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    Clinical Trial Results:
    A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

    Summary
    EudraCT number
    		 2007-005478-29
    Trial protocol
    		 FR   DE   SE   GB   BE   ES   IT  
    Global end of trial date
    31 Dec 2009

    Results information
    Results version number
    		 v1(current)
    This version publication date
    11 Apr 2020
    First version publication date
    11 Apr 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    PTC124-GD-007-DMD
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00592553
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    PTC Therapeutics, Inc.
    Sponsor organisation address
    100 Corporate Court, South Plainfield, United States, NJ 07080
    Public contact
    Medical Information, PTC Therapeutics, Inc., +353 1-866-562-4620, medinfo@ptcbio.com
    Scientific contact
    Medical Information, PTC Therapeutics International Limited, +353 19068700, medinfo@ptcbio.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?
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    05 Feb 2010
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Dec 2009
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to determine the effect of ataluren on ambulation.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki (revised version of Edinburgh, Scotland, 2000) and in conformance with the International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidance documents.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    29 Feb 2008
    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 States: 78
    Country: Number of subjects enrolled
    United Kingdom: 21
    Country: Number of subjects enrolled
    Italy: 13
    Country: Number of subjects enrolled
    Australia: 11
    Country: Number of subjects enrolled
    Germany: 11
    Country: Number of subjects enrolled
    Canada: 10
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    Sweden: 8
    Country: Number of subjects enrolled
    Spain: 6
    Country: Number of subjects enrolled
    Belgium: 5
    Country: Number of subjects enrolled
    Israel: 3
    Worldwide total number of subjects
    174
    EEA total number of subjects
    72
    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)
    156
    Adolescents (12-17 years)
    17
    Adults (18-64 years)
    1
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 185 participants were screened for eligibility, of which 11 participants did not meet entry criteria.

    Pre-assignment
    Screening details
    		 A total of 174 eligible participants were randomized in 1:1:1 ratio to receive either placebo, low-dose ataluren, or high-dose ataluren.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Carer, Assessor, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 High-Dose Ataluren
    Arm description
    		 Participants received ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ataluren
    Investigational medicinal product code
    PTC124
    Other name
    Pharmaceutical forms
    Granules for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Ataluren was administered as per the dose and schedule specified in the respective arms.

    Arm title
    		 Low-Dose Ataluren
    Arm description
    		 Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ataluren
    Investigational medicinal product code
    PTC124
    Other name
    Pharmaceutical forms
    Granules for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Ataluren was administered as per the dose and schedule specified in the respective arms.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    PTC124
    Other name
    Pharmaceutical forms
    Granules for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo matched to ataluren was administered as per the dose and schedule specified in the respective arms.

    Number of subjects in period 1
    		High-Dose Ataluren Low-Dose Ataluren Placebo
    Started
    60
    57
    57
    As-treated Population
    60
    57
    57
    ITT Population
    60
    57
    57
    Completed
    59
    57
    57
    Not completed
    1
    0
    0
         Protocol Noncompliance
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    High-Dose Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for 48 weeks.

    Reporting group title
    Low-Dose Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.

    Reporting group values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo Total
    Number of subjects
    		 60 57 57 174
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 8.4 ( 2.53 ) 8.8 ( 2.91 ) 8.3 ( 2.33 ) -
    Sex: Female, Male
    Units: Subjects
        Female
    		 0 0 0 0
        Male
    		 60 57 57 174
    6-Minute Walk Distance (6MWD)
    The 6MWD test was performed in a 30-meters-long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
    Units: meters
        arithmetic mean (standard deviation)
    		 358.2 ( 103.97 ) 350.0 ( 97.55 ) 359.6 ( 87.67 ) -

    End points

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    End points reporting groups
    Reporting group title
    High-Dose Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for 48 weeks.

    Reporting group title
    Low-Dose Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.

    Primary: Change From Baseline in 6MWD at Week 48

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    End point title
    		 Change From Baseline in 6MWD at Week 48
    End point description
    The 6MWD test was performed in a 30-meters-long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Intent-to-treat (ITT) population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘Overall number of participants analysed‘ signifies participants evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    59
    55
    55
    Units: meters
        arithmetic mean (standard deviation)
    -41.81 ( 89.234 )
    -12.86 ( 72.007 )
    -42.56 ( 90.046 )
    Statistical analysis title
    		 High-Dose Ataluren versus Placebo
    Statistical analysis description
    Analysis was performed using mixed model for repeated measures (MMRM) method including rank transformed 6MWD as the dependent variable; and rank transformed baseline 6MWD, treatment, visit, age (less than [<] 9 years versus [vs.] greater than or equal to [>=] 9 years) and corticosteroid use (yes vs. no) stratification factors, and interaction between treatment and visit as independent variables.
    Comparison groups
    High-Dose Ataluren v Placebo
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.4756
    Method
    		 Mixed models analysis
    Parameter type
    		 Least Square (LS) Mean Difference
    Point estimate
    -30.52
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -114.8
         upper limit
    		 53.75
    Variability estimate
    Standard error of the mean
    Dispersion value
    42.68
    Statistical analysis title
    		 Low-Dose Ataluren versus Placebo
    Statistical analysis description
    Analysis was performed using MMRM method including rank transformed 6MWD as the dependent variable; and rank transformed baseline 6MWD, treatment, visit, age (< 9 years vs. >= 9 years) and corticosteroid use (yes vs. no) stratification factors, and interaction between treatment and visit as independent variables.
    Comparison groups
    Low-Dose Ataluren v Placebo
    Number of subjects included in analysis
    110
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.149
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    62.65
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -22.66
         upper limit
    		 147.96
    Variability estimate
    Standard error of the mean
    Dispersion value
    43.21

    Secondary: Change From Baseline in Time to Stand From Supine Position at Week 48

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    End point title
    		 Change From Baseline in Time to Stand From Supine Position at Week 48
    End point description
    If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported. ITT population: all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. ‘n’= participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n=60, 57, 57)
    12.25 ( 11.191 )
    10.80 ( 9.924 )
    11.50 ( 11.440 )
        Change at Week 48 (n=59, 57, 56)
    3.00 ( 5.686 )
    3.23 ( 5.761 )
    3.24 ( 7.253 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Walk/Run 10 Meters at Week 48

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    End point title
    		 Change From Baseline in Time to Walk/Run 10 Meters at Week 48
    End point description
    If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n‘ signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n=60, 57, 57)
    7.80 ( 5.243 )
    7.45 ( 4.373 )
    6.86 ( 2.813 )
        Change at Week 48 (n=59, 57, 56)
    2.37 ( 6.149 )
    1.68 ( 5.617 )
    3.03 ( 6.691 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Climb 4 Stairs at Week 48

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    End point title
    		 Change From Baseline in Time to Climb 4 Stairs at Week 48
    End point description
    If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n‘ signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n=60, 57, 57)
    7.63 ( 7.522 )
    6.94 ( 6.474 )
    6.04 ( 5.661 )
        Change at Week 48 (n=59, 57, 56)
    3.51 ( 6.794 )
    2.39 ( 4.618 )
    4.79 ( 7.949 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Descend 4 Stairs at Week 48

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    End point title
    		 Change From Baseline in Time to Descend 4 Stairs at Week 48
    End point description
    If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n‘ signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n= 60, 57, 57)
    6.75 ( 7.219 )
    6.08 ( 5.985 )
    5.52 ( 5.753 )
        Change at Week 48 (n=59, 57, 56)
    2.95 ( 7.323 )
    2.41 ( 6.162 )
    4.03 ( 7.828 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, and Shoulder Abduction at Week 48, as Assessed by Myometry

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    End point title
    		 Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, and Shoulder Abduction at Week 48, as Assessed by Myometry
    End point description
    Upper and lower extremity myometry was performed using a myometer following standardized procedures. Muscle groups evaluated included knee flexors, knee extensors, elbow flexors, elbow extensors, and shoulder abductors. Bilateral assessments were done and 3 measurements were recorded from each muscle group on each side if possible. Mean values for the left and right sides were calculated. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n‘ signifies participants evaluable for specified categories.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: pounds
    arithmetic mean (standard deviation)
        Baseline: Knee flexion (n=60,57,57)
    12.45 ( 4.684 )
    12.08 ( 4.217 )
    11.06 ( 3.494 )
        Change at Week 48: Knee flexion (n=59,57,55)
    0.39 ( 3.148 )
    -0.07 ( 3.511 )
    0.38 ( 2.991 )
        Baseline: Knee extension (n=60,57,56)
    12.71 ( 7.910 )
    12.81 ( 5.753 )
    12.96 ( 6.162 )
        Change at Week 48: Knee extension (n=59,57,55)
    -0.59 ( 3.511 )
    -0.63 ( 3.616 )
    -1.85 ( 3.899 )
        Baseline: Elbow flexion (n=60,57,57)
    8.72 ( 4.709 )
    7.66 ( 3.154 )
    8.14 ( 2.972 )
        Change at Week 48: Elbow flexion (n=59,57,56)
    -0.50 ( 1.832 )
    -0.10 ( 1.680 )
    -0.35 ( 1.807 )
        Baseline: Elbow extension (n=60,57,57)
    6.81 ( 3.815 )
    6.19 ( 3.083 )
    6.77 ( 2.785 )
        Change at Week 48: Elbow extension (n=59,57,56)
    -0.28 ( 1.473 )
    0.10 ( 1.493 )
    -0.51 ( 2.333 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mean Activity Period/Day/Visit at Week 48, as Assessed by Step Activity Monitoring (SAM)

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    End point title
    		 Change From Baseline in Mean Activity Period/Day/Visit at Week 48, as Assessed by Step Activity Monitoring (SAM)
    End point description
    Participants were instructed to continue to wear SAM (a pedometer) for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is the leg motion that begins when foot with SAM leaves the floor and ends when same foot touches the floor again (a stride generally equals 2 steps). Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data. For each day, an active period was defined as the first time after 3:00 AM that greater than (>) 2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was less than (<) 50% of mean active period across all days for that participant's visit. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n'=participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: minutes
    arithmetic mean (standard deviation)
        Baseline (n= 59, 53, 54)
    756.84 ( 84.612 )
    761.86 ( 76.984 )
    751.71 ( 60.513 )
        Change at Week 48 (n= 56, 53, 49)
    0.87 ( 57.833 )
    -22.23 ( 84.759 )
    -19.91 ( 91.960 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 48, as Assessed by SAM

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    End point title
    		 Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 48, as Assessed by SAM
    End point description
    Participants were instructed to continue to wear SAM (a pedometer) for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when foot with SAM leaves the floor and ends when the same foot touches the floor again (a stride generally equals 2 steps). Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of mean active period across all days for that participant's visit. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'n'= participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: steps/day
    arithmetic mean (standard deviation)
        Baseline (n=59, 53, 54)
    5302.31 ( 1907.058 )
    4870.13 ( 2165.522 )
    5602.31 ( 2023.543 )
        Change at Week 48 (n=56, 53, 49)
    -615.14 ( 1468.452 )
    -676.46 ( 1717.535 )
    -908.34 ( 1999.969 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 48, as Assessed by SAM

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    End point title
    		 Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 48, as Assessed by SAM
    End point description
    Participants were instructed to continue to wear SAM (a pedometer) for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (a stride generally equals 2 steps). Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: steps/hour
    arithmetic mean (standard deviation)
        Baseline (n=59, 53, 54)
    423.67 ( 168.754 )
    383.62 ( 161.911 )
    446.37 ( 160.666 )
        Change at Week 48 (n=56, 53, 49)
    -44.51 ( 125.154 )
    -42.23 ( 126.429 )
    -59.62 ( 153.054 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 48, as Assessed by SAM

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    End point title
    		 Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 48, as Assessed by SAM
    End point description
    Participants were instructed to continue to wear SAM (a pedometer) for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (a stride generally equals 2 steps). Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable for specified categories.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: steps
    arithmetic mean (standard deviation)
        Baseline: 10-min step count (n=59,55,54)
    35.77 ( 10.222 )
    32.24 ( 11.374 )
    36.76 ( 8.935 )
        Change at Week 48: 10-min step count (n=56,54,49)
    -2.77 ( 8.569 )
    -2.79 ( 6.355 )
    -3.97 ( 9.720 )
        Baseline: 20-min step count (n=59,55,54)
    29.13 ( 9.272 )
    25.68 ( 10.038 )
    29.74 ( 8.205 )
        Change at Week 48: 20-min step count (n=56,54,49)
    -2.49 ( 7.407 )
    -2.40 ( 5.806 )
    -3.55 ( 8.677 )
        Baseline: 30-min step count (n=59,55,54)
    25.00 ( 8.053 )
    22.08 ( 9.206 )
    25.70 ( 7.350 )
        Change at Week 48: 30-min step count (n=56,54,49)
    -2.08 ( 6.519 )
    -2.31 ( 5.505 )
    -3.03 ( 7.604 )
        Baseline: 60-min step count (n=59,55,54)
    18.58 ( 6.210 )
    16.52 ( 7.199 )
    19.50 ( 5.887 )
        Change at Week 48: 60-min step count (n=56,54,49)
    -1.50 ( 5.177 )
    -1.85 ( 4.616 )
    -2.33 ( 6.241 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Percentage of Time During the Active Period Spent at Low Activity (Less Than or Equal to [≤] 15 steps/minute), Medium Activity (16-30 steps/minute), and High Activity (Greater Than [>]30 Steps/minute) at Week 48

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    End point title
    		 Change From Baseline in Percentage of Time During the Active Period Spent at Low Activity (Less Than or Equal to [≤] 15 steps/minute), Medium Activity (16-30 steps/minute), and High Activity (Greater Than [>]30 Steps/minute) at Week 48
    End point description
    Participants were instructed to continue to wear SAM (a pedometer) for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is the leg motion that begins when foot with SAM leaves the floor and ends when same foot touches the floor again (a stride generally equals 2 steps). Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data. For each day, an active period was defined as the first time after 3:00 AM that greater than (>) 2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was less than (<) 50% of mean active period across all days for that participant's visit. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n'=participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: percentage of time
    arithmetic mean (standard deviation)
        Baseline: Low activity (n=59,53,54)
    32.91 ( 7.842 )
    32.38 ( 8.213 )
    32.86 ( 6.239 )
        Change at Week 48: Low activity (n=56,53,49)
    -2.06 ( 7.903 )
    -1.12 ( 8.220 )
    -1.11 ( 5.586 )
        Baseline: Medium activity (n=59,53,54)
    11.11 ( 4.013 )
    10.00 ( 3.656 )
    11.84 ( 4.304 )
        Change at Week 48: Medium activity (n=56,53,49)
    -1.35 ( 3.348 )
    -0.69 ( 3.828 )
    -1.92 ( 4.178 )
        Baseline: High activity (n=59,53,54)
    6.59 ( 4.077 )
    5.78 ( 3.785 )
    7.17 ( 3.700 )
        Change at Week 48: High activity (n=54,52,48)
    -0.66 ( 2.790 )
    -0.96 ( 2.828 )
    -1.03 ( 3.783 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 48

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    End point title
    		 Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 48
    End point description
    PedsQL includes generic core module (including physical, emotional, social and school functioning scales) comprises 23 questions and fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. Examples of items in each of generic core module scales include: “It is hard for me to run”; “I feel sad or blue”; “I cannot do things that other kids my age can do;” and “It is hard to pay attention in class.” Each of the generic core module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating better health-related quality of life. ITT population: all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable for specified categories.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline: Physical function score (n=58,56,56)
    63.63 ( 20.029 )
    59.27 ( 22.782 )
    61.87 ( 19.411 )
        Change at Week 48: Physical function (n=54,55,50)
    -0.94 ( 19.125 )
    2.37 ( 25.105 )
    -1.00 ( 24.022 )
        Baseline: Emotional function score (n=58,55,56)
    73.92 ( 20.418 )
    73.70 ( 20.223 )
    70.13 ( 19.332 )
        Change at Week 48: Emotional function (n=54,54,50)
    2.36 ( 16.742 )
    -1.83 ( 23.725 )
    4.30 ( 22.315 )
        Baseline: Social function score (n=58,55,55)
    67.50 ( 21.749 )
    65.09 ( 18.421 )
    63.36 ( 20.476 )
        Change at Week 48: Social function (n=54,54,51)
    5.37 ( 20.463 )
    3.89 ( 21.841 )
    7.75 ( 18.870 )
        Baseline: School function score (n=58,55,54)
    67.72 ( 19.276 )
    64.55 ( 20.396 )
    64.65 ( 17.841 )
        Change at Week 48: School function (n=52,54,49)
    3.61 ( 13.008 )
    6.11 ( 23.765 )
    4.06 ( 23.244 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Parent/Caregiver- Reported HRQL as measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 48

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    End point title
    		 Change From Baseline in Parent/Caregiver- Reported HRQL as measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 48
    End point description
    PedsQL includes generic core module (including physical, emotional, social and school functioning scales) comprises 23 questions and fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. Examples of items in each of generic core module scales include: “It is hard for me to run”; “I feel sad or blue”; “I cannot do things that other kids my age can do;” and “It is hard to pay attention in class.” Each of the generic core module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating better health-related quality of life. ITT population: all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable for specified categories.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline: Physical function score (n=60,56,57)
    56.15 ( 19.955 )
    54.96 ( 20.592 )
    51.47 ( 19.274 )
        Change at Week 48: Physical function (n=59,56,57)
    0.03 ( 17.088 )
    -3.10 ( 16.550 )
    0.23 ( 23.712 )
        Baseline: Emotional function score (n=60,56,57)
    70.08 ( 16.836 )
    69.11 ( 18.711 )
    65.96 ( 17.964 )
        Change at Week 48: Emotional function (n=59,56,57)
    4.07 ( 15.382 )
    3.39 ( 18.068 )
    1.21 ( 17.794 )
        Baseline: Social function score (n=60,56,57)
    61.58 ( 15.825 )
    62.77 ( 16.540 )
    55.79 ( 18.269 )
        Change at Week 48: Social function (n=59,56,57)
    -0.40 ( 18.470 )
    -1.09 ( 14.268 )
    3.71 ( 14.130 )
        Baseline: School function score (n=60,56,57)
    66.17 ( 18.258 )
    66.16 ( 16.320 )
    61.93 ( 13.587 )
        Change at Week 48: School function (n=58,56,56)
    2.73 ( 18.490 )
    -2.32 ( 15.462 )
    3.48 ( 13.913 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Participant-Reported HRQL as measured by the Total Fatigue Scale Score at Week 48

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    End point title
    		 Change From Baseline in Participant-Reported HRQL as measured by the Total Fatigue Scale Score at Week 48
    End point description
    The fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. Fatigue-specific module obtains information relating to items such as: “I feel too tired to do things that I like to do”; “I spend a lot of time in bed”; and “I have trouble remembering more than one thing at a time;” Each of the fatigue-specific module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating less fatigue. Total score was the sum of all items over the number of items answered on all scales. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n= 58, 55, 54)
    69.47 ( 16.525 )
    71.62 ( 16.474 )
    69.70 ( 15.263 )
        Change at Week 48 (n= 55, 54, 52)
    6.95 ( 13.460 )
    0.45 ( 23.068 )
    3.92 ( 16.512 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Parent/Caregiver-Reported HRQL as measured by the Total Fatigue Scale Score at Week 48

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    End point title
    		 Change From Baseline in Parent/Caregiver-Reported HRQL as measured by the Total Fatigue Scale Score at Week 48
    End point description
    The fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. Fatigue-specific module obtains information relating to items such as: “I feel too tired to do things that I like to do”; “I spend a lot of time in bed”; and “I have trouble remembering more than one thing at a time;” Each of the fatigue-specific module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating less fatigue. Total score was the sum of all items over the number of items answered on all scales. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n=60, 56, 57)
    73.39 ( 13.671 )
    70.71 ( 12.720 )
    68.27 ( 13.170 )
        Change at Week 48 (n=58,54, 57)
    1.97 ( 13.873 )
    1.27 ( 12.095 )
    2.51 ( 12.039 )
    No statistical analyses for this end point

    Secondary: Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score

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    End point title
    		 Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score
    End point description
    TSQM consisted of 14 questions(Q) in 4 domains: Effectiveness (Q 1-3 scored as 1 [extremely dissatisfied] to 7 [extremely satisfied]), Side Effects (Q 4 scored:0 [no] or 1 [yes]; Q 5 scored:1 [extreme bothersome] to 5 [not at all bothersome]; Q 6 - 8 scored:1 [great deal] to 5 [not at all]), Convenience (Q 9 and 10 scored:1 [extreme difficult] to 7[extreme easy]; Q 11 scored:1 [extremely inconvenient] to 5 [extremely convenient]) and Global Satisfaction (Q 12 scored:1 [not at all confident] to 7 [extreme confident]; Q 13 scored:1 [not at all certain] to 5[extremely certain]; Q 14 scored:1 [extremely dissatisfied] to 5 [extremely satisfied]). Scores of each of the domains were added together and an algorithm was used to create a score of 0-100, with higher scores=better treatment satisfaction. ITT population:participants who were randomized & received any study drug; had a valid baseline, & at least 1 valid post-baseline 6MWD value. ‘n’=participants evaluable for specified categories.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: units on a scale
    arithmetic mean (standard deviation)
        Effectiveness score (n=53,55,53)
    55.97 ( 27.796 )
    54.60 ( 22.307 )
    51.26 ( 23.536 )
        Side-effects score (n=55,56,55)
    96.36 ( 11.199 )
    97.77 ( 7.578 )
    96.89 ( 8.874 )
        Convenience score (n=57,56,55)
    55.85 ( 17.008 )
    58.23 ( 19.040 )
    60.91 ( 16.665 )
        Global satisfaction score (n=55,56,55)
    61.04 ( 25.967 )
    61.19 ( 23.691 )
    57.56 ( 21.851 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Participant/Caregiver-Reported Number of Daily Accidental Falls at Week 48

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    End point title
    		 Change From Baseline in Participant/Caregiver-Reported Number of Daily Accidental Falls at Week 48
    End point description
    Number of falls was determined by daily diary records maintained by participants and/or parent/caregivers. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'n' signifies participants evaluable for this endpoint at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: falls/day
    arithmetic mean (standard deviation)
        Baseline (n=54, 48, 48)
    0.40 ( 0.597 )
    0.27 ( 0.480 )
    0.54 ( 0.943 )
        Change at Week 48 (n=52, 47, 44)
    -0.10 ( 0.466 )
    -0.06 ( 0.501 )
    0.20 ( 1.282 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 48

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    End point title
    		 Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 48
    End point description
    Basic attention and working memory was measured using the digit span task. A series of digits (0-9) were presented to the child in an auditory format only. The task had 2 parts; in the forward condition, the child was requested to repeat back the digits in the order they were presented and in the backward condition, he was requested to reverse the order of presentation. A raw score of the total number of correct responses was converted to an age-scaled-score (z-score) by subtracting the corresponding mean and dividing by the corresponding standard deviation of a reference population for that age. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n’=participants evaluable for this endpoint for specified categories.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: z-score
    arithmetic mean (standard deviation)
        Baseline: Forward condition (n=59,56,55)
    3.59 ( 2.554 )
    2.89 ( 2.180 )
    2.84 ( 1.675 )
        Change at Week 48: Forward condition (n=57,54,52)
    0.39 ( 1.677 )
    0.50 ( 1.767 )
    0.40 ( 1.550 )
        Baseline: Backward condition (n=59,56,54)
    1.73 ( 1.846 )
    1.70 ( 1.868 )
    1.59 ( 1.460 )
        Change at Week 48: Backward condition (57,54,51)
    0.56 ( 1.500 )
    0.33 ( 1.441 )
    0.59 ( 1.203 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Heart Rate Before, During, and After Each 6MWT at Week 48, as Assessed by Heart Rate Monitoring With the Polar® RS400

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    End point title
    		 Change From Baseline in Heart Rate Before, During, and After Each 6MWT at Week 48, as Assessed by Heart Rate Monitoring With the Polar® RS400
    End point description
    Heart rate was measured with a Polar RS400 heart rate monitor, which consists of a transmitter strap worn around the chest and a wristwatch receiver. Monitor produces a digital text file with 1 value per minute that represents mean heart rate for that minute. Mean heart rates values were collected prior to, during, and after the 6MWD. The participant rested for 5 minutes in a sitting position prior to the 6MWD, and the mean heart rate for the last minute of this rest period was collected and documented as the resting heart rate. During the 6MWT, the mean heart rate was collected and documented as the active heart rate. After completing the 6MWT and resting for 3 minutes, the mean heart rate for 1 minute was collected and documented as the recovery heart rate. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, ‘n'=participants evaluable for specified categories.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: beats/minute
    arithmetic mean (standard deviation)
        Baseline: Resting heart rate (n=60,57,57)
    105.47 ( 12.908 )
    109.70 ( 9.908 )
    104.07 ( 11.588 )
        Change at Week 48: Resting heart rate (n=54,53,50)
    -0.63 ( 13.008 )
    -0.36 ( 10.223 )
    -0.26 ( 15.614 )
        Baseline: Active heart rate (n=60,56,57)
    142.67 ( 18.070 )
    141.77 ( 15.574 )
    136.67 ( 20.713 )
        Change at Week 48: Active heart rate (n=53,49,48)
    -5.00 ( 21.976 )
    2.39 ( 19.095 )
    1.65 ( 21.295 )
        Baseline: Recovery heart rate (n=60,56,57)
    109.90 ( 12.633 )
    113.48 ( 10.340 )
    107.86 ( 12.066 )
        Change at Week 48:Recovery heart rate (n=53,49,48)
    -0.23 ( 13.475 )
    -1.33 ( 13.270 )
    0.54 ( 15.181 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 48

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    End point title
    		 Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 48
    End point description
    Blood samples collected for chemistry assays were used to quantify serum CK concentrations. Serum CK was assessed as a potential biomarker for muscle fragility, with a reduction in serum CK considered to be a positive outcome. ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'n' signifies participants evaluable for this endpoint at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: units/liter (U/L)
    arithmetic mean (standard deviation)
        Baseline (n= 60, 57, 57)
    10853.65 ( 6251.136 )
    12084.70 ( 7772.631 )
    10569.60 ( 6488.477 )
        Change at Week 48 (n= 58, 57, 55)
    -1680.09 ( 4264.769 )
    -2146.32 ( 7151.944 )
    -1235.13 ( 4323.943 )
    No statistical analyses for this end point

    Secondary: Percent Change From Pre-Treatment Visit (1 Week Prior to Baseline Visit) in Biceps Muscle Dystrophin Expression at Post-Treatment Visit (Week 36), as Determined by Immunofluorescence

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    End point title
    		 Percent Change From Pre-Treatment Visit (1 Week Prior to Baseline Visit) in Biceps Muscle Dystrophin Expression at Post-Treatment Visit (Week 36), as Determined by Immunofluorescence
    End point description
    Immunofluorescence evidence of a change in dystrophin expression on biceps muscle biopsy was defined as an increase in the staining of the sarcolemmal membrane with an antibody to the C-terminal portion of the dystrophin protein (excluding revertant fibers) between the pre-treatment (1 week prior to Baseline visit) and post-treatment (Week 36) biopsies. The biceps muscle was biopsied from one arm for confirmation of the absence or reduced levels of dystrophin prior to treatment initiation and from the other arm to assess for production of dystrophin post-treatment. As-treated population included all randomized participants who actually received any study treatment. Here, ‘n' signifies participants evaluable for this endpoint at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Pre-Treatment (1 week prior to baseline), post-treatment (Week 36)
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: percent change
    arithmetic mean (standard deviation)
        Pre-treatment (n=60,55,56)
    336.096 ( 138.9753 )
    359.797 ( 142.7361 )
    357.271 ( 139.6650 )
        Percent change post-treatment (n=59,55,56)
    -1.278 ( 27.7432 )
    -2.128 ( 28.8287 )
    -0.898 ( 19.2112 )
    No statistical analyses for this end point

    Other pre-specified: Percentage of Participants With Treatment-Emergent Adverse Events (AEs)

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    End point title
    		 Percentage of Participants With Treatment-Emergent Adverse Events (AEs)
    End point description
    An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. Treatment-emergent adverse event (TEAE) was defined as an adverse event that occurred or worsened in the period extending from first dose of study drug to 6 weeks after the last dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. As-treated population included all randomized participants who actually received any study treatment.
    End point type
    Other pre-specified
    End point timeframe
    Baseline up to Week 54
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: percentage of participants
        number (not applicable)
    95.0
    96.5
    98.2
    No statistical analyses for this end point

    Other pre-specified: Study Drug Compliance

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    End point title
    		 Study Drug Compliance
    End point description
    Study drug compliance was assessed by participant daily diary and quantification of used and unused study drug. Compliance was assessed in terms of the percentage of drug actually taken relative to the amount that should have been taken during the study. As-treated population included all randomized participants who actually received any study treatment.
    End point type
    Other pre-specified
    End point timeframe
    Baseline to Week 48
    End point values
    		 High-Dose Ataluren Low-Dose Ataluren Placebo
    Number of subjects analysed
    60
    57
    57
    Units: percentage of drug
        median (full range (min-max))
    97.87 (34.9 to 99.6)
    97.03 (64.5 to 99.8)
    97.74 (76.6 to 99.9)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to 6 weeks after the last dose of study drug (Week 54)
    Adverse event reporting additional description
    As-treated population included all randomized participants who actually received any study treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    9.1
    Reporting groups
    Reporting group title
    High-Dose Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for 48 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.

    Reporting group title
    Low-Dose Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.

    Serious adverse events
    		 High-Dose Ataluren Placebo Low-Dose Ataluren
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 60 (3.33%)
    3 / 57 (5.26%)
    2 / 57 (3.51%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (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
    Lower limb fracture
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (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
    Supraventricular tachycardia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (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
    Nervous system disorders
    Grand mal convulsion
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (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
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (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
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (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
    Varicella
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (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
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 High-Dose Ataluren Placebo Low-Dose Ataluren
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    58 / 60 (96.67%)
    57 / 57 (100.00%)
    56 / 57 (98.25%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Melanocytic naevus
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Skin papilloma
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    1
    1
    3
    Vascular disorders
    Flushing
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Hypertension
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    4 / 57 (7.02%)
         occurrences all number
    0
    1
    6
    Hypotension
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Pallor
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Surgical and medical procedures
    Endodontic procedure
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Tooth extraction
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Adhesion
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Asthenia
         subjects affected / exposed
    4 / 60 (6.67%)
    2 / 57 (3.51%)
    3 / 57 (5.26%)
         occurrences all number
    5
    2
    4
    Disease progression
         subjects affected / exposed
    5 / 60 (8.33%)
    6 / 57 (10.53%)
    4 / 57 (7.02%)
         occurrences all number
    5
    6
    4
    Energy increased
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Fatigue
         subjects affected / exposed
    6 / 60 (10.00%)
    2 / 57 (3.51%)
    2 / 57 (3.51%)
         occurrences all number
    6
    3
    2
    Feeling abnormal
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Feeling hot
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Gait disturbance
         subjects affected / exposed
    4 / 60 (6.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    4
    1
    0
    Ill-defined disorder
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    4
    0
    0
    Malaise
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    0
    1
    2
    Pain
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    8 / 60 (13.33%)
    12 / 57 (21.05%)
    14 / 57 (24.56%)
         occurrences all number
    10
    14
    21
    Thirst
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Hypersensitivity
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    2
    0
    0
    Seasonal allergy
         subjects affected / exposed
    0 / 60 (0.00%)
    4 / 57 (7.02%)
    1 / 57 (1.75%)
         occurrences all number
    0
    4
    1
    Reproductive system and breast disorders
    Balanitis
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Genital erythema
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Pruritus genital
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Testicular pain
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Bronchial hyperreactivity
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Cough
         subjects affected / exposed
    15 / 60 (25.00%)
    13 / 57 (22.81%)
    9 / 57 (15.79%)
         occurrences all number
    27
    19
    14
    Dyspnoea
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Epistaxis
         subjects affected / exposed
    3 / 60 (5.00%)
    1 / 57 (1.75%)
    3 / 57 (5.26%)
         occurrences all number
    7
    1
    6
    Nasal congestion
         subjects affected / exposed
    6 / 60 (10.00%)
    5 / 57 (8.77%)
    5 / 57 (8.77%)
         occurrences all number
    8
    9
    8
    Nasal discomfort
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    4 / 60 (6.67%)
    4 / 57 (7.02%)
    6 / 57 (10.53%)
         occurrences all number
    7
    7
    7
    Productive cough
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Pulmonary congestion
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Rhinitis allergic
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    3
    Rhinorrhoea
         subjects affected / exposed
    1 / 60 (1.67%)
    6 / 57 (10.53%)
    4 / 57 (7.02%)
         occurrences all number
    1
    8
    7
    Sinus congestion
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    2
    1
    Sneezing
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Throat irritation
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    2
    Upper respiratory tract congestion
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    2
    0
    Wheezing
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    4
    1
    0
    Psychiatric disorders
    Abnormal behaviour
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Aggression
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Agitation
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    3
    0
    1
    Anger
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Anxiety
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    0
    2
    2
    Attention deficit/hyperactivity disorder
         subjects affected / exposed
    3 / 60 (5.00%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    3
    0
    2
    Depression
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Emotional disorder
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Encopresis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Initial insomnia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Insomnia
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    3
    0
    Mood swings
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Obsessive-compulsive disorder
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Oppositional defiant disorder
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Sleep disorder
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Somnambulism
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Stress
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Tic
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    2
    0
    Investigations
    Blood aldosterone increased
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Blood bicarbonate abnormal
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Blood bicarbonate decreased
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Blood calcium increased
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Blood magnesium increased
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Blood pressure increased
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Immunoglobulins decreased
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Renin increased
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Thyroxine free increased
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Thyroxine increased
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Urine colour abnormal
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    3
    1
    Weight decreased
         subjects affected / exposed
    5 / 60 (8.33%)
    1 / 57 (1.75%)
    6 / 57 (10.53%)
         occurrences all number
    5
    1
    6
    Weight increased
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Injury, poisoning and procedural complications
    Accident
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Accidental overdose
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Arthropod bite
         subjects affected / exposed
    1 / 60 (1.67%)
    3 / 57 (5.26%)
    1 / 57 (1.75%)
         occurrences all number
    1
    3
    2
    Arthropod sting
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Back injury
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Contusion
         subjects affected / exposed
    8 / 60 (13.33%)
    4 / 57 (7.02%)
    8 / 57 (14.04%)
         occurrences all number
    9
    4
    10
    Chillblains
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    2
    Excoriation
         subjects affected / exposed
    3 / 60 (5.00%)
    4 / 57 (7.02%)
    0 / 57 (0.00%)
         occurrences all number
    3
    6
    0
    Eye injury
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Face injury
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Fall
         subjects affected / exposed
    7 / 60 (11.67%)
    7 / 57 (12.28%)
    11 / 57 (19.30%)
         occurrences all number
    14
    7
    15
    Femur fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    2
    0
    Foot fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Hand fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Head injury
         subjects affected / exposed
    2 / 60 (3.33%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    3
    0
    0
    Humerus fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Iliotibial band syndrome
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Incision site erythema
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Joint injury
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    5 / 57 (8.77%)
         occurrences all number
    0
    1
    5
    Joint sprain
         subjects affected / exposed
    4 / 60 (6.67%)
    1 / 57 (1.75%)
    4 / 57 (7.02%)
         occurrences all number
    4
    1
    4
    Laceration
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    2
    0
    0
    Limb injury
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    5 / 57 (8.77%)
         occurrences all number
    0
    1
    6
    Lower limb fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Lumbar vertebral fracture
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Muscle strain
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Post procedural discomfort
         subjects affected / exposed
    3 / 60 (5.00%)
    0 / 57 (0.00%)
    6 / 57 (10.53%)
         occurrences all number
    3
    0
    8
    Post procedural haematoma
         subjects affected / exposed
    4 / 60 (6.67%)
    2 / 57 (3.51%)
    3 / 57 (5.26%)
         occurrences all number
    4
    2
    3
    Post procedural swelling
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Procedural pain
         subjects affected / exposed
    11 / 60 (18.33%)
    10 / 57 (17.54%)
    10 / 57 (17.54%)
         occurrences all number
    17
    14
    13
    Scratch
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Skin laceration
         subjects affected / exposed
    0 / 60 (0.00%)
    3 / 57 (5.26%)
    0 / 57 (0.00%)
         occurrences all number
    0
    3
    0
    Spinal compression fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    2
    Sunburn
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Suture rupture
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Thermal burn
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Tibia fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    2
    0
    Tooth fracture
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Tooth injury
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Traumatic haematoma
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Wound
         subjects affected / exposed
    2 / 60 (3.33%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    2
    2
    0
    Wound dehiscence
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Wrist fracture
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Congenital, familial and genetic disorders
    Kidney malformation
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Macroglossia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    Arrhythmia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Dilatation ventricular
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Left ventricular hypertrophy
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Palpitations
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    2
    1
    1
    Sinus bradycardia
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Supraventricular extrasystoles
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Supraventricular tachycardia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Areflexia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Convulsion
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Dizziness
         subjects affected / exposed
    3 / 60 (5.00%)
    4 / 57 (7.02%)
    3 / 57 (5.26%)
         occurrences all number
    3
    5
    4
    Headache
         subjects affected / exposed
    16 / 60 (26.67%)
    14 / 57 (24.56%)
    23 / 57 (40.35%)
         occurrences all number
    85
    25
    69
    Hypertonia
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Hypotonia
         subjects affected / exposed
    2 / 60 (3.33%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    2
    0
    1
    Lethargy
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Migraine
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    2
    0
    4
    Psychomotor hyperactivity
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Sinus headache
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Syncope
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Unresponsive to stimuli
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    2 / 57 (3.51%)
         occurrences all number
    0
    2
    2
    Microcytic anaemia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Ear congestion
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    0
    0
    2
    Ear pain
         subjects affected / exposed
    1 / 60 (1.67%)
    3 / 57 (5.26%)
    2 / 57 (3.51%)
         occurrences all number
    1
    3
    3
    Motion sickness
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Tinnitus
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    2
    0
    Tympanic membrane hyperaemia
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    2
    1
    5
    Tympanic membrane perforation
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Eye disorders
    Abnormal sensation in eye
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Cataract
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Conjunctivitis
         subjects affected / exposed
    3 / 60 (5.00%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    3
    1
    2
    Conjunctivitis allergic
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Eye pain
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Eye pruritus
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Eye swelling
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Eyelid cyst
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Hypermetropia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Ocular hyperaemia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Vision blurred
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Visual impairment
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    2 / 60 (3.33%)
    4 / 57 (7.02%)
    0 / 57 (0.00%)
         occurrences all number
    2
    4
    0
    Abdominal pain
         subjects affected / exposed
    12 / 60 (20.00%)
    4 / 57 (7.02%)
    8 / 57 (14.04%)
         occurrences all number
    23
    10
    8
    Abdominal pain lower
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    13 / 60 (21.67%)
    9 / 57 (15.79%)
    9 / 57 (15.79%)
         occurrences all number
    32
    13
    18
    Abdominal tenderness
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    2
    1
    0
    Aerophagia
         subjects affected / exposed
    1 / 60 (1.67%)
    2 / 57 (3.51%)
    1 / 57 (1.75%)
         occurrences all number
    1
    2
    1
    Anal pruritus
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Aphthous stomatitis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Chapped lips
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Constipation
         subjects affected / exposed
    4 / 60 (6.67%)
    2 / 57 (3.51%)
    2 / 57 (3.51%)
         occurrences all number
    6
    2
    3
    Diarrhoea
         subjects affected / exposed
    18 / 60 (30.00%)
    15 / 57 (26.32%)
    11 / 57 (19.30%)
         occurrences all number
    39
    20
    15
    Dyspepsia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Dysphagia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Enteritis
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    2
    1
    0
    Faecal incontinence
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Flatulence
         subjects affected / exposed
    10 / 60 (16.67%)
    4 / 57 (7.02%)
    5 / 57 (8.77%)
         occurrences all number
    10
    4
    5
    Frequent bowel movements
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    1
    0
    Gastritis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Gingival bleeding
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Gingivitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Haematemesis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Ileus
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Lip dry
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Mouth ulceration
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Nausea
         subjects affected / exposed
    10 / 60 (16.67%)
    7 / 57 (12.28%)
    8 / 57 (14.04%)
         occurrences all number
    16
    9
    16
    Oral pain
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Reflux oesophagitis
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Regurgitation
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    1
    0
    4
    Stomach discomfort
         subjects affected / exposed
    5 / 60 (8.33%)
    0 / 57 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    7
    0
    4
    Tooth disorder
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Tooth impacted
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Tooth loss
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Toothache
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    1
    1
    2
    Vomiting
         subjects affected / exposed
    28 / 60 (46.67%)
    22 / 57 (38.60%)
    33 / 57 (57.89%)
         occurrences all number
    72
    48
    88
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Decubitus ulcer
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Dermatitis allergic
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Dermatitis contact
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Dry skin
         subjects affected / exposed
    0 / 60 (0.00%)
    3 / 57 (5.26%)
    2 / 57 (3.51%)
         occurrences all number
    0
    3
    2
    Ecchymosis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Eczema
         subjects affected / exposed
    2 / 60 (3.33%)
    2 / 57 (3.51%)
    1 / 57 (1.75%)
         occurrences all number
    3
    2
    1
    Erythema
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    1
    0
    4
    Exfoliative rash
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Heat rash
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Hyperhidrosis
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Hyperkeratosis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Ingrowing nail
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Keratosis pilaris
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Nail disorder
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Night sweats
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Pruritus
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    2
    0
    Rash
         subjects affected / exposed
    8 / 60 (13.33%)
    5 / 57 (8.77%)
    6 / 57 (10.53%)
         occurrences all number
    8
    6
    7
    Rash erythematous
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Rash follicular
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Rash generalised
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Rash maculo-papular
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Rash papular
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    2
    1
    0
    Scar
         subjects affected / exposed
    5 / 60 (8.33%)
    4 / 57 (7.02%)
    4 / 57 (7.02%)
         occurrences all number
    9
    7
    8
    Skin discolouration
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Skin irritation
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Skin lesion
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Urticaria
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    1
    1
    1
    Enuresis
         subjects affected / exposed
    3 / 60 (5.00%)
    0 / 57 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    4
    0
    4
    Incontinence
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Myoglobinuria
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    3
    Pollakiuria
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    2
    1
    1
    Pyelocaliectasis
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Renal cyst
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Residual urine
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Strangury
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Ureteric dilatation
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Urinary incontinence
         subjects affected / exposed
    2 / 60 (3.33%)
    2 / 57 (3.51%)
    2 / 57 (3.51%)
         occurrences all number
    2
    2
    2
    Urine abnormality
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Endocrine disorders
    Goitre
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    7 / 60 (11.67%)
    2 / 57 (3.51%)
    2 / 57 (3.51%)
         occurrences all number
    9
    2
    2
    Back pain
         subjects affected / exposed
    6 / 60 (10.00%)
    5 / 57 (8.77%)
    9 / 57 (15.79%)
         occurrences all number
    15
    10
    9
    Coccydynia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Groin pain
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    3
    0
    Joint contracture
         subjects affected / exposed
    3 / 60 (5.00%)
    0 / 57 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    3
    0
    3
    Joint swelling
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Lordosis
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    2
    1
    0
    Muscle contracture
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Muscle spasms
         subjects affected / exposed
    1 / 60 (1.67%)
    5 / 57 (8.77%)
    3 / 57 (5.26%)
         occurrences all number
    1
    8
    4
    Muscle tightness
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    1
    1
    2
    Muscular weakness
         subjects affected / exposed
    5 / 60 (8.33%)
    2 / 57 (3.51%)
    3 / 57 (5.26%)
         occurrences all number
    5
    2
    3
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    1
    1
    4
    Musculoskeletal pain
         subjects affected / exposed
    2 / 60 (3.33%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    2
    1
    1
    Myalgia
         subjects affected / exposed
    2 / 60 (3.33%)
    2 / 57 (3.51%)
    3 / 57 (5.26%)
         occurrences all number
    2
    2
    3
    Neck pain
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Osteoporosis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Pain in extremity
         subjects affected / exposed
    8 / 60 (13.33%)
    6 / 57 (10.53%)
    8 / 57 (14.04%)
         occurrences all number
    16
    7
    12
    Scoliosis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Tendinous contracture
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Tendon disorder
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    1
    1
    1
    Trendelenburg's symptom
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    2
    0
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 60 (0.00%)
    5 / 57 (8.77%)
    2 / 57 (3.51%)
         occurrences all number
    0
    5
    2
    Catheter site infection
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Croup infectious
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Cystitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Ear infection
         subjects affected / exposed
    4 / 60 (6.67%)
    4 / 57 (7.02%)
    3 / 57 (5.26%)
         occurrences all number
    5
    4
    4
    Eczema infected
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Enterobiasis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    1
    0
    1
    Eyelid infection
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Fungal infection
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Gastroenteritis
         subjects affected / exposed
    6 / 60 (10.00%)
    4 / 57 (7.02%)
    10 / 57 (17.54%)
         occurrences all number
    7
    7
    10
    Gastroenteritis viral
         subjects affected / exposed
    3 / 60 (5.00%)
    3 / 57 (5.26%)
    4 / 57 (7.02%)
         occurrences all number
    3
    4
    4
    Genital candidiasis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Herpes zoster
         subjects affected / exposed
    0 / 60 (0.00%)
    2 / 57 (3.51%)
    0 / 57 (0.00%)
         occurrences all number
    0
    2
    0
    Hordeolum
         subjects affected / exposed
    2 / 60 (3.33%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    4
    0
    0
    Influenza
         subjects affected / exposed
    7 / 60 (11.67%)
    8 / 57 (14.04%)
    6 / 57 (10.53%)
         occurrences all number
    9
    9
    8
    Infusion site infection
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Lice infestation
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Localised infection
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    1
    1
    1
    Measles
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Molluscum contagiosum
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    1
    0
    3
    Nasopharyngitis
         subjects affected / exposed
    10 / 60 (16.67%)
    13 / 57 (22.81%)
    13 / 57 (22.81%)
         occurrences all number
    15
    17
    22
    Oral candidiasis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    2
    0
    0
    Oral herpes
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Otitis externa
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Otitis media
         subjects affected / exposed
    2 / 60 (3.33%)
    4 / 57 (7.02%)
    0 / 57 (0.00%)
         occurrences all number
    2
    4
    0
    Paronychia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    4 / 57 (7.02%)
         occurrences all number
    0
    1
    4
    Pharyngitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    0
    0
    2
    Pharyngitis streptococcal
         subjects affected / exposed
    2 / 60 (3.33%)
    3 / 57 (5.26%)
    1 / 57 (1.75%)
         occurrences all number
    3
    3
    3
    Pharyngotonsillitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Pneumonia
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    1 / 57 (1.75%)
         occurrences all number
    0
    1
    1
    Post procedural infection
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Postoperative wound infection
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    3
    0
    0
    Rhinitis
         subjects affected / exposed
    3 / 60 (5.00%)
    2 / 57 (3.51%)
    7 / 57 (12.28%)
         occurrences all number
    4
    2
    11
    Roseola
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Scarlet fever
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Sinusitis
         subjects affected / exposed
    2 / 60 (3.33%)
    4 / 57 (7.02%)
    1 / 57 (1.75%)
         occurrences all number
    2
    7
    1
    Skin infection
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Tinea capitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Tinea infection
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Tinea pedis
         subjects affected / exposed
    1 / 60 (1.67%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    1
    4
    0
    Tonsillitis
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    2 / 57 (3.51%)
         occurrences all number
    0
    0
    2
    Upper respiratory tract infection
         subjects affected / exposed
    12 / 60 (20.00%)
    12 / 57 (21.05%)
    9 / 57 (15.79%)
         occurrences all number
    18
    17
    16
    Urinary tract infection
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0
    Varicella
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    2 / 57 (3.51%)
         occurrences all number
    0
    1
    2
    Viral infection
         subjects affected / exposed
    3 / 60 (5.00%)
    1 / 57 (1.75%)
    3 / 57 (5.26%)
         occurrences all number
    4
    1
    3
    Viral pharyngitis
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    2 / 60 (3.33%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    2
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    5 / 60 (8.33%)
    2 / 57 (3.51%)
    5 / 57 (8.77%)
         occurrences all number
    6
    2
    6
    Dehydration
         subjects affected / exposed
    0 / 60 (0.00%)
    0 / 57 (0.00%)
    1 / 57 (1.75%)
         occurrences all number
    0
    0
    1
    Hyperuricaemia
         subjects affected / exposed
    1 / 60 (1.67%)
    0 / 57 (0.00%)
    0 / 57 (0.00%)
         occurrences all number
    1
    0
    0
    Iron deficiency
         subjects affected / exposed
    0 / 60 (0.00%)
    1 / 57 (1.75%)
    0 / 57 (0.00%)
         occurrences all number
    0
    1
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Dec 2007
    The changes implemented with this amendment are: - Information resulting from in vivo general toxicology studies of ataluren dosing for one month in mice was updated; - Information resulting from in vitro studies of human cytochrome P450 inhibition and induction was updated.
    28 Apr 2008
    The changes implemented with this amendment are: - Clarified that muscle dystrophin levels may be absent or reduced in Duchenne/Becker muscular dystrophy (DBMD) participants; - Clarified the required phenotypic evidence of DBMD by 9 years of age to ensure the enrollment of only BMD participants with more severe disease presentation and thereby limit the potential for a ceiling effect associated with the study-related efficacy evaluations; - Removed plasma renin as a laboratory parameter for potential exclusion from the study; - Clarified the duration of the follow-up period (that is, 6 weeks) requiring the use of contraceptives for sexually active participants; - Clarified the information regarding the metabolism of losartan by CYP2C9; - Clarified that participants must not use assistive devices during the 6MWD; - Clarified that blood pressure values collected during the 6MWD were not to be reconciled with blood pressure values collected with vital signs; - Clarified the wording to describe the methods used by the participants to complete the timed-function test (TFT) for running or walking 10 meters and climbing/descending stairs; - Clarified the wording to describe the documentation of the active heart rate during the 6MWD; - Clarified the site adverse event reporting requirements and de-identification of submitted source documentation to facilitate appropriate reporting by the sites; - Added and clarified assessments of 6MWD variability for upward adaption of sample size if variability was higher than expected; - Added a sensitivity analysis to determine the influence of assigning siblings to the same treatment group on the primary analysis; - Clarified the time-to-event analyses with regards to censoring; - Added a definition of treatment-emergent adverse events to clarify the safety analysis; - Corrected the number of participants for the interim efficacy and safety analysis from 100 to 90 participants (completing at least 24 weeks of treatment).
    27 Oct 2008
    The changes implemented with this amendment are: - Updated the required screening laboratory values based on experience to date in the DBMD study population. Thus, this amendment modified the screening laboratory parameters to allow for the inclusion of participants with clinically insignificant laboratory abnormalities and avoid unnecessary participant blood collection for retesting; - Modified the safety monitoring parameters and actions to be taken consistent with the changes to the laboratory entry criteria. This change was made to avoid the burden of unnecessary blood collection to reassess clinically inconsequential Grade 1 laboratory findings in this pediatric study population; - Removed the table describing the safety profile of ataluren; - Addressed the need for additional language to be inserted into the informed consent document encouraging participants and parents/guardians to avoid discussion of study-related procedures and study drug except with medical professionals involved in the study or in the care of the participant.
    04 Mar 2009
    The changes implemented with this amendment are: - Clarified that a participant is only required to walk for ≥75 meters during the 6MWD at Screening for the purpose of determining study eligibility, and that distances <75 meters are allowed for subsequent 6MWTs; - Revised the analysis plan for data derived from the SAM based on input from experts, recent literature, and assessment of initial pre-treatment data; - Updated the baseline 6MWD stratification from <270 meters and ≥270 meters to <350 meters and ≥350 meters. Prior to study start, the estimated mean 6MWD for the study population was ~270 meters; however, early assessment of pre-treatment 6MWD data (including 47 participants at Screening and 20 participants at Baseline visit) showed a mean 6MWD of ~350-360 meters; - Clarified that an overdose of study drug is the administration of a study drug dose >2 times the highest intended total daily dose level for this protocol; - Clarified the procedures for confirming Grade 1 or minor laboratory abnormalities; - Clarified that information regarding prior use of cardiac drugs for congestive heart failure (CHF) prophylaxis/treatment was also to be collected; - Clarified the parameters being measured for hematology laboratory assessments; - Clarified that not all muscle samples were to be processed and immunostained for various sarcoglycans and dystroglycan; rather, select samples may be assessed, as permitted by sample availability and observed dystrophin expression; - Revised the analysis plan for the 6MWD. Because the magnitudes of the changes in 6MWD were likely to be related to the baseline 6MWD, and because the MMRM was likely to have greater power than the repeated-measures analysis of covariance (RANOVA), the MMRM was to be used rather than the RANOVA.

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

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