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    Clinical Trial Results:
    Long-term, Interventional, Open Label Extension Study Evaluating the Safety of Tocilizumab Treatment in Patients with Polyarticular-Course Juvenile Idiopathic Arthritis From Germany who Completed the Global, Multinational Trial (WA19977)

    Summary
    EudraCT number
    		 2011-001097-25
    Trial protocol
    		 DE  
    Global end of trial date
    14 Aug 2013

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 May 2016
    First version publication date
    07 Aug 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ML25756
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01667471
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    F. Hoffmann-La Roche AG
    Sponsor organisation address
    Grenzacherstrasse 124, Basel, Switzerland, CH-4070
    Public contact
    Roche Trial Information Hotline, F. Hoffmann-La Roche AG , 41 61 6878333, global.trial_information@roche.com
    Scientific contact
    Roche Trial Information Hotline, F. Hoffmann-La Roche AG , 41 61 6878333, global.trial_information@roche.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
    14 Aug 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Aug 2013
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety of tocilizumab treatment in participants with Polyarticular-Course Juvenile Idiopathic Arthritis (pcJIA) from Germany who completed the WA19977 study and entered this extension.
    Protection of trial subjects
    The conduct of this clinical study met all local legal and regulatory requirements. The study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and the International Conference on Harmonization (ICH) guideline E6: Good Clinical Practice (GCP).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    18 Jan 2012
    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
    Germany: 7
    Worldwide total number of subjects
    7
    EEA total number of subjects
    7
    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)
    1
    Adolescents (12-17 years)
    4
    Adults (18-64 years)
    2
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 All participants who completed the core study WA19977 were considered for enrollment into this study if they fulfilled the respective requirements of study eligibility. Screening was conducted at Visit 1 during the week prior to enrollment visit.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Arm description
    		 Participants received tocilizumab 8 mg/kg intravenously (IV) every 4 weeks up to 104 weeks or until tocilizumab was commercially available for polyarticular-course Juvenile Idiopathic Arthritis (pcJIA).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Tocilizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received tocilizumab 8 mg/kg IV every 4 weeks.

    Number of subjects in period 1
    		Tocilizumab 8 milligrams per kilogram (mg/kg)
    Started
    7
    Completed
    7

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 All participants who received at least one dose of the study were included in the Safety Analysis Set.

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 7 7
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 14.4 ( 3.2 ) -
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 1 1

    End points

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    End points reporting groups
    Reporting group title
    Tocilizumab 8 milligrams per kilogram (mg/kg)
    Reporting group description
    		 Participants received tocilizumab 8 mg/kg intravenously (IV) every 4 weeks up to 104 weeks or until tocilizumab was commercially available for polyarticular-course Juvenile Idiopathic Arthritis (pcJIA).

    Primary: Number of Participants With Adverse Events of Special Interest and Study-Drug Related Adverse Events

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    End point title
    		 Number of Participants With Adverse Events of Special Interest and Study-Drug Related Adverse Events [1]
    End point description
    Adverse Events (AEs) and Serious Adverse Events (SAEs) were recorded from the first day of tocilizumab administration until 4 weeks after administration of the last dose of tocilizumab.
    End point type
    Primary
    End point timeframe
    Baseline and every 4 weeks up to Week 108
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary objective of this study was Safety. No statistical analysis was done for safety endpoints.
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [2]
    Units: participants
    number (not applicable)
        Drug related AEs
    6
        Drug related SAEs
    0
        AEs of special interest
    1
        Drug related AEs of special interest
    1
    Notes
    [2] - Safety Analysis Set
    No statistical analyses for this end point

    Primary: Number of AEs of Special Interest and Study Drug Related AEs

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    End point title
    		 Number of AEs of Special Interest and Study Drug Related AEs [3]
    End point description
    AEs and SAEs were recorded from the first day of tocilizumab administration until 4 weeks after administration of the last dose of tocilizumab.
    End point type
    Primary
    End point timeframe
    Baseline and every 4 weeks up to Week 108
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary objective of this study was Safety. No statistical analysis was done for safety endpoints.
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [4]
    Units: adverse events
    number (not applicable)
        Drug-related AEs
    22
        Drug-related SAEs
    0
        AEs of special interest
    2
        Drug-related AEs of special interest
    2
    Notes
    [4] - Safety Analysis Set
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit

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    End point title
    		 Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
    End point description
    The six JIA ACR components comprised of: 1) Physician's global assessment of disease activity, 2) Parent/Participant's global assessment of overall well-being, 3) Maximum number of joints with active arthritis, 4) Number of joints with limitation of movement, 5) Erythrocyte Sedimentation Rate (ESR) and/or C-reactive Protein (CRP), and 6) Childhood Health Assessment Questionnaire - Disease Index (CHAQ-DI). At an assessment visit a JIA ACR30/50/70/90 response in comparison to Baseline was defined as: At least three of the six JIA ACR core components improving by at least 30 percent (%), 50%, 70%, or 90% and no more than one of the remaining JIA ACR core components worsening by more than 30%.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 36, 48, 60, 72 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [5]
    Units: percentage of participants
    number (not applicable)
        Baseline JIA ACR30 (n=7)
    85.7
        Week 12 JIA ACR30 (n=7)
    100
        Week 24 JIA ACR30 (n=7)
    100
        Week 36 JIA ACR30 (n=7)
    100
        Week 48 JIA ACR30 (n=6)
    100
        Week 60 JIA ACR30 (n=6)
    100
        Week 72 JIA ACR30 (n=2)
    100
        Week 108 JIA ACR30 (n=7)
    100
        Baseline JIA ACR50 (n=7)
    85.7
        Week 12 JIA ACR50 (n=7)
    100
        Week 24 JIA ACR50 (n=7)
    100
        Week 36 JIA ACR50 (n=7)
    100
        Week 48 JIA ACR50 (n=6)
    100
        Week 60 JIA ACR50 (n=6)
    100
        Week 72 JIA ACR50 (n=2)
    100
        Week 108 JIA ACR50 (n=7)
    100
        Baseline JIA ACR70 (n=6)
    85.7
        Week 12 JIA ACR70 (n=7)
    100
        Week 24 JIA ACR70 (n=7)
    100
        Week 36 JIA ACR70 (n=7)
    100
        Week 48 JIA ACR70 (n=6)
    100
        Week 60 JIA ACR70 (n=6)
    100
        Week 72 JIA ACR70 (n=2)
    100
        Week 108 JIA ACR70 (n=7)
    100
        Baseline JIA ACR90 (n=6)
    85.7
        Week 12 JIA ACR90 (n=7)
    85.7
        Week 24 JIA ACR90 (n=7)
    85.7
        Week 36 JIA ACR90 (n=7)
    71.4
        Week 48 JIA ACR90 (n=6)
    83.3
        Week 60 JIA ACR90 (n=6)
    66.7
        Week 72 JIA ACR90 (n=2)
    100
        Week 108 JIA ACR90 (n=7)
    85.7
    Notes
    [5] - n = number of participants analyzed for the given parameter at the specified visit.
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Inactive Disease by Visit

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    End point title
    		 Percentage of Participants With Inactive Disease by Visit
    End point description
    A participant was defined to show inactive disease if all of the following criteria were applied: 1) No joints with active arthritis (no joints with swelling and no joints with lack of motion), 2) No fever, rash, serositis, splenomegaly, or generalized lymphadenopathy attributable to JIA, 3) No active uveitis, 4) ESR and/or CRP within normal range, and 5) Physician's global assessment of disease activity equals (=) 0 millimeters (mm) on a Visual analog scale (VAS).
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 36, 48, 60, 72 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [6]
    Units: percentage of participants
    number (not applicable)
        Baseline(n=7)
    57.1
        Week 12 (n=7)
    42.9
        Week 24 (n=7)
    14.3
        Week 36 (n=7)
    42.9
        Week 48 (n=6)
    16.7
        Week 60 (n=6)
    33.3
        Week 72 (n=2)
    50
        Week 108 (n=7)
    57.1
    Notes
    [6] - n = number of participants analyzed for the given parameter at the specified visit.
    No statistical analyses for this end point

    Secondary: Percentage of Participants Achieving Clinical Remission (CR) at Each Visit

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    End point title
    		 Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
    End point description
    CR was defined as clinical remission with medication (CRem). A participant was in CR if inactive disease was observed for a minimum of 6 consecutive months.
    End point type
    Secondary
    End point timeframe
    Baseline, Screening, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [7]
    Units: percentage of participants
    number (not applicable)
        Baseline (n=7)
    0
        Screening (n=7)
    0
        Week 4 (n=7)
    0
        Week 8 (n=7)
    0
        Week 12 (n=7)
    0
        Week 16 (n=7)
    0
        Week 20 (n=7)
    0
        Week 24 (n=7)
    0
        Week 28 (n=7)
    14.3
        Week 32 (n=7)
    14.3
        Week 36 (n=7)
    14.3
        Week 40 (n=6)
    16.7
        Week 44 (n=6)
    16.7
        Week 48 (n=6)
    16.7
        Week 52 (n=6)
    16.7
        Week 56 (n=6)
    16.7
        Week 60 (n=6)
    0
        Week 64 (n=5)
    0
        Week 68 (n=3)
    0
        Week 72 (n=2)
    0
        Week 76 (n=1)
    0
        Week 108 (n=7)
    0
    Notes
    [7] - n = number of participants analyzed for the given parameter at the specified visit.
    No statistical analyses for this end point

    Secondary: Physicians Assessment of Global Activity (VAS)

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    End point title
    		 Physicians Assessment of Global Activity (VAS)
    End point description
    The participant's treating physician provided a rating of the participant's arthritis disease activity on a 0 to 100 mm horizontal scale. The extreme left end of the line represented 'arthritis inactive' (ie, symptom-free and no arthritis symptoms) and the extreme right end represented 'arthritis very active'. A higher score indicated more disease activity.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [8]
    Units: mm
    arithmetic mean (standard deviation)
        Baseline (n=7)
    12 ( 30.4 )
        Week 12 (n=7)
    2.3 ( 2.9 )
        Week 24 (n=7)
    5.4 ( 6.1 )
        Week 28 (n=1)
    1 ( 0 )
        Week 32 (n=2)
    29 ( 8.5 )
        Week 36 (n=7)
    5 ( 7.9 )
        Week 48 (n=6)
    5.7 ( 8.2 )
        Week 60 (n=6)
    5.7 ( 8.7 )
        Week 72 (n=2)
    1 ( 1.4 )
        Week 108 (n=7)
    5 ( 8.7 )
    Notes
    [8] - Safety Analysis Set; number (n) = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: Parent or Participant's Assessment of Global Activity (VAS)

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    End point title
    		 Parent or Participant's Assessment of Global Activity (VAS)
    End point description
    The participant or parent/guardian, as appropriate, provided a rating of the participant's well-being on a 0 to 100 mm horizontal scale. The extreme left end of the line represented 'very well' (ie, symptom-free and no arthritis disease activity) and the extreme right end represented 'very poor' (ie, maximum arthritis disease activity). A higher score indicated poorer well-being.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [9]
    Units: mm
    arithmetic mean (standard deviation)
        Baseline (n=7)
    10.1 ( 20 )
        Week 12 (n=7)
    7.1 ( 8.1 )
        Week 24 (n=7)
    16.4 ( 19.9 )
        Week 28 (n=1)
    1 ( 0 )
        Week 32 (n=2)
    22.5 ( 24.7 )
        Week 36 (n=7)
    12.1 ( 18.4 )
        Week 48 (n=6)
    11.7 ( 13.9 )
        Week 60 (n=6)
    10.2 ( 13.8 )
        Week 72 (n=2)
    3.5 ( 4.9 )
        Week 108 (n=7)
    9.7 ( 16 )
    Notes
    [9] - Safety Analysis Set; n = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: Number of Joints With Active Arthritis

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    End point title
    		 Number of Joints With Active Arthritis
    End point description
    Joints with active arthritis were defined as joints with swelling or pain, and limited of motion. The maximum number of joints with active arthritis was 71.The joint assessment was performed by an independent assessor who was not the treating physician and who was blinded to all other aspects of the participant's efficacy and safety data.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [10]
    Units: joints
    arithmetic mean (standard deviation)
        Baseline (n=7)
    0.9 ( 2.3 )
        Week 12 (n=7)
    0.3 ( 0.8 )
        Week 24 (n=7)
    1 ( 2.6 )
        Week 28 (n=1)
    1 ( 0 )
        Week 32 (n=2)
    1.5 ( 0.7 )
        Week 36 (n=7)
    1.1 ( 2.2 )
        Week 48 (n=6)
    0 ( 0 )
        Week 60 (n=6)
    0.3 ( 0.8 )
        Week 72 (n=2)
    0 ( 0 )
        Week 108 (n=7)
    0.3 ( 0.8 )
    Notes
    [10] - Safety Analysis Set; n = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: Number of Joints With Lack of Motion

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    End point title
    		 Number of Joints With Lack of Motion
    End point description
    Joints with lack of movement were assessed. The maximum number of joints with lack of movement was 67. The joint assessment was performed by an independent assessor who was not the treating physician and who was blinded to all other aspects of the participant's efficacy and safety data.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [11]
    Units: joints
    arithmetic mean (standard deviation)
        Baseline (n=7)
    1.4 ( 2.7 )
        Week 12 (n=7)
    2.6 ( 5.1 )
        Week 24 (n=7)
    2.4 ( 3.2 )
        Week 28 (n=1)
    1 ( 0 )
        Week 32 (n=2)
    5.5 ( 7.8 )
        Week 36 (n=7)
    1.7 ( 2.4 )
        Week 48 (n=6)
    2.7 ( 5.2 )
        Week 60 (n=6)
    1.8 ( 3 )
        Week 72 (n=2)
    0 ( 0 )
        Week 108 (n=7)
    2.9 ( 4.8 )
    Notes
    [11] - Safety Analysis Set; n = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: Erythrocyte Sedimentation Rate (ESR)

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    End point title
    		 Erythrocyte Sedimentation Rate (ESR)
    End point description
    ESR is a marker of inflammation and was measured as millimeters per hour (mm/h).
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8,12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [12]
    Units: mm/h
    arithmetic mean (standard deviation)
        Baseline (n=7)
    4.9 ( 5.1 )
        Week 4 (n=7)
    3 ( 1.9 )
        Week 8 (n=7)
    2.9 ( 2.3 )
        Week 12 (n=7)
    3 ( 2 )
        Week 16 (n=7)
    8.6 ( 16.1 )
        Week 20 (n=7)
    2.6 ( 0.8 )
        Week 24 (n=7)
    2.7 ( 1.4 )
        Week 28 (n=5)
    3.4 ( 3.1 )
        Week 32 (n=7)
    4.3 ( 4.2 )
        Week 36 (n=7)
    4.3 ( 2.5 )
        Week 40 (n=6)
    2 ( 1.8 )
        Week 44 (n=6)
    2.7 ( 1.5 )
        Week 48 (n=6)
    3.8 ( 1.5 )
        Week 52 (n=6)
    3.2 ( 3.6 )
        Week 56 (n=6)
    2.3 ( 1.6 )
        Week 60 (n=6)
    10.2 ( 12.7 )
        Week 64 (n=5)
    3.2 ( 2.4 )
        Week 68 (n=3)
    10 ( 7.2 )
        Week 72 (n=2)
    4.5 ( 0.7 )
        Week 76 (n=1)
    2 ( 0 )
        Week 108 (n=7)
    4.3 ( 2.8 )
    Notes
    [12] - Safety Analysis Set; n = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: CHAQ-DI Score

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    End point title
    		 CHAQ-DI Score
    End point description
    The CHAQ-DI questionnaire consisted of 30 questions referring to eight domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities. Each domain had at least two component questions and if applicable to the participant there were four possible responses (0 = without any difficulty, 1 = with some difficulty, 2 = with much difficulty, 3 = unable to do). The CHAQ-DI score is the sum of the domain scores divided by the number of domains that have a non-missing score. This overall score ranges from 0 (best) to 3 (worst).
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72, and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [13]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline (n=7)
    0 ( 0 )
        Week 12 (n=7)
    0.04 ( 0.09 )
        Week 24 (n=7)
    0.14 ( 0.2 )
        Week 28 (n=1)
    0 ( 0 )
        Week 32 (n=2)
    0.19 ( 0.27 )
        Week 36 (n=7)
    0.13 ( 0.33 )
        Week 48 (n=6)
    0 ( 0 )
        Week 60 (n=6)
    0.21 ( 0.51 )
        Week 72 (n=2)
    0 ( 0 )
        Week 108 (n=7)
    0 ( 0 )
    Notes
    [13] - Safety Analysis Set; n = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: Parent or Participant's Assessment of Pain (VAS)

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    End point title
    		 Parent or Participant's Assessment of Pain (VAS)
    End point description
    Parents or participants rated participant's pain by placing a horizontal line on a VAS of 0 (no pain)- 100 mm (severe pain).
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72, and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [14]
    Units: mm
    arithmetic mean (standard deviation)
        Baseline (n=7)
    10.9 ( 21.8 )
        Week 12 (n=7)
    7 ( 10 )
        Week 24 (n=7)
    13.7 ( 16.7 )
        Week 28 (n=1)
    1 ( 0 )
        Week 32 (n=2)
    28.5 ( 17.7 )
        Week 36 (n=7)
    10.6 ( 15.9 )
        Week 48 (n=6)
    12.3 ( 13.6 )
        Week 60 (n=6)
    10.2 ( 13.8 )
        Week 72 (n=2)
    4 ( 5.7 )
        Week 108 (n=7)
    9.9 ( 15.9 )
    Notes
    [14] - Safety Analysis Set; n = number of participants analyzed at the specified visit.
    No statistical analyses for this end point

    Secondary: CRP Levels

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    End point title
    		 CRP Levels
    End point description
    CRP an acute phase protein, is a marker of inflammation. CRP was measured as milligrams per deciliter (mg/dL).
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8,12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76 and 108
    End point values
    		 Tocilizumab 8 milligrams per kilogram (mg/kg)
    Number of subjects analysed
    7 [15]
    Units: mg/dL
    arithmetic mean (standard deviation)
        Baseline (n=7)
    0.03 ( 0.01 )
        Week 4 (n=7)
    0.05 ( 0.07 )
        Week 8 (n=7)
    0.02 ( 0.02 )
        Week 12 (n=7)
    0.03 ( 0.01 )
        Week 16 (n=7)
    0.23 ( 0.54 )
        Week 20 (n=7)
    0.05 ( 0.07 )
        Week 24 (n=7)
    0.03 ( 0.02 )
        Week 28 (n=6)
    0.02 ( 0.02 )
        Week 32 (n=7)
    0.03 ( 0.02 )
        Week 36 (n=7)
    0.02 ( 0.02 )
        Week 40 (n=6)
    0.03 ( 0.02 )
        Week 44 (n=6)
    0.03 ( 0.02 )
        Week 48 (n=6)
    0.03 ( 0.02 )
        Week 52 (n=6)
    0.02 ( 0.02 )
        Week 56 (n=6)
    0.03 ( 0.02 )
        Week 60 (n=6)
    0.38 ( 0.9 )
        Week 64 (n=5)
    0.03 ( 0.03 )
        Week 68 (n=3)
    0.09 ( 0.1 )
        Week 72 (n=2)
    0.04 ( 0.04 )
        Week 76 (n=1)
    0.05 ( 0 )
        Week 108 (n=7)
    0.05 ( 0.06 )
    Notes
    [15] - Safety Analysis Set; n = number of participants analyzed at the specifies visit.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were collected from the date of screening until Week 108.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    16.0
    Reporting groups
    Reporting group title
    Tocilizumab
    Reporting group description
    		 Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.

    Serious adverse events
    		 Tocilizumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 7 (14.29%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Tocilizumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 7 (100.00%)
    Investigations
    Biopsy kidney
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    White blood cell count decreased
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Excoriation
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Fall
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Ligament sprain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Cardiac disorders
    Aortic valve incompetence
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Mitral valve incompetence
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    General disorders and administration site conditions
    Injection site swelling
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Local swelling
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Vaccination site reaction
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 7 (28.57%)
         occurrences all number
    2
    Abdominal pain upper
         subjects affected / exposed
    2 / 7 (28.57%)
         occurrences all number
    2
    Constipation
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Epigastric discomfort
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Gastritis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    2 / 7 (28.57%)
         occurrences all number
    2
    Vomiting
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    3
    Oropharyngeal pain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Tonsillar disorder
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Psoriasis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Urticaria
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 7 (57.14%)
         occurrences all number
    6
    Foot deformity
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Joint effusion
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Juvenile idiopathic arthritis
         subjects affected / exposed
    3 / 7 (42.86%)
         occurrences all number
    4
    Pain in extremity
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Infections and infestations
    Acute tonsillitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Bronchitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Cystitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Gastroenteritis
         subjects affected / exposed
    4 / 7 (57.14%)
         occurrences all number
    4
    Herpes simplex
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Lice infestation
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Otitis externa
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Rhinitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Scarlet fever
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 7 (42.86%)
         occurrences all number
    4
    Vulvovaginal mycotic infection
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Dec 2011
    Changes were made to the Screening and Exclusion criteria and definition of adverse events of special interest was modified. The amended text included that in case of pregnancy, the treatment must be permanently discontinued.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    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
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