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    Clinical Trial Results:
    Protocol TRO19622CLEQ1275-1 (WN29836) Phase II, Multicenter, Randomized, Adaptive, Double-Blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients

    Summary
    EudraCT number
    		 2010-020386-24
    Trial protocol
    		 FR   NL   BE   DE   IT   GB  
    Global end of trial date
    09 Oct 2013

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Jan 2018
    First version publication date
    25 Jan 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WN29836
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01302600
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    F. Hoffmann-La Roche AG
    Sponsor organisation address
    Grenzacherstrasse 124, Basel, Switzerland, CH-4070
    Public contact
    Medical Communications, F. Hoffmann-La Roche AG, +41 616878333, global.trial_information@roche.com
    Scientific contact
    Medical Communications, F. Hoffmann-La Roche AG, +41 616878333, global.trial_information@roche.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    09 Oct 2013
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Oct 2013
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Oct 2013
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objectives of this study were to assess the efficacy and safety of olesoxime 10 milligrams per kilogram (mg/kg) once daily liquid suspension formulation in spinal muscular atrophy (SMA) Type 2 or Type 3 non-ambulant subjects, aged 3 - 25 years.
    Protection of trial subjects
    Each subject, or the subject's representative, signed an informed consent form before participating in the study.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    18 Nov 2011
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 16
    Country: Number of subjects enrolled
    France: 29
    Country: Number of subjects enrolled
    Germany: 20
    Country: Number of subjects enrolled
    Italy: 52
    Country: Number of subjects enrolled
    Netherlands: 9
    Country: Number of subjects enrolled
    Poland: 20
    Country: Number of subjects enrolled
    United Kingdom: 19
    Worldwide total number of subjects
    165
    EEA total number of subjects
    165
    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)
    108
    Adolescents (12-17 years)
    36
    Adults (18-64 years)
    21
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Eligible subjects included 3 to 25 year-old patients with Type 2 or non-ambulant Type 3 SMA.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Matching placebo, once a day for 104 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo, once a day for 104 weeks.

    Arm title
    		 Olesoxime
    Arm description
    		 Olesoxime, 10 mg/kg body weight once a day for 104 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Olesoxime
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Olesoxime, 10 mg/kg body weight once a day for 104 weeks.

    Number of subjects in period 1
    		Placebo Olesoxime
    Started
    57
    108
    Completed
    50
    98
    Not completed
    7
    10
         Consent withdrawn by subject
    1
    3
         Adverse event, non-fatal
    2
    4
         Death
    1
    1
         Non-compliance with study drug
    1
    -
         Reason not specified
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matching placebo, once a day for 104 weeks.

    Reporting group title
    Olesoxime
    Reporting group description
    		 Olesoxime, 10 mg/kg body weight once a day for 104 weeks.

    Reporting group values
    		 Placebo Olesoxime Total
    Number of subjects
    		 57 108 165
    Age Categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 30 78 108
        Adolescents (12-17 years)
    		 20 16 36
        Adults (18-64 years)
    		 7 14 21
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 11.2 ( 6.0 ) 9.3 ( 5.7 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 32 51 83
        Male
    		 25 57 82

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matching placebo, once a day for 104 weeks.

    Reporting group title
    Olesoxime
    Reporting group description
    		 Olesoxime, 10 mg/kg body weight once a day for 104 weeks.

    Primary: Mean Change from Baseline to Week 104 in the Motor Function Measure (MFM) Dimensions 1 and 2 (D1+D2) Score

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    End point title
    		 Mean Change from Baseline to Week 104 in the Motor Function Measure (MFM) Dimensions 1 and 2 (D1+D2) Score
    End point description
    The MFM is an assessment of functional motor abilities of subjects with neuromuscular disease comprising items rated on a 4-point scale (0 to 3). The MFM items are grouped into three functional dimensions D1, D2 and D3. Patient MFM scores were normalized to the maximum achievable score. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Primary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: score
        least squares mean (standard error)
    -1.82 ( 0.901 )
    0.18 ( 0.717 )
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Placebo v Olesoxime
    Number of subjects included in analysis
    160
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0676
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Change from Baseline in MFM Total Score at Week 104

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    End point title
    		 Change from Baseline in MFM Total Score at Week 104
    End point description
    The MFM is an assessment of functional motor abilities of subjects with neuromuscular disease comprising items rated on a 4-point scale (0 to 3). The MFM items are grouped into three functional dimensions D1, D2 and D3. Patient MFM scores were normalized to the maximum achievable score. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: score
        least squares mean (standard error)
    -1.45 ( 0.943 )
    0.59 ( 0.751 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Hammersmith Functional Motor Scale (HFMS) at Week 91

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    End point title
    		 Change from Baseline in Hammersmith Functional Motor Scale (HFMS) at Week 91
    End point description
    The HFMS consists of 20 items, assessing the subject’s ability to perform various activities. Each activity is scored on a 3-point scoring system, with a score of 2 for unaided, 1 for assistance, and 0 for inability. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 91
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: score
        least squares mean (standard error)
    -1.72 ( 0.515 )
    -0.78 ( 0.416 )
    No statistical analyses for this end point

    Secondary: Time to 4-Point Decrease on the HFMS Score

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    End point title
    		 Time to 4-Point Decrease on the HFMS Score
    End point description
    The HFMS consists of 20 items, assessing the subject’s ability to perform various activities. Each activity is scored on a 3-point scoring system, with a score of 2 for unaided, 1 for assistance, and 0 for inability. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available. 9999=not estimable.
    End point type
    Secondary
    End point timeframe
    Up to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: weeks
        median (confidence interval 95%)
    9999 (91.4 to 9999)
    9999 (9999 to 9999)
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with Response for MFM D1+D2 Score at Week 104

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    End point title
    		 Percentage of Subjects with Response for MFM D1+D2 Score at Week 104
    End point description
    The MFM is an assessment of functional motor abilities of subjects with neuromuscular disease comprising items rated on a 4-point scale (0 to 3). The MFM items are grouped into three functional dimensions D1, D2 and D3. Patient MFM scores were normalized to the maximum achievable score. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: percentage of subjects
        number (not applicable)
    38.6
    54.4
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with Response for MFM Total Score at Week 104

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    End point title
    		 Percentage of Subjects with Response for MFM Total Score at Week 104
    End point description
    The MFM is an assessment of functional motor abilities of subjects with neuromuscular disease comprising items rated on a 4-point scale (0 to 3). The MFM items are grouped into three functional dimensions D1, D2 and D3. Patient MFM scores were normalized to the maximum achievable score. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: percentage of subjects
        number (not applicable)
    38.6
    56.3
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with Response for HFMS Score at Week 91

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    End point title
    		 Percentage of Subjects with Response for HFMS Score at Week 91
    End point description
    The HFMS consists of 20 items, assessing the subject’s ability to perform various activities. Each activity is scored on a 3-point scoring system, with a score of 2 for unaided, 1 for assistance, and 0 for inability. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 91
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: percentage of subjects
        number (not applicable)
    28.1
    49.5
    No statistical analyses for this end point

    Secondary: Change from Baseline in Compound Muscle Action Potential (CMAP) at Week 104

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    End point title
    		 Change from Baseline in Compound Muscle Action Potential (CMAP) at Week 104
    End point description
    CMAP is an electromyography investigation (electrical study of muscle function). The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Change from Baseline in Compound Muscle Action Potential (CMAP) at Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: millivolts (mV)
        least squares mean (standard error)
    -0.16 ( 0.294 )
    -0.07 ( 0.214 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Motor Unit Number Estimation (MUNE) at Week 104

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    End point title
    		 Change from Baseline in Motor Unit Number Estimation (MUNE) at Week 104
    End point description
    MUNE is a technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: mune
        least squares mean (standard error)
    -6.69 ( 5.106 )
    -4.51 ( 3.867 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Pediatric Quality of Life Inventory (PedsQL) Neuromuscular Module Score - Patient Report

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    End point title
    		 Change from Baseline in Pediatric Quality of Life Inventory (PedsQL) Neuromuscular Module Score - Patient Report
    End point description
    The PedsQL Neuromuscular Module Scales are comprised of parallel child self-report and parent proxy-report formats for children ages 5-18 years, and a parent proxy-report format for children ages 2-4 years. The PedsQL scale assess: the disease process and associated symptoms; the subject’s ability to communicate with healthcare providers and others about his/her illness; and items related to family financial and social support systems. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    47
    80
    Units: score
        least squares mean (standard error)
    -3.49 ( 2.061 )
    -3.24 ( 1.729 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in PedsQL Neuromuscular Module Score - Parent/Caregiver Report at Week 104

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    End point title
    		 Change from Baseline in PedsQL Neuromuscular Module Score - Parent/Caregiver Report at Week 104
    End point description
    The PedsQL Neuromuscular Module Scales are comprised of parallel child self-report and parent proxy-report formats for children ages 5-18 years, and a parent proxy-report format for children ages 2-4 years. The PedsQL scale assess: the disease process and associated symptoms; the subject’s ability to communicate with healthcare providers and others about his/her illness; and items related to family financial and social support systems. The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: score
        least squares mean (standard error)
    -5.67 ( 1.954 )
    -2.06 ( 1.564 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Forced Expiratory Vital Capacity (FVC)/Theoretical Capacity (TC) at Week 104

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    End point title
    		 Change from Baseline in Forced Expiratory Vital Capacity (FVC)/Theoretical Capacity (TC) at Week 104
    End point description
    Pulmonary Function was assessed in subjects 5 years or older by measuring FVC (as percent predicted for age and height). The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    47
    80
    Units: percent
        least squares mean (standard error)
    6.16 ( 2.601 )
    4.28 ( 2.316 )
    No statistical analyses for this end point

    Secondary: Clinical Global Impression of Change - Patient/Caregiver Assessment at Week 104

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    End point title
    		 Clinical Global Impression of Change - Patient/Caregiver Assessment at Week 104
    End point description
    The CGI-C measures global improvement or change and is rated on a 7-point scale, with scores ranging from 1 (very much improved) through to 7 (very much worse). The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: percentage of subjects
    number (not applicable)
        Very much improved
    0.0
    1.0
        Much improved
    6.1
    3.1
        Minimally improved
    16.3
    20.8
        No change
    59.2
    66.7
        Minimally worse
    12.2
    7.3
        Much worse
    6.1
    1.0
        Very much worse
    0.0
    0.0
    No statistical analyses for this end point

    Secondary: Clinical Global Impression of Change at Week 104 - Physician Assessment

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    End point title
    		 Clinical Global Impression of Change at Week 104 - Physician Assessment
    End point description
    The CGI-C measures global improvement or change and is rated on a 7-point scale, with scores ranging from 1 (very much improved) through to 7 (very much worse). The full analysis set (FAS) included all randomized subjects who received at least one dose of study drug and who had at least one post-randomization assessment of MFM available.
    End point type
    Secondary
    End point timeframe
    Week 104
    End point values
    		 Placebo Olesoxime
    Number of subjects analysed
    57
    103
    Units: percentage of subjects
    number (not applicable)
        Very much improved
    0.0
    0.0
        Much improved
    6.0
    1.0
        Minimally improved
    8.0
    16.7
        No change
    66.0
    75.0
        Minimally worse
    18.0
    7.3
        Much worse
    2.0
    0.0
        Very much worse
    0.0
    0.0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 2 years.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matching placebo, once a day for 104 weeks.

    Reporting group title
    Olesoxime
    Reporting group description
    		 Olesoxime, 10 mg/kg body weight once a day for 104 weeks.

    Serious adverse events
    		 Placebo Olesoxime
    Total subjects affected by serious adverse events
         subjects affected / exposed
    29 / 57 (50.88%)
    34 / 108 (31.48%)
         number of deaths (all causes)
    1
    1
         number of deaths resulting from adverse events
    Surgical and medical procedures
    Appendicectomy
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Arthrodesis
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Device therapy
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fracture treatment
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrostomy
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hospitalisation
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Mechanical ventilation
         subjects affected / exposed
    1 / 57 (1.75%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Scoliosis surgery
         subjects affected / exposed
    2 / 57 (3.51%)
    5 / 108 (4.63%)
         occurrences causally related to treatment / all
    0 / 2
    1 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Spinal fusion surgery
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Spinal operation
         subjects affected / exposed
    1 / 57 (1.75%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Spinal rod insertion
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tenotomy
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tooth extraction
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Chest pain
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    2 / 57 (3.51%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Unevaluable event
         subjects affected / exposed
    0 / 57 (0.00%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Atelectasis
         subjects affected / exposed
    1 / 57 (1.75%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cough
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Dyspnoea
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypoventilation
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lung disorder
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Obstructive airways disorder
         subjects affected / exposed
    0 / 57 (0.00%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Pleurisy
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    2 / 57 (3.51%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 2
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Restrictive pulmonary disease
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tonsillar hypertrophy
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tracheal disorder
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    2 / 57 (3.51%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    2 / 2
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bronchoscopy
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Coagulation factor VIII level decreased
         subjects affected / exposed
    2 / 57 (3.51%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 57 (1.75%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Oxygen saturation decreased
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary function test
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sleep study
         subjects affected / exposed
    0 / 57 (0.00%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Transaminases increased
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Concussion
         subjects affected / exposed
    0 / 57 (0.00%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fall
         subjects affected / exposed
    2 / 57 (3.51%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 57 (0.00%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fibula fracture
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fracture displacement
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Greenstick fracture
         subjects affected / exposed
    1 / 57 (1.75%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Head injury
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Humerus fracture
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Road traffic accident
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Congenital, familial and genetic disorders
    Cryptorchism
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frenulum breve
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac arrest
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Nervous system disorders
    Neuralgia
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Subarachnoid haemorrhage
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tremor
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Coagulopathy
         subjects affected / exposed
    2 / 57 (3.51%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    2 / 2
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Abdominal pain upper
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ketonuria
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Kyphoscoliosis
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Scoliosis
         subjects affected / exposed
    1 / 57 (1.75%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bronchitis
         subjects affected / exposed
    1 / 57 (1.75%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    4 / 57 (7.02%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    1 / 5
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    3 / 57 (5.26%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Meningitis
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pharyngotonsillitis
         subjects affected / exposed
    1 / 57 (1.75%)
    0 / 108 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    6 / 57 (10.53%)
    7 / 108 (6.48%)
         occurrences causally related to treatment / all
    0 / 7
    5 / 9
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    0 / 57 (0.00%)
    4 / 108 (3.70%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tooth abscess
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 57 (1.75%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 57 (1.75%)
    2 / 108 (1.85%)
         occurrences causally related to treatment / all
    0 / 2
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypoglycaemia
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolic acidosis
         subjects affected / exposed
    0 / 57 (0.00%)
    1 / 108 (0.93%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo Olesoxime
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    52 / 57 (91.23%)
    96 / 108 (88.89%)
    Investigations
    Weight increased
         subjects affected / exposed
    3 / 57 (5.26%)
    4 / 108 (3.70%)
         occurrences all number
    3
    4
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    5 / 57 (8.77%)
    8 / 108 (7.41%)
         occurrences all number
    6
    11
    Nervous system disorders
    Headache
         subjects affected / exposed
    13 / 57 (22.81%)
    22 / 108 (20.37%)
         occurrences all number
    19
    57
    General disorders and administration site conditions
    Pain
         subjects affected / exposed
    0 / 57 (0.00%)
    6 / 108 (5.56%)
         occurrences all number
    0
    6
    Pyrexia
         subjects affected / exposed
    15 / 57 (26.32%)
    33 / 108 (30.56%)
         occurrences all number
    22
    57
    Unevaluable event
         subjects affected / exposed
    5 / 57 (8.77%)
    3 / 108 (2.78%)
         occurrences all number
    5
    3
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    3 / 57 (5.26%)
    3 / 108 (2.78%)
         occurrences all number
    1
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    10 / 57 (17.54%)
    20 / 108 (18.52%)
         occurrences all number
    18
    39
    Abdominal pain upper
         subjects affected / exposed
    4 / 57 (7.02%)
    8 / 108 (7.41%)
         occurrences all number
    4
    11
    Constipation
         subjects affected / exposed
    4 / 57 (7.02%)
    5 / 108 (4.63%)
         occurrences all number
    6
    5
    Diarrhoea
         subjects affected / exposed
    12 / 57 (21.05%)
    18 / 108 (16.67%)
         occurrences all number
    16
    22
    Nausea
         subjects affected / exposed
    3 / 57 (5.26%)
    9 / 108 (8.33%)
         occurrences all number
    4
    18
    Toothache
         subjects affected / exposed
    5 / 57 (8.77%)
    1 / 108 (0.93%)
         occurrences all number
    5
    1
    Vomiting
         subjects affected / exposed
    16 / 57 (28.07%)
    25 / 108 (23.15%)
         occurrences all number
    25
    38
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    16 / 57 (28.07%)
    31 / 108 (28.70%)
         occurrences all number
    30
    48
    Oropharyngeal pain
         subjects affected / exposed
    9 / 57 (15.79%)
    16 / 108 (14.81%)
         occurrences all number
    11
    30
    Respiratory tract congestion
         subjects affected / exposed
    3 / 57 (5.26%)
    3 / 108 (2.78%)
         occurrences all number
    4
    6
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    7 / 57 (12.28%)
    2 / 108 (1.85%)
         occurrences all number
    7
    2
    Back pain
         subjects affected / exposed
    3 / 57 (5.26%)
    3 / 108 (2.78%)
         occurrences all number
    3
    4
    Joint contracture
         subjects affected / exposed
    4 / 57 (7.02%)
    5 / 108 (4.63%)
         occurrences all number
    4
    5
    Pain in extremity
         subjects affected / exposed
    5 / 57 (8.77%)
    14 / 108 (12.96%)
         occurrences all number
    6
    17
    Scoliosis
         subjects affected / exposed
    4 / 57 (7.02%)
    12 / 108 (11.11%)
         occurrences all number
    5
    13
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    17 / 57 (29.82%)
    17 / 108 (15.74%)
         occurrences all number
    21
    24
    Ear infection
         subjects affected / exposed
    2 / 57 (3.51%)
    9 / 108 (8.33%)
         occurrences all number
    2
    11
    Gastroenteritis
         subjects affected / exposed
    8 / 57 (14.04%)
    16 / 108 (14.81%)
         occurrences all number
    12
    21
    Influenza
         subjects affected / exposed
    9 / 57 (15.79%)
    10 / 108 (9.26%)
         occurrences all number
    14
    26
    Lower respiratory tract infection
         subjects affected / exposed
    3 / 57 (5.26%)
    3 / 108 (2.78%)
         occurrences all number
    3
    12
    Nasopharyngitis
         subjects affected / exposed
    15 / 57 (26.32%)
    25 / 108 (23.15%)
         occurrences all number
    34
    55
    Pharyngitis
         subjects affected / exposed
    6 / 57 (10.53%)
    15 / 108 (13.89%)
         occurrences all number
    8
    19
    Pneumonia
         subjects affected / exposed
    3 / 57 (5.26%)
    3 / 108 (2.78%)
         occurrences all number
    3
    5
    Respiratory tract infection
         subjects affected / exposed
    6 / 57 (10.53%)
    15 / 108 (13.89%)
         occurrences all number
    10
    30
    Rhinitis
         subjects affected / exposed
    6 / 57 (10.53%)
    14 / 108 (12.96%)
         occurrences all number
    9
    18
    Upper respiratory tract infection
         subjects affected / exposed
    13 / 57 (22.81%)
    22 / 108 (20.37%)
         occurrences all number
    33
    36
    Urinary tract infection
         subjects affected / exposed
    4 / 57 (7.02%)
    4 / 108 (3.70%)
         occurrences all number
    4
    4
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    3 / 57 (5.26%)
    1 / 108 (0.93%)
         occurrences all number
    3
    3

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

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

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

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