: Baseline - 6 Minutes Walk Test

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    End point title
    		 Baseline - 6 Minutes Walk Test
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    3
    15
    21
    Units: meter
        arithmetic mean (standard deviation)
    334 ± 227.119
    360 ± 51.403
    363.8 ± 157.818
    359 ± 151.553
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 144 - 6 Minutes Walk Test

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    End point title
    		 Change from Baseline to Week 144 - 6 Minutes Walk Test
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    2
    11
    16
    Units: meter
        arithmetic mean (standard deviation)
    -36 ± 90.508
    -120.75 ± 67.529
    9.5 ± 77.572
    -15.31 ± 86.181
    No statistical analyses for this end point

    Secondary: Baseline - Maximum Voluntary Ventilation (MVV)

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    End point title
    		 Baseline - Maximum Voluntary Ventilation (MVV)
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [11]
    3
    15
    18
    Units: L/min
        arithmetic mean (standard deviation)
    ±
    76 ± 41.037
    68.08 ± 26.74
    69.4 ± 28.217
    Notes
    [11] - NA
    No statistical analyses for this end point

    Secondary: Change from Baseline at Week 144 - Maximum Voluntary Ventilation (MVV)

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    End point title
    		 Change from Baseline at Week 144 - Maximum Voluntary Ventilation (MVV)
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [12]
    2
    11
    13
    Units: L/min
        arithmetic mean (standard deviation)
    ±
    13.5 ± 13.435
    2.24 ± 11.635
    3.97 ± 12.073
    Notes
    [12] - NA
    No statistical analyses for this end point

    Secondary: Baseline - Percent Predicted Upright FVC

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    End point title
    		 Baseline - Percent Predicted Upright FVC
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    3
    15
    21
    Units: Percent
        arithmetic mean (standard deviation)
    69.33 ± 19.732
    67.33 ± 26.577
    57.13 ± 18.677
    60.33 ± 19.518
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 144 - Percent Predicted Upright FVC

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    End point title
    		 Change from Baseline to Week 144 - Percent Predicted Upright FVC
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    2
    11
    16
    Units: percent
        arithmetic mean (standard deviation)
    -8.33 ± 7.095
    -0.5 ± 4.95
    -2 ± 8
    -3 ± 7.633
    No statistical analyses for this end point

    Secondary: Baseline - Percent Predicted Supine FVC

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    End point title
    		 Baseline - Percent Predicted Supine FVC
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    3
    12
    18
    Units: percent
        arithmetic mean (standard deviation)
    36.67 ± 17.954
    47.67 ± 32.578
    44.25 ± 21.55
    43.56 ± 21.794
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 144 - Percent Predicted Supine FVC

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    End point title
    		 Change from Baseline to Week 144 - Percent Predicted Supine FVC
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    2
    7
    12
    Units: percent
        arithmetic mean (standard deviation)
    -4.33 ± 9.452
    4.5 ± 26.163
    -3.43 ± 6.901
    -2.33 ± 10.714
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 144 - Urine Tetrasaccharide

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    End point title
    		 Change from Baseline to Week 144 - Urine Tetrasaccharide
    End point description
    21 patients analyzed for this endpoint transitioned from the POM-001 protocol. The baseline data use to calculate the change from baseline Urine Tetrasaccharide was derived from the POM-001 study.
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [13]
    2
    10
    12
    Units: mmol/mol
        arithmetic mean (standard deviation)
    ±
    -0.2 ± 3.394
    -1.07 ± 0.975
    -0.93 ± 1.393
    Notes
    [13] - NA
    No statistical analyses for this end point

    Secondary: Baseline - IGF-I

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    End point title
    		 Baseline - IGF-I
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [14]
    0 [15]
    11
    11
    Units: nmol/L
        arithmetic mean (standard deviation)
    ±
    ±
    20.26 ± 6.465
    20.26 ± 6.465
    Notes
    [14] - NA
    [15] - NA
    No statistical analyses for this end point

    Secondary: Week 144 - IGF-I

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    End point title
    		 Week 144 - IGF-I
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    2
    2
    11
    15
    Units: nmol/L
        arithmetic mean (standard deviation)
    26.98 ± 1.379
    18.4 ± 7.078
    17.62 ± 5.878
    18.97 ± 6.247
    No statistical analyses for this end point

    Secondary: Baseline - IGF-II

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    End point title
    		 Baseline - IGF-II
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [16]
    1
    6
    7
    Units: nmol/L
        arithmetic mean (standard deviation)
    ±
    702 ± 0
    496.83 ± 113.417
    526.14 ± 129.355
    Notes
    [16] - 20 mg/kg dose group suggested the most efficacious activity compared to other dose groups studied.
    No statistical analyses for this end point

    Secondary: Week 144 - IGF-II

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    End point title
    		 Week 144 - IGF-II
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    2
    11
    11
    Units: nmol/L
        arithmetic mean (standard deviation)
    467.67 ± 117.142
    559.5 ± 140.714
    698.82 ± 228.948
    639.75 ± 216.405
    No statistical analyses for this end point

    Secondary: Baseline - IGFBP3

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    End point title
    		 Baseline - IGFBP3
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    0 [17]
    0 [18]
    11
    11
    Units: nmol/L
        arithmetic mean (standard deviation)
    ±
    ±
    150.45 ± 23.914
    150.45 ± 23.914
    Notes
    [17] - 20 mg/kg dose group suggested the most efficacious activity compared to other dose groups studied.
    [18] - 20 mg/kg dose group suggested the most efficacious activity compared to other dose groups studied.
    No statistical analyses for this end point

    Secondary: Week 144 - IGFBP3

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    End point title
    		 Week 144 - IGFBP3
    End point description
    End point type
    Secondary
    End point timeframe
    0-144 weeks
    End point values
    		 5 mg/kg 10 mg/kg 20 mg/kg Full Analysis Set
    Number of subjects analysed
    3
    2
    11
    16
    Units: nmol/L
        arithmetic mean (standard deviation)
    157.33 ± 41.477
    209 ± 39.598
    146.64 ± 33.587
    156.44 ± 39.05
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Study Period
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    5 mg/kg
    Reporting group description
    		 -

    Reporting group title
    10 mg/kg
    Reporting group description
    		 -

    Reporting group title
    20 mg/kg
    Reporting group description
    		 -

    Serious adverse events
    		 5 mg/kg 10 mg/kg 20 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 3 (33.33%)
    7 / 15 (46.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tendon rupture
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure acute
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebrovascular accident
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Presyncope
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Anaphylactoid reaction
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Chronic respiratory failure
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Stridor
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Prerenal failure
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypoglycaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 5 mg/kg 10 mg/kg 20 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    3 / 3 (100.00%)
    15 / 15 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    0
    0
    8
    Haematoma
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Hot flush
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    4 / 15 (26.67%)
         occurrences all number
    1
    1
    4
    Hypertension
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    6
    Hypotension
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    6
    Pallor
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    3
    General disorders and administration site conditions
    Application site erythema
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Asthenia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Catheter site erythema
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Catheter site inflammation
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Catheter site pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Chest discomfort
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    1
    0
    19
    Chest pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Chills
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    5
    Discomfort
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Extravasation
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Fatigue
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 3 (0.00%)
    8 / 15 (53.33%)
         occurrences all number
    18
    0
    16
    Feeling abnormal
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Feeling cold
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    10
    Feeling hot
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    0
    1
    7
    Feeling of body temperature change
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Gait disturbance
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Influenza like illness
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Infusion site extravasation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Infusion site pain
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Infusion site swelling
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Malaise
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    1
    0
    5
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    2
    Oedema peripheral
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    8
    Pain
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    2
    Peripheral swelling
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    3
    Pyrexia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Temperature intolerance
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Tenderness
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    2
    0
    Vessel puncture site bruise
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Chronic respiratory failure
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Cough
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    8
    Dyspnoea
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    9 / 15 (60.00%)
         occurrences all number
    1
    0
    26
    Epistaxis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Hypoxia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Nasal congestion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Oropharyngeal pain
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    5 / 15 (33.33%)
         occurrences all number
    1
    2
    12
    Paranasal sinus hypersecretion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Pleuritic pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Productive cough
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    2
    0
    Pulmonary congestion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Rales
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Respiratory failure
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Tachypnoea
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    40
    Wheezing
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    4
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Confusional state
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Mood swings
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Insomnia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    2 / 15 (13.33%)
         occurrences all number
    2
    1
    2
    Nervousness
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    4 / 15 (26.67%)
         occurrences all number
    4
    1
    6
    Restlessness
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    2
    Sleep terror
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Product issues
    Device occlusion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Investigations
    Ammonia increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Bacterial test positive
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Blood calcium decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Blood glucose decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    7 / 15 (46.67%)
         occurrences all number
    0
    0
    14
    Blood immunoglobulin E increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Cardiac murmur
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Complement factor increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Lipase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Pulmonary physical examination abnormal
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Troponin increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Tryptase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 3 (33.33%)
    6 / 15 (40.00%)
         occurrences all number
    5
    1
    9
    Epicondylitis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Eye contusion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Fall
         subjects affected / exposed
    3 / 3 (100.00%)
    2 / 3 (66.67%)
    7 / 15 (46.67%)
         occurrences all number
    22
    5
    26
    Head injury
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Joint injury
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Laceration
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Muscle contusion
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Muscle strain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Post procedural contusion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Post procedural haemorrhage
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Procedural pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    2
    Skin abrasion
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 3 (33.33%)
    1 / 15 (6.67%)
         occurrences all number
    5
    1
    1
    Skin wound
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Tendon injury
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Thermal burn
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Tooth fracture
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    3
    Wound
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Cardiac failure
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Diastolic dysfunction
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Left ventricular hypertrophy
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Sinus tachycardia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    4
    Tachycardia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    5 / 15 (33.33%)
         occurrences all number
    1
    1
    49
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Burning sensation
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    2
    Dizziness
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    8 / 15 (53.33%)
         occurrences all number
    1
    5
    11
    Headache
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 3 (33.33%)
    9 / 15 (60.00%)
         occurrences all number
    7
    4
    30
    Hypoaesthesia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    3
    Lethargy
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Migraine
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Paraesthesia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Presyncope
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Restless legs syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Sciatica
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Sinus headache
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Tremor
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Tunnel vision
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Ear and labyrinth disorders
    Ear discomfort
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Tinnitus
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Vertigo positional
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Eye disorders
    Blindness transient
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Ocular hyperaemia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Visual impairment
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    3
    Abdominal distension
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Abdominal pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    3
    Abdominal pain lower
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Abdominal pain upper
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    5
    Abdominal tenderness
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Constipation
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    4
    Dental caries
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Diarrhoea
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    7 / 15 (46.67%)
         occurrences all number
    0
    0
    13
    Dyspepsia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Flatulence
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    3
    Irritable bowel syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    3
    Nausea
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    6 / 15 (40.00%)
         occurrences all number
    1
    1
    29
    Painful defaecation
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Rectal haemorrhage
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Salivary hypersecretion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Tooth loss
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Toothache
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    5
    Vomiting
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    4
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Cold sweat
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    3
    Decubitus ulcer
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Dermal cyst
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    4
    0
    0
    Erythema
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    4
    Hyperhidrosis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    0
    0
    14
    Night sweats
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Pruritus
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    3
    Rash
         subjects affected / exposed
    1 / 3 (33.33%)
    2 / 3 (66.67%)
    1 / 15 (6.67%)
         occurrences all number
    1
    5
    1
    Rash macular
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Rash papular
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Rash pruritic
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    2
    1
    0
    Rosacea
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Skin lesion
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Skin mass
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Skin ulcer
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Urticaria
         subjects affected / exposed
    3 / 3 (100.00%)
    1 / 3 (33.33%)
    2 / 15 (13.33%)
         occurrences all number
    8
    1
    3
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    2
    Renal impairment
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Endocrine disorders
    Androgen deficiency
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 3 (33.33%)
    7 / 15 (46.67%)
         occurrences all number
    8
    1
    18
    Back pain
         subjects affected / exposed
    3 / 3 (100.00%)
    1 / 3 (33.33%)
    7 / 15 (46.67%)
         occurrences all number
    6
    1
    10
    Bursitis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Flank pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Groin pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Intervertebral disc protrusion
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Joint swelling
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Muscle spasms
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    0
    9
    Muscle twitching
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Muscular weakness
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    2
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Musculoskeletal discomfort
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Musculoskeletal pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    3
    Myalgia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    4
    Neck pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    4
    Osteopenia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Pain in extremity
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    8 / 15 (53.33%)
         occurrences all number
    1
    1
    14
    Spinal pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Tendonitis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Blister infected
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Bronchitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Ear infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Fungal infection
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Gastroenteritis viral
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Herpes simplex
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    Influenza
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    0
    3
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    4 / 15 (26.67%)
         occurrences all number
    1
    1
    9
    Lower respiratory tract infection viral
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    Nasopharyngitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    4 / 15 (26.67%)
         occurrences all number
    0
    1
    7
    Rhinitis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    2
    Sinusitis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
    2 / 3 (66.67%)
    7 / 15 (46.67%)
         occurrences all number
    3
    6
    11
    Urinary tract infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    1 / 15 (6.67%)
         occurrences all number
    0
    2
    1
    Viral infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    1 / 15 (6.67%)
         occurrences all number
    0
    2
    2
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Glucose tolerance impaired
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Hyperglycaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    Hyperkalaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Hypoglycaemia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    14 / 15 (93.33%)
         occurrences all number
    19
    30
    106
    Iron deficiency
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Mar 2012
    -Pharmacokinetic assessments have been added to Week 24 (or at time of early discontinuation) for transition patients in POM-002. Additional assessments (hematology, chemistry panel, lipase, and a brief physical examination) have been added to Week -2, Day of transition, and Weeks +2 and +4 for the transition patients. -Testing of C3, C4, and CH50 at the time of an infusion-associated reaction (IAR) has been added. In addition, serum will be obtained every 6 weeks for storage for testing of C3, C4, and CH50 and other possible immunologic and inflammatory markers in the event a patient experiences an IAR. -Added option to increase BMN 701 infusion time as required. -Added option to use anti-inflammatory medications (e.g., ibuprofen) as an infusion pretreatment at the Investigator’s discretion. -Added assays of insulin-like growth factor (IGF) analytes (IGF-I, IGF-II, and IGFBP3) in serum samples being collected at study visits.
    07 Dec 2012
    -Optional desensitization regimen has been added to provide an additional treatment option for patients who experience infusion-associated reactions (IARs) that meet certain criteria. -Study size has been changed from 30-45 patients to approximately 22 patients initially, and the number of sites participating from 20 to approximately 12 initially. -The primary study objectives have been modified to include evaluation of the long-term safety and tolerability both during and following BMN 701 administration.
    12 Aug 2013
    -An additional requirement has been added that equipment (including epinephrine) for emergency resuscitation be readily available during infusions of BMN 701. -If a patient experiences a severe IAR or an IAR requiring cessation of the BMN 701 infusion, blood is to be drawn for additional antibody assessments 4 hours (± 15 minutes) after the cessation of the infusion instead of “shortly” after the cessation of the infusion. -A new exploratory objective has been added to study biochemical, molecular, cellular, and genetic/genomic aspects relevant to Pompe disease from blood and urine samples obtained from patients in the study. -Laboratory testing for hemoglobin A1c levels has been added at the Week 12, Week 24, Termination, and 4-Week Post-Dose Follow-Up Visits.
    15 May 2015
    -During the Transition Period visits where PK sampling is performed, an additional PK timepoint has been added at 4 hours post-infusion completion. In addition, glucose monitoring has been added at all post-completion timepoints (0.25, 0.5, 1, 2, 3, 4, 6, and 8 hours). -Clarified that urine tetrasaccharide and blood samples for evaluation of anti-drug antibodies will be done at the Week 24 assessment visit following the Transition Period to the new BMN 701 drug product. -Stopping criteria concerning hypoglycemia have been added. -The possible length of the study has been increased from 240 weeks (10 cycles) to 480 weeks (20 cycles).

    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.
    The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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