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    Clinical Trial Results:
    A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

    Summary
    EudraCT number
    2017-003568-10
    Trial protocol
    SE   GB  
    Global end of trial date
    27 Oct 2021

    Results information
    Results version number
    v1(current)
    This version publication date
    11 May 2022
    First version publication date
    11 May 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VBP15-LTE
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03038399
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    ReveraGen BioPharma Inc.
    Sponsor organisation address
    155 Gibbs St. Suite 433, Rockville, United States, 20850
    Public contact
    Chief Operating Officer, ReveraGen BioPharma Inc., +1 215 680 8286, jesse.damsker@reveragen.com
    Scientific contact
    Chief Operating Officer, 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
    27 Oct 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-month Treatment Period, in young boys with DMD who completed protocol VBP15-003;
    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
    02 Feb 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Sweden: 4
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    Canada: 7
    Country: Number of subjects enrolled
    United States: 18
    Country: Number of subjects enrolled
    Australia: 6
    Country: Number of subjects enrolled
    Israel: 5
    Worldwide total number of subjects
    46
    EEA total number of subjects
    4
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    46
    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 VBP15-003 trial are able to participate in the VBP15-LTE trial

    Pre-assignment
    Screening details
    Subject has previously completed study VBP15-003 up to and including the Week 24 Final assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering.

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Dose Level Group 1
    Arm description
    Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Arm title
    Dose Level Group 2
    Arm description
    Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Arm title
    Dose Level Group 3
    Arm description
    Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Arm title
    Dose Level Group 4
    Arm description
    Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Number of subjects in period 1
    Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
    Started
    11
    12
    12
    11
    Completed
    11
    12
    12
    11
    Period 2
    Period 2 title
    Treatment Period
    Is this the baseline period?
    No
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Dose Level Group 1
    Arm description
    Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Arm title
    Dose Level Group 2
    Arm description
    Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Arm title
    Dose Level Group 3
    Arm description
    Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Arm title
    Dose Level Group 4
    Arm description
    Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day
    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
    4% wt/wt oral suspension; administered daily

    Number of subjects in period 2 [1]
    Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
    Started
    11
    12
    12
    11
    Completed
    0
    0
    11
    32
    Not completed
    11
    23
    27
    9
         Desire to be eligible for other clinical trials
    -
    -
    -
    3
         Transferred to other arm/group
    11
    23
    27
    4
         undue study burden
    -
    -
    -
    1
         loss of muscle strength
    -
    -
    -
    1
    Joined
    0
    11
    26
    30
         Transferred in from other group/arm
    -
    11
    26
    30
    Notes
    [1] - The number of subjects transferring in and out of the arms in the period are not the same. It is expected the net number of transfers in and out of the arms in a period, will be zero.
    Justification: subjects were allowed to escalate and de-escalate dose levels throughout the trial

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dose Level Group 1
    Reporting group description
    Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day

    Reporting group title
    Dose Level Group 2
    Reporting group description
    Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day

    Reporting group title
    Dose Level Group 3
    Reporting group description
    Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day

    Reporting group title
    Dose Level Group 4
    Reporting group description
    Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day

    Reporting group values
    Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4 Total
    Number of subjects
    11 12 12 11 46
    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)
    11 12 12 11 46
        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
    11 12 12 11 46

    End points

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    End points reporting groups
    Reporting group title
    Dose Level Group 1
    Reporting group description
    Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day

    Reporting group title
    Dose Level Group 2
    Reporting group description
    Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day

    Reporting group title
    Dose Level Group 3
    Reporting group description
    Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day

    Reporting group title
    Dose Level Group 4
    Reporting group description
    Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day
    Reporting group title
    Dose Level Group 1
    Reporting group description
    Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day

    Reporting group title
    Dose Level Group 2
    Reporting group description
    Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day

    Reporting group title
    Dose Level Group 3
    Reporting group description
    Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day

    Reporting group title
    Dose Level Group 4
    Reporting group description
    Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day

    Primary: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03

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    End point title
    Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03 [1]
    End point description
    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- month Treatment Period, in boys ages 4-7 years with DMD; 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);
    End point type
    Primary
    End point timeframe
    24 months
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: no statistical analysis
    End point values
    Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
    Number of subjects analysed
    11
    23
    38
    41
    Units: subjects
    4
    14
    29
    39
    No statistical analyses for this end point

    Primary: Total Number of Adverse Events as Assessed by CTCAE Version 4.03

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    End point title
    Total Number of Adverse Events as Assessed by CTCAE Version 4.03 [2]
    End point description
    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-month Treatment Period, in boys ages 4-7 years with DMD. 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).
    End point type
    Primary
    End point timeframe
    24 months
    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: no statistical analysis
    End point values
    Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
    Number of subjects analysed
    11
    23
    38
    41
    Units: events
    14
    34
    202
    300
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    24 months
    Adverse event reporting additional description
    Adverse events, including Serious Adverse Events (SAEs will be assessed at each study visit and recorded throughout the 24 months treatment period.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Dose Level Group 1
    Reporting group description
    -

    Reporting group title
    Dose Level Group 2
    Reporting group description
    -

    Reporting group title
    Dose Level Group 3
    Reporting group description
    -

    Reporting group title
    Dose Level Group 4
    Reporting group description
    -

    Serious adverse events
    Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    0 / 38 (0.00%)
    1 / 41 (2.44%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Renal and urinary disorders
    Myoglobinuria
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    1 / 41 (2.44%)
         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
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         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 Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 11 (36.36%)
    14 / 23 (60.87%)
    29 / 38 (76.32%)
    39 / 41 (95.12%)
    Investigations
    Weight increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    2 / 38 (5.26%)
    10 / 41 (24.39%)
         occurrences all number
    0
    0
    2
    10
    Blood triglycerides increased
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    3 / 41 (7.32%)
         occurrences all number
    0
    0
    0
    3
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    4 / 38 (10.53%)
    6 / 41 (14.63%)
         occurrences all number
    0
    1
    4
    6
    Joint injury
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    2 / 38 (5.26%)
    0 / 41 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Limb injury
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    3 / 38 (7.89%)
    0 / 41 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Arthropod bite
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 23 (4.35%)
    1 / 38 (2.63%)
    1 / 41 (2.44%)
         occurrences all number
    1
    1
    1
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    4 / 38 (10.53%)
    7 / 41 (17.07%)
         occurrences all number
    0
    0
    4
    7
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    1 / 38 (2.63%)
    3 / 41 (7.32%)
         occurrences all number
    0
    1
    1
    3
    Influenza like illness
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    0 / 38 (0.00%)
    3 / 41 (7.32%)
         occurrences all number
    0
    1
    0
    3
    Pyrexia
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    9 / 38 (23.68%)
    8 / 41 (19.51%)
         occurrences all number
    0
    0
    9
    8
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 23 (8.70%)
    5 / 38 (13.16%)
    6 / 41 (14.63%)
         occurrences all number
    0
    2
    5
    6
    Medical device site joint pain
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Medical device site rash
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Ear and labyrinth disorders
    Ear Pain
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    5 / 38 (13.16%)
    4 / 41 (9.76%)
         occurrences all number
    0
    0
    5
    4
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    2 / 38 (5.26%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    2
    0
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    6 / 41 (14.63%)
         occurrences all number
    0
    0
    0
    6
    Abdominal pain upper
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    1 / 38 (2.63%)
    5 / 41 (12.20%)
         occurrences all number
    0
    0
    1
    5
    Constipation
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    3 / 38 (7.89%)
    8 / 41 (19.51%)
         occurrences all number
    0
    0
    3
    8
    Diarrhoea
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    1 / 38 (2.63%)
    3 / 41 (7.32%)
         occurrences all number
    0
    1
    1
    3
    Vomiting
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    5 / 38 (13.16%)
    7 / 41 (17.07%)
         occurrences all number
    1
    0
    5
    7
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 23 (4.35%)
    7 / 38 (18.42%)
    8 / 41 (19.51%)
         occurrences all number
    1
    1
    7
    8
    Oropharyngeal pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    1 / 38 (2.63%)
    3 / 41 (7.32%)
         occurrences all number
    0
    1
    1
    3
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 23 (8.70%)
    0 / 38 (0.00%)
    1 / 41 (2.44%)
         occurrences all number
    0
    2
    0
    1
    Urticaria
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    1 / 41 (2.44%)
         occurrences all number
    0
    0
    0
    1
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Anxiety
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    1 / 38 (2.63%)
    3 / 41 (7.32%)
         occurrences all number
    0
    0
    1
    3
    Osteopenia
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    4 / 41 (9.76%)
         occurrences all number
    0
    0
    0
    4
    Pain in extremity
         subjects affected / exposed
    0 / 11 (0.00%)
    3 / 23 (13.04%)
    5 / 38 (13.16%)
    6 / 41 (14.63%)
         occurrences all number
    0
    3
    5
    6
    Back pain
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    2 / 38 (5.26%)
    1 / 41 (2.44%)
         occurrences all number
    0
    0
    2
    1
    Infections and infestations
    Ear Infection
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    2 / 38 (5.26%)
    4 / 41 (9.76%)
         occurrences all number
    0
    1
    2
    4
    Gastroenteritis
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    3 / 38 (7.89%)
    3 / 41 (7.32%)
         occurrences all number
    0
    0
    3
    3
    Influenza
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    3 / 38 (7.89%)
    6 / 41 (14.63%)
         occurrences all number
    0
    1
    3
    6
    Nasopharyngitis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 23 (4.35%)
    7 / 38 (18.42%)
    12 / 41 (29.27%)
         occurrences all number
    0
    1
    7
    12
    Otitis media
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    3 / 38 (7.89%)
    2 / 41 (4.88%)
         occurrences all number
    0
    0
    3
    2
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 23 (8.70%)
    2 / 38 (5.26%)
    3 / 41 (7.32%)
         occurrences all number
    1
    2
    2
    3
    Sinusitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    1 / 38 (2.63%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Body tinea
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 11 (0.00%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    2 / 41 (4.88%)
         occurrences all number
    0
    0
    0
    2
    Hand-foot-and-mouth disease
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 23 (0.00%)
    0 / 38 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    1
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Oct 2018
    1. To update Section 1 Introduction with safety results from Phase II studies in DMD boys (VBP15-002 and VBP15-003) 2. To update Section 1.5 Overall Benefit/Risk 3. To update Section 13 References 4. To clarify that dose may be escalated incrementally to 6.0 mg/kg/day 5. To clarify the circumstances under which study drug dose should be interrupted, de-escalated, or discontinued 6. To allow dose de-escalation from 6.0 mg/kg/day to 2.0 mg/kg/day to be followed by dose escalation to 4.0 mg/kg/day,balancing efficacy and safety concerns, in the opinion of the Investigator 7. To prohibit use of Exondys 51, Translarna, and other medications indicated for treatment of DMD during the study 8. To add details of Data and Safety Monitoring Board (DSMB) responsibilities 9. To add collection of 8.5 mL of blood at the Month 24 Visit for deoxyribonucleic acid (DNA) testing for established genetic modifiers of DMD 10. To add spine x-ray for detection of fracture at the Month 24 Visit 11. To add hand x-ray for assessment of bone age at the Month 24 Visit 12. To remove the designation of BMI z-score as the primary safety outcome 13. To update the primary efficacy endpoint 14. To remove comparison of vamorolone with prednisone-treated historical control data for serum PD biomarkers of safety 15. To add comparison of vamorolone to prednisone- and deflazacort-treated historical control data for bone age, spine fracture, and height z-score, and comparison to deflazacort-treated historical control data for BMI z-score 16. To update the statistical methodology and composition of the control populations 17. To clarify that collection of the Pediatric Outcomes Data Collection Instrument (PODCI) does not need to be repeated at the VBP15-LTE Baseline Visit if the Baseline Visit occurs ≤ 28 days after the date of the VBP15-003 Week 24 Visit 18. To clarify the PD biomarkers to be evaluated

    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

    Online references

    http://www.ncbi.nlm.nih.gov/pubmed/32956407
    http://www.ncbi.nlm.nih.gov/pubmed/35076703
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