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    Clinical Trial Results:
    A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

    Summary
    EudraCT number
    		 2016-004263-38
    Trial protocol
    		 SE   GB  
    Global end of trial date
    26 Apr 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Mar 2019
    First version publication date
    30 Mar 2019
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    VBP15-003
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02760277
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    ReveraGen BioPharma Inc.
    Sponsor organisation address
    155 Gibbs Street, Rockville, United States, 20850
    Public contact
    Vice President, Operations, ReveraGen BioPharma Inc., +1 215 680 8286, jesse.damsker@reveragen.com
    Scientific contact
    Vice President, Operations, ReveraGen BioPharma Inc., +1 215 680 8286, jesse.damsker@reveragen.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001794-PIP02-16
    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
    14 Sep 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    26 Apr 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Apr 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objectives: 1. To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, in boys ages 4-7 years with DMD; 2. To compare the efficacy, as measured by the Time to Stand Test (TTSTAND), of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. untreated DMD historical controls in boys ages 4-7 years with DMD; and 3. To compare the safety, as measured by body mass index (BMI) z-score, of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls in boys ages 4-7 years with DMD.
    Protection of trial subjects
    The trial will be conducted in accordance with the International Conference on Harmonisation E6 Guideline for Good Clinical Practice; The United States FDA Code of Federal Regulations, Title 21 CFR Part 312, and the US Health Insurance Portability and Accountability Act of 1996. The Parent/guardian of each participant must consent in writing for participant to be enrolled.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 Jul 2016
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 2 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 20
    Country: Number of subjects enrolled
    Canada: 7
    Country: Number of subjects enrolled
    Israel: 5
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    Sweden: 4
    Country: Number of subjects enrolled
    Australia: 5
    Worldwide total number of subjects
    48
    EEA total number of subjects
    11
    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)
    48
    Adolescents (12-17 years)
    0
    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
    		 Only those who have participated in the VBP13-003 trial are able to participate in the 003 trial.

    Pre-assignment
    Screening details
    		 Subject has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment.

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Dose level group 1
    Arm description
    		 0.25 mg/kg/day for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Vamorolone 4% oral suspension

    Arm title
    		 Dose level Group 2
    Arm description
    		 0.75 mg/kg/day for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Vamorolone 4% oral suspension

    Arm title
    		 Dose level Group 3
    Arm description
    		 2.0 mg/kg/day for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Vamorolone 4% suspension

    Arm title
    		 Dose level Group 4
    Arm description
    		 6.0 mg/kg/day for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Vamorolone 4% oral suspension

    Number of subjects in period 1
    		Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Started
    12
    12
    12
    12
    Completed
    12
    12
    12
    12

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dose level group 1
    Reporting group description
    		 0.25 mg/kg/day for 24 weeks

    Reporting group title
    Dose level Group 2
    Reporting group description
    		 0.75 mg/kg/day for 24 weeks

    Reporting group title
    Dose level Group 3
    Reporting group description
    		 2.0 mg/kg/day for 24 weeks

    Reporting group title
    Dose level Group 4
    Reporting group description
    		 6.0 mg/kg/day for 24 weeks

    Reporting group values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4 Total
    Number of subjects
    		 12 12 12 12 48
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0
        Children (2-11 years)
    		 12 12 12 12 48
        Adolescents (12-17 years)
    		 0 0 0 0 0
        Adults (18-64 years)
    		 0 0 0 0 0
        From 65-84 years
    		 0 0 0 0 0
        85 years and over
    		 0 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0 0 0
        Male
    		 12 12 12 12 48

    End points

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    End points reporting groups
    Reporting group title
    Dose level group 1
    Reporting group description
    		 0.25 mg/kg/day for 24 weeks

    Reporting group title
    Dose level Group 2
    Reporting group description
    		 0.75 mg/kg/day for 24 weeks

    Reporting group title
    Dose level Group 3
    Reporting group description
    		 2.0 mg/kg/day for 24 weeks

    Reporting group title
    Dose level Group 4
    Reporting group description
    		 6.0 mg/kg/day for 24 weeks

    Primary: Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03

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    End point title
    		 Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03 [1]
    End point description
    Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug; To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- week Treatment Period, in boys ages 4-7 years with DMD.
    End point type
    Primary
    End point timeframe
    24 weeks
    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: Treatment levels were not compared.
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: Number of Events Reported
        Total Number of AEs
    48
    44
    54
    73
        Total Number of TEAEs
    48
    44
    54
    72
        Subjects with Any TEAE
    10
    10
    11
    11
        Subjects with Any Drug Related TEAE
    1
    2
    4
    5
        Subjects with Any CTCAE Grade 3 or Higher TEAE
    0
    0
    0
    2
        Discontinuation of Study Drug due to TEAE
    0
    0
    0
    0
        Subjects with Any Serious TEAE
    0
    1
    0
    2
        Death
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Muscle function measured by Time to Stand Test (TTSTAND)- Velocity

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    End point title
    		 Muscle function measured by Time to Stand Test (TTSTAND)- Velocity
    End point description
    To compare the efficacy, as measured by the Time to Stand Test (TTSTAND), of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. untreated DMD historical controls in boys ages 4-7 years with DMD
    End point type
    Primary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: rises/second
    arithmetic mean (standard deviation)
        002 Baseline
    0.18 ( 0.065 )
    0.24 ( 0.090 )
    0.22 ( 0.082 )
    0.19 ( 0.056 )
        003 Baseline
    0.15 ( 0.045 )
    0.22 ( 0.077 )
    0.24 ( 0.078 )
    0.22 ( 0.070 )
        003 Week 12
    0.18 ( 0.072 )
    0.23 ( 0.102 )
    0.24 ( 0.089 )
    0.22 ( 0.075 )
        003 Week 12 Change from 002 Baseline
    -0.01 ( 0.061 )
    0.00 ( 0.054 )
    0.02 ( 0.066 )
    0.02 ( 0.034 )
        003 Week 24
    0.18 ( 0.081 )
    0.24 ( 0.114 )
    0.26 ( 0.108 )
    0.24 ( 0.086 )
        003 Week 24 Change from 002 Baseline
    -0.01 ( 0.066 )
    0.00 ( 0.062 )
    0.05 ( 0.061 )
    0.04 ( 0.045 )
    Statistical analysis title
    		 Week 24 Change from 002 baseline
    Comparison groups
    Dose level Group 2 v Dose level group 1 v Dose level Group 3 v Dose level Group 4
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0192 [2]
    Method
    		 Mixed models analysis
    Confidence interval
    Notes
    [2] - Group 1 compared to Group 3

    Primary: Safety as Measured by BMI Z-score

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    End point title
    		 Safety as Measured by BMI Z-score
    End point description
    To compare the safety, as measured by body mass index (BMI) z-score, of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls in boys ages 4-7 years with DMD.
    End point type
    Primary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: unitless
    arithmetic mean (standard deviation)
        002 Baseline
    1.165 ( 0.6219 )
    0.703 ( 1.0738 )
    1.200 ( 0.5325 )
    0.695 ( 0.7189 )
        003 Week 12
    1.103 ( 0.6457 )
    0.494 ( 1.0680 )
    1.261 ( 0.3981 )
    1.011 ( 0.7034 )
        003 Week 12 Change from 002 Baseline
    -0.062 ( 0.2438 )
    -0.209 ( 0.4078 )
    0.062 ( 0.3886 )
    0.174 ( 0.5826 )
        003 Week 24
    1.004 ( 0.6381 )
    0.493 ( 1.1696 )
    1.242 ( 0.4596 )
    1.330 ( 0.5857 )
        003 Week 24 Change from 002 Baseline
    -0.161 ( 0.3234 )
    -0.210 ( 0.3629 )
    0.043 ( 0.3849 )
    0.493 ( 0.6363 )
    Statistical analysis title
    		 Week 24 Change from 002 baseline
    Comparison groups
    Dose level group 1 v Dose level Group 2 v Dose level Group 3 v Dose level Group 4
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.003 [3]
    Method
    		 Mixed models analysis
    Confidence interval
    Notes
    [3] - Dose level group 1 vs. dose level group 4

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: pg/mL
    arithmetic mean (standard deviation)
        002 Baseline
    18.3 ( 2.96 )
    18.0 ( 6.88 )
    21.1 ( 6.13 )
    19.3 ( 8.67 )
        003 Baseline
    15.9 ( 4.52 )
    18.6 ( 4.56 )
    18.2 ( 5.29 )
    18.4 ( 9.73 )
        003 Week 8
    13.0 ( 6.25 )
    7.1 ( 5.84 )
    7.8 ( 4.42 )
    6.5 ( 5.23 )
        003 Week 8 Change from 002 Baseline
    -5.3 ( 6.92 )
    -10.5 ( 9.32 )
    -13.3 ( 7.33 )
    -13.5 ( 7.84 )
        003 Week 8 Percent Change from 002 Baseline
    -27.1 ( 40.94 )
    -54.2 ( 36.34 )
    -61.9 ( 21.34 )
    -67.7 ( 24.09 )
        003 Week 16
    12.2 ( 4.93 )
    9.0 ( 5.86 )
    9.0 ( 14.18 )
    9.0 ( 4.49 )
        003 Week 16 Change from 002 Baseline
    -6.2 ( 6.34 )
    -9.1 ( 9.89 )
    -12.0 ( 15.42 )
    -12.2 ( 8.16 )
        003 Week 16 Percent Change from 002 Baseline
    -30.6 ( 35.64 )
    -42.8 ( 44.91 )
    -55.7 ( 64.72 )
    -56.0 ( 22.61 )
        003 Week 24
    19.8 ( 6.32 )
    14.0 ( 3.88 )
    15.7 ( 9.63 )
    11.3 ( 7.52 )
        003 Week 24 Change from 002 Baseline
    0.6 ( 6.63 )
    -4.0 ( 5.48 )
    -5.4 ( 11.09 )
    -6.3 ( 7.78 )
        003 Week 24 Percent Change from 002 Baseline
    4.9 ( 37.93 )
    -18.6 ( 19.99 )
    -21.3 ( 50.74 )
    -34.3 ( 49.52 )
        003 Week 26-29
    9.5 ( 7.26 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Change from 002 Baseline
    -7.8 ( 4.19 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Percent Change from 002 Baseline
    -49.5 ( 33.00 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: mg/dL
    arithmetic mean (standard deviation)
        002 Baseline
    87.5 ( 9.44 )
    88.9 ( 18.71 )
    89.3 ( 7.91 )
    92.3 ( 8.19 )
        003 Week 12
    81.5 ( 5.61 )
    81.7 ( 4.35 )
    84.3 ( 8.13 )
    86.5 ( 5.57 )
        003 Week 12 Change from 002 Baseline
    -6.8 ( 8.29 )
    -7.6 ( 19.22 )
    -5.1 ( 9.01 )
    -5.2 ( 9.21 )
        003 Week 12 Percent Change from 002 Baseline
    -7.0 ( 8.95 )
    -5.8 ( 13.93 )
    -5.3 ( 9.41 )
    -5.0 ( 9.95 )
        003 Week 24
    80.8 ( 6.56 )
    80.8 ( 4.08 )
    81.3 ( 7.94 )
    84.6 ( 6.53 )
        003 Week 24 Change from 002 Baseline
    -6.3 ( 11.97 )
    -9.0 ( 20.87 )
    -8.1 ( 10.28 )
    -7.8 ( 9.44 )
        003 Week 24 Percent Change from 002 Baseline
    -6.1 ( 13.69 )
    -7.0 ( 15.41 )
    -8.6 ( 10.73 )
    -7.9 ( 9.96 )
    No statistical analyses for this end point

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: UIU/mL
    arithmetic mean (standard deviation)
        002 Baseline
    5.54 ( 3.651 )
    3.09 ( 2.033 )
    3.40 ( 1.548 )
    3.96 ( 2.027 )
        003 Week 12
    4.17 ( 3.167 )
    2.97 ( 1.669 )
    3.89 ( 2.189 )
    6.97 ( 3.526 )
        003 Week 12 Change from 002 Baseline
    -1.13 ( 3.822 )
    -0.14 ( 1.756 )
    0.49 ( 2.592 )
    2.97 ( 2.277 )
        003 Week 12 Percent Change from 002 Baseline
    -12.70 ( 50.989 )
    9.35 ( 64.450 )
    50.96 ( 135.187 )
    85.79 ( 76.416 )
        003 Week 24
    4.23 ( 2.560 )
    3.12 ( 1.788 )
    4.82 ( 3.393 )
    7.21 ( 2.374 )
        003 Week 24 Change from 002 Baseline
    -1.67 ( 4.478 )
    0.34 ( 2.898 )
    1.36 ( 3.262 )
    3.26 ( 2.862 )
        003 Week 24 Percent Change from 002 Baseline
    -15.83 ( 50.754 )
    53.32 ( 121.763 )
    60.57 ( 114.769 )
    121.34 ( 107.414 )
    No statistical analyses for this end point

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: Percent Hemoglobin A1C
    arithmetic mean (standard deviation)
        002 Baseline
    5.18 ( 0.260 )
    5.22 ( 0.244 )
    5.19 ( 0.124 )
    5.23 ( 0.231 )
        003 Week 8
    5.18 ( 0.226 )
    5.33 ( 0.250 )
    5.28 ( 0.204 )
    5.25 ( 0.216 )
        003 Week 8 Change from 002 Baseline
    0.00 ( 0.128 )
    0.12 ( 0.153 )
    0.09 ( 0.198 )
    0.00 ( 0.100 )
        003 Week 8 Percent Change from 002 Baseline
    0.06 ( 2.470 )
    2.28 ( 3.008 )
    1.79 ( 3.864 )
    0.02 ( 1.927 )
        003 Week 16
    5.26 ( 0.239 )
    5.35 ( 0.238 )
    5.26 ( 0.156 )
    5.31 ( 0.270 )
        003 Week 16 Change from 002 Baseline
    0.08 ( 0.204 )
    0.14 ( 0.225 )
    0.07 ( 0.130 )
    0.05 ( 0.113 )
        003 Week 16 Percent Change from 002 Baseline
    1.70 ( 3.989 )
    2.72 ( 4.457 )
    1.30 ( 2.505 )
    1.03 ( 2.147 )
        003 Week 24
    5.15 ( 0.302 )
    5.22 ( 0.221 )
    5.13 ( 0.160 )
    5.24 ( 0.254 )
        003 Week 24 Change from 002 Baseline
    -0.10 ( 0.220 )
    0.00 ( 0.186 )
    -0.07 ( 0.130 )
    -0.02 ( 0.154 )
        003 Week 24 Percent Change from 002 Baseline
    -1.89 ( 4.081 )
    0.08 ( 3.628 )
    -1.27 ( 2.525 )
    -0.33 ( 2.895 )
        003 Week 26-29
    5.07 ( 0.208 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Change from 002 Baseline
    -0.07 ( 0.208 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Percent Change from 002 Baseline
    -1.25 ( 3.942 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: ng/mL
    arithmetic mean (standard deviation)
        002 Baseline
    37.94 ( 11.622 )
    35.66 ( 6.800 )
    41.17 ( 5.617 )
    44.36 ( 5.979 )
        003 Baseline
    39.20 ( 14.136 )
    41.84 ( 8.552 )
    47.91 ( 6.648 )
    42.81 ( 10.851 )
        003 Week 8
    36.21 ( 10.374 )
    41.78 ( 13.856 )
    44.45 ( 7.439 )
    41.55 ( 5.446 )
        003 Week 8 Change from 002 Baseline
    -1.60 ( 8.849 )
    6.13 ( 10.440 )
    3.28 ( 8.325 )
    -2.01 ( 8.898 )
        003 Week 8 Percent Change from 002 Baseline
    -1.16 ( 22.132 )
    16.51 ( 27.717 )
    9.34 ( 23.470 )
    -2.82 ( 20.662 )
        003 Week 16
    39.01 ( 9.620 )
    42.23 ( 9.393 )
    44.60 ( 9.534 )
    39.39 ( 6.972 )
        003 Week 16 Change from 002 Baseline
    1.07 ( 9.916 )
    6.57 ( 6.709 )
    3.43 ( 10.718 )
    -4.17 ( 8.494 )
        003 Week 16 Percent Change from 002 Baseline
    7.48 ( 31.539 )
    18.93 ( 20.384 )
    10.09 ( 26.893 )
    -8.52 ( 19.797 )
        003 Week 24
    38.80 ( 6.292 )
    51.41 ( 11.265 )
    51.98 ( 9.372 )
    49.08 ( 7.771 )
        003 Week 24 Change from 002 Baseline
    -1.34 ( 11.289 )
    15.75 ( 10.211 )
    10.81 ( 7.542 )
    5.29 ( 7.858 )
        003 Week 24 Percent Change from 002 Baseline
    2.52 ( 26.344 )
    46.30 ( 32.470 )
    26.61 ( 17.650 )
    13.10 ( 19.573 )
        003 Week 26-29
    40.10 ( 18.729 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Change from 002 Baseline
    -1.23 ( 17.943 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Percent Change from 002 Baseline
    -0.58 ( 49.574 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: ng/mL
    arithmetic mean (standard deviation)
        002 Baseline
    555.8 ( 184.72 )
    480.7 ( 118.20 )
    508.2 ( 94.36 )
    511.5 ( 106.50 )
        003 Baseline
    573.8 ( 251.02 )
    489.3 ( 121.66 )
    492.0 ( 81.92 )
    566.3 ( 149.32 )
        003 Week 8
    511.6 ( 190.94 )
    459.8 ( 101.93 )
    485.2 ( 105.12 )
    402.7 ( 70.46 )
        003 Week 8 Change from 002 Baseline
    -20.3 ( 120.32 )
    -22.9 ( 128.79 )
    -23.0 ( 96.84 )
    -105.6 ( 121.07 )
        003 Week 8 Percent Change from 002 Baseline
    -3.5 ( 24.14 )
    -1.1 ( 27.39 )
    -2.8 ( 20.99 )
    -18.1 ( 19.46 )
        003 Week 16
    481.9 ( 159.93 )
    431.8 ( 81.25 )
    455.7 ( 99.50 )
    488.5 ( 130.11 )
        003 Week 16 Change from 002 Baseline
    -73.8 ( 109.31 )
    -42.4 ( 109.07 )
    -52.5 ( 104.05 )
    -19.8 ( 130.12 )
        003 Week 16 Percent Change from 002 Baseline
    -12.1 ( 18.25 )
    -5.1 ( 25.62 )
    -8.6 ( 20.91 )
    -2.3 ( 26.63 )
        003 Week 24
    457.1 ( 129.21 )
    471.1 ( 121.10 )
    565.5 ( 158.89 )
    526.2 ( 130.18 )
        003 Week 24 Change from 002 Baseline
    -30.8 ( 113.64 )
    2.1 ( 165.16 )
    57.3 ( 150.36 )
    8.7 ( 88.95 )
        003 Week 24 Percent Change from 002 Baseline
    -6.2 ( 22.42 )
    6.3 ( 38.81 )
    13.2 ( 30.95 )
    2.2 ( 18.51 )
        003 Week 26-29
    619.0 ( 379.07 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Change from 002 Baseline
    -152.0 ( 331.46 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Percent Change from 002 Baseline
    -16.5 ( 42.70 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Serum Pharmacodynamics Biomarkers Measured by Levels of CTX

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    End point title
    		 Serum Pharmacodynamics Biomarkers Measured by Levels of CTX
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: pg/mLd
    arithmetic mean (standard deviation)
        002 Baseline
    871.0 ( 160.85 )
    935.8 ( 286.50 )
    936.8 ( 256.25 )
    889.3 ( 186.68 )
        003 Baseline
    915.9 ( 263.13 )
    964.4 ( 319.26 )
    949.8 ( 303.76 )
    989.2 ( 216.29 )
        003 Week 8
    897.1 ( 365.45 )
    933.3 ( 330.20 )
    928.3 ( 333.11 )
    825.5 ( 164.36 )
        003 Week 8 Change from 002 Baseline
    26.1 ( 368.76 )
    -31.6 ( 236.34 )
    -8.5 ( 248.82 )
    -59.6 ( 259.08 )
        003 Week 8 Percent Change from 002 Baseline
    3.9 ( 39.76 )
    -1.2 ( 26.23 )
    0.2 ( 26.94 )
    -2.4 ( 30.39 )
        003 Week 16
    885.4 ( 261.53 )
    912.8 ( 305.08 )
    939.8 ( 157.17 )
    953.7 ( 199.53 )
        003 Week 16 Change from 002 Baseline
    -2.6 ( 304.50 )
    -46.3 ( 321.06 )
    3.0 ( 244.17 )
    102.3 ( 230.44 )
        003 Week 16 Percent Change from 002 Baseline
    2.2 ( 33.71 )
    -1.2 ( 32.64 )
    5.4 ( 26.64 )
    15.6 ( 35.06 )
        003 Week 24
    1109.3 ( 287.92 )
    1235.6 ( 295.79 )
    1248.7 ( 308.90 )
    1237.0 ( 277.20 )
        003 Week 24 Change from 002 Baseline
    212.3 ( 318.86 )
    295.6 ( 357.93 )
    346.5 ( 327.16 )
    321.4 ( 264.65 )
        003 Week 24 Percent Change from 002 Baseline
    26.2 ( 35.61 )
    39.8 ( 43.14 )
    43.0 ( 38.40 )
    37.5 ( 31.68 )
        003 Week 26-29
    1059.3 ( 536.26 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Change from 002 Baseline
    569.5 ( 28.99 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
        003 Week 26-29 Percent Change from 002 Baseline
    73.4 ( 18.15 )
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity

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    End point title
    		 Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Climb Test (TTCLIMB) in boys ages 4-7 years with DMD.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: tasks/ second
    arithmetic mean (standard deviation)
        002 Baseline
    0.20 ( 0.054 )
    0.29 ( 0.147 )
    0.29 ( 0.164 )
    0.24 ( 0.086 )
        003 Baseline
    0.20 ( 0.065 )
    0.29 ( 0.168 )
    0.31 ( 0.144 )
    0.25 ( 0.082 )
        003 Week 12
    0.21 ( 0.064 )
    0.34 ( 0.238 )
    0.31 ( 0.157 )
    0.26 ( 0.095 )
        003 Week 12 Change from 002 Baseline
    0.01 ( 0.044 )
    0.05 ( 0.115 )
    0.02 ( 0.107 )
    0.02 ( 0.051 )
        003 Week 24
    0.20 ( 0.071 )
    0.30 ( 0.166 )
    0.34 ( 0.148 )
    0.29 ( 0.097 )
        003 Week 24 Change from 002 Baseline
    0.00 ( 0.076 )
    0.01 ( 0.066 )
    0.04 ( 0.090 )
    0.05 ( 0.061 )
    No statistical analyses for this end point

    Secondary: Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity

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    End point title
    		 Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Run/Walk Test (TTRW) in boys ages 4-7 years with DMD.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: meters/second
    arithmetic mean (standard deviation)
        002 Baseline
    1.60 ( 0.312 )
    1.77 ( 0.367 )
    1.84 ( 0.347 )
    1.64 ( 0.279 )
        003 Baseline
    1.57 ( 0.371 )
    1.78 ( 0.414 )
    1.86 ( 0.418 )
    1.72 ( 0.295 )
        003 Week 12
    1.54 ( 0.306 )
    1.77 ( 0.550 )
    1.97 ( 0.503 )
    1.88 ( 0.341 )
        003 Week 12 Change from 002 Baseline
    -0.06 ( 0.261 )
    0.00 ( 0.307 )
    0.13 ( 0.316 )
    0.26 ( 0.297 )
        003 Week 24
    1.55 ( 0.384 )
    1.84 ( 0.486 )
    1.90 ( 0.321 )
    1.89 ( 0.378 )
        003 Week 24 Change from 002 Baseline
    -0.05 ( 0.311 )
    0.06 ( 0.210 )
    0.06 ( 0.210 )
    0.27 ( 0.254 )
    No statistical analyses for this end point

    Secondary: Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)

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    End point title
    		 Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by North Star Ambulatory Assessment (NSAA) in boys ages 4-7 years with DMD.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: total score
    arithmetic mean (standard deviation)
        002 Baseline
    19.0 ( 5.13 )
    20.5 ( 5.58 )
    20.0 ( 4.95 )
    19.7 ( 4.94 )
        003 Baseline
    20.1 ( 7.30 )
    20.8 ( 5.66 )
    21.7 ( 3.87 )
    20.4 ( 4.01 )
        003 Week 12
    19.3 ( 5.60 )
    21.2 ( 6.45 )
    21.0 ( 5.13 )
    20.4 ( 5.41 )
        003 Week 12 Change from 002 Baseline
    0.3 ( 2.06 )
    0.7 ( 2.71 )
    1.0 ( 2.56 )
    0.5 ( 2.38 )
        003 Week 24
    19.8 ( 7.09 )
    21.6 ( 7.23 )
    22.3 ( 3.80 )
    22.3 ( 5.76 )
        003 Week 24 Change from 002 Baseline
    0.8 ( 2.83 )
    1.1 ( 2.94 )
    2.3 ( 1.78 )
    2.5 ( 2.62 )
    No statistical analyses for this end point

    Secondary: Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT)

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    End point title
    		 Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT)
    End point description
    To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by 6-minute Walk Test (6MWT) in boys ages 4-7 years with DMD.
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 Dose level group 1 Dose level Group 2 Dose level Group 3 Dose level Group 4
    Number of subjects analysed
    12
    12
    12
    12
    Units: meters
    arithmetic mean (standard deviation)
        002 Baseline
    316.2 ( 59.47 )
    331.5 ( 52.76 )
    353.9 ( 65.40 )
    336.8 ( 63.18 )
        003 Baseline
    294.3 ( 60.62 )
    332.2 ( 56.83 )
    341.1 ( 49.42 )
    335.1 ( 80.13 )
        003 Week 12
    312.9 ( 60.93 )
    358.7 ( 71.47 )
    393.7 ( 59.72 )
    369.9 ( 69.47 )
        003 Week 12 Change from 002 Baseline
    6.0 ( 28.81 )
    20.8 ( 38.09 )
    39.8 ( 35.61 )
    27.6 ( 42.0 )
        003 Week 24
    306.2 ( 68.08 )
    350.4 ( 64.23 )
    383.1 ( 63.38 )
    372.6 ( 69.12 )
        003 Week 24 Change from 002 Baseline
    -11.6 ( 29.45 )
    18.9 ( 41.08 )
    29.2 ( 35.91 )
    43.9 ( 43.72 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events, including Serious Adverse Events (SAEs), and concomitant medications will be assessed at each study visit and recorded throughout the study starting from Treatment Period Day 1.
    Adverse event reporting additional description
    Adverse events will be summarized overall and by dose level, system organ class (SOC) and preferred term (using the Medical Dictionary for Regulatory Activities [MedDRA]); by dose level and relationship to study medication; and by dose level and intensity (CTCAE grade).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Dose Group 1
    Reporting group description
    		 -

    Reporting group title
    Dose Group 2
    Reporting group description
    		 -

    Reporting group title
    Dose Group 3
    Reporting group description
    		 -

    Reporting group title
    Dose Group 4
    Reporting group description
    		 -

    Serious adverse events
    		 Dose Group 1 Dose Group 2 Dose Group 3 Dose Group 4
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Reproductive system and breast disorders
    Testicular torsion
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Hypoxia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Dose Group 1 Dose Group 2 Dose Group 3 Dose Group 4
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 12 (83.33%)
    10 / 12 (83.33%)
    11 / 12 (91.67%)
    11 / 12 (91.67%)
    General disorders and administration site conditions
    Gait disturbance
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    2 / 12 (16.67%)
    7 / 12 (58.33%)
    5 / 12 (41.67%)
    3 / 12 (25.00%)
         occurrences all number
    2
    7
    5
    3
    Thirst
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Reproductive system and breast disorders
    Testicular torsion
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Cough
         subjects affected / exposed
    1 / 12 (8.33%)
    4 / 12 (33.33%)
    1 / 12 (8.33%)
    3 / 12 (25.00%)
         occurrences all number
    1
    4
    1
    3
    Epistaxis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hypoxia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Nasal congestion
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Productive cough
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Upper-airway cough syndrome
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Psychiatric disorders
    abnormal behaviour
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Anxiety
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Emotional disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    1
    Insomnia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Personality change
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Stereotypy
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Tic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Investigations
    Blood cortisol abnormal
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Urine output increased
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Weight increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Arthropod sting
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Foot fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Human bite
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Laceration
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Muscle strain
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    0
    1
    Tendon injury
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Upper limb fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Cardiac disorders
    Left ventricular dysfunction
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    2
    1
    Presyncope
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Psychomotor hyperactivity
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    1
    Eye disorders
    Excessive eye blinking
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Abdominal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Abdominal Pain upper
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    1
    2
    Constipation
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
         occurrences all number
    1
    0
    1
    2
    Diarrhoea
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    1 / 12 (8.33%)
         occurrences all number
    2
    0
    2
    1
    Faeces discoloured
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Lip swelling
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    3 / 12 (25.00%)
    2 / 12 (16.67%)
         occurrences all number
    1
    1
    3
    2
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Dermatitis contact
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eczema
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hypertrichosis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    2
    Rash
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Rash pruritic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Swelling face
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Uriticaria
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    0
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Back pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Myalgia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    2
    Pain in jaw
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Tendon pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Conjunctivitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    2
    1
    Ear infection
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    1
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal infection
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hordeolum
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Influenza
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    1
    0
    0
    3
    Otitis media
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Pharyngitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pharyngitis streptococcal
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pneumonia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Sinusitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Sinusitis bacterial
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Staphylococcal skin infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Tooth infection
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    1
    0
    Viral infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    4 / 12 (33.33%)
    4 / 12 (33.33%)
    7 / 12 (58.33%)
    5 / 12 (41.67%)
         occurrences all number
    4
    4
    7
    5
    Enterobiasis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Eye infection
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Hyperlipidaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    1
    Hypoglycaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Vitamin D deficiency
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    Neck pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0

    More information

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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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