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    Clinical Trial Results:
    A phase II open-label, multiple dose study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of vamorolone in boys ages 2 to <4 years and 7 to <18 years with Duchenne Muscular Dystrophy (DMD)

    Summary
    EudraCT number
    		 2025-000201-16
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    17 Jun 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    01 Aug 2025
    First version publication date
    01 Aug 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    VBP15-006
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05185622
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND: 118,942
    Sponsors
    Sponsor organisation name
    Santhera Pharmaceuticals (Schweiz) AG
    Sponsor organisation address
    Hohenrainstr. 24, Pratteln, Switzerland, 4133
    Public contact
    Chief Medical Officer, Santhera Pharmaceuticals (Schweiz) AG, office@santhera.com
    Scientific contact
    Chief Medical Officer, Santhera Pharmaceuticals (Schweiz) AG, office@santhera.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    07 Nov 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Jun 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Jun 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a 3-month treatment period in boys ages 2 to <4 and 7 to <18 years with DMD.
    Protection of trial subjects
    No specific measures.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Mar 2022
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Ethical reason
    Long term follow-up duration
    		 5 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 54
    Worldwide total number of subjects
    54
    EEA total number of subjects
    0
    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)
    42
    Adolescents (12-17 years)
    12
    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
    		 -

    Pre-assignment
    Screening details
    		 The study comprised a 5-week Pretreatment Screening Period; a 1-day Pretreatment Baseline Period. A total of 57 subjects were screened. Three subjects did not enter the Treatment Period due to failure to meet the inclusion criteria.

    Period 1
    Period 1 title
    5-week Pretreatment Screening Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Screening
    Arm description
    		 -
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Screening
    Started
    54
    Completed
    54
    Period 2
    Period 2 title
    1-day Pretreatment Baseline Period
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Age 2-<4 yrs - vamorolone 2 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    2 mg / kg

    Arm title
    		 Age 2-<4 yrs - vamorolone 6 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    6 mg / kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    2 mg / kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    6 mg / kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    2 mg / kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    6 mg / kg

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: Baseline Period is Period 2, when the assessments prior to 1st dose happen
    Number of subjects in period 2
    		Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Started
    10
    10
    6
    6
    6
    16
    Completed
    10
    10
    6
    6
    6
    16
    Period 3
    Period 3 title
    3-month open-label Treatment Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Age 2-<4 yrs - vamorolone 2 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    2 mg/kg

    Arm title
    		 Age 2-<4 yrs - vamorolone 6 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    6 mg/kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    2 mg/kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    6 mg/kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    2 mg/kg

    Arm title
    		 Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    vamorolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    6 mg/kg

    Number of subjects in period 3
    		Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Started
    10
    10
    6
    6
    6
    16
    Completed
    10
    10
    6
    6
    6
    16

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Age 2-<4 yrs - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 2-<4 yrs - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg Total
    Number of subjects
    		 10 10 6 6 6 16 54
    Age categorical
    Units: Subjects
        Age 2 to <4 Years
    		 10 10 0 0 0 0 20
        Age 7 to <18 Years
    		 0 0 6 6 6 16 34
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 3.3 ( 0.45 ) 3.5 ( 0.32 ) 9.9 ( 3.57 ) 8.3 ( 1.48 ) 10.7 ( 3.61 ) 13.2 ( 2.70 ) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0 0 0 0 0
        Male
    		 10 10 6 6 6 16 54
    Race
    Units: Subjects
        Asian
    		 5 1 1 2 2 3 14
        Black or African American
    		 0 1 0 0 0 0 1
        White
    		 5 8 5 3 3 13 37
        Multiple
    		 0 0 0 1 0 0 1
        Unknown
    		 0 0 0 0 1 0 1
    Age at 1st symptoms
    Units: months
        arithmetic mean (standard deviation)
    		 20.8 ( 5.67 ) 14.2 ( 4.47 ) 30.0 ( 15.18 ) 51.0 ( 23.92 ) 32.2 ( 14.26 ) 42.8 ( 27.59 ) -
    Subject analysis sets

    Subject analysis set title
    Safety set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects who receive at least one dose of study medication. Is the analysis set for all analyses, except PK analyses.

    Subject analysis set title
    PK set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects who receive at least one dose of vamorolone study medication and have sufficient data for PK analysis. Is the analysis set for PK analyses

    Subject analysis sets values
    		 Safety set PK set
    Number of subjects
    54
    54
    Age categorical
    Units: Subjects
        Age 2 to <4 Years
    20
    20
        Age 7 to <18 Years
    34
    34
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    8.4 ( 4.68 )
    8.4 ( 4.68 )
    Gender categorical
    Units: Subjects
        Female
    0
    0
        Male
    54
    54
    Race
    Units: Subjects
        Asian
    14
    14
        Black or African American
    1
    1
        White
    37
    37
        Multiple
    1
    1
        Unknown
    1
    1
    Age at 1st symptoms
    Units: months
        arithmetic mean (standard deviation)
    31.7 ( 21.88 )
    31.7 ( 21.88 )

    End points

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    End points reporting groups
    Reporting group title
    Screening
    Reporting group description
    		 -
    Reporting group title
    Age 2-<4 yrs - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 2-<4 yrs - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Reporting group description
    		 -
    Reporting group title
    Age 2-<4 yrs - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 2-<4 yrs - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Subject analysis set title
    Safety set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects who receive at least one dose of study medication. Is the analysis set for all analyses, except PK analyses.

    Subject analysis set title
    PK set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects who receive at least one dose of vamorolone study medication and have sufficient data for PK analysis. Is the analysis set for PK analyses

    Primary: Overview of treatment-emergent adverse events (TEAEs)

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    End point title
    		 Overview of treatment-emergent adverse events (TEAEs) [1]
    End point description
    End point type
    Primary
    End point timeframe
    3-month treatment period
    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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg Safety set
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    54
    Units: Number
        Total number of subjects with TEAEs
    7
    9
    6
    4
    3
    12
    41
        With drug related TEAEs
    1
    7
    3
    4
    1
    8
    24
        With severe TEAEs
    0
    0
    0
    1
    0
    1
    2
        With TEAEs leading to permanent discontinuation
    0
    0
    0
    0
    0
    0
    0
        With TEAEs leading to temporary interruption
    0
    0
    0
    0
    0
    1
    1
        With serious TEAEs
    0
    0
    0
    1
    0
    1
    2
    No statistical analyses for this end point

    Primary: Change in Height (Absolute) From Baseline

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    End point title
    		 Change in Height (Absolute) From Baseline [2]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    14
    Units: cm
        arithmetic mean (standard deviation)
    1.98 ( 1.146 )
    2.35 ( 0.568 )
    0.55 ( 0.557 )
    2.29 ( 1.609 )
    0.60 ( 0.594 )
    1.27 ( 1.313 )
    No statistical analyses for this end point

    Primary: Change in Height (Percentile) From Baseline

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    End point title
    		 Change in Height (Percentile) From Baseline [3]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [3] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    14
    Units: %
        arithmetic mean (standard deviation)
    1.17 ( 11.538 )
    2.09 ( 5.634 )
    -3.89 ( 3.907 )
    2.02 ( 8.338 )
    -3.69 ( 3.408 )
    -0.30 ( 1.390 )
    No statistical analyses for this end point

    Primary: Change in Height (Z-score) From Baseline

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    End point title
    		 Change in Height (Z-score) From Baseline [4]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    14
    Units: SD
        arithmetic mean (standard deviation)
    0.04 ( 0.361 )
    0.14 ( 0.201 )
    -0.12 ( 0.111 )
    0.07 ( 0.253 )
    -0.13 ( 0.159 )
    0.00 ( 0.194 )
    No statistical analyses for this end point

    Primary: Change in Weight (Absolute) From Baseline

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    End point title
    		 Change in Weight (Absolute) From Baseline [5]
    End point description
    End point type
    Primary
    End point timeframe
    week 12
    Notes
    [5] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: cm
        arithmetic mean (standard deviation)
    0.43 ( 0.776 )
    0.65 ( 0.911 )
    -0.18 ( 3.259 )
    3.47 ( 3.133 )
    1.53 ( 1.188 )
    1.94 ( 2.598 )
    No statistical analyses for this end point

    Primary: Change in Weight (Percentile) From Baseline

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    End point title
    		 Change in Weight (Percentile) From Baseline [6]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: %
        arithmetic mean (standard deviation)
    -1.44 ( 12.835 )
    -1.97 ( 12.504 )
    -4.00 ( 11.672 )
    15.20 ( 2.083 )
    2.12 ( 4.235 )
    4.22 ( 8.566 )
    No statistical analyses for this end point

    Primary: Change in Weight (Z-score) From Baseline

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    End point title
    		 Change in Weight (Z-score) From Baseline [7]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [7] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: SD
        arithmetic mean (standard deviation)
    -0.01 ( 0.406 )
    0.05 ( 0.430 )
    -0.03 ( 0.438 )
    0.55 ( 0.159 )
    0.11 ( 0.147 )
    0.22 ( 0.353 )
    No statistical analyses for this end point

    Primary: Change in Body Mass Index (BMI) (Absolute) From Baseline

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    End point title
    		 Change in Body Mass Index (BMI) (Absolute) From Baseline [8]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [8] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    14
    Units: kg/m2
        arithmetic mean (standard deviation)
    -0.09 ( 0.933 )
    -0.04 ( 0.665 )
    0.05 ( 1.394 )
    1.53 ( 1.385 )
    0.82 ( 0.784 )
    0.60 ( 1.471 )
    No statistical analyses for this end point

    Primary: Change in Body Mass Index (BMI) (Percentile) From Baseline

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    End point title
    		 Change in Body Mass Index (BMI) (Percentile) From Baseline [9]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [9] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    14
    Units: %
        arithmetic mean (standard deviation)
    -2.85 ( 24.623 )
    -1.61 ( 12.509 )
    -3.00 ( 15.781 )
    10.78 ( 7.312 )
    6.22 ( 6.849 )
    4.44 ( 11.498 )
    No statistical analyses for this end point

    Primary: Change in Body Mass Index (BMI) (Z-score) From Baseline

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    End point title
    		 Change in Body Mass Index (BMI) (Z-score) From Baseline [10]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    14
    Units: SD
        arithmetic mean (standard deviation)
    -0.05 ( 0.764 )
    -0.02 ( 0.495 )
    0.27 ( 0.771 )
    1.10 ( 0.584 )
    0.29 ( 0.317 )
    0.29 ( 0.483 )
    No statistical analyses for this end point

    Primary: Change in Blood Pressure (BP) from Baseline

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    End point title
    		 Change in Blood Pressure (BP) from Baseline [11]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [11] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    9
    10
    6
    6
    6
    15
    Units: mmHg
    arithmetic mean (standard deviation)
        Systolic BP
    -2.33 ( 12.923 )
    -2.20 ( 11.233 )
    -2.50 ( 6.504 )
    5.00 ( 13.266 )
    2.50 ( 5.958 )
    0.07 ( 11.591 )
        Diastolic BP
    -0.44 ( 8.248 )
    2.00 ( 8.179 )
    -3.17 ( 7.333 )
    5.80 ( 6.979 )
    1.50 ( 6.950 )
    -2.40 ( 11.915 )
    No statistical analyses for this end point

    Primary: Number of Participants With Treatment Emergent Cushingoid Features

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    End point title
    		 Number of Participants With Treatment Emergent Cushingoid Features [12]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to Week 12
    Notes
    [12] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: Number
    0
    0
    0
    1
    0
    0
    No statistical analyses for this end point

    Primary: Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result

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    End point title
    		 Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result [13]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to Week 12
    Notes
    [13] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: Number
        Cortisol decreased
    0
    1
    2
    0
    0
    2
        Blood glucose decreased
    0
    0
    0
    0
    0
    1
        Blood insulin in creased
    0
    0
    0
    0
    0
    1
        Blood TSH increased
    0
    0
    0
    0
    0
    1
        Thyroxin free increased
    0
    0
    0
    0
    0
    1
    No statistical analyses for this end point

    Primary: Categorical Analysis of QTcF

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    End point title
    		 Categorical Analysis of QTcF [14]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [14] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    5
    15
    Units: Number
        =< 450 msec
    10
    10
    6
    5
    5
    15
        > 450 msec
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Number of Subjects With Cataract

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    End point title
    		 Number of Subjects With Cataract [15]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline and Week 12
    Notes
    [15] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: Number
        No cataract at Baseline
    9
    9
    6
    6
    4
    8
        No cataract at Week 12
    0
    4
    6
    5
    4
    7
        Cataract at Baseline
    0
    0
    0
    0
    2
    8
        Cataract at Week12
    0
    0
    0
    0
    1
    7
    No statistical analyses for this end point

    Primary: Number of Subjects With Glaucoma

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    End point title
    		 Number of Subjects With Glaucoma [16]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline and Week 12
    Notes
    [16] - 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: Only descriptive statistics were planned and performed
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: Number
        No Glaucoma at Baseline
    9
    10
    6
    6
    4
    8
        No Glaucoma at Week 12
    9
    4
    6
    5
    4
    7
        Suspected Glaucoma at Baseline
    0
    0
    0
    0
    0
    1
        Suspected Glaucoma at Week 12
    0
    0
    0
    0
    0
    0
        Glaucoma at Baseline
    0
    0
    0
    0
    0
    0
        Glaucoma at Week 12
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 - Age 7-<18 yrs

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    End point title
    		 Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 - Age 7-<18 yrs
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 2
    End point values
    		 Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    6
    6
    6
    16
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 1 - predose
    0 ( 0.00 )
    0 ( 0.00 )
    0 ( 0.00 )
    0 ( 0.00 )
        Day 1 - 1h
    142.5 ( 225.74 )
    519.1 ( 437.26 )
    142.5 ( 225.74 )
    519.1 ( 437.26 )
        Day 1 - 2h
    216.4 ( 181.19 )
    780.5 ( 457.91 )
    216.4 ( 181.19 )
    780.5 ( 457.91 )
        Day 1 - 4h
    163.8 ( 103.74 )
    711.1 ( 353.60 )
    163.8 ( 103.74 )
    711.1 ( 353.60 )
        Day 1 - 6h
    148.0 ( 109.33 )
    389.1 ( 307.57 )
    148.0 ( 109.33 )
    389.1 ( 307.57 )
        Day 1 - 8h
    78.5 ( 41.66 )
    170.6 ( 183.67 )
    78.5 ( 41.66 )
    170.6 ( 183.67 )
        Week 2 - predose
    0.7 ( 2.06 )
    2.9 ( 5.22 )
    0.7 ( 2.06 )
    2.9 ( 5.22 )
        Week 2 - 1h
    192.0 ( 199.92 )
    463.7 ( 348.19 )
    192.0 ( 199.92 )
    463.7 ( 348.19 )
        Week 2 - 2h
    217.8 ( 149.31 )
    686.3 ( 369.69 )
    217.8 ( 149.31 )
    686.3 ( 369.69 )
        Week 2 - 4h
    207.6 ( 167.99 )
    603.0 ( 346.88 )
    207.6 ( 167.99 )
    603.0 ( 346.88 )
        Week 2 - 6h
    158.9 ( 105.34 )
    300.2 ( 206.18 )
    158.9 ( 105.34 )
    300.2 ( 206.18 )
        Week 2 - 8h
    55.8 ( 31.81 )
    174.7 ( 137.78 )
    55.8 ( 31.81 )
    174.7 ( 137.78 )
    No statistical analyses for this end point

    Secondary: Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 - Age 2-<4 yrs

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    End point title
    		 Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 - Age 2-<4 yrs
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 2
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    Units: ng / mL
    arithmetic mean (standard deviation)
        Day 1 - predose
    0.0 ( 0.00 )
    0 ( 0.00 )
        Day 1 - 1h
    198.3 ( 196.67 )
    471.3 ( 333.61 )
        Day 1 - 2h
    236.7 ( 153.58 )
    769.3 ( 527.67 )
        Day 1 - 6h
    125.7 ( 84.83 )
    280.0 ( 249.99 )
        Week 2 - predose
    0 ( 0.00 )
    0 ( 0.00 )
        Week 2 - 1h
    346.0 ( 329.08 )
    409.8 ( 385.65 )
        Week 2 - 2h
    360.4 ( 222.93 )
    829.4 ( 740.83 )
        Week 2 - 6h
    134.5 ( 110.99 )
    272.0 ( 109.71 )
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in Bayley-III Gross Motor Scale

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    End point title
    		 Change From Baseline in Bayley-III Gross Motor Scale
    End point description
    Test only applied to 2 to 4 year old subjects
    End point type
    Other pre-specified
    End point timeframe
    Week 12
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg
    Number of subjects analysed
    9
    10
    Units: scale unit
        arithmetic mean (standard deviation)
    0.44 ( 1.130 )
    2.50 ( 1.716 )
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in Morning Cortisol Concentration

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    End point title
    		 Change From Baseline in Morning Cortisol Concentration
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Week 12
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    8
    6
    6
    6
    15
    Units: nmol/mL
        arithmetic mean (standard deviation)
    -184.700 ( 138.2020 )
    -217.750 ( 103.9832 )
    -110.667 ( 85.8596 )
    -248.667 ( 186.9060 )
    12.000 ( 49.3356 )
    -34.933 ( 42.1485 )
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1], Osteocalcin and Serum Aminoterminal Propeptide of Type I Collagen [P1NP], )

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    End point title
    		 Change From Baseline in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1], Osteocalcin and Serum Aminoterminal Propeptide of Type I Collagen [P1NP], )
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Week 12
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    9
    6
    5
    6
    16
    Units: ng/L, ug/L
    arithmetic mean (standard deviation)
        CTX1 (ng/L)
    28.300 ( 387.1423 )
    138.000 ( 257.6640 )
    93.500 ( 235.7446 )
    101.800 ( 365.3542 )
    631.833 ( 155.3621 )
    273.000 ( 335.4937 )
        Osteocalcin (ug/L)
    -3.340 ( 21.0550 )
    2.422 ( 10.6960 )
    14.817 ( 18.7328 )
    -1.120 ( 15.8528 )
    31.700 ( 11.6915 )
    16.546 ( 9.1486 )
        P1NP (ug/L)
    -39.870 ( 188.7787 )
    14.478 ( 117.5704 )
    19.067 ( 78.6139 )
    -82.840 ( 178.0726 )
    318.850 ( 99.0038 )
    149.671 ( 106.2874 )
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in Insulin Resistance Biomarkers - Part 1 (Glucose, Hemoglobin A1c [HbA1c])

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    End point title
    		 Change From Baseline in Insulin Resistance Biomarkers - Part 1 (Glucose, Hemoglobin A1c [HbA1c])
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Week 12
    End point values
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    10
    6
    6
    6
    16
    Units: mmol/L, fraction of 1
    arithmetic mean (standard deviation)
        Glucose (mmol/L)
    -0.160 ( 0.2912 )
    -0.243 ( 0.6309 )
    -0.063 ( 0.3524 )
    -0.335 ( 0.3815 )
    0.195 ( 0.5727 )
    -0.254 ( 0.3909 )
        HbA1c (fraction of 1)
    0.001 ( 0.0026 )
    0.000 ( 0.0015 )
    0.000 ( 0.0016 )
    0.000 ( 0.0010 )
    0.000 ( 0.0011 )
    -0.001 ( 0.0012 )
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in Insulin Resistance Biomarkers - Part 2 (Insulin)

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    End point title
    		 Change From Baseline in Insulin Resistance Biomarkers - Part 2 (Insulin)
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Week 12
    End point values
    		 Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Number of subjects analysed
    10
    6
    6
    16
    Units: pmol/L
        arithmetic mean (standard deviation)
    6.333 ( 20.5183 )
    0.667 ( 33.0051 )
    46.333 ( 51.9256 )
    -26.333 ( 51.1493 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Until study completion (Week 12) [SAEs were reported until 30 days after last dose of study drug]
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.0
    Reporting groups
    Reporting group title
    Age 2-<4 yrs - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 2-<4 yrs - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Reporting group description
    		 -

    Serious adverse events
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    vomiting
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Rhabdomyolisis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Age 2-<4 yrs - vamorolone 2 mg/kg Age 2-<4 yrs - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid untreated - vamorolone 6 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 2 mg/kg Age 7-<18 yrs, corticosteroid treated - vamorolone 6 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 10 (70.00%)
    9 / 10 (90.00%)
    6 / 6 (100.00%)
    4 / 6 (66.67%)
    3 / 6 (50.00%)
    12 / 16 (75.00%)
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Energy increased
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Fatigue
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 10 (10.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    2
    1
    1
    0
    0
    1
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Epistaxis
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    1
    2
    0
    0
    1
    0
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Behaviour disorder
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Compulsive lip biting
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Disruptive mood dysregulation disorder
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 16 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Irritability
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Mood swings
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Investigations
    Blood glucose decreased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Blood insulin increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Blood thyroid stimulating hormone increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Thyroxine free increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Cortisol decreased
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 16 (12.50%)
         occurrences all number
    0
    1
    2
    0
    0
    2
    Weight increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Fall
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 10 (20.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    1
    2
    0
    1
    2
    0
    Head injury
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Joint injury
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Skin abrasion
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    4 / 16 (25.00%)
         occurrences all number
    0
    1
    3
    0
    0
    5
    Hypoaesthesia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Eye disorders
    Cataract
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 10 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    2
    0
    0
    0
    3
    Abnormal faeces
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 10 (0.00%)
    3 / 10 (30.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    3
    0
    0
    0
    0
    Faeces discoloured
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Toothache
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 10 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Vomiting
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 10 (10.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    2 / 16 (12.50%)
         occurrences all number
    1
    1
    1
    2
    0
    2
    Skin and subcutaneous tissue disorders
    Miliaria
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Rash
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Urticaria
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Endocrine disorders
    Adrenal insufficiency
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Adrenal suppression
         subjects affected / exposed
    0 / 10 (0.00%)
    5 / 10 (50.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    5
    0
    1
    0
    1
    Cushingoid
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Back pain
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    2 / 16 (12.50%)
         occurrences all number
    0
    1
    0
    0
    1
    2
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 10 (20.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    2
    0
    0
    2
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 10 (10.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    4 / 10 (40.00%)
    3 / 10 (30.00%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    2 / 16 (12.50%)
         occurrences all number
    8
    3
    2
    0
    1
    2
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 10 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    14 Apr 2022
    VBP15-006-A1 (Version 1.1) Reasons for Protocol Amendment #1: 1. To clarify vamorolone is a 4.0% wt/vol suspension and provide details of vamorolone packaging 2. To clarify that age of enrollment will be collected and date of birth will not 3. To clarify standing height will be measured in all participants who can stand independently 4. To remove glutamate dehydrogenase (GLDH) from laboratory evaluations 5. To clarify vamorolone administration will take place at the study site during the Week 6 visit 6. To specify subjects should be fasted ≥6 hours prior to all study visits when blood will be drawn
    09 Feb 2023
    VBP15-006-A2 (Version 1.0) Reasons for Protocol Amendment #2: The following changes have been included in this amendment and will be implemented following the standard approval process: • To make editorial modifications regarding transfer of Sponsor from ReveraGen BioPharma, Inc. to Santhera Pharmaceutical (Switzerland) Ltd. • To update section 1 following Investigator’s Brochure revision (version 12) • To update the reference list • To update Dosing Recommendation for children weighing 50 kg or more • To describe the procedure of the dose tapering after discontinuation of vamorolone • To include ambulatory status recording as part of physical examination at Baseline • To define a new age subgroup (12 to <18 years) in which 10 additional glucocorticoid-treated subjects will be recruited, to the vamorolone daily dose of 6 mg/kg and to add exploration of the gonadal and thyroid axis biomarkers in this subgroup. To update the total number of participating subjects accordingly. • To clarify that date of birth may be collected where local regulations allow.
    11 Apr 2023
    VBP15-006-A3 (Version 1.0) Reasons for Protocol Amendment #3: PPD, the central laboratory was providing an EDTA-1ML K2 (Plastic) tube for collection of specific samples. However the manufacturer of this item has informed PPD that they are discontinuing the manufacture of this item globally, effective immediately. As such and since there is no equivalent tube with 1 mL volume, PPD needs to replace it with an alternate tube EDTA-1.2 mL S-Monovette K2 (Plastic). As a consequence, the volumes of blood collected at the study visits need to be revised. The following changes have been included in this amendment and will be implemented following the standard approval process: • Replace the 1 mL EDTA blood sampling tube by the 1.2 mL and revise the sampling plan (HbA1c is analyzed from the hematology sample)

    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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