Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

    Summary
    EudraCT number
    2017-002704-27
    Trial protocol
    BE   SE   NL   CZ   GR   ES   GB  
    Global end of trial date
    06 May 2022

    Results information
    Results version number
    v1(current)
    This version publication date
    15 Dec 2022
    First version publication date
    15 Dec 2022
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    VBP15-004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03439670
    WHO universal trial number (UTN)
    -
    Other trial identifiers
    FDA IND Number: 118942
    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
    06 May 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 May 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and 2. To evaluate the safety and tolerability of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg in ambulant boys ages 4 to <7 years with DMD.
    Protection of trial subjects
    The trial will be conducted in accordance with the International Conference on Harmonisation E6 Guideline for Good Clinical Practice; The United States FDA Code of Federal Regulations, Title 21 CFR Part 312, and the US Health Insurance Portability and Accountability Act of 1996. The Parent/guardian of each participant must consent in writing for participant to be enrolled.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    01 Mar 2018
    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
    Czechia: 8
    Country: Number of subjects enrolled
    Greece: 5
    Country: Number of subjects enrolled
    Netherlands: 7
    Country: Number of subjects enrolled
    Spain: 7
    Country: Number of subjects enrolled
    Sweden: 3
    Country: Number of subjects enrolled
    United Kingdom: 30
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Australia: 9
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Canada: 14
    Country: Number of subjects enrolled
    United States: 30
    Worldwide total number of subjects
    121
    EEA total number of subjects
    36
    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)
    121
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    The target population for this study was boys with DMD between 4 to <7 years who were corticosteroid naïve and ambulatory.

    Period 1
    Period 1 title
    Period 1
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Treatment Group 1
    Arm description
    Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    vamorolone 1.33% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 2.0 mg/kg were administered a vamorolone 1.33% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 2
    Arm description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    vamorolone 4.0% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 6.0 mg/kg group were administered a vamorolone 4.0% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 3
    Arm description
    Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    5 mg prednisone tablets
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Prednisone 0.75 mg/kg were administered orally as tablet(s). Prednisone was supplied as 5 mg tablets; the number of prednisone or matching placebo tablets that were administered was based on the subject’s weight at the previous visit. Following tablet administration, the subject drank approximately 50 mL (ie, approximately 2 ounces) of water.

    Investigational medicinal product name
    vamorolone 1.33% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 2.0 mg/kg were administered a vamorolone 1.33% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 4
    Arm description
    Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.
    Arm type
    Active comparator

    Investigational medicinal product name
    5 mg prednisone tablets
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Prednisone 0.75 mg/kg were administered orally as tablet(s). Prednisone was supplied as 5 mg tablets; the number of prednisone or matching placebo tablets that were administered was based on the subject’s weight at the previous visit. Following tablet administration, the subject drank approximately 50 mL (ie, approximately 2 ounces) of water.

    Investigational medicinal product name
    Placebo for vamorolone suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 5
    Arm description
    Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Placebo for vamorolone suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Investigational medicinal product name
    vamorolone 1.33% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 2.0 mg/kg were administered a vamorolone 1.33% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 6
    Arm description
    Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Placebo for vamorolone suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Investigational medicinal product name
    vamorolone 4.0% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 6.0 mg/kg group were administered a vamorolone 4.0% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Number of subjects in period 1
    Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
    Started
    30
    30
    15
    16
    15
    15
    Completed
    30
    30
    15
    16
    15
    15
    Period 2
    Period 2 title
    Period 2
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Treatment Group 1
    Arm description
    Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    vamorolone 1.33% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 2.0 mg/kg were administered a vamorolone 1.33% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 2
    Arm description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    vamorolone 4.0% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 6.0 mg/kg group were administered a vamorolone 4.0% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 3
    Arm description
    Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    vamorolone 1.33% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 2.0 mg/kg were administered a vamorolone 1.33% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Investigational medicinal product name
    5 mg prednisone tablets
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Prednisone 0.75 mg/kg were administered orally as tablet(s). Prednisone was supplied as 5 mg tablets; the number of prednisone or matching placebo tablets that were administered was based on the subject’s weight at the previous visit. Following tablet administration, the subject drank approximately 50 mL (ie, approximately 2 ounces) of water.

    Arm title
    Treatment Group 4
    Arm description
    Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    vamorolone 4.0% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 6.0 mg/kg group were administered a vamorolone 4.0% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 5
    Arm description
    Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Placebo for vamorolone suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Investigational medicinal product name
    vamorolone 1.33% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 2.0 mg/kg were administered a vamorolone 1.33% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Arm title
    Treatment Group 6
    Arm description
    Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Placebo for vamorolone suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Investigational medicinal product name
    vamorolone 4.0% wt/wt oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in the vamorolone 6.0 mg/kg group were administered a vamorolone 4.0% wt/wt oral suspension. The same volume of either vamorolone 2 or 6 mg/kg or its placebo were orally administered using a volumetric syringe with a breakfast that included at least 8 g of fat; the dose administered was based on the subject’s weight at the previous visit. Following administration of the study drug suspension, the volumetric syringe was filled once with water and the water was orally administered.

    Number of subjects in period 2
    Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
    Started
    30
    30
    15
    16
    15
    15
    Completed
    28
    26
    15
    15
    14
    14
    Not completed
    2
    4
    0
    1
    1
    1
         Consent withdrawn by subject
    2
    2
    -
    -
    -
    -
         Physician decision
    -
    1
    -
    -
    -
    -
         WITHDRAWN DUE TO GH DEFICIENCY
    -
    -
    -
    -
    -
    1
         Adverse event, non-fatal
    -
    1
    -
    1
    -
    -
         AMBIGUOUS VARICELLA ZOSTER IMMUNITY
    -
    -
    -
    -
    1
    -

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Treatment Group 1
    Reporting group description
    Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 2
    Reporting group description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 3
    Reporting group description
    Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 4
    Reporting group description
    Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 5
    Reporting group description
    Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 6
    Reporting group description
    Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.

    Reporting group values
    Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6 Total
    Number of subjects
    30 30 15 16 15 15 121
    Age categorical
    Units: Subjects
        In utero
    0 0 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0 0 0 0 0
        Newborns (0-27 days)
    0 0 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0 0 0 0 0
        Children (2-11 years)
    30 30 15 16 15 15 121
        Adolescents (12-17 years)
    0 0 0 0 0 0 0
        Adults (18-64 years)
    0 0 0 0 0 0 0
        From 65-84 years
    0 0 0 0 0 0 0
        85 years and over
    0 0 0 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    5.29 (4.1 to 7.0) 5.42 (4.1 to 6.8) 5.41 (4.0 to 6.9) 5.61 (4.5 to 7.0) 5.35 (4.3 to 6.5) 5.44 (4.1 to 7.0) -
    Gender categorical
    Units: Subjects
        Female
    0 0 0 0 0 0 0
        Male
    30 30 15 16 15 15 121
    Subject analysis sets

    Subject analysis set title
    Analysis Group 1
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Patients enrolled in Analysis Group 1 (placebo comparator group) will receive placebo for 24 weeks.

    Subject analysis set title
    Analysis Group 2
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks

    Subject analysis sets values
    Analysis Group 1 Analysis Group 2
    Number of subjects
    28
    27
    Age categorical
    Units: Subjects
        In utero
        Preterm newborn infants (gestational age < 37 wks)
        Newborns (0-27 days)
        Infants and toddlers (28 days-23 months)
        Children (2-11 years)
        Adolescents (12-17 years)
        Adults (18-64 years)
        From 65-84 years
        85 years and over
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    5.40 (4.1 to 7.0)
    5.42 (4.1 to 6.8)
    Gender categorical
    Units: Subjects
        Female
        Male

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Treatment Group 1
    Reporting group description
    Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 2
    Reporting group description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 3
    Reporting group description
    Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 4
    Reporting group description
    Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 5
    Reporting group description
    Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 6
    Reporting group description
    Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.
    Reporting group title
    Treatment Group 1
    Reporting group description
    Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 2
    Reporting group description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 3
    Reporting group description
    Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 4
    Reporting group description
    Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 5
    Reporting group description
    Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 6
    Reporting group description
    Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.

    Subject analysis set title
    Analysis Group 1
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Patients enrolled in Analysis Group 1 (placebo comparator group) will receive placebo for 24 weeks.

    Subject analysis set title
    Analysis Group 2
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks

    Primary: TTSTAND Velocity Change from Baseline to Week 24: Vamorolone 6 mg/kg versus Placebo

    Close Top of page
    End point title
    TTSTAND Velocity Change from Baseline to Week 24: Vamorolone 6 mg/kg versus Placebo
    End point description
    End point type
    Primary
    End point timeframe
    Week 24
    End point values
    Analysis Group 1 Analysis Group 2
    Number of subjects analysed
    28
    27
    Units: rises/second
        arithmetic mean (standard deviation)
    -0.007 ( 0.0628 )
    0.054 ( 0.0666 )
    Statistical analysis title
    Primary endpoint analysis
    Statistical analysis description
    For EMA, change from baseline in TTSTAND velocity was compared using the REML-based MMRM and multiple imputation assuming missing not at random (MNAR).
    Comparison groups
    Analysis Group 2 v Analysis Group 1
    Number of subjects included in analysis
    55
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0018
    Method
    Mixed models analysis
    Confidence interval

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    48 weeks
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    21.1
    Reporting groups
    Reporting group title
    Treatment Group 1
    Reporting group description
    Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 2
    Reporting group description
    Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 3
    Reporting group description
    Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 4
    Reporting group description
    Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 5
    Reporting group description
    Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.

    Reporting group title
    Treatment Group 6
    Reporting group description
    Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.

    Serious adverse events
    Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 28 (7.14%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Gastroenteritis viral/viral gasteroenteritis
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         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: 5%
    Non-serious adverse events
    Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    26 / 28 (92.86%)
    26 / 28 (92.86%)
    15 / 15 (100.00%)
    13 / 15 (86.67%)
    12 / 14 (85.71%)
    14 / 14 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Vascular disorders
    Hot flush
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Peripheral coldness
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    7 / 28 (25.00%)
    3 / 28 (10.71%)
    3 / 15 (20.00%)
    1 / 15 (6.67%)
    3 / 14 (21.43%)
    3 / 14 (21.43%)
         occurrences all number
    8
    3
    5
    1
    3
    6
    Asthenia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    2 / 15 (13.33%)
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    2
    2
    0
    0
    Impaired healing
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Thirst
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    5 / 28 (17.86%)
    3 / 28 (10.71%)
    2 / 15 (13.33%)
    4 / 15 (26.67%)
    0 / 14 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    8
    3
    2
    4
    0
    3
    Nasal congestion
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 28 (7.14%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    2
    1
    2
    0
    0
    Rhinorrhoea
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    2
    1
    0
    2
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    0
    1
    1
    0
    2
    Psychiatric disorders
    Abnormal behaviour
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 28 (3.57%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    2
    1
    2
    0
    2
    1
    Aggression
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    2
    1
    0
    3
    1
    0
    Irritability
         subjects affected / exposed
    0 / 28 (0.00%)
    3 / 28 (10.71%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    3
    1
    0
    0
    1
    Sleep disorder
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    1
    0
    1
    0
    Anger
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Dysphemia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Emotional disorder
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Enuresis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Initial insomnia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Insomnia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Mood swings
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    2
    1
    0
    Oppositional defiant disorder
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Personality change
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Trichotillomania
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Epistaxis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    1
    1
    0
    Night sweats
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Investigations
    Blood uric acid increased
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    0
    0
    3
    0
    1
    Protein Urine Present
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    3 / 15 (20.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    1
    3
    0
    0
    Weight increased
         subjects affected / exposed
    1 / 28 (3.57%)
    3 / 28 (10.71%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    1
    3
    1
    1
    1
    1
    Cortisol decreased
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    1
    0
    0
    0
    1
    Bacterial test postive
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Blood pressure increased
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    1
    0
    1
    0
    Blood triglycerides increased
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Electrocardiogram abnormal
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Glycosylated haemoglobin increased
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Lipase decreased
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Urine analysis abnormal
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Weight decreased
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 28 (7.14%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    1
    2
    0
    0
    1
    3
    Contusion
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    1
    1
    0
    0
    0
    Fall
         subjects affected / exposed
    2 / 28 (7.14%)
    4 / 28 (14.29%)
    2 / 15 (13.33%)
    3 / 15 (20.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    2
    5
    3
    5
    0
    1
    Ligament strain
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    0
    0
    1
    0
    Back injury
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Muscle strain
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Vaccination complication
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 28 (10.71%)
    2 / 28 (7.14%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    3 / 14 (21.43%)
         occurrences all number
    3
    2
    1
    1
    0
    7
    Psychomotor hyperactivity
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    1
    2
    0
    0
    Dizziness
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Poor quality sleep
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Seizure
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Syncope
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Blood and lymphatic system disorders
    Leukopenia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    2
    1
    3
    0
    0
    Tympanic membrane perforation
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    3 / 28 (10.71%)
    3 / 28 (10.71%)
    2 / 15 (13.33%)
    1 / 15 (6.67%)
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    3
    3
    3
    1
    3
    0
    Abdominal Pain upper
         subjects affected / exposed
    1 / 28 (3.57%)
    3 / 28 (10.71%)
    3 / 15 (20.00%)
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    2
    6
    3
    3
    1
    1
    Constipation
         subjects affected / exposed
    3 / 28 (10.71%)
    3 / 28 (10.71%)
    0 / 15 (0.00%)
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    5
    4
    0
    2
    1
    1
    Diarrhoea
         subjects affected / exposed
    3 / 28 (10.71%)
    5 / 28 (17.86%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    3
    7
    2
    1
    0
    1
    Vomiting
         subjects affected / exposed
    6 / 28 (21.43%)
    6 / 28 (21.43%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    3 / 14 (21.43%)
         occurrences all number
    8
    6
    1
    0
    0
    4
    Mouth Ulceration
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 28 (7.14%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Toothache
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    1
    0
    0
    0
    Diarrhoea haemorrhagic
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Flatulance
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Hepatobiliary disorders
    Hepatomegaly
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Hypertrichosis
         subjects affected / exposed
    1 / 28 (3.57%)
    3 / 28 (10.71%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    3
    1
    1
    0
    1
    Rash
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 28 (7.14%)
    2 / 15 (13.33%)
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    2 / 14 (14.29%)
         occurrences all number
    2
    2
    2
    2
    1
    3
    Eczema
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Perioral dermatitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    2
    0
    1
    0
    0
    Pruritis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    1
    0
    0
    0
    Dry skin
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Erythema
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    Rash macropapular
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Urticaria
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Viral rash
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    1
    1
    0
    0
    Proteinuria
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    4 / 28 (14.29%)
    9 / 28 (32.14%)
    4 / 15 (26.67%)
    4 / 15 (26.67%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    4
    9
    4
    4
    1
    1
    Growth hormone deficiency
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    2 / 28 (7.14%)
    3 / 28 (10.71%)
    4 / 15 (26.67%)
    4 / 15 (26.67%)
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    2
    3
    5
    4
    2
    0
    Back pain
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 28 (7.14%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    1
    3
    0
    1
    0
    1
    Athralgia
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    1
    0
    0
    0
    Muscle spasms
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    1
    0
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    0
    0
    0
    2
    Costrochondritis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Joint contracture
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Joint swelling
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Muscle atrophy
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    Muscular weakness
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Musculoskeletal pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Vertebral wedging
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Agitation
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    1
    1
    0
    0
    Infections and infestations
    Gasteroenteritis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    2
    0
    1
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 28 (7.14%)
    4 / 28 (14.29%)
    3 / 15 (20.00%)
    2 / 15 (13.33%)
    1 / 14 (7.14%)
    2 / 14 (14.29%)
         occurrences all number
    2
    8
    5
    8
    2
    5
    Upper respiratory tract infection
         subjects affected / exposed
    10 / 28 (35.71%)
    4 / 28 (14.29%)
    2 / 15 (13.33%)
    3 / 15 (20.00%)
    3 / 14 (21.43%)
    2 / 14 (14.29%)
         occurrences all number
    12
    7
    2
    7
    6
    5
    Rhinitis
         subjects affected / exposed
    2 / 28 (7.14%)
    4 / 28 (14.29%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    3
    5
    0
    0
    0
    3
    Enterobiasis
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 28 (7.14%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    2
    0
    0
    0
    0
    Conjunctivitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Ear Infection
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    1
    1
    0
    2
    1
    2
    Gastroenteritis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    2
    0
    1
    1
    Tonsilitis
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    0
    0
    2
    0
    Viral Infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    COVID-19
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Cystitis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Fungal Infection
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gastroenteritis viral/viral gasteroenteritis
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    3
    0
    1
    0
    0
    0
    Hand-foot-and-mouth disease
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Impetigo
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Influenza
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    1
    0
    2
    0
    Moluscum contagiosum
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Otitis media bacterial
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    4
    0
    2
    0
    0
    1
    Pneumonia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    Respiratory track infection viral
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Steptococcal infection
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Tooth infection
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 28 (7.14%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    2
    0
    1
    1
    0
    Vitamin D Deficiency
         subjects affected / exposed
    2 / 28 (7.14%)
    3 / 28 (10.71%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    5
    1
    2
    0
    0
    Decreased appetite
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Hypertriglyceridaemia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Overweight
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Polydipsia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    04 May 2018
    1. To update blood volumes collected for clinical laboratory testing and PD biomarker evaluation; 2. To clarify that the blood and urine samples collected at the Screening Visit for clinical laboratory testing do not need to be collected after the subject has fasted; 3. To clarify that vamorolone should be used with caution with any drug metabolized by CYP3A4; 4. To clarify storage and use of blood samples for future exploratory research studies; 5. To remove administration of the Ease of Study Medication Administration Assessment from Day 1 Visit assessments; 6. To change the region imaged by spine x-ray from T4-L4 to T4-L5; 7. To clarify endpoints and revise the statistical methodology; 8. To clarify the definition of time windows around study visits; 9. To clarify that fractures reported during the Screening Period using the Fracture Questionnaire will be recorded as Medical History; 10. To change the name of the Synacthen Test to the ACTH Stimulation Test and to clarify that Cosyntropin (also known as tetracosactide) is administered to stimulate cortisol response; 11. To clarify that the ACTH Stimulation Test will include insertion of a saline lock; 12. To clarify that ACTH Stimulation testing should be initiated as close to 8 AM local time as possible, and that the time 0 blood sample for cortisol measurement should be collected immediately prior to Cosyntropin administration; 13. To change the name of the Extremity Fracture Questionnaire to Fracture Questionnaire to more accurately reflect the nature of the fractures (vertebral and non-vertebral) to be collected; 14. To revise the definition of protocol deviation/violation; 15. To clarify that GLP studies were conducted in, or inspected by, a country that has implemented the Organisation for Economic Cooperation and Development (OECD) Mutual Acceptance of Data (MAD) system; 16. To update Section 1.5 Overall Benefit/Risk with clinical data; 17. To update version of the Investigator’s Brochure re
    05 Mar 2019
    1. To correct contact information for the Medical Monitor; 2. To update Section 1.3 Clinical Experience with results from the VBP15-002 and VBP15-003 clinical trials in DMD boys; 3. To update Section 1.5 Overall Benefit/Risk; 4. To revise exclusion criterion #7 regarding prior use of glucocorticoids; 5. To add an exclusion criterion excluding use of live attenuated vaccines within 14 days prior to first dose of study medication; 6. To exclude subjects who have siblings currently enrolled in, or intending to enroll in during the subject’s participation in this study, any vamorolone study or Expanded Access Program; 7. To delete use of the Child Behavior Checklist behavioral assessment tool; 8. To clarify that the PARS III assessment tool is a measure of behavior/neuropsychology, and the PODCI is a measure of physical functioning; 9. To specify that the PODCI results will be analyzed for vamorolone vs. placebo, and the PARS III results will be analyzed for vamorolone vs. prednisone; 17. To clarify that subjects who opt to continue treatment with vamorolone at study completion may be given access to vamorolone through an additional vamorolone study or general access program; 18. To remove the responsibility of the DSMB to review accumulating study efficacy data, in response to FDA feedback; 19. To clarify the circumstances under which additional subjects may be enrolled; 20. To clarify that an email with each subject randomization number will not be sent to the site investigator at the time of randomization; 21. To clarify the blinding status following the Week 24 analyses; 22. To add sensitivity analyses for missing data; 23. To add the 6MWT (comparison of vamorolone vs. prednisone) to the sequential testing procedure for the secondary efficacy endpoints; 24. To specify that statistical analyses will be performed using SAS® version 9.4 or later; 25. To define time windows around scheduled study visits; 26. To update the Schedule of Study Activities to clarify
    21 May 2019
    1. To revise Inclusion Criterion #7 to clarify that subjects with abnormal and clinically-significant vitamin D levels will not be excluded from randomization; 2. To revise Inclusion Criterion #8 to allow subjects who have had 2 doses of varicella vaccine prior to randomization, with or without serologic evidence of immunity, to be eligible for randomization to treatment; 3. To revise Sections 6.1 and 7.2.5 to reflect that varicella immunity may be demonstrated by a positive anti-varicella antibody titer or documentation of 2 doses of varicella vaccine; 4. To revise Section 5.6 to clarify instructions for study drug dose interruption; and 5. To correct typographical error in the Synopsis, Exclusion Criterion #7 to make consistent with Section 4.3, Exclusion Criterion #7.
    28 Aug 2020
    1. To revise one of the primary objectives of the study to compare the efficacy of vamorolone administered orally at a dose of 6.0 mg/kg/day vs. placebo over a 24-week treatment period; 2. To revise the primary efficacy endpoint to TTSTAND velocity, comparison of vamorolone 6.0 mg/kg/day vs. placebo in change from baseline to Week 24, to align with the primary objective; 3. To add an additional secondary objective of the study to compare the efficacy of vamorolone administered orally at a dose of 2.0 mg/kg/day vs. placebo over a 24-week treatment period; 4. To delete a secondary objective of the study comparing the efficacy of vamorolone 2.0 mg/kg/day vs. 6.0 mg/kg/day over 24 weeks; 5. To revise the list of safety endpoints to include linear growth velocity, and to clarify the endpoints for BMI z-score and ACTH Stimulation Test; 6. To revise the secondary efficacy endpoints for Treatment Period #1; 7. To add exploratory efficacy endpoints for Treatment Period #1; 8. To add comparison of each vamorolone group to the placebo group for PARS III; 9. To clarify Ease of Study Drug Administration exploratory endpoint; 10. To revise the methodology for sample size calculation, in consideration of the revised primary efficacy endpoint; 11. To add a Per Protocol Population for statistical analyses; 12. To revise the multiple testing procedures for the efficacy endpoints; 13. To revise the statistical methodology for efficacy and safety analyses; 14. To clarify the circumstances under which hospitalizations should be considered serious adverse events; 15. To add assessment of suicidality and abuse potential associated with treatment from examination of adverse event data; 16. To clarify that demographic and baseline characteristics summary tables will not be presented by age stratification;

    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
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA