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    Clinical Trial Results:
    A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy

    Summary
    EudraCT number
    2010-024566-22
    Trial protocol
    FR   Outside EU/EEA  
    Global end of trial date
    28 Feb 2012

    Results information
    Results version number
    v1(current)
    This version publication date
    03 Aug 2019
    First version publication date
    03 Aug 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    DMD114118
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    GlaxoSmithKline
    Sponsor organisation address
    980 Great West Road, Brentford, Middlesex, United Kingdom,
    Public contact
    GSK Respoonse Centre, GlaxoSmithKline, 1 8664357343, GSKClinicalSupportHD@GSK.com
    Scientific contact
    GSK Respoonse 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-PIP01-09
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    02 Aug 2012
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    28 Feb 2012
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Feb 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the pharmacokinetics, safety and tolerability of GSK2402968 after a single subcutaneous administration at different dose levels in non-ambulatory subjects with Duchenne muscular dystrophy.
    Protection of trial subjects
    This study was performed in compliance with Good Clinical Practices and GlaxoSmithKline Standard Operating Procedures for all processes involved, including the archiving of essential documents.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    12 Jul 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
    France: 4
    Country: Number of subjects enrolled
    United States: 16
    Worldwide total number of subjects
    20
    EEA total number of subjects
    4
    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)
    3
    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 at 2 study centers in United States and France.

    Pre-assignment
    Screening details
    A total of 21 subjects were screened and 20 subjects randomized into the study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Single dose of placebo sterile solution for subcutaneous injection.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Single dose of placebo sterile solution for subcutaneous injection.

    Arm title
    3mg/kg GSK2402968
    Arm description
    Single dose of 3mg/kg GSK2402968 sterile solution for subcutaneous injection
    Arm type
    Experimental

    Investigational medicinal product name
    GSK2402968
    Investigational medicinal product code
    Other name
    Drisapersen
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Single dose of 3mg/kg GSK2402968 sterile solution for subcutaneous injection

    Arm title
    6mg/kg GSK2402968
    Arm description
    Single dose of 6mg/kg GSK2402968 sterile solution for subcutaneous injection
    Arm type
    Experimental

    Investigational medicinal product name
    GSK2402968
    Investigational medicinal product code
    Other name
    Drisapersen
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Single dose of 6mg/kg GSK2402968 sterile solution for subcutaneous injection

    Arm title
    9mg/kg GSK2402968
    Arm description
    Single dose of 9mg/kg GSK2402968 sterile solution for subcutaneous injection.
    Arm type
    Experimental

    Investigational medicinal product name
    GSK2402968
    Investigational medicinal product code
    Other name
    Drisapersen
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Single dose of 9mg/kg GSK2402968 sterile solution for subcutaneous injection

    Number of subjects in period 1
    Placebo 3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Started
    5
    6
    6
    3
    Completed
    5
    6
    6
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Single dose of placebo sterile solution for subcutaneous injection.

    Reporting group title
    3mg/kg GSK2402968
    Reporting group description
    Single dose of 3mg/kg GSK2402968 sterile solution for subcutaneous injection

    Reporting group title
    6mg/kg GSK2402968
    Reporting group description
    Single dose of 6mg/kg GSK2402968 sterile solution for subcutaneous injection

    Reporting group title
    9mg/kg GSK2402968
    Reporting group description
    Single dose of 9mg/kg GSK2402968 sterile solution for subcutaneous injection.

    Reporting group values
    Placebo 3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968 Total
    Number of subjects
    5 6 6 3 20
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    12.2 ± 0.84 13.8 ± 1.72 13.3 ± 1.21 10.3 ± 1.53 -
    Gender categorical
    Units: Subjects
        Female
    0 0 0 0 0
        Male
    5 6 6 3 20
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    0 2 0 0 2
        Not Hispanic or Latino
    4 4 5 1 14
        Unknown or Not Reported
    1 0 1 2 4
    Race
    Units: Subjects
        White - Arabic/North African Heritage
    0 0 2 0 2
        White - White/Caucasian/European Heritage
    4 6 3 1 14
        Not Reported
    1 0 1 2 4
    Body Mass Index (BMI)
    Units: kg/m^2
        arithmetic mean (standard deviation)
    23.68 ± 11.12 22.73 ± 6.14 29.07 ± 6.85 22.49 ± 3.54 -
    Weight
    Units: kg
        arithmetic mean (standard deviation)
    49.1 ± 14.00 50.45 ± 13.52 59.08 ± 12.09 49.73 ± 8.62 -
    Height
    Units: cm
        arithmetic mean (standard deviation)
    147.6 ± 23.88 149.5 ± 10.15 143.5 ± 7.26 147.3 ± 7.51 -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Single dose of placebo sterile solution for subcutaneous injection.

    Reporting group title
    3mg/kg GSK2402968
    Reporting group description
    Single dose of 3mg/kg GSK2402968 sterile solution for subcutaneous injection

    Reporting group title
    6mg/kg GSK2402968
    Reporting group description
    Single dose of 6mg/kg GSK2402968 sterile solution for subcutaneous injection

    Reporting group title
    9mg/kg GSK2402968
    Reporting group description
    Single dose of 9mg/kg GSK2402968 sterile solution for subcutaneous injection.

    Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Follow-Up Adverse events (AE's)

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    End point title
    Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Follow-Up Adverse events (AE's) [1]
    End point description
    Safety Population are all subjects who received a dose of study medication. Adverse Events (AEs) classified into treatment emergent AEs and follow-up AEs. Treatment emergent AEs occurring from the start of study treatment up to and including Day 28. Follow-up AEs are those AEs beginning from Day 29 up to and including the final follow-up contact. Adverse Events of special interest resulting from any of the Laboratory Safety Parameter Stopping Criteria for hepatic or renal toxicity, thrombocytes, inflammation and coagulation occurring and also any AEs resulting from injection site reactions.
    End point type
    Primary
    End point timeframe
    Up to 5 months
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No Statistical Analysis for safety evaluation.
    End point values
    Placebo 3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Number of subjects analysed
    5
    6
    6
    3
    Units: participants
        Treatment Emergent
    2
    6
    6
    3
        Follow-up
    2
    3
    4
    0
        Drug related Adverse Events
    1
    5
    5
    3
        AE's of Special Interest - Injection Site Reaction
    1
    4
    5
    3
        AE's of Special Interest - Renal Toxicity
    0
    0
    0
    1
        AE's of Special Interest - Inflammation
    0
    0
    1
    3
        Deaths
    0
    0
    0
    0
        Serious Adverse Events by Phase
    0
    0
    0
    0
        Adverse Events Leading to Withdrawal
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Maximum Observed Plasma Concentration (Cmax) of GSK2402968

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    End point title
    Maximum Observed Plasma Concentration (Cmax) of GSK2402968 [2] [3]
    End point description
    Pharmacokinetic Parameters Cmax is the observed maximum plasma concentration post-dose. PK Population subjects in the ‘Safety’ population for whom a pharmacokinetic sample was obtained and analysed. This population was used for all PK summaries.
    End point type
    Primary
    End point timeframe
    At 0.25, 0.5, 1, 3, 6, 9, 24hrs, Days 4, 8 and 29-35
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    [3] - 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 Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    End point values
    3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Number of subjects analysed
    6
    6
    3
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    4990 ± 36.0
    8140 ± 25.9
    8940 ± 20.8
    No statistical analyses for this end point

    Primary: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration [AUC(0-t)] of GSK2402968

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    End point title
    Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Concentration [AUC(0-t)] of GSK2402968 [4] [5]
    End point description
    Pharmacokinetic Parameters [AUC(0-t)] is Area under the plasma concentration-time curve from Time 0 to last measured concentration PK Population subjects
    End point type
    Primary
    End point timeframe
    At 0.25, 0.5, 1, 3, 6, 9, 24hrs, Days 4, 8 and 29-35
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    [5] - 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 Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    End point values
    3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Number of subjects analysed
    6
    6
    3
    Units: ng.hr/mL
        geometric mean (geometric coefficient of variation)
    48100 ± 15.4
    98800 ± 28.0
    107000 ± 9.1
    No statistical analyses for this end point

    Primary: Area Under the Plasma Concentration-time Curve From Time 0 to 24 hours post-dose [AUC(0-24)] of GSK2402968

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    End point title
    Area Under the Plasma Concentration-time Curve From Time 0 to 24 hours post-dose [AUC(0-24)] of GSK2402968 [6] [7]
    End point description
    Pharmacokinetic Parameters [AUC(0-24h)] is the Area under the plasma concentration-time curve from Time 0 to 24 hours post-dose PK Population subjects
    End point type
    Primary
    End point timeframe
    At 0.25, 0.5, 1, 3, 6, 9, 24hrs, Days 4, 8 and 29-35
    Notes
    [6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    [7] - 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 Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    End point values
    3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Number of subjects analysed
    6
    6
    3
    Units: ng.hr/mL
        geometric mean (geometric coefficient of variation)
    44600 ± 18.5
    87800 ± 33.4
    97800 ± 10.7
    No statistical analyses for this end point

    Primary: Area Under the Plasma Concentration-time Curve From Time 0 to 7 days post-dose [AUC(0-7d)] of GSK2402968

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    End point title
    Area Under the Plasma Concentration-time Curve From Time 0 to 7 days post-dose [AUC(0-7d)] of GSK2402968 [8] [9]
    End point description
    Pharmacokinetic Parameters [AUC(0-7d)] is Area under the plasma concentration-time curve from Time 0 to 7 days post-dose PK Population subjects
    End point type
    Primary
    End point timeframe
    At 0.25, 0.5, 1, 3, 6, 9, 24hrs, Days 4, 8 and 29-35
    Notes
    [8] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    [9] - 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 Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    End point values
    3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Number of subjects analysed
    6
    6
    3
    Units: ng.hr/mL
        geometric mean (geometric coefficient of variation)
    45500 ± 16.0
    87300 ± 18.7
    112000 ± 0.6
    No statistical analyses for this end point

    Primary: Time of maximum plasma concentration (tmax) of GSK2402968

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    End point title
    Time of maximum plasma concentration (tmax) of GSK2402968 [10] [11]
    End point description
    Pharmacokinetic Parameters (tmax) is the time of maximum plasma concentration post-dose. PK Population subjects
    End point type
    Primary
    End point timeframe
    At 0.25, 0.5, 1, 3, 6, 9, 24hrs, Days 4, 8 and 29-35
    Notes
    [10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    [11] - 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 Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    End point values
    3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Number of subjects analysed
    6
    6
    3
    Units: hr^2
        median (full range (min-max))
    3.01 (2.97 to 5.78)
    3.00 (2.98 to 6.00)
    6.00 (3.00 to 6.00)
    No statistical analyses for this end point

    Primary: Apparent plasma clearance (CL/F) of GSK2402968

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    End point title
    Apparent plasma clearance (CL/F) of GSK2402968 [12] [13]
    End point description
    Pharmacokinetic Parameters (CL/F) is the Apparent plasma clearance PK Population subjects
    End point type
    Primary
    End point timeframe
    At 0.25, 0.5, 1, 3, 6, 9, 24hrs, Days 4, 8 and 29-35
    Notes
    [12] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    [13] - 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 Statistical Analysis is not the comparision between the doses, it is to Estimate Dose Proportionality after Single Dose.
    End point values
    3mg/kg GSK2402968 6mg/kg GSK2402968
    Number of subjects analysed
    4
    1
    Units: mL/hr
        arithmetic mean (standard deviation)
    3700.6 ± 834.94
    3770.8 ± 0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 5 months
    Adverse event reporting additional description
    Adverse Events (AEs) for this study were classified into treatment emergent AEs and follow-up AEs. Treatment emergent AEs were defined as AEs occurring from the start of study treatment up to and including Day 28. Follow-up AEs were defined as those AEs beginning from Day 29 up to and including the final follow-up contact.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    14.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    -

    Reporting group title
    3mg/kg GSK2402968
    Reporting group description
    -

    Reporting group title
    6mg/kg GSK2402968
    Reporting group description
    -

    Reporting group title
    9mg/kg GSK2402968
    Reporting group description
    -

    Serious adverse events
    Placebo 3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Placebo 3mg/kg GSK2402968 6mg/kg GSK2402968 9mg/kg GSK2402968
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 5 (40.00%)
    6 / 6 (100.00%)
    6 / 6 (100.00%)
    3 / 3 (100.00%)
    Investigations
    Alpha 1 microglobulin increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    0
    1
    Immunology test abnormal
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    0
    1
    C-reactive protein increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    2
    1
    Upper respiratory tract congestion
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Headache
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    2 / 3 (66.67%)
         occurrences all number
    1
    1
    2
    2
    Eye disorders
    Conjunctivitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    General disorders and administration site conditions
    Injection site discolouration
         subjects affected / exposed
    1 / 5 (20.00%)
    4 / 6 (66.67%)
    5 / 6 (83.33%)
    3 / 3 (100.00%)
         occurrences all number
    1
    4
    8
    3
    Injection site erythema
         subjects affected / exposed
    0 / 5 (0.00%)
    3 / 6 (50.00%)
    1 / 6 (16.67%)
    2 / 3 (66.67%)
         occurrences all number
    0
    3
    1
    3
    Injection site haematoma
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    2 / 6 (33.33%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    2
    1
    Injection site inflammation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    3 / 3 (100.00%)
         occurrences all number
    0
    0
    1
    3
    Injection site induration
         subjects affected / exposed
    1 / 5 (20.00%)
    3 / 6 (50.00%)
    4 / 6 (66.67%)
    2 / 3 (66.67%)
         occurrences all number
    1
    3
    5
    2
    Injection site oedema
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 3 (66.67%)
         occurrences all number
    0
    0
    0
    3
    Injection site pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Injection site pruritus
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Injection site warmth
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    3 / 3 (100.00%)
         occurrences all number
    0
    0
    1
    3
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Insomnia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    1
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Infections and infestations
    Sinusitis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    3
    0
    Bronchitis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 May 2010
    Protocol Amendment 1 was a global amendment for all countries, all centers that was released prior to any screening into the study. The purpose of this amendment was to: 1. Increase the number of subjects from 24 to 32, with 6 active and 2 placebo subjects per cohort 2. Increase time between cohorts to a minimum of 14 days 3. Remove guarantee of a follow-on study 4. Clarify the role of the Independent Data Monitoring Committee (IDMC) 5. Exclusion Criterion #5: Reduce the exclusionary period for idebenone and Coenzyme Q10 to 1 month
    12 Jan 2011
    Protocol Amendment 2 was a country specific amendment for France. This amendment was approved after 13 subjects had been randomized and the purpose of this amendment was to: Add Inclusion Criterion #11, a social security entry criterion specific to French subjects
    20 Apr 2011
    Protocol Amendment 3 was a global amendment for all countries, all centers. This amendment was approved after 14 subjects had been randomized and the amendment was at the request of the French regulatory authority, Afsaaps, to: Add inflammatory markers as a dose limiting criterion.
    20 Aug 2011
    Protocol Amendment 4 was a global amendment for all countries, all centers. This amendment was approved after 18 subjects had been randomized and the purpose of this amendment was to: 1. Update the safety monitoring criteria, following discussions with the FDA, to add Complement Factor C3 and clarify C Reactive Protein (CRP) testing is high sensitivity CRP (hsCRP). This ensured consistency across the GSK2402968 program. 2. Remove Inclusion Criterion #8 regarding contraceptive requirements. Additional data became available which supported removal of need to use contraception.

    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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