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   44334   clinical trials with a EudraCT protocol, of which   7366   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:
    PROSPECTIVE, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED PHASE III STUDY EVALUATING EFFICACY AND SAFETY OF OCTAGAM 10% IN PATIENTS WITH DERMATOMYOSITIS

    Summary
    EudraCT number
    		 2016-002902-37
    Trial protocol
    		 CZ   DE   HU   NL   RO  
    Global end of trial date
    05 Nov 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 Nov 2020
    First version publication date
    20 Nov 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GAM10-08
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02728752
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND: 16925
    Sponsors
    Sponsor organisation name
    Octapharma Pharmazeutika Produktionsges.m.b.H.
    Sponsor organisation address
    Oberlaaer Strasse 235, Vienna, Austria, 1100
    Public contact
    Global Clinical Project Manager, Octapharma Pharmazeutika Produktionsges.m.b.H., 0043 1 61032 1168, clinical.department@octapharma.com
    Scientific contact
    Global Clinical Project Manager, Octapharma Pharmazeutika Produktionsges.m.b.H., 0043 1 61032 1168, clinical.department@octapharma.com
    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
    03 Jul 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Nov 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To provide confirmatory data on the beneficial effect of 2.0 g/kg of Octagam 10% given every 4 weeks compared with placebo in subjects with active DM based on the percentage of responders at Week 16.
    Protection of trial subjects
    This trial was conducted in accordance to the principles of ICH- GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the Declaration of Helsinki and national regulatory requirements. Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and risk factors associated with the investigational medicinal product. Study safety was assessed such as monitoring of AEs and SAEs, monitoring of safety lab results, concomitant medication and vital signs.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    27 Feb 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    Czech Republic: 12
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Hungary: 15
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    Romania: 2
    Country: Number of subjects enrolled
    Russian Federation: 21
    Country: Number of subjects enrolled
    Ukraine: 8
    Country: Number of subjects enrolled
    United States: 27
    Worldwide total number of subjects
    95
    EEA total number of subjects
    38
    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)
    75
    From 65 to 84 years
    20
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Patients with a diagnosis of definite or probable Dermatomyositis according to the Bohan and Peter criteria were were screened according to predefined in- and exclusion criteria.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Subjects received 4 infusions of placebo every 4 weeks during the blinded First Period (up to week 16). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period continued to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorated also during Octagam 10% treatment at 2 consecutive visits were droped-out after response assessment at Week 16 and did not enter the Extension Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received 4 infusions of placebo every 4 weeks during the blinded First Period (up to week 16). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to Octagam 10%.

    Arm title
    		 Octagam 10%
    Arm description
    		 Subjects received 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period continued receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration were droped-out after response assessment at Week 16 and did not enter the Extension Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Octagam 10%
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period continued receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration were droped-out after response assessment at Week 16 and did not enter the Extension Period.

    Number of subjects in period 1
    		Placebo Octagam 10%
    Started
    48
    47
    Completed
    35
    34
    Not completed
    13
    13
         Patient decision
    4
    4
         Adverse event, non-fatal
    7
    5
         other reason
    2
    3
         Administrative reason
    -
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received 4 infusions of placebo every 4 weeks during the blinded First Period (up to week 16). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period continued to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorated also during Octagam 10% treatment at 2 consecutive visits were droped-out after response assessment at Week 16 and did not enter the Extension Period.

    Reporting group title
    Octagam 10%
    Reporting group description
    		 Subjects received 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period continued receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration were droped-out after response assessment at Week 16 and did not enter the Extension Period.

    Reporting group values
    		 Placebo Octagam 10% Total
    Number of subjects
    		 48 47 95
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 51.35 (22 to 79) 54.04 (22 to 77) -
    Gender categorical
    Units: Subjects
        Female
    		 35 36 71
        Male
    		 13 11 24

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received 4 infusions of placebo every 4 weeks during the blinded First Period (up to week 16). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period continued to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorated also during Octagam 10% treatment at 2 consecutive visits were droped-out after response assessment at Week 16 and did not enter the Extension Period.

    Reporting group title
    Octagam 10%
    Reporting group description
    		 Subjects received 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period continued receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who were stable on 2.0 g/kg Octagam 10% could be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration were droped-out after response assessment at Week 16 and did not enter the Extension Period.

    Subject analysis set title
    Full Analysis Set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The full analysis set (FAS) is defined according to the intention-to-treat principle and consists of all randomized subjects. It is expected that the FAS will coincide with the safety set.

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The safety analysis set (SAF) consists of all subjects who received at least part of one infusion of Octagam or placebo.

    Subject analysis set title
    Time to Minimal Improvement Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Time to Minimal Improvement - From Start of First Treatment in Overall Period

    Subject analysis set title
    Time to Minimal Improvement Octagam 10%
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Time to Minimal Improvement - From Start of First Treatment in Overall Period

    Subject analysis set title
    Time to Moderate Improvement Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Time to Moderate Improvement - From Start of First Treatment in Overall Period

    Subject analysis set title
    Time to Moderate Improvement Octagam 10%
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Time to Moderate Improvement - From Start of First Treatment in Overall Period

    Subject analysis set title
    Time to Major Improvement Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Time to Major Improvement - From Start of First Treatment in Overall Period

    Subject analysis set title
    Time to Major Improvement Octagam 10%
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Time to Major Improvement - From Start of First Treatment in Overall Period

    Primary: Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS)

    		 Close Top of page
    End point title
    		 Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS)
    End point description
    Proportion of responders in the 2.0 g/kg Octagam 10% and placebo arms at Week 16 relative to baseline (Week 0). A responder being defined as a patient with an increase of ≥20 points on the Total Improvement Score (TIS, a scale from 0 to 100; 20-39 points being minimal improvement, 40-59 points being moderate improvement, and ≥60 points being major improvement Full Analysis Set : Octagam (N=47) Placebo (N=48)
    End point type
    Primary
    End point timeframe
    At week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    48
    47
    Units: Patients
    number (not applicable)
        Number of patients
    21
    37
        (%)
    43.75
    78.72
    Statistical analysis title
    		 Proportion of Responders
    Statistical analysis description
    The primary endpoint measure ‘response’ was assessed at Week 16 based on the TIS score. The proportion of responders within both treatment groups was compared by the Cochran-Mantel-Haenszel test, stratified by GDA at baseline (randomization stratum), using a two-sided alpha level of 0.05. The primary analysis was to be considered a success if the proportion of responders was significantly higher in the octagam 10% group compared to the placebo group.
    Comparison groups
    Placebo v Octagam 10%
    Number of subjects included in analysis
    95
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0008
    Method
    		 Cochran-Mantel-Haenszel
    Parameter type
    		 Difference in Response Rate
    Point estimate
    34.97
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 16.7
         upper limit
    		 53.24

    Secondary: Proportion of TIS Responders by Improvement Category at Week 16

    		 Close Top of page
    End point title
    		 Proportion of TIS Responders by Improvement Category at Week 16
    End point description
    The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement. Full Analysis Set : Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    16 weeks
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    48
    47
    Units: patients
    number (not applicable)
        Primary
    21
    37
        Primary (%)
    43.75
    78.72
        At least moderate improvement
    11
    32
        At least moderate improvement (%)
    22.92
    68.09
        At least major improvement
    4
    15
        At least major improvement (%)
    8.33
    31.91
    No statistical analyses for this end point

    Secondary: Proportion of TIS Responders by Improvement Category at Week 40

    		 Close Top of page
    End point title
    		 Proportion of TIS Responders by Improvement Category at Week 40
    End point description
    The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement. Forty-five patients in the octagam 10% group and 46 patients in the placebo group completed the First Period and entered the 24-week Extension Period during which all patients received open-label octagam 10% treatment. Full Analysis Set : Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    40 weeks
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    46
    45
    Units: Patients
    number (not applicable)
        At least minimal improvement
    32
    32
        At least minimal improvement (%)
    69.57
    71.11
        At least moderate improvement
    28
    26
        At least moderate improvement (%)
    60.87
    57.78
        At least major improvement
    14
    17
        At least major improvement (%)
    30.43
    37.78
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
    End point description
    The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron’s papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed. Full Analysis Set : Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    First 16 weeks
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
    arithmetic mean (standard deviation)
        Total Activity Score
    -1.16 ( 7.000 )
    -9.36 ( 10.542 )
        Total Damage Score
    -0.02 ( 0.771 )
    -0.67 ( 1.871 )
    No statistical analyses for this end point

    Secondary: Mean Change From End of First Period (Week 16) to End of Extension Period (Week 40) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

    		 Close Top of page
    End point title
    		 Mean Change From End of First Period (Week 16) to End of Extension Period (Week 40) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
    End point description
    The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron’s papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From week 16 to Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    33
    34
    Units: Score
    arithmetic mean (standard deviation)
        Total Activity Score
    -8.52 ( 11.344 )
    -1.79 ( 4.169 )
        Total Damage Score
    -0.24 ( 0.969 )
    0.26 ( 2.220 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: SF-36v2 Health Survey

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: SF-36v2 Health Survey
    End point description
    The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index. Different numbers of patients analyzed in each group due to patient discontinuation. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    43
    Units: Score
    arithmetic mean (standard deviation)
        Physical Functioning
    2.27 ( 4.598 )
    7.06 ( 8.220 )
        Role Physical
    1.88 ( 8.154 )
    4.49 ( 8.444 )
        Bodily Pain
    1.29 ( 8.089 )
    5.29 ( 9.632 )
        General Health
    3.20 ( 6.732 )
    5.46 ( 6.431 )
        Vitality
    4.22 ( 7.540 )
    6.63 ( 9.458 )
        Social Functioning
    2.56 ( 7.686 )
    4.66 ( 10.401 )
        Role Emotional
    0.65 ( 9.157 )
    3.24 ( 8.162 )
        Mental Health
    1.89 ( 7.340 )
    3.95 ( 7.024 )
        Physical Health Score
    2.39 ( 5.945 )
    6.27 ( 8.720 )
        Mental Health Score
    1.95 ( 7.580 )
    3.36 ( 7.399 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: SF-36v2 Health Survey

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: SF-36v2 Health Survey
    End point description
    The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: Score
    arithmetic mean (standard deviation)
        Physical Functioning
    6.65 ( 8.258 )
    8.77 ( 10.930 )
        Role Physical
    6.80 ( 9.945 )
    8.32 ( 10.870 )
        Bodily Pain
    6.09 ( 11.597 )
    6.22 ( 12.895 )
        General Health
    6.74 ( 6.808 )
    6.42 ( 10.170 )
        Vitality
    9.25 ( 9.742 )
    8.65 ( 11.385 )
        Social Functioning
    8.31 ( 11.272 )
    7.82 ( 12.537 )
        Role Emotional
    5.07 ( 11.118 )
    3.17 ( 12.580 )
        Mental Health
    7.92 ( 10.202 )
    6.31 ( 8.974 )
        Physical Health Score
    6.31 ( 8.598 )
    8.41 ( 11.858 )
        Mental Health Score
    7.39 ( 10.154 )
    4.83 ( 9.631 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Physician’s Global Disease Activity

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Physician’s Global Disease Activity
    End point description
    10 cm VAS assessing global disease activity from “No evidence of disease activity” to “Extremely active or severe disease activity”; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis). Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
        arithmetic mean (standard deviation)
    -0.60 ( 1.815 )
    -2.39 ( 1.987 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Physician’s Global Disease Activity

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Physician’s Global Disease Activity
    End point description
    10 cm VAS assessing global disease activity from “No evidence of disease activity” to “Extremely active or severe disease activity”; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis). Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: Score
        arithmetic mean (standard deviation)
    -2.93 ( 1.888 )
    -3.06 ( 1.817 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: Patient Global Disease Activity

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: Patient Global Disease Activity
    End point description
    Patient’s Global Disease Activity (10cm VAS assessing the overall activity of the patient’s disease today from “No evidence of disease activity” to “Extremely active or severe disease activity”, Disease Activity being active inflammation in the patient’s muscles, skin, joints, intestines, heart, lungs or other parts of the body, which can improve when treated with medicines). 0 is lowest score and 10 is highest score. Higher score associated with worse outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
        arithmetic mean (standard deviation)
    -1.11 ( 2.094 )
    -2.19 ( 2.276 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: Patient Global Disease Activity

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: Patient Global Disease Activity
    End point description
    Patient’s Global Disease Activity (10cm VAS assessing the overall activity of the patient’s disease today from “No evidence of disease activity” to “Extremely active or severe disease activity”, Disease Activity being active inflammation in the patient’s muscles, skin, joints, intestines, heart, lungs or other parts of the body, which can improve when treated with medicines). 0 is lowest score and 10 is highest score. Higher score associated with worse outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40.
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    33
    Units: Score
        arithmetic mean (standard deviation)
    -2.77 ( 2.133 )
    -2.71 ( 2.651 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: MMT-8

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: MMT-8
    End point description
    Manual Muscle Testing - MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 – 150]. Higher score associated with better outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
        arithmetic mean (standard deviation)
    3.21 ( 9.390 )
    14.38 ( 14.581 )
    No statistical analyses for this end point

    Secondary: Measure Title Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: MMT-8

    		 Close Top of page
    End point title
    		 Measure Title Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: MMT-8
    End point description
    Manual Muscle Testing - MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 – 150]. Higher score associated with better outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    Time Frame From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    33
    Units: Score
        arithmetic mean (standard deviation)
    12.00 ( 7.491 )
    20.09 ( 14.486 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: Health Assessment Questionnaire

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: Health Assessment Questionnaire
    End point description
    Health Assessment Questionnaire (HAQ); a generic rather than a disease-specific instrument; comprised of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8). Assessment completed by patients. Lowest score 0 highest score 24. Higher score associated with worse outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
        arithmetic mean (standard deviation)
    -0.16 ( 0.366 )
    -0.56 ( 0.590 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: Health Assessment Questionnaire

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: Health Assessment Questionnaire
    End point description
    Health Assessment Questionnaire (HAQ); a generic rather than a disease-specific instrument; comprised of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8). Assessment completed by patients. Lowest score 0 highest score 24. Higher score associated with worse outcome. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    34
    33
    Units: Score
        arithmetic mean (standard deviation)
    -0.54 ( 0.524 )
    -0.66 ( 0.805 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Alanine Aminotransferase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Alanine Aminotransferase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    42
    Units: U/L
        arithmetic mean (standard deviation)
    -4.47 ( 29.796 )
    -8.52 ( 18.474 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Alanine Aminotransferase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Alanine Aminotransferase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    33
    Units: U/L
        arithmetic mean (standard deviation)
    -4.06 ( 14.787 )
    -7.15 ( 18.480 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Aspartate Aminotransferase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Aspartate Aminotransferase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    44
    Units: U/L
        arithmetic mean (standard deviation)
    -3.07 ( 31.317 )
    -7.82 ( 26.139 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Aspartate Aminotransferase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Aspartate Aminotransferase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: U/L
        arithmetic mean (standard deviation)
    -2.20 ( 16.421 )
    -7.76 ( 26.431 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Lactate Dehydrogenase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Lactate Dehydrogenase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: U/L
        arithmetic mean (standard deviation)
    -19.79 ( 142.012 )
    -11.04 ( 165.252 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Lactate Dehydrogenase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Lactate Dehydrogenase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40.
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: U/L
        arithmetic mean (standard deviation)
    -33.43 ( 63.651 )
    -59.21 ( 92.801 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Aldolase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Aldolase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    41
    42
    Units: U/L
        arithmetic mean (standard deviation)
    -2.51 ( 12.077 )
    -0.48 ( 7.743 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Aldolase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Aldolase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    32
    Units: U/L
        arithmetic mean (standard deviation)
    -0.59 ( 7.839 )
    -1.48 ( 3.644 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Creatine Kinase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in Enzymes (Creatine Kinase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    44
    Units: U/L
        arithmetic mean (standard deviation)
    -352.79 ( 2141.534 )
    -169.20 ( 434.224 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Creatine Kinase)

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in Enzymes (Creatine Kinase)
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial till Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: U/L
        arithmetic mean (standard deviation)
    -56.54 ( 556.928 )
    -169.38 ( 449.210 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: Extra-muscular Activity

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to End of First Period (Week 16) in: Extra-muscular Activity
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial until Week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
        arithmetic mean (standard deviation)
    -0.94 ( 2.287 )
    -2.18 ( 2.134 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: Extra-muscular Activity

    		 Close Top of page
    End point title
    		 Mean Change From Baseline (Week 0) to Extension Period (Week 40) in: Extra-muscular Activity
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    From start of the trial until Week 40
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: Score
        arithmetic mean (standard deviation)
    -2.64 ( 2.092 )
    -2.67 ( 2.061 )
    No statistical analyses for this end point

    Secondary: Mean TIS From Baseline (Week 0) to End of First Period (Week 16)

    		 Close Top of page
    End point title
    		 Mean TIS From Baseline (Week 0) to End of First Period (Week 16)
    End point description
    The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    Up to 16 weeks
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    43
    45
    Units: Score
        arithmetic mean (standard deviation)
    21.57 ( 20.185 )
    48.44 ( 24.444 )
    No statistical analyses for this end point

    Secondary: Mean TIS From Baseline (Week 0) to End of Extension Period (Week 40)

    		 Close Top of page
    End point title
    		 Mean TIS From Baseline (Week 0) to End of Extension Period (Week 40)
    End point description
    The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement. Full Analysis Set : Octagam (N=47) Placebo (N=48) Different numbers of patients analyzed in each group due to patient discontinuation.
    End point type
    Secondary
    End point timeframe
    Up to 40 weeks
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    35
    34
    Units: Score
        arithmetic mean (standard deviation)
    51.07 ( 18.314 )
    55.44 ( 21.712 )
    No statistical analyses for this end point

    Secondary: Time to Minimal Improvement in TIS

    		 Close Top of page
    End point title
    		 Time to Minimal Improvement in TIS
    End point description
    When interpreting these time to event evaluations, one has to keep in mind that eventually all patients were treated with octagam 10%, so that differences in time to response for the Overall Period reflect this delayed start of treatment rather than the true difference between treatment with octagam 10% and placebo. Full Analysis Set: Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    Up to 40 weeks
    End point values
    		 Time to Minimal Improvement Placebo Time to Minimal Improvement Octagam 10%
    Number of subjects analysed
    41
    47
    Units: days
        number (not applicable)
    114
    35
    No statistical analyses for this end point

    Secondary: Time to Moderate Improvement in TIS

    		 Close Top of page
    End point title
    		 Time to Moderate Improvement in TIS
    End point description
    When interpreting these time to event evaluations, one has to keep in mind that eventually all patients were treated with octagam 10%, so that differences in time to response for the Overall Period reflect this delayed start of treatment rather than the true difference between treatment with octagam 10% and placebo. Full Analysis Set: Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    up to 40 weeks
    End point values
    		 Time to Moderate Improvement Placebo Time to Moderate Improvement Octagam 10%
    Number of subjects analysed
    36
    39
    Units: days
        number (not applicable)
    197
    85
    No statistical analyses for this end point

    Secondary: Time to Confirmed Deterioration in the First Period

    		 Close Top of page
    End point title
    		 Time to Confirmed Deterioration in the First Period
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    Up to week 16
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    48
    47
    Units: days
    number (not applicable)
        Number of patients analyzed
    3
    0
        Time
    117
    0
    No statistical analyses for this end point

    Secondary: Time to Confirmed Deterioration Overall

    		 Close Top of page
    End point title
    		 Time to Confirmed Deterioration Overall
    End point description
    Full Analysis Set : Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    Up to 40 weeks
    End point values
    		 Placebo Octagam 10%
    Number of subjects analysed
    48 [1]
    47 [2]
    Units: days
    number (not applicable)
        Number of patients analyzed
    3
    1
        Time
    0
    0
    Notes
    [1] - No result due to limited sample size
    [2] - No result due to limited sample size
    No statistical analyses for this end point

    Secondary: Time to Major Improvement in TIS

    		 Close Top of page
    End point title
    		 Time to Major Improvement in TIS
    End point description
    When interpreting these time to event evaluations, one has to keep in mind that eventually all patients were treated with octagam 10%, so that differences in time to response for the Overall Period reflect this delayed start of treatment rather than the true difference between treatment with octagam 10% and placebo. Full Analysis Set: Octagam (N=47) Placebo (N=48)
    End point type
    Secondary
    End point timeframe
    Up to 40 weeks
    End point values
    		 Time to Major Improvement Placebo Time to Major Improvement Octagam 10%
    Number of subjects analysed
    20
    25
    Units: days
        number (not applicable)
    283
    283
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    40 weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    First Period Placebo
    Reporting group description
    		 -

    Reporting group title
    Overall Period Octagam
    Reporting group description
    		 -

    Serious adverse events
    		 First Period Placebo Overall Period Octagam
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 48 (4.17%)
    14 / 95 (14.74%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Squamous cell carcinoma
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypertension
         subjects affected / exposed
    1 / 48 (2.08%)
    0 / 95 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure congestive
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sinus tachycardia
         subjects affected / exposed
    1 / 48 (2.08%)
    0 / 95 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ventricular extrasystoles
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebral infarction
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cerebrovascular accident
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypoaesthesia
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Loss of consciousness
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Condition aggravated
         subjects affected / exposed
    0 / 48 (0.00%)
    2 / 95 (2.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Dyspnoea
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 48 (0.00%)
    3 / 95 (3.16%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Atypical pneumonia
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Escherichia bacteraemia
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 48 (0.00%)
    1 / 95 (1.05%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    0 / 48 (0.00%)
    2 / 95 (2.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tropical spastic paresis
         subjects affected / exposed
    1 / 48 (2.08%)
    0 / 95 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 First Period Placebo Overall Period Octagam
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    28 / 48 (58.33%)
    83 / 95 (87.37%)
    Investigations
    Blood pressure increased
         subjects affected / exposed
    3 / 48 (6.25%)
    4 / 95 (4.21%)
         occurrences all number
    4
    8
    Body temperature increased
         subjects affected / exposed
    0 / 48 (0.00%)
    5 / 95 (5.26%)
         occurrences all number
    0
    8
    Coombs test positive
         subjects affected / exposed
    0 / 48 (0.00%)
    5 / 95 (5.26%)
         occurrences all number
    0
    5
    Vascular disorders
    Hypertension
         subjects affected / exposed
    3 / 48 (6.25%)
    5 / 95 (5.26%)
         occurrences all number
    3
    6
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    2 / 48 (4.17%)
    5 / 95 (5.26%)
         occurrences all number
    2
    6
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 48 (8.33%)
    46 / 95 (48.42%)
         occurrences all number
    10
    102
    Dizziness
         subjects affected / exposed
    3 / 48 (6.25%)
    5 / 95 (5.26%)
         occurrences all number
    3
    6
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 48 (6.25%)
    17 / 95 (17.89%)
         occurrences all number
    3
    26
    Condition aggravated
         subjects affected / exposed
    9 / 48 (18.75%)
    9 / 95 (9.47%)
         occurrences all number
    22
    13
    Fatigue
         subjects affected / exposed
    4 / 48 (8.33%)
    5 / 95 (5.26%)
         occurrences all number
    4
    6
    Chills
         subjects affected / exposed
    1 / 48 (2.08%)
    7 / 95 (7.37%)
         occurrences all number
    2
    12
    Chest pain
         subjects affected / exposed
    3 / 48 (6.25%)
    3 / 95 (3.16%)
         occurrences all number
    3
    3
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    2 / 48 (4.17%)
    18 / 95 (18.95%)
         occurrences all number
    2
    30
    Vomiting
         subjects affected / exposed
    0 / 48 (0.00%)
    9 / 95 (9.47%)
         occurrences all number
    0
    12
    Constipation
         subjects affected / exposed
    0 / 48 (0.00%)
    5 / 95 (5.26%)
         occurrences all number
    0
    5
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    1 / 48 (2.08%)
    5 / 95 (5.26%)
         occurrences all number
    1
    5
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 48 (2.08%)
    6 / 95 (6.32%)
         occurrences all number
    1
    6
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 48 (8.33%)
    7 / 95 (7.37%)
         occurrences all number
    5
    8
    Myalgia
         subjects affected / exposed
    0 / 48 (0.00%)
    9 / 95 (9.47%)
         occurrences all number
    0
    12
    Pain in extremity
         subjects affected / exposed
    0 / 48 (0.00%)
    6 / 95 (6.32%)
         occurrences all number
    0
    9
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 48 (2.08%)
    6 / 95 (6.32%)
         occurrences all number
    1
    8

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Jul 2017
    Amendment 3: -Following a recommendation of the Steering Committee inclusion of patients with Dermatomyositis who are under no current treatment due to intolerance and/or non-responsiveness to DM-related medication has been added -upon recommendation by the Steering Committee exclusion criterion was amended to shorten the waiting period for patients after excision of basal or squamous cell skin cancer -Following a recommendation by the Steering Committee Subjects diagnosed with Juvenile Dermatomyositis were excluded from the study. - further specification of some exclusion criteria -Maximally allowed stable dose as concomitant therapy for Hydroxychloroquine was re-defined -in accoedance with the Steering Committee the definition of “Confirmed deterioration” was updated -As requested by the study’s Independent Adjudication Committee Thyroid-stimulating hormone (TSH) was added to the clinical chemistry test panel - Details on breaking the blind have been udated upon request of the Czech Regulatory Authority
    18 Jul 2018
    Amendment 4: -The maximum infusion rate has been reduced to 0.04 mL/kg/min upon request of FDA -Duration of infusion cycles for extension period changed from 2 days to 2-5 days -Upon FDA request a new stopping rule was implemented for TEEs observed on or after the cut-off date of 04-Jul-2018 (since introduction of reduced maximum infusion rate of 0.04 mL/kg/min) -Thromboembolic events will be reported as SAE (medical occurrence that at any dose is another important medical event) - Exclusion criterion with reference to forbidden medication has been added for clarification -Time window defined for Direct Coombs’ test taken prior infusion cycle has been extended for administrative reason
    05 Nov 2018
    Amendment 6: -Exclusion criterion was extended so that patients with any prior TEE event, regardless of the onset of the event, are not be eligible for participation in the study -References were added to explicitly state the higher risk for thromboembolic events in “newly” diagnosed patients with DM. -For the safety of patients with risk factors for thromboembolic events or patients who are in the initial disease stage after diagnosis the Permitted Concomitant Therapy Section has been amended wit the information that TEE prophylaxis, if deemed necessary by the investigator, is allowed as a precautionary measure and should follow standard of care. Its use must be documented.
    12 Mar 2019
    Amendment 7: Early Termination section has been updated based on the opinion of the IDMC. TEEs with a likely temporal and cause-effect relationship which occur in a single subject within a short clinical time period should be counted as a single adverse events of special interest (AESI) including for the calculations performed for stopping rules.
    03 Jul 2019
    Amendment 8: -As requested by FDA the definition of responders in study protocol was reworded and the primary endpoint was reworded subsequently in order to be unambiguous

    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-Thu May 01 04:31:14 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA