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    Clinical Trial Results:
    A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy.

    Summary
    EudraCT number
    		 2010-020069-26
    Trial protocol
    		 DE   FR   NL   BE   IT   PL   CZ   ES   NO   DK   HU   Outside EU/EEA  
    Global end of trial date
    28 Jun 2013

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Oct 2019
    First version publication date
    26 Oct 2019
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    DMD114044
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01254019
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    GlaxoSmithKline
    Sponsor organisation address
    980 Great West Road, Brentford, Middlesex, United Kingdom,
    Public contact
    GSK Response Centre, GlaxoSmithKline, 1 8664357343, GSKClinicalSupportHD@GSK.com
    Scientific contact
    GSK Response Centre, GlaxoSmithKline, 1 8664357343, GSKClinicalSupportHD@GSK.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000746-PIP01-04
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    28 Jun 2013
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Jun 2013
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the efficacy of subcutaneous 6 mg/kg GSK2402968 versus placebo administered over 48 weeks in ambulant participants with DMD.
    Protection of trial subjects
    Not Applicable
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Dec 2010
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 5 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Argentina: 6
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    Brazil: 18
    Country: Number of subjects enrolled
    Canada: 20
    Country: Number of subjects enrolled
    Chile: 7
    Country: Number of subjects enrolled
    Czech Republic: 7
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    France: 16
    Country: Number of subjects enrolled
    Germany: 22
    Country: Number of subjects enrolled
    Italy: 26
    Country: Number of subjects enrolled
    Japan: 14
    Country: Number of subjects enrolled
    Korea, Republic of: 6
    Country: Number of subjects enrolled
    Netherlands: 6
    Country: Number of subjects enrolled
    Norway: 3
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    Russian Federation: 8
    Country: Number of subjects enrolled
    Spain: 11
    Country: Number of subjects enrolled
    Taiwan: 3
    Country: Number of subjects enrolled
    Turkey: 5
    Worldwide total number of subjects
    186
    EEA total number of subjects
    99
    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)
    169
    Adolescents (12-17 years)
    17
    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
    		 This study was conducted across 44 centers in 19 countries from 02 December 2010 to 28 June 2013.

    Pre-assignment
    Screening details
    		 A total of 186 participants were randomized which included males with a maximum age of 16 years, no adults were included in this study.

    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 single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo was administered as a single dose sterile solution via subcutaneous route preferably in the morning over the 48 week treatment period.

    Arm title
    		 GSK2402968 6mg/kg/week
    Arm description
    		 Participants received single dose of GSK2402968 6 milligrams per kilogram per week (mg/kg/week) subcutaneous injection preferably in the morning over the 48 week treatment period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    GSK2402968
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    GSK2402968 was administered as a single dose of GSK2402968 6 milligrams per kilogram per week (mg/kg/week) via subcutaneous route preferably in the morning over the 48 week treatment period.

    Number of subjects in period 1
    		Placebo GSK2402968 6mg/kg/week
    Started
    61
    125
    Completed
    60
    121
    Not completed
    1
    4
         Consent withdrawn by subject
    -
    2
         Adverse Event
    -
    2
         Protocol deviation
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.

    Reporting group title
    GSK2402968 6mg/kg/week
    Reporting group description
    		 Participants received single dose of GSK2402968 6 milligrams per kilogram per week (mg/kg/week) subcutaneous injection preferably in the morning over the 48 week treatment period.

    Reporting group values
    		 Placebo GSK2402968 6mg/kg/week Total
    Number of subjects
    		 61 125 186
    Age categorical
    Units: Subjects
        Overall Participants
    		 61 125 186
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 8.0 ± 2.37 8.3 ± 2.43 -
    Sex: Female, Male
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 61 125 186
    Race/Ethnicity, Customized
    Units: Subjects
        African American/African Heritage|
    		 1 0 1
        Asian - Central/South Asian Heritage|
    		 1 3 4
        Asian - East Asian Heritage|
    		 3 6 9
        Asian - Japanese Heritage|
    		 5 9 14
        Asian - South East Asian Heritage|
    		 0 2 2
        White - Arabic/North African Heritage|
    		 4 5 9
        White - White/Caucasian/European Heritage|
    		 46 95 141
        White - Mixed Race|
    		 0 1 1
        Mixed Race|
    		 1 4 5

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.

    Reporting group title
    GSK2402968 6mg/kg/week
    Reporting group description
    		 Participants received single dose of GSK2402968 6 milligrams per kilogram per week (mg/kg/week) subcutaneous injection preferably in the morning over the 48 week treatment period.

    Primary: Change from Baseline in muscle function using the 6 Minute Walking Distance (6MWD) test assessed at Week 48

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    End point title
    		 Change from Baseline in muscle function using the 6 Minute Walking Distance (6MWD) test assessed at Week 48
    End point description
    During the 6MWD, participants were asked to walk, at their own preferred speed, up and down a fixed distance until they were told to stop after 6 minutes. The participants were warned of the time and were told that they may stop earlier if they feel unable to continue. The total distance walked within 6 minutes (or until the participant stopped in case of early termination of the test), the 6MWD, was recorded in meters as well as any falls. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48. The intent-to-treat (ITT) population comprised all participants who received atleast one dose of study medication and for whom atleast one post-Baseline efficacy assessment was available. A mixed model with repeated measures (MMRM) analysis was performed, including all available data at each visit.
    End point type
    Primary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    59 [1]
    117 [2]
    Units: Meters
        least squares mean (standard error)
    -52.65 ± 10.423
    -42.32 ± 7.378
    Notes
    [1] - ITT Population
    [2] - ITT Population
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    176
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.415 [3]
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    10.334
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -14.645
         upper limit
    		 35.312
    Notes
    [3] - Statistical significance was assessed at the 5% level.

    Secondary: Change from Baseline in the linearized North Star Ambulatory Assessment (NSAA) total score at Week 48

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    End point title
    		 Change from Baseline in the linearized North Star Ambulatory Assessment (NSAA) total score at Week 48
    End point description
    The NSAA is a functional scale devised from the Hammersmith Scale of Motor Ability specifically for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was achieved by adding the responses of all activities, ranging from 0 to 34, with a score of 34 implying normal function and lower score implying more severe symptoms. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48. A positive change from Baseline indicated improvement.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    58 [4]
    117 [5]
    Units: Scores on a scale
        least squares mean (standard error)
    -6.7 ± 1.43
    -7.2 ± 1.01
    Notes
    [4] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [5] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    175
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.757
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.53
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.95
         upper limit
    		 2.88

    Secondary: Change from Baseline in the 4 stair climb (ascent) velocity at Week 48

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    End point title
    		 Change from Baseline in the 4 stair climb (ascent) velocity at Week 48
    End point description
    The participant was asked to ascend four steps. Time was recorded with a stopwatch from the initiation of movement until the participant stood on the fourth step. A flight of steps with handrail was used for this test. Number of stairs ascended per second was calculated as 4 divided by the time to ascend 4 complete stairs. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    55 [6]
    111 [7]
    Units: Stairs per second
        least squares mean (standard error)
    -0.12 ± 0.049
    -0.14 ± 0.035
    Notes
    [6] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [7] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    166
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.718
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.021
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.137
         upper limit
    		 0.095

    Secondary: Change from Baseline in the 10-meter walk/run velocity at Week 48

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    End point title
    		 Change from Baseline in the 10-meter walk/run velocity at Week 48
    End point description
    The participant was instructed to perform the test bare foot. No aids or orthoses were allowed. The participant was asked to traverse a marked 10-meter measured walkway as quickly as he safely could. Time was recorded to one tenth of a second with a stopwatch from when his first foot crossed the start line until when the second foot crossed the finish line. Baseline was defined as participant's randomization assessment at visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    58 [8]
    117 [9]
    Units: Meters per second
        least squares mean (standard error)
    -0.20 ± 0.050
    -0.21 ± 0.035
    Notes
    [8] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [9] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    175
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.881
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.009
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.129
         upper limit
    		 0.111

    Secondary: Change from Baseline in the timed function test Rise from floor at Week 48

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    End point title
    		 Change from Baseline in the timed function test Rise from floor at Week 48
    End point description
    The participant stood from a standardized supine position as quickly as possible when told to go. Time was recorded with a stopwatch from the initiation of movement until the assumption of upright standing. No aids or orthoses were allowed. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    44 [10]
    91 [11]
    Units: Seconds
        least squares mean (standard error)
    7.48 ± 2.080
    6.36 ± 1.463
    Notes
    [10] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [11] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    135
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.658 [12]
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    -1.115
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -6.097
         upper limit
    		 3.866
    Notes
    [12] - Negative difference compared to placebo represents benefit over placebo.

    Secondary: Change from Baseline in the 4 stair climb (descent) velocity at Week 48

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    End point title
    		 Change from Baseline in the 4 stair climb (descent) velocity at Week 48
    End point description
    The participant was asked to descend four steps. Time was recorded with a stopwatch from the initiation of movement until the participant stood on the fourth step. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    55 [13]
    109 [14]
    Units: Stairs per second
        least squares mean (standard error)
    -0.15 ± 0.052
    -0.11 ± 0.037
    Notes
    [13] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [14] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    164
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.513 [15]
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.041
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.082
         upper limit
    		 0.164
    Notes
    [15] - Positive difference compared to placebo represents benefit over placebo

    Secondary: Change from Baseline in muscle strength (total score) at Week 48

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    End point title
    		 Change from Baseline in muscle strength (total score) at Week 48
    End point description
    Muscle strength was recorded by handheld myometry using a micro force evaluation testing 2 (FET2) myometer. Upper and lower limb proximal muscles were evaluated including knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors. The muscle strength total score (pounds) was the sum of the 12 individual muscle strength tests. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    58 [16]
    118 [17]
    Units: lbs
        least squares mean (standard error)
    -1.21 ± 2.729
    -2.18 ± 1.926
    Notes
    [16] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [17] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    176
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.769 [18]
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.965
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.446
         upper limit
    		 5.516
    Notes
    [18] - Positive difference compared to placebo represents benefit over placebo

    Secondary: Kaplan-Meier Estimates for Time to loss of ambulation

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    End point title
    		 Kaplan-Meier Estimates for Time to loss of ambulation
    End point description
    All participants were ambulant when entered into the study; however they could have become non-ambulant at some time during the study. The date was recorded and the variable time to loss of ambulation was calculated as: time to loss of ambulation = date of loss of ambulation – date of first dose. Median and interquartile range i.e. 1st and 3rd quartile is presented. 99999 indicates calculation of statistics was not possible due to insufficient events.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [19]
    125 [20]
    Units: Days
        median (inter-quartile range (Q1-Q3))
    99999 (99999 to 99999)
    99999 (390.0 to 99999)
    Notes
    [19] - ITT Population
    [20] - ITT Population
    No statistical analyses for this end point

    Secondary: Number of participants who experienced accidental falls during 6MWD assessments at Week 48

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    End point title
    		 Number of participants who experienced accidental falls during 6MWD assessments at Week 48
    End point description
    The number of accidental falls occurring during the 6MWD were counted. Data has been presented for the number of participants who experienced accidental falls (from 0 to 1) during the 6MWD assessment. Only those participants with data available at the indicated time points were analyzed.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    53 [21]
    103 [22]
    Units: Participants
        Number of falls=0|
    48
    102
        Number of falls=1|
    5
    1
    Notes
    [21] - ITT Population
    [22] - ITT Population
    No statistical analyses for this end point

    Secondary: Change from Baseline in creatine kinase serum concentrations at Week 48

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    End point title
    		 Change from Baseline in creatine kinase serum concentrations at Week 48
    End point description
    Creatine kinase is a muscle-specific enzyme; its level in serum is considered to reflect the extent of muscle damage. In the blood samples drawn to this purpose, the serum level of creatine kinase were measured. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    60 [23]
    118 [24]
    Units: International units per liter
        least squares mean (standard error)
    -1228.5 ± 500.59
    -5273.5 ± 359.05
    Notes
    [23] - ITT population. MMRM analysis was performed, including all available data at each visit.
    [24] - ITT population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    178
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001 [25]
    Method
    		 Mixed Effect Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    -4044.99
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5232.21
         upper limit
    		 -2857.77
    Notes
    [25] - No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.

    Secondary: Change from Baseline in Pulmonary Function test Forced vital capacity (FVC) and Forced expiratory volume in 1 second (FEV1) at Week 48

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    End point title
    		 Change from Baseline in Pulmonary Function test Forced vital capacity (FVC) and Forced expiratory volume in 1 second (FEV1) at Week 48
    End point description
    The FEV1 is the volume of air forcefully exhaled in 1 second, whereas the FVC is the volume of air that can be maximally forcefully exhaled using non-invasive spirometry was conducted to determine actual and percentage values for FVC and FEV1. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48. Only those participants available at the specified time points were analyzed (indicated by n=X in category titles).
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [26]
    125 [27]
    Units: Liters
    arithmetic mean (standard deviation)
        FVC at Week 48; n=58, 121|
    0.118 ± 0.1847
    0.087 ± 0.2337
        FEV1 at Week 48; n=55, 116|
    0.126 ± 0.2530
    0.049 ± 0.2997
    Notes
    [26] - ITT Population
    [27] - ITT Population
    No statistical analyses for this end point

    Secondary: Number of participants with Identified Mutation: DMD Exon 51 skip (upon muscle biopsies) at Week 48

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    End point title
    		 Number of participants with Identified Mutation: DMD Exon 51 skip (upon muscle biopsies) at Week 48
    End point description
    Biopsies were taken from their tibialis anterior muscle and few were taken from quadriceps. Total muscle ribonucleic acid (RNA) was isolated from muscle tissue sections and was analyzed by reverse transcriptase polymer chain reaction (RT-PCR). RT-PCR analysis focused on the area flanking the targeted exon 51 was performed to detect specific exon 51 skipping in muscle. Depending on the participants mutation different sets of DMD-gene specific RT and PCR primers were used. Sequence analysis was performed on isolated PCR products to confirm specific exon 51 skip band detection. Only those participants with data available at the indicated time points were analyzed.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    59 [28]
    119 [29]
    Units: Participants
        DMD Exon 51 skip, Band detected|
    48
    106
        DMD Exon 51 skip, No Band detected|
    9
    9
        DMD Exon 51 skip, No result|
    0
    2
        DMD Exon 51 skip, Not analyzed|
    2
    2
    Notes
    [28] - ITT Population
    [29] - ITT Population
    No statistical analyses for this end point

    Secondary: Change from Baseline in Pediatric Quality of Life (PedsQL) Total Score at Week 48

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    End point title
    		 Change from Baseline in Pediatric Quality of Life (PedsQL) Total Score at Week 48
    End point description
    PedsQL version 3.0 scale is used to measure pediatric quality of life in children with neuromuscular disorders. The 25-item PedsQL encompasses 3 scales About My/My Childs Neuromuscular Disease (17 items), Communication (3 items), About Our Family Resources (5 items). A 5-point response scale is utilized (where 0=never a problem; 4=almost always a problem). It was assessed both by child and parent. PedsQL total score was calculated by reverse scoring individual items and linearly transforming the score to a 0-100 scale, where higher scores indicated better health-related quality of life. To reverse score individual items, the 0-4 scale items were transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, and 4=0. Total score was then calculated as sum of items divided by number of items answered. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48. A positive change from Baseline indicated improvement.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [30]
    125 [31]
    Units: Scores on a scale
    arithmetic mean (standard deviation)
        Assesor child at week 48; n=57, 114|
    0.52 ± 11.313
    1.36 ± 11.360
        Assesor parent at week 48; n=58, 117|
    -0.11 ± 11.064
    -1.19 ± 11.269
    Notes
    [30] - ITT population. Only those participants available at the specified time points were analyzed.
    [31] - ITT population. Only those participants available at the specified time points were analyzed.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Pulmonary Function test peak cough flow (PCF) and peak flow (PF) at Week 48

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    End point title
    		 Change from Baseline in Pulmonary Function test peak cough flow (PCF) and peak flow (PF) at Week 48
    End point description
    The PF also called peak expiratory flow rate (PEFR) is a participants maximum speed of expiration, as measured with a peak flow meter, a small, hand-held device used to monitor a participants ability to breathe out air. PCF was measured for participants wearing a nose clip and performing a maximum cough into a pocket peak flow meter. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [32]
    125 [33]
    Units: Liters per minute
    arithmetic mean (standard deviation)
        PF at Week 48; n=59, 121|
    25.810 ± 41.5342
    11.603 ± 42.6964
        PCF at Week 48; n=59, 120|
    22.7 ± 42.46
    10.7 ± 45.04
    Notes
    [32] - ITT population. Only those participants available at the specified time points were analyzed.
    [33] - ITT population. Only those participants available at the specified time points were analyzed.
    No statistical analyses for this end point

    Secondary: Number of participants who showed improvement on Clinician Global Impression of Improvement (CGI-I) scale at Week 48

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    End point title
    		 Number of participants who showed improvement on Clinician Global Impression of Improvement (CGI-I) scale at Week 48
    End point description
    The CGI-I is scored based on the clinician’s reflection of the participant’s current overall clinical condition compared to the overall clinical condition just prior to the initiation of medication use (i.e., the period prior to Randomization). The CGI-I is rated without regard to the clinician’s belief that any clinical changes are or are not due to medication and without consideration of the etiology of the symptoms. The CGI-I is measured on a 7-point Likert scale (where 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse). The score ranged from 1-7, where lower score indicated more improvement and higher score indicated less improvement.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    57 [34]
    116 [35]
    Units: Participants
        Very much improved|
    0
    3
        Much improved|
    1
    9
        Minimally improved|
    2
    23
        No change|
    27
    41
        Minimally worse|
    20
    30
        Much worse|
    6
    10
        Very much worse|
    1
    0
    Notes
    [34] - ITT population. Only those participants with data available at specified time points were analyzed
    [35] - ITT population. Only those participants with data available at specified time points were analyzed
    No statistical analyses for this end point

    Secondary: Change from Baseline in Health Utilities Index (HUI) Scores at Week 48

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    End point title
    		 Change from Baseline in Health Utilities Index (HUI) Scores at Week 48
    End point description
    A 15-item HUI questionnaire assessed Health-related quality of life (HRQoL). Responses from 15-item HUI were used to quantify HRQoL according to 2 health status classification systems, HUI Mark 2 (HUI2) and HUI Mark 3 (HUI3). HUI2 assessed 7 HRQoL dimensions: sensation, mobility, emotion, cognition, self care, pain and fertility. HUI3 assessed 8 HRQoL dimensions: vision, hearing, speech, ambulation, dexterity, emotion, cognition and pain. Both HUI2 (range from -0.03 to 1.0) and HUI3 (range from -0.36 to 1.0) utility scores were calculated using algorithms incorporating community-derived preference weights. A utility value of 1.0 represented perfect health and a utility value of 0.0 represented death. Lowest possible HUI2 score was -0.03 and for HUI3 score was -0.36, where scores less than 0 represented health states considered worse than death. Change from Baseline was calculated by subtracting Baseline value from Week 48 value. A positive change from Baseline indicated improvement.
    End point type
    Secondary
    End point timeframe
    Baseline (Randomization Visit, Day 0) and Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [36]
    125 [37]
    Units: Scores on a scale
    least squares mean (standard error)
        HUI2 at Week 48; n=53, 112|
    -0.052 ± 0.0190
    -0.023 ± 0.0133
        HUI3 at Week 48; n=56, 116|
    -0.061 ± 0.0266
    -0.056 ± 0.0188
    Notes
    [36] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    [37] - ITT Population. MMRM analysis was performed, including all available data at each visit.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo, HUI2 at Week 48
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    186
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.207 [38]
    Method
    		 Mixed Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.0288
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.0161
         upper limit
    		 0.0738
    Notes
    [38] - No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.
    Statistical analysis title
    		 GSK2402968 6mg/kg/week Vs Placebo, HUI3 at Week 48
    Comparison groups
    Placebo v GSK2402968 6mg/kg/week
    Number of subjects included in analysis
    186
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.88 [39]
    Method
    		 Mixed Model for Repeated Measures
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.0048
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.058
         upper limit
    		 0.0676
    Notes
    [39] - No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.

    Secondary: Number of participants with adverse events (AE) and severe adverse events (SAE)

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    End point title
    		 Number of participants with adverse events (AE) and severe adverse events (SAE)
    End point description
    An AE is any untoward medical occurrence in a clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect.
    End point type
    Secondary
    End point timeframe
    Up to Follow-up (Week 68)
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [40]
    125 [41]
    Units: Participants
        Any AE|
    58
    123
        Any SAE|
    5
    13
    Notes
    [40] - Safety Population comprised of all participants who received at least one dose of study medication
    [41] - Safety Population comprised of all participants who received at least one dose of study medication
    No statistical analyses for this end point

    Secondary: Number of participants with vital sign data for systolic blood pressure (SBP) and diastolic blood pressure (DBP) and heart rate (HR) of potential clinical concern (PCC) at any visit post-Baseline

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    End point title
    		 Number of participants with vital sign data for systolic blood pressure (SBP) and diastolic blood pressure (DBP) and heart rate (HR) of potential clinical concern (PCC) at any visit post-Baseline
    End point description
    Blood pressure SBP, DBP and HR were recorded after five minutes of rest in a semi-supine position. The following changes from Baseline (Day 0) in vital signs were considered to be of potential clinical concern: DBP was defined as high (increase from Baseline >=20 and >=40 millimeters of mercury [mmHg] and low (decrease from Baseline >=20 and >=40 mmHg), SBP high (increase from Baseline >=10 and >=20 mmHg and low (decrease from Baseline >=10 and >=20 mmHg) and for HR high (increase from Baseline >=20 and >=40 beats per minute [bpm] and low (decrease from Baseline >=20 and >=40 bpm). Only those parameters for which a value of PCC was reported at any visit post-Baseline is presented.
    End point type
    Secondary
    End point timeframe
    Up to Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [42]
    125 [43]
    Units: Participants
        DBP, High at any visit post-Baseline|
    22
    52
        HR, low at any visit post-Baseline|
    19
    49
        HR, high at any visit post-Baseline|
    18
    32
        SBP, low at any visit post-Baseline|
    8
    7
        SBP, high at any visit post-Baseline|
    28
    72
    Notes
    [42] - Safety Population
    [43] - Safety Population
    No statistical analyses for this end point

    Secondary: Number of participants with abnormal-clinically significant Electrocardiogram (ECG) findings at any visit post-Baseline

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    End point title
    		 Number of participants with abnormal-clinically significant Electrocardiogram (ECG) findings at any visit post-Baseline
    End point description
    ECG measurements were carried out and the clinical interpretation of the ECG by the investigator was recorded as normal, abnormal but not clinically significant and abnormal clinically significant. The PCC ranges include, QT interval corrected for heart rate by Bazett’s formula (QTcB) or QT interval corrected for heart rate by Fridericia’s formula (QTcF) >450 milliseconds and any increase from Baseline of QTcB or QTcF. Participants were categorized as abnormal clinically significant based on the investigator’s judgment and PCC ranges. Data has been presented for number of participants with abnormal clinically significant findings at any visit post-Baseline.
    End point type
    Secondary
    End point timeframe
    Up to Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [44]
    125 [45]
    Units: Participants
    0
    5
    Notes
    [44] - Safety Population
    [45] - Safety Population
    No statistical analyses for this end point

    Secondary: Number of participants with hematology parameters of PCC at any visit post-Baseline

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    End point title
    		 Number of participants with hematology parameters of PCC at any visit post-Baseline
    End point description
    Laboratory samples were collected for analysis of hematology parameters. The PCC values for hematology parameters: hematocrit was 1.02 x Upper limit of normal (ULN), for hemoglobin was 1.03 x ULN, for lymphocytes was 0.81 x lower limit of normal (LLN), for platelet count was 0.67 x LLN and 1.57 x ULN, for total neutrophils was 0.83 x LLN, and that for white blood cell count was 0.67 x LLN and value of 1.82 x ULN. Only those parameters for which a value of PCC was reported at any visit post-Baseline have been presented.
    End point type
    Secondary
    End point timeframe
    Up to Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [46]
    125 [47]
    Units: Participants
        Hematocrit > reference range high|
    12
    27
        Hemoglobin > reference range high|
    2
    6
        Lymphocytes < reference range low|
    6
    9
        Platelet count > reference range high|
    1
    0
        Platelet count < reference range low|
    0
    1
        Total neutrophils < reference range low|
    8
    9
        White blood cell count < reference range low|
    3
    4
    Notes
    [46] - Safety Population
    [47] - Safety Population
    No statistical analyses for this end point

    Secondary: Number of participants with coagulation parameters of PCC at any visit post-Baseline

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    End point title
    		 Number of participants with coagulation parameters of PCC at any visit post-Baseline
    End point description
    Laboratory samples were collected for analysis of coagulation parameters. The PCC values for coagulation parameters activated partial thromboplastin time (aPTT) was 1.5 x ULN and aPTT ratio also known as international normalized ratio (INR) was 1.2 x ULN. Only those parameters for which a value of PCC was reported at any visit post-Baseline is presented.
    End point type
    Secondary
    End point timeframe
    Up to Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [48]
    125 [49]
    Units: Participants
        aPTT > reference range high|
    6
    6
        INR > reference range high|
    11
    32
    Notes
    [48] - Safety Population
    [49] - Safety Population
    No statistical analyses for this end point

    Secondary: Number of participants with clinical chemistry parameters of PCC at any visit post-Baseline

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    End point title
    		 Number of participants with clinical chemistry parameters of PCC at any visit post-Baseline
    End point description
    Laboratory samples were collected for analysis of chemistry parameters. The PCC values for chemistry parameters for alanine amino transferase (ALT) plus total bilirubin (TB) was >=1.5 x ULN for TB and >=2 x ULN for ALT, for albumin was 0.86 x LLN, for asparatate amino transferase (AST) was >=2 x ULN, for calcium was 0.91 x LLN and 1.06 x ULN, for glucose was 0.71 x LLN and 1.41 x ULN, for phosphorus was 0.80 x LLN and 1.14 x ULN, for sodium was 0.96 x LLN and 1.03 x ULN, for potassium was 0.86 x LLN and 1.10 x ULN and that for alkaline phosphatase was >=2x ULN. Only those parameters for which a value of PCC was reported at any visit post-Baseline is presented.
    End point type
    Secondary
    End point timeframe
    Up to Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [50]
    125 [51]
    Units: Participants
        ALT plus TB > reference range high|
    0
    2
        ALT > reference range high|
    61
    125
        Albumin < reference range low|
    0
    1
        AST > reference range high|
    61
    125
        Calcium > reference range high|
    1
    3
        Calcium < reference range low|
    0
    3
        Glucose > reference range high|
    3
    5
        Glucose < reference range low|
    0
    2
        Phosphorus > reference range high|
    1
    8
        Sodium > reference range high|
    2
    9
        Potassium > reference range high|
    0
    0
        Potassium < reference range low|
    0
    0
        Alkaline phosphatase > reference range high|
    0
    0
    Notes
    [50] - Safety Population
    [51] - Safety Population
    No statistical analyses for this end point

    Secondary: Number of participants with urinalysis data outside the reference range (>reference range high) at any visit post- Baseline

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    End point title
    		 Number of participants with urinalysis data outside the reference range (>reference range high) at any visit post- Baseline
    End point description
    Urine samples were collected for analysis of abnormal urine parameters. Quantitative examination included the assessment for urine albumin excretion rate, urine alpha-1-microglobulin, urine creatinine excretion-24 hour and urine protein excretion-24 hour. Only those parameters for which a value of >reference range high was reported at any visit post-Baseline is presented.
    End point type
    Secondary
    End point timeframe
    Up to Week 48
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    61 [52]
    125 [53]
    Units: Participants
        Urine Albumin excretion rate > reference range|
    1
    15
        Urine Protein excretion-24 hour > reference range
    4
    54
    Notes
    [52] - Safety Population. Only those participants available at the specified time points were analyzed.
    [53] - Safety Population. Only those participants available at the specified time points were analyzed.
    No statistical analyses for this end point

    Secondary: Plasma Concentrations of GSK2402968 Following Subcutaneous Administration

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    End point title
    		 Plasma Concentrations of GSK2402968 Following Subcutaneous Administration
    End point description
    Blood samples for pharmacokinetic assessment were taken at Week 0 (Randomization) at 0.5, 1, 3 hours post-dose and at Week 8,12, 24, 36 and 47 at pre-dose, and between 1 and 4 hours post-dose. Data has been presented for plasma concentrations of GSK2402968 following subcutaneous administration. The pharmacokinetic population comprised all participants who were randomized to the study and from whom at least one blood sample was obtained for assessment of GSK2402968 concentration. Only those participants available at the specified time points were analyzed (indicated by n=X in category titles).
    End point type
    Secondary
    End point timeframe
    Randomization (Week 0 at 0.5, 1 and 3 hours), Week 8 (pre-dose, 1-4 hours), Week 12 (pre-dose, 1-4 hours), Week 24 (pre-dose, 1-4 hours), Week 36 (pre-dose, 1-4 hours), Week 47 (pre-dose, 1-4 hours)
    End point values
    		 Placebo GSK2402968 6mg/kg/week
    Number of subjects analysed
    0 [54]
    125 [55]
    Units: Nanograms per millimeter
    median (full range (min-max))
        Week 0, 0.5 hours; n=120|
    ( to )
    3349.595 (572.07 to 10408.94)
        Week 0, 1 hour; n=122|
    ( to )
    4946.950 (1094.90 to 13159.51)
        Week 0, 3 hour; n=121|
    ( to )
    5932.740 (1794.00 to 12765.26)
        Week 8, Pre-dose; n=47|
    ( to )
    12.730 (0.00 to 4424.28)
        Week 8, 1-4 hours; n=47|
    ( to )
    5100.220 (7.62 to 10471.64)
        Week 12, Pre-dose; n=47|
    ( to )
    19.090 (4.60 to 50.95)
        Week 12, 1-4 hours; n=47|
    ( to )
    5613.040 (10.89 to 10583.33)
        Week 24, Pre-dose; n=49|
    ( to )
    40.920 (0.00 to 186.87)
        Week 24, 1-4 hours; n=49|
    ( to )
    6141.580 (40.65 to 11902.15)
        Week 36, Pre-dose; n=38|
    ( to )
    55.185 (22.64 to 6590.81)
        Week 36, 1-4 hours; n=37|
    ( to )
    5755.950 (68.84 to 12967.56)
        Week 47, Pre-dose; n=106|
    ( to )
    61.395 (3.86 to 5922.31)
        Week 47, 1-4 hours; n=99|
    ( to )
    5266.240 (46.42 to 13841.11)
    Notes
    [54] - Cannot measure plasma concentrations of verum in the placebo group
    [55] - Pharmacokinetic Population
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to Follow-up (Week 68)
    Adverse event reporting additional description
    The Safety Population was used for analysis.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    16.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.

    Reporting group title
    GSK2402968 6mg/kg/week
    Reporting group description
    		 Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.

    Serious adverse events
    		 Placebo GSK2402968 6mg/kg/week
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 61 (8.20%)
    13 / 125 (10.40%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    International normalised ratio increased
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Head injury
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lumbar vertebral fracture
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Patella fracture
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 125 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Toxicity to various agents
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 125 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Wound dehiscence
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac fibrillation
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myocardial ischaemia
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Benign intracranial hypertension
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Intercostal neuralgia
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 125 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Intracranial venous sinus thrombosis
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Spinal pain
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Enteritis
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Glomerulonephritis
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 125 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 125 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 125 (0.80%)
         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: 5%
    Non-serious adverse events
    		 Placebo GSK2402968 6mg/kg/week
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    58 / 61 (95.08%)
    123 / 125 (98.40%)
    Investigations
    Protein urine present
         subjects affected / exposed
    4 / 61 (6.56%)
    17 / 125 (13.60%)
         occurrences all number
    9
    30
    Red blood cells urine positive
         subjects affected / exposed
    4 / 61 (6.56%)
    14 / 125 (11.20%)
         occurrences all number
    5
    18
    Cystatin C increased
         subjects affected / exposed
    2 / 61 (3.28%)
    14 / 125 (11.20%)
         occurrences all number
    4
    18
    Red blood cells urine
         subjects affected / exposed
    4 / 61 (6.56%)
    11 / 125 (8.80%)
         occurrences all number
    6
    21
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    2 / 61 (3.28%)
    11 / 125 (8.80%)
         occurrences all number
    3
    18
    Blood fibrinogen decreased
         subjects affected / exposed
    6 / 61 (9.84%)
    3 / 125 (2.40%)
         occurrences all number
    8
    3
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    12 / 61 (19.67%)
    27 / 125 (21.60%)
         occurrences all number
    16
    54
    Contusion
         subjects affected / exposed
    7 / 61 (11.48%)
    8 / 125 (6.40%)
         occurrences all number
    10
    19
    Excoriation
         subjects affected / exposed
    3 / 61 (4.92%)
    7 / 125 (5.60%)
         occurrences all number
    3
    9
    Ligament sprain
         subjects affected / exposed
    3 / 61 (4.92%)
    7 / 125 (5.60%)
         occurrences all number
    4
    7
    Nervous system disorders
    Headache
         subjects affected / exposed
    11 / 61 (18.03%)
    33 / 125 (26.40%)
         occurrences all number
    27
    97
    General disorders and administration site conditions
    Injection site erythema
         subjects affected / exposed
    4 / 61 (6.56%)
    62 / 125 (49.60%)
         occurrences all number
    6
    494
    Injection site discolouration
         subjects affected / exposed
    2 / 61 (3.28%)
    41 / 125 (32.80%)
         occurrences all number
    3
    230
    Pyrexia
         subjects affected / exposed
    15 / 61 (24.59%)
    27 / 125 (21.60%)
         occurrences all number
    23
    42
    Injection site pain
         subjects affected / exposed
    2 / 61 (3.28%)
    23 / 125 (18.40%)
         occurrences all number
    3
    41
    Injection site reaction
         subjects affected / exposed
    1 / 61 (1.64%)
    24 / 125 (19.20%)
         occurrences all number
    3
    105
    Injection site bruising
         subjects affected / exposed
    6 / 61 (9.84%)
    11 / 125 (8.80%)
         occurrences all number
    18
    168
    Injection site induration
         subjects affected / exposed
    0 / 61 (0.00%)
    17 / 125 (13.60%)
         occurrences all number
    0
    35
    Injection site pruritus
         subjects affected / exposed
    0 / 61 (0.00%)
    17 / 125 (13.60%)
         occurrences all number
    0
    34
    Injection site haematoma
         subjects affected / exposed
    2 / 61 (3.28%)
    9 / 125 (7.20%)
         occurrences all number
    4
    21
    Injection site atrophy
         subjects affected / exposed
    0 / 61 (0.00%)
    9 / 125 (7.20%)
         occurrences all number
    0
    12
    Injection site urticaria
         subjects affected / exposed
    0 / 61 (0.00%)
    9 / 125 (7.20%)
         occurrences all number
    0
    38
    Injection site swelling
         subjects affected / exposed
    0 / 61 (0.00%)
    8 / 125 (6.40%)
         occurrences all number
    0
    20
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    4 / 61 (6.56%)
    6 / 125 (4.80%)
         occurrences all number
    5
    7
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    13 / 61 (21.31%)
    28 / 125 (22.40%)
         occurrences all number
    18
    53
    Diarrhoea
         subjects affected / exposed
    9 / 61 (14.75%)
    23 / 125 (18.40%)
         occurrences all number
    20
    38
    Abdominal pain
         subjects affected / exposed
    7 / 61 (11.48%)
    14 / 125 (11.20%)
         occurrences all number
    11
    22
    Abdominal pain upper
         subjects affected / exposed
    3 / 61 (4.92%)
    9 / 125 (7.20%)
         occurrences all number
    19
    12
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    12 / 61 (19.67%)
    24 / 125 (19.20%)
         occurrences all number
    18
    30
    Epistaxis
         subjects affected / exposed
    5 / 61 (8.20%)
    11 / 125 (8.80%)
         occurrences all number
    6
    15
    Oropharyngeal pain
         subjects affected / exposed
    1 / 61 (1.64%)
    10 / 125 (8.00%)
         occurrences all number
    1
    15
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    6 / 61 (9.84%)
    3 / 125 (2.40%)
         occurrences all number
    7
    3
    Erythema
         subjects affected / exposed
    1 / 61 (1.64%)
    7 / 125 (5.60%)
         occurrences all number
    1
    40
    Pruritus
         subjects affected / exposed
    1 / 61 (1.64%)
    7 / 125 (5.60%)
         occurrences all number
    1
    8
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    11 / 61 (18.03%)
    42 / 125 (33.60%)
         occurrences all number
    18
    99
    Haematuria
         subjects affected / exposed
    5 / 61 (8.20%)
    21 / 125 (16.80%)
         occurrences all number
    11
    43
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    12 / 61 (19.67%)
    11 / 125 (8.80%)
         occurrences all number
    28
    21
    Back pain
         subjects affected / exposed
    5 / 61 (8.20%)
    8 / 125 (6.40%)
         occurrences all number
    7
    12
    Arthralgia
         subjects affected / exposed
    1 / 61 (1.64%)
    10 / 125 (8.00%)
         occurrences all number
    1
    12
    Muscle spasms
         subjects affected / exposed
    4 / 61 (6.56%)
    5 / 125 (4.00%)
         occurrences all number
    6
    5
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    25 / 61 (40.98%)
    38 / 125 (30.40%)
         occurrences all number
    45
    68
    Upper respiratory tract infection
         subjects affected / exposed
    8 / 61 (13.11%)
    15 / 125 (12.00%)
         occurrences all number
    18
    26
    Gastroenteritis
         subjects affected / exposed
    6 / 61 (9.84%)
    15 / 125 (12.00%)
         occurrences all number
    5
    19
    Rhinitis
         subjects affected / exposed
    3 / 61 (4.92%)
    10 / 125 (8.00%)
         occurrences all number
    5
    14
    Influenza
         subjects affected / exposed
    4 / 61 (6.56%)
    7 / 125 (5.60%)
         occurrences all number
    4
    7
    Ear infection
         subjects affected / exposed
    5 / 61 (8.20%)
    4 / 125 (3.20%)
         occurrences all number
    5
    4
    Pharyngitis
         subjects affected / exposed
    4 / 61 (6.56%)
    5 / 125 (4.00%)
         occurrences all number
    6
    5
    Bronchitis
         subjects affected / exposed
    4 / 61 (6.56%)
    4 / 125 (3.20%)
         occurrences all number
    5
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Sep 2010
    This amendment applied to all sites and countries and: corrected previous errors discovered post finalization of original protocol; added further justification to the selection of 6 mg/kg weekly dosing; added new wording regarding the rotation of injection sites; changed frequency of collection of dystrophin antibodies to reduce the blood volumes; expanded the window for performing muscle biopsy; changed the timing for the 24 hour urine collection timing; clarified wording around the use of concomitant medications; added language clarifying that dual-energy X-ray absorptiometry (DEXA) should not be performed when there may be confounders to interpretation (e.g. on boys with metal rods/metal implants which could create artefacts); added language around stopping and follow-up criteria for laboratory safety parameters related to liver, renal inflammation and disseminated intravascular coagulation (DIC); revised statistical analysis sections; updated the Sponsor Information Page and added additional Quality of Life instrument, the Health Utilities Index (HUI).
    21 Jun 2011
    This amendment applied to all countries and all sites and was implemented to update the safety monitoring criteria, to remove the requirement for contraception, added MRI sub-study in Brazil and Argentina, updated the study design and End of Treatment section to allow for subjects who withdraw early from the study for safety reasons to enter the extension study. This allowed subjects for whom the risk:benefit assessment supports continued treatment to have further access to drisapersen. This amendment also implemented the addition of two secondary endpoints as requested by European Medicines Agency (EMA)
    31 Oct 2011
    This country/site specific amendment was implemented to comply with local requirements in Italy related to the requirement for contraception
    21 Jun 2012
    This amendment updated renal safety monitoring criteria, reporting updates for serious adverse events (SAEs), and added clarification to the DIC criteria. Medical Monitor contact information was also updated as there was a change to the Medical Monitor.

    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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    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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