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    Clinical Trial Results:
    A double blind, randomized, placebo-controlled study to evaluate safety, tolerability, efficacy and preliminary dose-response of BAF312 in patients with active dermatomyositis

    Summary
    EudraCT number
    2013-001799-39
    Trial protocol
    HU   CZ   PL   BE  
    Global end of trial date
    17 Feb 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    01 Dec 2016
    First version publication date
    01 Dec 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CBAF312X2206
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02029274
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    17 Feb 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Feb 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the efficacy of different doses of BAF312 after 6 months of treatment in active DM patients as assessed by manual muscle testing using the MMT-24 scoring system
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    15 Nov 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 10
    Country: Number of subjects enrolled
    Czech Republic: 4
    Country: Number of subjects enrolled
    Japan: 3
    Worldwide total number of subjects
    17
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    17
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered to all randomized at the dose of 2 mg daily .

    Pre-assignment
    Screening details
    Participants were randomized to each treatment group in a1:1:1:1 ratio.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    BAF312 0.5mg
    Arm description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Siponimod
    Investigational medicinal product code
    BAF312
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Arm title
    BAF312 2mg
    Arm description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Siponimod
    Investigational medicinal product code
    BAF312
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Arm title
    BAF312 10 mg
    Arm description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Siponimod
    Investigational medicinal product code
    BAF312
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Arm title
    Placebo
    Arm description
    During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Number of subjects in period 1
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo
    Started
    5
    4
    4
    4
    Completed
    4
    4
    2
    2
    Not completed
    1
    0
    2
    2
         Adverse event, non-fatal
    1
    -
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    BAF312 0.5mg
    Reporting group description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group title
    BAF312 2mg
    Reporting group description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group title
    BAF312 10 mg
    Reporting group description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group title
    Placebo
    Reporting group description
    During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group values
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo Total
    Number of subjects
    5 4 4 4 17
    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)
    0 0 0 0 0
        Adolescents (12-17 years)
    0 0 0 0 0
        Adults (18-64 years)
    5 4 4 4 17
        From 65-84 years
    0 0 0 0 0
        85 years and over
    0 0 0 0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    51.8 ± 16.72 44 ± 6.98 51.8 ± 4.79 48 ± 10.61 -
    Gender, Male/Female
    Units: Subjects
        Female
    4 2 4 3 13
        Male
    1 2 0 1 4

    End points

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    End points reporting groups
    Reporting group title
    BAF312 0.5mg
    Reporting group description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group title
    BAF312 2mg
    Reporting group description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group title
    BAF312 10 mg
    Reporting group description
    During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Reporting group title
    Placebo
    Reporting group description
    During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.

    Primary: Change from baseline in Manual Muscle Testing - 24 muscles (MMT-24) score

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    End point title
    Change from baseline in Manual Muscle Testing - 24 muscles (MMT-24) score
    End point description
    Each muscles tested was evaluated on a 0 - 10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. The total MMT24 score ranged from 0 - 240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.
    End point type
    Primary
    End point timeframe
    6 months
    End point values
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo
    Number of subjects analysed
    3
    4
    3
    3
    Units: score on a scale
        least squares mean (standard error)
    28.286 ± 8.1539
    12.367 ± 7.0967
    14.026 ± 8.1541
    27.735 ± 8.2175
    Statistical analysis title
    Change from baseline in MMT-24 score
    Comparison groups
    Placebo v BAF312 0.5mg
    Number of subjects included in analysis
    6
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.9621
    Method
    Repeated measures analysis
    Parameter type
    Mean difference (net)
    Point estimate
    0.551
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -22.447
         upper limit
    23.549
    Variability estimate
    Standard error of the mean
    Dispersion value
    11.5519
    Statistical analysis title
    Change from baseline in MMT-24 score
    Comparison groups
    BAF312 2mg v Placebo
    Number of subjects included in analysis
    7
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.1637
    Method
    Repeated measures analysis
    Parameter type
    Mean difference (net)
    Point estimate
    -15.368
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -37.128
         upper limit
    6.391
    Variability estimate
    Standard error of the mean
    Dispersion value
    10.9297
    Statistical analysis title
    Change from baseline in MMT-24 score
    Comparison groups
    Placebo v BAF312 10 mg
    Number of subjects included in analysis
    6
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.2409
    Method
    Repeated measures
    Parameter type
    Mean difference (net)
    Point estimate
    -13.709
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -36.806
         upper limit
    9.388
    Variability estimate
    Standard error of the mean
    Dispersion value
    11.6016

    Secondary: BAF312 plasma concentration

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    End point title
    BAF312 plasma concentration
    End point description
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo
    Number of subjects analysed
    0 [1]
    0 [2]
    0 [3]
    0 [4]
    Units: ng/mL
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    ±
    Notes
    [1] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    [2] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    [3] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    [4] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    No statistical analyses for this end point

    Secondary: Peripheral blood lymphocyte counts

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    End point title
    Peripheral blood lymphocyte counts
    End point description
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo
    Number of subjects analysed
    0 [5]
    0 [6]
    0 [7]
    0 [8]
    Units: Percent
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    ±
    Notes
    [5] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    [6] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    [7] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    [8] - Study was terminated prematurely due to futility. Primary outcome disclosed only.
    No statistical analyses for this end point

    Secondary: Change from baseline in Manual Muscle Testing - 24 muscles (MMT-24) score

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    End point title
    Change from baseline in Manual Muscle Testing - 24 muscles (MMT-24) score
    End point description
    End point type
    Secondary
    End point timeframe
    3 months
    End point values
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo
    Number of subjects analysed
    0 [9]
    0 [10]
    0 [11]
    0 [12]
    Units: score on a scale
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    ±
    Notes
    [9] - The study was prematurely terminated based on the results of an interim analysis.
    [10] - The study was prematurely terminated based on the results of an interim analysis.
    [11] - The study was prematurely terminated based on the results of an interim analysis.
    [12] - The study was prematurely terminated based on the results of an interim analysis.
    No statistical analyses for this end point

    Secondary: 6 Minutes Walking Distance (6-MWD) test

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    End point title
    6 Minutes Walking Distance (6-MWD) test
    End point description
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    BAF312 0.5mg BAF312 2mg BAF312 10 mg Placebo
    Number of subjects analysed
    0 [13]
    0 [14]
    0 [15]
    0 [16]
    Units: meters
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    ±
    Notes
    [13] - The study was prematurely terminated based on the results of an interim analysis.
    [14] - The study was prematurely terminated based on the results of an interim analysis.
    [15] - The study was prematurely terminated based on the results of an interim analysis.
    [16] - The study was prematurely terminated based on the results of an interim analysis.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Period 1 Placebo
    Reporting group description
    Period 1 Placebo

    Reporting group title
    Period 1 BAF312 0.5 mg/day
    Reporting group description
    Period 1 BAF312 0.5 mg/day

    Reporting group title
    Period 1 BAF312 2 mg/day
    Reporting group description
    Period 1 BAF312 2 mg/day

    Reporting group title
    Period 1 BAF312 10 mg/day
    Reporting group description
    Period 1 BAF312 10 mg/day

    Reporting group title
    Period 2 Placebo /BAF312 2 mg/day
    Reporting group description
    Period 2 Placebo /BAF312 2 mg/day

    Reporting group title
    Period 2 BAF312 0.5 mg/day/BAF312 2 mg/day
    Reporting group description
    Period 2 BAF312 0.5 mg/day/BAF312 2 mg/day

    Reporting group title
    Period 2 BAF312 2 mg/day/BAF312 2 mg/day
    Reporting group description
    Period 2 BAF312 2 mg/day/BAF312 2 mg/day

    Reporting group title
    Period 2 BAF312 10 mg/day/BAF312 2 mg/day
    Reporting group description
    Period 2 BAF312 10 mg/day/BAF312 2 mg/day

    Serious adverse events
    Period 1 Placebo Period 1 BAF312 0.5 mg/day Period 1 BAF312 2 mg/day Period 1 BAF312 10 mg/day Period 2 Placebo /BAF312 2 mg/day Period 2 BAF312 0.5 mg/day/BAF312 2 mg/day Period 2 BAF312 2 mg/day/BAF312 2 mg/day Period 2 BAF312 10 mg/day/BAF312 2 mg/day
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    1 / 4 (25.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Laceration
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Procedural pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    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
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    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
    0 / 0
    0 / 0
    Nervous system disorders
    Subarachnoid haemorrhage
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Syncope
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Dermatomyositis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    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
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    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
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Period 1 Placebo Period 1 BAF312 0.5 mg/day Period 1 BAF312 2 mg/day Period 1 BAF312 10 mg/day Period 2 Placebo /BAF312 2 mg/day Period 2 BAF312 0.5 mg/day/BAF312 2 mg/day Period 2 BAF312 2 mg/day/BAF312 2 mg/day Period 2 BAF312 10 mg/day/BAF312 2 mg/day
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
    4 / 4 (100.00%)
    4 / 4 (100.00%)
    2 / 5 (40.00%)
    1 / 4 (25.00%)
    4 / 4 (100.00%)
    2 / 4 (50.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Surgical and medical procedures
    Internal limiting membrane peeling
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Fibroadenoma of breast
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    2 / 4 (50.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    0
    0
    Fatigue
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    2 / 4 (50.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    0
    0
    0
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Oedema peripheral
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    1
    0
    Pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Peripheral swelling
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Psychiatric disorders
    Confusional state
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Post procedural inflammation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Investigations
    Carbon monoxide diffusing capacity decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Lymphocyte count decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    1
    Pulmonary function test abnormal
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Allergic sinusitis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    0
    Cough
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Nasal congestion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Productive cough
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Pulmonary congestion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Pulmonary hypertension
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Nervous system disorders
    Dizziness postural
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    Exertional headache
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Headache
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    2 / 4 (50.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    2 / 4 (50.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    3
    0
    0
    2
    1
    Migraine
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    1
    0
    Somnolence
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Syncope
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Transient global amnesia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    0
    Tremor
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Eye disorders
    Blepharospasm
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Chorioretinal atrophy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Conjunctival haemorrhage
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Eye swelling
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Eyelid oedema
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    0
    Retinal vein occlusion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Vitreous detachment
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Vitreous floaters
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Vertigo
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Diarrhoea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    1
    0
    Gingival recession
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    Nausea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Dermatitis atopic
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    0
    Ecchymosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    2
    Eczema
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    0
    0
    Generalised erythema
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    Pruritus
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Pruritus generalised
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    Rash
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    0
    Skin fissures
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    0
    0
    Muscle spasms
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    2 / 4 (50.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    4
    0
    0
    0
    0
    Muscular weakness
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    2 / 4 (50.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    0
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Musculoskeletal pain
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Osteonecrosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Pain in extremity
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    2
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Cellulitis orbital
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Dacryocystitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    Herpes simplex
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Herpes zoster
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Influenza
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    3 / 4 (75.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    6
    0
    0
    0
    0
    0
    Oral candidiasis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Pharyngitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Skin infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    0
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    1 / 4 (25.00%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    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
    16 Dec 2013
    The reason for amending the protocol was the expansion of the study to other countries (Japan) where the MCT system was not approved yet. The cardiac monitoring was performed with a Holter ECG instead of the device planned in the initial sites. The technical properties of the Holter device require 4 additional visits to be added to the schedule, in order to apply the Holter device. Furthermore, typos were corrected and clarifications added.
    15 Apr 2014
    The reason for amending the protocol was the addition of recent findings in a mouse carcinogenicity study. The additions made to the protocol reflected the changes made in the Investigator’s Brochure version 11 and the patients’ ICF. A section of efficacy data in PM/DM was updated with final data of a completed PoC trial that had become available. The upper age limit was increased from 65 years to 70 years based on recent investigator feedback, to better accommodate the patient population in need for new therapy. Based on the large safety database with more than 1500 patients exposed to BAF312 in 15 completed and 8 ongoing trials (as of 5 March 2014), the risk-benefit profile of BAF312 was considered suitable for patients up to the age of 70. The study stopping rules were modified to allow for a full safety review prior to discontinuation of all patients in case of 2 patients experiencing a study drug related adverse event, as specified in the stopping rules. Considering the relatively long treatment duration of 48 weeks and 56 patients to be enrolled, chances were high to observe SAEs that could be judged as related because a relation could never be completely excluded. In such a situation it could be unethical to discontinue treatment immediately in all patients; in particular in those who were benefiting, at least until a full safety review had been conducted.
    01 Jul 2015
    The purpose of this amendment was to update the eligibility criteria, to adapt the protocol to newly available information, and to include a broader population that better reflects the patient population in need of new therapeutic options. Furthermore, typing errors were corrected, and clarifications were added.
    27 Jul 2015
    The purpose of this amendment was to update the contact data for the Translational Medicine Expert, and to correct inconsistencies between synopsis and protocol body. Furthermore, Figure 3-1 was updated regarding time windows.

    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.
    The study was terminated prematurely after an interim analysis for futility. The study did not provide any evidence for efficacy of BAF312 in dermatomyositis. There were no safety concerns.
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