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    Clinical Trial Results:
    A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Distrophinopathy

    Summary
    EudraCT number
    		 2012-004527-20
    Trial protocol
    		 BE   CZ   SE   GB   DE   IT   ES   PL   FR  
    Global end of trial date
    20 Aug 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Aug 2020
    First version publication date
    02 Aug 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PTC124-GD-020-DMD
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01826487
    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., +011 44 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)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000115-PIP01-07
    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
    23 Sep 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Aug 2015
    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 ability of ataluren to slow disease progression as assessed by ambulatory decline (decrease in 6-minute walk distance [6MWD]).
    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
    26 Mar 2013
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 35 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 68
    Country: Number of subjects enrolled
    Spain: 22
    Country: Number of subjects enrolled
    France: 17
    Country: Number of subjects enrolled
    Turkey: 16
    Country: Number of subjects enrolled
    Chile: 14
    Country: Number of subjects enrolled
    Germany: 13
    Country: Number of subjects enrolled
    United Kingdom: 13
    Country: Number of subjects enrolled
    Italy: 11
    Country: Number of subjects enrolled
    Australia: 10
    Country: Number of subjects enrolled
    Poland: 8
    Country: Number of subjects enrolled
    Canada: 7
    Country: Number of subjects enrolled
    Sweden: 7
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Brazil: 5
    Country: Number of subjects enrolled
    Korea, Republic of: 4
    Country: Number of subjects enrolled
    Switzerland: 3
    Country: Number of subjects enrolled
    Czech Republic: 3
    Country: Number of subjects enrolled
    Israel: 3
    Worldwide total number of subjects
    230
    EEA total number of subjects
    100
    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)
    209
    Adolescents (12-17 years)
    21
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 291 participants were screened for eligibility, of which 61 participants did not meet entry criteria.

    Pre-assignment
    Screening details
    		 A total of 230 eligible participants were randomized in 1:1 ratio to receive either placebo or ataluren. 2 participants, 1 in each treatment arm, were excluded from intent-to-treat (ITT) population; as they did not have at least 1 valid post-baseline 6MWD value, a requirement for inclusion in ITT population.

    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
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matched to ataluren orally 3 times a day (TID) at morning, midday, and evening for 48 weeks.
    Arm type
    		 Placebo

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

    Arm title
    		 Ataluren
    Arm description
    		 Participants received ataluren suspension orally TID, 10 milligrams/kilogram (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 will be administered as per the dose and schedule specified in the arm.

    Number of subjects in period 1
    		Placebo Ataluren
    Started
    115
    115
    As-treated Population
    115
    115
    ITT Population
    114
    114
    Completed
    111
    110
    Not completed
    4
    5
         Consent withdrawn by subject
    1
    3
         Adverse event, non-fatal
    1
    1
         Lost to follow-up
    1
    -
         Protocol deviation
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to ataluren orally 3 times a day (TID) at morning, midday, and evening for 48 weeks.

    Reporting group title
    Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally TID, 10 milligrams/kilogram (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 values
    		 Placebo Ataluren Total
    Number of subjects
    		 115 115 230
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 9.0 ± 1.65 8.9 ± 1.79 -
    Sex: Female, Male
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 115 115 230
    Baseline 6MWD
    The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is 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: Subjects
        <300 meters
    		 22 25 47
        >=300 to <400 meters
    		 52 47 99
        >=400 meters
    		 41 43 84
    6MWD
    6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is 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)
    		 362.69 ± 81.424 364.04 ± 73.342 -
    Time to Walk/Run 10 Meters
    Units: seconds
        arithmetic mean (standard deviation)
    		 6.83 ± 2.924 6.66 ± 3.078 -
    Time to Climb 4 Stairs
    Number of participants analyzed were 112 for each arm.
    Units: seconds
        arithmetic mean (standard deviation)
    		 6.76 ± 7.287 5.99 ± 5.347 -
    Time to Descend 4 Stairs
    Number of participants analyzed were 109 and 112 for Placebo and Ataluren arm respectively.
    Units: seconds
        arithmetic mean (standard deviation)
    		 5.05 ± 5.362 5.07 ± 5.157 -
    Physical Function Total Score as Measured by North Star Ambulatory Assessment (NSAA)
    Number of participants analyzed were 114 for each arm.
    Units: units on a scale
        arithmetic mean (standard deviation)
    		 60.2 ± 18.37 60.9 ± 17.97 -
    Pediatric Outcomes Data Collection Instrument (PODCI) Transfers/Basic Mobility Score
    PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity & Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Each domain was scored from 0 (poor outcome/worse health) to 100(the highest level of functioning & least pain). Number of participants analyzed were 114 for each arm.
    Units: units on a scale
        arithmetic mean (standard deviation)
    		 81.4 ± 15.79 83.9 ± 13.10 -
    Pediatric Outcomes Data Collection Instrument (PODCI) Sports/Physical Functioning Score
    PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity & Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 (poor outcome/worse health) to 100(the highest level of functioning & least pain). Number of participants analyzed were 114 for each arm.
    Units: units on a scale
        arithmetic mean (standard deviation)
    		 56.0 ± 20.94 56.2 ± 18.94 -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to ataluren orally 3 times a day (TID) at morning, midday, and evening for 48 weeks.

    Reporting group title
    Ataluren
    Reporting group description
    		 Participants received ataluren suspension orally TID, 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) 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 is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is 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. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline and Week 48 6MWD values are each the average of two valid 6MWD values, or a single available value if one was missing. 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. Multiple imputation was applied to impute missing values within the treatment groups.
    End point type
    Primary
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    114
    114
    Units: meters
        least squares mean (standard error)
    -60.67 ± 9.323
    -47.69 ± 9.247
    Statistical analysis title
    		 Placebo versus Ataluren
    Statistical analysis description
    Analysis was performed using analysis of covariance (ANCOVA) method including stratification factors for age (less than [<] 9 years versus [vs.] greater than or equal to [>=] 9 years), duration of use of corticosteroids at baseline (approx. >=6 to <12 months vs. >=12 months), and baseline 6MWD category (>=350 meters vs <350 meters), as well as baseline 6MWD as covariate.
    Comparison groups
    Placebo v Ataluren
    Number of subjects included in analysis
    228
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.213 [1]
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (final values)
    Point estimate
    12.98
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.44
         upper limit
    		 33.39
    Variability estimate
    Standard error of the mean
    Dispersion value
    10.415
    Notes
    [1] - Threshold for significance at 0.05. Secondary endpoints were tested for significance, only if the primary endpoint was statistically significant.

    Secondary: Time to 10 Percent (%) Persistent Worsening in 6MWD

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    End point title
    		 Time to 10 Percent (%) Persistent Worsening in 6MWD
    End point description
    6MWD test was performed in a 30 meters long flat corridor, where participant was instructed to walk as far as possible, back and forth around two cones, with permission to slow down, rest, or stop. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during test. Time to 10% persistent worsening in 6MWD: last time that 6MWD was not 10% worse than baseline. For participants who did not have 10% 6MWD worsening or who were removed from study, time to 10% 6MWD worsening was censored at the time of last 6MWD test. Participants who became non-ambulatory were considered to have 10% worsening. 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. Multiple imputation was applied to impute missing value. ‘n‘=participants evaluable for specified categories. '99999'=data not calculated due to smaller number of participant with an event.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    114
    114
    Units: days
    median (confidence interval 95%)
        <300 meters (n=21,24)
    56 (1.0 to 111.0)
    164 (1.0 to 225.0)
        >=300 to <400 meters (n=52,47)
    280 (169.0 to 99999)
    99999 (280.0 to 99999)
        >=400 meters (n=41,43)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    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
    The method of walk/run used by participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support; 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). If time taken to perform a test exceeded 30 seconds or if a participant could not perform test due to disease progression, a value of 30 seconds was used. A cumulative 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, ‘Overall number of participants analyzed‘ signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    110
    109
    Units: seconds
        arithmetic mean (standard deviation)
    3.47 ± 6.393
    2.27 ± 5.216
    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
    The method of climbing used by participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs “marking time” (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs “marking time” (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Climbs 4 standard stairs “marking time”, not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. A cumulative 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, ‘Overall number of participants analyzed‘ signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    103
    105
    Units: seconds
        arithmetic mean (standard deviation)
    4.46 ± 7.310
    2.65 ± 5.297
    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
    The method of descending used by participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs “marking time” (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs “marking time”, using one arm on one handrail; 4. Descends 4 standard stairs “marking time” (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. A cumulative 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, ‘Overall number of participants analyzed‘ signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    100
    106
    Units: seconds
        arithmetic mean (standard deviation)
    3.97 ± 7.854
    2.15 ± 5.306
    No statistical analyses for this end point

    Secondary: 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
    Secondary
    End point timeframe
    Baseline up to Week 54
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    115
    115
    Units: percentage of participants
        number (not applicable)
    87.8
    89.6
    No statistical analyses for this end point

    Other pre-specified: Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel

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    End point title
    		 Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel
    End point description
    At screening or baseline, participant and/or parent/caregiver were asked to identify any activities of daily living (ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by participant’s DMD. At post-baseline (Week 48), same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within following categories: physical functioning(PF); general energy level(EL); cognition/school function(C/SF); emotional/social functioning(E/SocF); and sleep. Changes were reported on a 5-point Likert scale:1=much worse,2=slightly worse,3=unchanged,4=slightly better, or 5=much better. 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 for specified categories.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    114
    114
    Units: participants
        PF:Upper Extremity Activity-Much better (n=82,88)
    1
    4
        PF:Walking-Much better (n=112,108)
    5
    8
        PF:Climbing Stairs-Much better (n=105,110)
    4
    4
        PF:Other-Much better (n=65,68)
    4
    5
        C/SF-Much better (n=92,104)
    6
    5
        E/SocF-Much better (n=94,105)
    3
    8
        General EL-Much better (n=83,89)
    3
    8
        Sleep-Much better (n=85,91)
    3
    3
        Other-Much better (n=23,23)
    0
    1
        PF:Upper Extremity Activity-Slight better(n=82,88)
    7
    7
        PF:Walking-Slightly better (n=112,108)
    13
    16
        PF:Climbing Stairs-Slightly better (n=105,110)
    8
    13
        PF:Other-Slightly better (n=65,68)
    9
    10
        C/SF-Slightly better (n=92,104)
    12
    20
        E/SocF-Slightly better (n=94,105)
    9
    21
        General EL-Slightly better (n=83,89)
    12
    12
        Sleep-Slightly better (n=85,91)
    4
    8
        Other-Slightly better (n=23,23)
    5
    6
        PF:Upper Extremity Activity-Unchanged (n=82,88)
    67
    73
        PF:Walking-Unchanged (n=112,108)
    57
    60
        PF:Climbing Stairs-Unchanged (n=105,110)
    61
    65
        PF:Other-Unchanged (n=65,68)
    38
    44
        C/SF-Unchanged (n=92,104)
    71
    74
        E/SocF-Unchanged (n=94,105)
    75
    68
        General EL-Unchanged (n=83,89)
    54
    63
        Sleep-Unchanged (n=85,91)
    73
    76
        Other-Unchanged (n=23,23)
    15
    13
        PF:Upper Extremity Activity-Slight worse (n=82,88)
    6
    2
        PF:Walking-Slightly worse (n=112,108)
    19
    21
        PF:Climbing Stairs-Slightly worse (n=105,110)
    17
    15
        PF:Other-Slightly worse (n=65,68)
    7
    8
        C/SF-Slightly worse (n=92,104)
    3
    3
        E/SocF-Slightly worse (n=94,105)
    5
    6
        General EL-Slightly worse (n=83,89)
    11
    5
        Sleep-Slightly worse (n=85,91)
    5
    2
        Other-Slightly worse (n=23,23)
    2
    3
        PF:Upper Extremity Activity-Much worse (n=82,88)
    1
    2
        PF:Walking-Much worse (n=112,108)
    18
    3
        PF:Climbing Stairs-Much worse (n=105,110)
    15
    13
        PF:Other-Much worse (n=65,68)
    7
    1
        C/SF-Much worse (n=92,104)
    0
    2
        E/SocF-Much worse (n=94,105)
    2
    2
        General EL-Much worse (n=83,89)
    3
    1
        Sleep-Much worse (n=85,91)
    0
    2
        Other-Much worse (n=23,23)
    1
    0
    No statistical analyses for this end point

    Other pre-specified: Ataluren Plasma Concentration

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    End point title
    		 Ataluren Plasma Concentration [2]
    End point description
    Plasma samples for the determination of ataluren concentrations were analyzed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation of 0.5 micrograms/milliliter (mcg/mL). As-treated population included all randomized participants who actually received any study treatment. Here, ‘n‘ signifies participants evaluable at specified timepoint.
    End point type
    Other pre-specified
    End point timeframe
    Weeks 8, 16, 24, 32, 40, and 48
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint is reporting statistics for Ataluren arm only.
    End point values
    		 Ataluren
    Number of subjects analysed
    115
    Units: mcg/mL
    arithmetic mean (standard deviation)
        Week 08 (n=113)
    4.230 ± 5.3913
        Week 16 (n=113)
    3.429 ± 3.9275
        Week 24 (n=112)
    3.323 ± 3.6135
        Week 32 (n=113)
    3.480 ± 3.1053
        Week 40 (n=111)
    3.997 ± 4.7615
        Week 48 (n=110)
    3.544 ± 3.8082
    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 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
    		 Placebo Ataluren
    Number of subjects analysed
    115
    115
    Units: percentage of drug
        arithmetic mean (standard deviation)
    95.1 ± 9.43
    95.7 ± 7.57
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48

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    End point title
    		 Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48
    End point description
    NSAA is a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. It comprised tests for 17 abilities: ability to stand, rise from floor, get from lying to sitting, get from sitting to standing, raise one’s head, stand on one’s heels, hop, jump, and run. For each activity, a score of 0,1, or 2 was recorded, with 0=unable to achieve independently,1=modified method but achieves goal independently, or 2 =normal- achieves goal independently. Sum of these scores(except for 'raise one's head' activity) was reported as ordinal total score, which was transformed to a linear total score ranging from 0(worst) to 100(best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0. 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. ‘Number of participants analyzed‘=participants evaluable for this endpoint.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    108
    106
    Units: units on a scale
        arithmetic mean (standard deviation)
    -8.4 ± 10.65
    -6.3 ± 10.64
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48

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    End point title
    		 Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48
    End point description
    Changes in health-related quality of life(HRQL) were measured via PODCI questionnaire. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. Following PODCI domains were prespecified in protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 to 100, with 0=poor outcome/worse health, while 100=the highest level of functioning and least pain. 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. ‘Number of participants analyzed‘=participants evaluable for this endpoint.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 48
    End point values
    		 Placebo Ataluren
    Number of subjects analysed
    110
    109
    Units: units on a scale
    arithmetic mean (standard deviation)
        Transfers/Basic Mobility Score
    -8.8 ± 15.80
    -6.6 ± 14.76
        Sports/Physical Functioning Score
    -7.3 ± 15.87
    -5.6 ± 15.91
    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
    15.1
    Reporting groups
    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
    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
    		 Placebo Ataluren
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 115 (3.48%)
    4 / 115 (3.48%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Post-traumatic pain
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lower limb fracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Myocarditis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Adenoidal hypertrophy
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nasal turbinate hypertrophy
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hepatic function abnormal
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Tendon disorder
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bronchiolitis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Placebo Ataluren
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    99 / 115 (86.09%)
    103 / 115 (89.57%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Hypotension
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Aortic dilatation
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Flushing
         subjects affected / exposed
    3 / 115 (2.61%)
    0 / 115 (0.00%)
         occurrences all number
    3
    0
    Haematoma
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    12 / 115 (10.43%)
    16 / 115 (13.91%)
         occurrences all number
    17
    20
    Disease progression
         subjects affected / exposed
    14 / 115 (12.17%)
    9 / 115 (7.83%)
         occurrences all number
    15
    9
    Abasia
         subjects affected / exposed
    0 / 115 (0.00%)
    3 / 115 (2.61%)
         occurrences all number
    0
    3
    Oedema peripheral
         subjects affected / exposed
    0 / 115 (0.00%)
    3 / 115 (2.61%)
         occurrences all number
    0
    3
    Fatigue
         subjects affected / exposed
    3 / 115 (2.61%)
    1 / 115 (0.87%)
         occurrences all number
    3
    1
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Influenza like illness
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Gait disturbance
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Malaise
         subjects affected / exposed
    3 / 115 (2.61%)
    0 / 115 (0.00%)
         occurrences all number
    3
    0
    Thirst
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Immune system disorders
    Allergy to chemicals
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Seasonal allergy
         subjects affected / exposed
    2 / 115 (1.74%)
    2 / 115 (1.74%)
         occurrences all number
    2
    2
    Rhinitis allergic
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Allergy to vaccine
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Drug hypersensitivity
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Reproductive system and breast disorders
    Genital discomfort
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    13 / 115 (11.30%)
    19 / 115 (16.52%)
         occurrences all number
    17
    23
    Oropharyngeal pain
         subjects affected / exposed
    6 / 115 (5.22%)
    7 / 115 (6.09%)
         occurrences all number
    9
    7
    Epistaxis
         subjects affected / exposed
    4 / 115 (3.48%)
    7 / 115 (6.09%)
         occurrences all number
    11
    10
    Nasal congestion
         subjects affected / exposed
    2 / 115 (1.74%)
    3 / 115 (2.61%)
         occurrences all number
    3
    5
    Rhinorrhoea
         subjects affected / exposed
    3 / 115 (2.61%)
    3 / 115 (2.61%)
         occurrences all number
    3
    4
    Wheezing
         subjects affected / exposed
    0 / 115 (0.00%)
    3 / 115 (2.61%)
         occurrences all number
    0
    3
    Asthmatic crisis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Productive cough
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Sneezing
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Dyspnoea
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Sinus congestion
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    3
    0
    Sleep apnoea syndrome
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Throat irritation
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Attention deficit/hyperactivity disorder
         subjects affected / exposed
    1 / 115 (0.87%)
    3 / 115 (2.61%)
         occurrences all number
    1
    3
    Aggression
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Sleep disorder
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    3
    Abnormal behaviour
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Negativism
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Obsessive-compulsive disorder
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Anxiety
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Dysphemia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Middle insomnia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Mood swings
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Investigations
    Renin increased
         subjects affected / exposed
    3 / 115 (2.61%)
    1 / 115 (0.87%)
         occurrences all number
    3
    1
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Arthropod sting
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Concussion
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Fall
         subjects affected / exposed
    20 / 115 (17.39%)
    21 / 115 (18.26%)
         occurrences all number
    31
    35
    Contusion
         subjects affected / exposed
    4 / 115 (3.48%)
    3 / 115 (2.61%)
         occurrences all number
    4
    3
    Ligament sprain
         subjects affected / exposed
    7 / 115 (6.09%)
    3 / 115 (2.61%)
         occurrences all number
    7
    3
    Laceration
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    3
    Post-traumatic pain
         subjects affected / exposed
    4 / 115 (3.48%)
    2 / 115 (1.74%)
         occurrences all number
    5
    2
    Spinal compression fracture
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    2
    Electrical burn
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Excoriation
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Fibula fracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Foot fracture
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Hand fracture
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Joint injury
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Muscle injury
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Muscle strain
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Scratch
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Tibia fracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Tooth injury
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Ulna fracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Upper limb fracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Head injury
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Limb injury
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Lip injury
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Thermal burn
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Cardiac disorders
    Right ventricular hypertrophy
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Left ventricular dysfunction
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Left ventricular hypertrophy
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Myocardial fibrosis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Palpitations
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Cardiomyopathy
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Wolff-Parkinson-White syndrome
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    21 / 115 (18.26%)
    21 / 115 (18.26%)
         occurrences all number
    29
    55
    Autism
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Coordination abnormal
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Epilepsy
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Migraine
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    2
    Dizziness
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hypotonia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Sinus headache
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Petit mal epilepsy
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Psychomotor hyperactivity
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Restless legs syndrome
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Tremor
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Splenomegaly
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Leukocytosis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Lymphadenopathy
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    4 / 115 (3.48%)
    0 / 115 (0.00%)
         occurrences all number
    4
    0
    Middle ear effusion
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Tympanic membrane disorder
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Astigmatism
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Cataract
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Conjunctivitis
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Eye allergy
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Conjunctivitis allergic
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Eye movement disorder
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Eyelid oedema
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Myopia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    10 / 115 (8.70%)
    20 / 115 (17.39%)
         occurrences all number
    13
    33
    Abdominal pain upper
         subjects affected / exposed
    13 / 115 (11.30%)
    9 / 115 (7.83%)
         occurrences all number
    17
    9
    Abdominal pain
         subjects affected / exposed
    5 / 115 (4.35%)
    7 / 115 (6.09%)
         occurrences all number
    10
    8
    Abdominal discomfort
         subjects affected / exposed
    0 / 115 (0.00%)
    3 / 115 (2.61%)
         occurrences all number
    0
    4
    Constipation
         subjects affected / exposed
    10 / 115 (8.70%)
    3 / 115 (2.61%)
         occurrences all number
    12
    4
    Flatulence
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    2
    Abnormal faeces
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Breath odour
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Dental caries
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Dry mouth
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    2
    Dyspepsia
         subjects affected / exposed
    3 / 115 (2.61%)
    1 / 115 (0.87%)
         occurrences all number
    3
    1
    Gastritis
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Anal fissure
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Food poisoning
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Haematochezia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Inguinal hernia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Irritable bowel syndrome
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    21 / 115 (18.26%)
    26 / 115 (22.61%)
         occurrences all number
    26
    44
    Nausea
         subjects affected / exposed
    7 / 115 (6.09%)
    7 / 115 (6.09%)
         occurrences all number
    8
    7
    Tooth crowding
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Malocclusion
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Odynophagia
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Oral pain
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Rectal haemorrhage
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Swollen tongue
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Toothache
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Hepatic function abnormal
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hepatic steatosis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Blister
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Acne
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Dermatitis allergic
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    4 / 115 (3.48%)
    4 / 115 (3.48%)
         occurrences all number
    5
    6
    Ingrowing nail
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Pruritus
         subjects affected / exposed
    2 / 115 (1.74%)
    2 / 115 (1.74%)
         occurrences all number
    2
    2
    Rash erythematous
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    2
    Dry skin
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Ecchymosis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Hair texture abnormal
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Hirsutism
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Seborrhoeic dermatitis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Skin burning sensation
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Skin lesion
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Skin mass
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Erythema
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Skin exfoliation
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Skin hyperpigmentation
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    1 / 115 (0.87%)
    7 / 115 (6.09%)
         occurrences all number
    1
    7
    Dysuria
         subjects affected / exposed
    3 / 115 (2.61%)
    2 / 115 (1.74%)
         occurrences all number
    3
    4
    Bladder trabeculation
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Enuresis
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    4
    Flank pain
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Hypercalciuria
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Myoglobinuria
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Nephrolithiasis
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Urinary incontinence
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    2
    Polyuria
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Urine abnormality
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    5
    2
    Renal cyst
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Pollakiuria
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Hyperaldosteronism
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    8 / 115 (6.96%)
    11 / 115 (9.57%)
         occurrences all number
    8
    15
    Arthralgia
         subjects affected / exposed
    5 / 115 (4.35%)
    6 / 115 (5.22%)
         occurrences all number
    5
    7
    Foot deformity
         subjects affected / exposed
    2 / 115 (1.74%)
    3 / 115 (2.61%)
         occurrences all number
    3
    3
    Growing pains
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Bone pain
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Pain in extremity
         subjects affected / exposed
    14 / 115 (12.17%)
    10 / 115 (8.70%)
         occurrences all number
    17
    12
    Myalgia
         subjects affected / exposed
    0 / 115 (0.00%)
    3 / 115 (2.61%)
         occurrences all number
    0
    4
    Musculoskeletal chest pain
         subjects affected / exposed
    2 / 115 (1.74%)
    2 / 115 (1.74%)
         occurrences all number
    2
    3
    Musculoskeletal pain
         subjects affected / exposed
    2 / 115 (1.74%)
    2 / 115 (1.74%)
         occurrences all number
    2
    2
    Joint contracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Muscle atrophy
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Muscle tightness
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Muscular weakness
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Osteoporosis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Scoliosis
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Tendinous contracture
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Joint crepitation
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Lordosis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Muscle spasms
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Osteopenia
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Infections and infestations
    Ear infection
         subjects affected / exposed
    1 / 115 (0.87%)
    6 / 115 (5.22%)
         occurrences all number
    1
    8
    Gastroenteritis
         subjects affected / exposed
    4 / 115 (3.48%)
    5 / 115 (4.35%)
         occurrences all number
    4
    7
    Influenza
         subjects affected / exposed
    5 / 115 (4.35%)
    3 / 115 (2.61%)
         occurrences all number
    8
    4
    Bronchitis
         subjects affected / exposed
    3 / 115 (2.61%)
    2 / 115 (1.74%)
         occurrences all number
    3
    2
    Conjunctivitis infective
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Fungal skin infection
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    4
    Gastroenteritis viral
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Gastrointestinal viral infection
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    3
    Hordeolum
         subjects affected / exposed
    3 / 115 (2.61%)
    2 / 115 (1.74%)
         occurrences all number
    3
    3
    Eye infection
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Gingivitis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Herpes simplex
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    2
    Lower respiratory tract infection
         subjects affected / exposed
    4 / 115 (3.48%)
    1 / 115 (0.87%)
         occurrences all number
    4
    2
    Lung infection
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Acute tonsillitis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Adenoiditis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal infection
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Gingival infection
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Helminthic infection
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Infected bites
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    22 / 115 (19.13%)
    24 / 115 (20.87%)
         occurrences all number
    33
    43
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 115 (5.22%)
    11 / 115 (9.57%)
         occurrences all number
    11
    16
    Rhinitis
         subjects affected / exposed
    4 / 115 (3.48%)
    8 / 115 (6.96%)
         occurrences all number
    4
    13
    Pharyngitis
         subjects affected / exposed
    4 / 115 (3.48%)
    4 / 115 (3.48%)
         occurrences all number
    5
    4
    Tonsillitis
         subjects affected / exposed
    2 / 115 (1.74%)
    3 / 115 (2.61%)
         occurrences all number
    3
    3
    Sinusitis
         subjects affected / exposed
    2 / 115 (1.74%)
    5 / 115 (4.35%)
         occurrences all number
    2
    6
    Viral infection
         subjects affected / exposed
    3 / 115 (2.61%)
    3 / 115 (2.61%)
         occurrences all number
    3
    3
    Nail infection
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    3
    Respiratory tract infection
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    2
    Pneumonia
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Otitis externa
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Otitis media
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    3
    1
    Parasitic gastroenteritis
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Skin infection
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Mycoplasma infection
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Oral candidiasis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Oral herpes
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Rubella
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Tracheitis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Urinary tract infection
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Metabolism and nutrition disorders
    Hypertriglyceridaemia
         subjects affected / exposed
    3 / 115 (2.61%)
    5 / 115 (4.35%)
         occurrences all number
    3
    5
    Decreased appetite
         subjects affected / exposed
    2 / 115 (1.74%)
    3 / 115 (2.61%)
         occurrences all number
    2
    3
    Electrolyte imbalance
         subjects affected / exposed
    1 / 115 (0.87%)
    2 / 115 (1.74%)
         occurrences all number
    1
    2
    Vitamin D deficiency
         subjects affected / exposed
    0 / 115 (0.00%)
    2 / 115 (1.74%)
         occurrences all number
    0
    2
    Abnormal weight gain
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Dyslipidaemia
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Fluid retention
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    3
    Hypercholesterolaemia
         subjects affected / exposed
    2 / 115 (1.74%)
    1 / 115 (0.87%)
         occurrences all number
    2
    1
    Hyperlipidaemia
         subjects affected / exposed
    1 / 115 (0.87%)
    1 / 115 (0.87%)
         occurrences all number
    1
    1
    Iron deficiency
         subjects affected / exposed
    0 / 115 (0.00%)
    1 / 115 (0.87%)
         occurrences all number
    0
    1
    Hyperglycaemia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hyperkalaemia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hyperuricaemia
         subjects affected / exposed
    2 / 115 (1.74%)
    0 / 115 (0.00%)
         occurrences all number
    2
    0
    Hypervitaminosis D
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hypoglycaemia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Hyponatraemia
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Increased appetite
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0
    Metabolic acidosis
         subjects affected / exposed
    1 / 115 (0.87%)
    0 / 115 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Jun 2013
    The changes implemented with this amendment are: - Incorporated feedback from the European Medicines Agency (EMA) related to the non-allowance of protocol waivers; - Added the timed test of rising from supine to stand; - Amended the criteria for withdrawal of a participant from the study due to the participant's condition substantially worsening after initiating study drug to include worsening of cardiac events such as QTc interval limits, new evidence of symptomatic cardiomyopathy, and significant decrease in left ventricular ejection fraction; - Deleted any requirement that there be any discussion between the investigator and the PTC Therapeutics medical monitor before unblinding a participant; Added information regarding benefits and risks of study participation.
    04 Mar 2014
    The changes implemented with this amendment are: - Updated information regarding ongoing ataluren studies; - Clarified inclusion criterion related to documentation of gene sequencing; - Clarified inclusion criterion related to no more than 20% change from screening to baseline 6MWD; - Updated the approximate number of investigator sites participating in study; - Updated the requirement for collection of date of birth for Screening; - Updated the actions to be taken in event of urine blood abnormalities; - Added information regarding renal abnormalities related to aminoglycosides; - Updated information regarding drugs metabolized by cytochrome P450 enzymes; - Updated information regarding potential drug interactions; - Updated schedule of events to clarify the timing of events such as randomization, study drug administration in clinic, study drug dispensing, and the Visit 2, Day 2 6MWT; - Updated the recommended sequence of study procedures at Visit 2 to 8, by updating sequence of events and timing of randomization; - Clarified the timing of randomization of a participant at Visit 2, Day 2; - Clarified End-of-Treatment Visit procedures for participants who discontinue prematurely; - Clarified Post-Treatment Visits procedures; - Added information regarding videotaping of the 6MWT, timed function tests, and NSAA; - Clarified the schedule for administration of Activities of Daily Living/Disease Symptom Survey at baseline; - Updated the blood volumes for the required blood draws; - Added information regarding blood pressure assessment; - Summarized information regarding tumor findings in rat studies as already included in the Ataluren Investigator Brochure; - Deleted any mention of forfeiture of participation in the open-label extension study for breaking the blind of the study; - Added information regarding benefits and risks of study participation.

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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