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    Clinical Trial Results:
    An open-label, multicenter, efficacy and safety pilot study of 6-month canakinumab treatment with up to 6-month follow-up and 24-month long-term treatment in patients with active Hyper-IgD Syndrome (HIDS)

    Summary
    EudraCT number
    		 2010-020904-31
    Trial protocol
    		 ES  
    Global end of trial date
    15 Jul 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Jul 2016
    First version publication date
    05 Aug 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CACZ885D2402
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01303380
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111 ,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111 ,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    15 Jul 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Jul 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the study was to assess reduction of the flare frequency after administration of canakinumab in subjects with active Hyper-IgD Syndrome (HIDS) during the 6-month treatment period compared to historical period (defined as the most recent 6 months in which the subject had not received drugs other than symptomatic treatment with Nonsteroidal anti-inflammatory drugs (NSAIDs) and/or steroids).
    Protection of trial subjects
    NSAIDs and corticosteroids were used as rescue medication. NSAIDs were allowed to treat the signs and symptoms of HIDS during acute flares as per discretion of the investigator. NSAIDs were used for symptomatic treatment of the initial qualified flare. Corticosteroids 0.5 milligram/kilogram (mg/kg) were allowed for new flares after the initial qualified flare.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    24 Mar 2011
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 9
    Worldwide total number of subjects
    9
    EEA total number of subjects
    9
    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)
    3
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    3
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at 3 centres in Spain.

    Pre-assignment
    Screening details
    		 A total of 10 subjects were screened, 9 of which entered in the treatment period of the study, one subject was considered to be a screening failure.

    Period 1
    Period 1 title
    Overall period
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    The study was open-label, hence no blinding was performed.

    Arms
    Arm title
    		 Canakinumab
    Arm description
    		 Subjects received body ­weight stratified dosage of canakinumab (4 mg/kg for subjects less than or equal to (≤) 40 kg or 300 mg for subjects more than (>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for subjects ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for subjects ≤40 kg) every 6 weeks, thereafter starting at Week 6 in subjects who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the subjects received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Canakinumab
    Investigational medicinal product code
    ACZ885
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Canakinumab 4 mg/kg (2 mg/kg - 6 mg/kg) for subjects ≤ 40 kg or 300 mg for subjects > 40 kg was injected via s.c. route every 6 weeks during 6 months of treatment.

    Number of subjects in period 1
    		Canakinumab
    Started
    9
    Completed
    8
    Not completed
    1
         Lack of compliance to the study procedures
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 Subjects received body ­weight stratified dosage of canakinumab (4 mg/kg for subjects less than or equal to (≤) 40 kg or 300 mg for subjects more than (>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for subjects ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for subjects ≤40 kg) every 6 weeks, thereafter starting at Week 6 in subjects who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the subjects received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.

    Reporting group values
    		 Canakinumab Total
    Number of subjects
    		 9 9
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 3 3
        Adolescents (12-17 years)
    		 3 3
        Adults (18-64 years)
    		 3 3
    Age continuous
    Units: years
        median (full range (min-max))
    		 17.3 (5 to 29) -
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 3 3

    End points

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    End points reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 Subjects received body ­weight stratified dosage of canakinumab (4 mg/kg for subjects less than or equal to (≤) 40 kg or 300 mg for subjects more than (>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for subjects ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for subjects ≤40 kg) every 6 weeks, thereafter starting at Week 6 in subjects who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the subjects received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.

    Primary: Number of flares per subject during historical period and treatment period

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    End point title
    		 Number of flares per subject during historical period and treatment period [1]
    End point description
    A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value > 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the subject has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids. The primary analysis was performed in the Full Analysis set (FAS) population defined as all subjects who received at least one dose of study treatment and had at least one post-baseline assessment.
    End point type
    Primary
    End point timeframe
    Historical period, Month 6 (Treatment period)
    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: Only descriptive summary statistics was planned for this outcome measure. 
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of flares
    median (full range (min-max))
        Historical period
    5 (0 to 2)
        6 Month treatment period
    0 (3 to 12)
    No statistical analyses for this end point

    Secondary: Number of flares per subject at Month 24

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    End point title
    		 Number of flares per subject at Month 24
    End point description
    A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L. The analysis was performed in the FAS population.
    End point type
    Secondary
    End point timeframe
    Month 12 to Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of flares
    median (full range (min-max))
        24 Month treatment period
    0 (0 to 1)
    No statistical analyses for this end point

    Secondary: Number of subjects who flared at treatment period, Month 12 and Month 24

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    End point title
    		 Number of subjects who flared at treatment period, Month 12 and Month 24
    End point description
    A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L. The analysis was performed in the FAS population.
    End point type
    Secondary
    End point timeframe
    Month 6 (Treatment period), Month 12 (Long-term treatment period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of subjects
        Baseline to Month 6
    2
        Month 6 to Month 12
    4
        Month 12 to Month 24
    2
    No statistical analyses for this end point

    Secondary: Number of subjects with flare events based on physician assessed HIDS flare severity score

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    End point title
    		 Number of subjects with flare events based on physician assessed HIDS flare severity score
    End point description
    Physician global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5-point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Any flare event [Day 1 (Baseline) to Month 24 (End of study)]
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of subjects
        Day 45, Absent (n=9)
    0
        Day 45, Minimal (n=9)
    0
        Day 45,Mild (n=9)
    0
        Day 45,Moderate (n=9)
    1
        Day 45,Severe (n=9)
    0
        Flare/Unscheduled visit 1, Absent (n=4)
    2
        Flare/Unscheduled visit 1, Minimal (n=4)
    0
        Flare/Unscheduled visit 1, Mild (n=4)
    1
        Flare/Unscheduled visit 1, Moderate (n=4)
    1
        Flare/Unscheduled visit 1, Severe (n=4)
    0
        Flare/Unscheduled visit 2, Absent (n=2)
    1
        Flare/Unscheduled visit 2, Minimal (n=2)
    0
        Flare/Unscheduled visit 2, Mild (n=2)
    1
        Flare/Unscheduled visit 2, Moderate (n=2)
    0
        Flare/Unscheduled visit 2, Severe (n=2)
    0
        Flare/Unscheduled visit 3, Absent (n=2)
    1
        Flare/Unscheduled visit 3, Minimal (n=2)
    0
        Flare/Unscheduled visit 3, Mild (n=2)
    0
        Flare/Unscheduled visit 3, Moderate (n=2)
    1
        Flare/Unscheduled visit 3, Severe (n=2)
    0
        End of Treatment period, Absent (n=7)
    0
        End of Treatment period, Minimal (n=7)
    0
        End of Treatment period, Mild (n=7)
    4
        End of Treatment period, Moderate (n=7)
    3
        End of Treatment period, Severe (n=7)
    0
        End of Follow-up period, Absent (n=8)
    0
        End of Follow-up period, Minimal (n=8)
    1
        End of Follow-up period, Mild (n=8)
    0
        End of Follow-up period, Moderate (n=8)
    0
        End of Follow-up period, Severe (n=8)
    0
        Long-term 12 M period, Flare 1, Absent (n=3)
    0
        Long-term 12 M period, Flare 1, Minimal (n=3)
    1
        Long-term 12 M period, Flare 1, Mild (n=3)
    1
        Long-term 12 M period, Flare 1, Moderate (n=3)
    1
        Long-term 12 M period, Flare 1, Severe (n=3)
    0
        Long-term 12 M period, Flare 2, Absent (n=1)
    0
        Long-term 12 M period, Flare 2, Minimal (n=1)
    0
        Long-term 12 M period, Flare 2, Mild (n=1)
    1
        Long-term 12 M period, Flare 2, Moderate (n=1)
    0
        Long-term 12 M period, Flare 2, Severe (n=1)
    0
        Long-term 12 M period, Flare 3, Absent (n=1)
    0
        Long-term 12 M period, Flare 3, Minimal (n=1)
    0
        Long-term 12 M period, Flare 3, Mild (n=1)
    1
        Long-term 12 M period, Flare 3, Moderate (n=1)
    0
        Long-term 12 M period, Flare 3, Severe (n=1)
    0
        Long-term 24 M period, Absent (n=2)
    1
        Long-term 24 M period, Minimal (n=2)
    0
        Long-term 24 M period, Mild (n=2)
    1
        Long-term 24 M period, Moderate (n=2)
    0
        Long-term 24 M period, Severe (n=2)
    0
    No statistical analyses for this end point

    Secondary: Number of subjects with flare events based on subject assessed HIDS flare severity score

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    End point title
    		 Number of subjects with flare events based on subject assessed HIDS flare severity score
    End point description
    Subject's global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5-point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe. Same investigator assessed the same subject throughout the study to ensure consistency between assessments. Investigators reviewed every subject’s diary at each visit after their own clinical assessment. The analysis was performed in the FAS population.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of subjects
        Treatment period, Absent
    0
        Treatment period, Minimal
    0
        Treatment period, Mild
    1
        Treatment period, Moderate
    1
        Treatment period, Severe
    0
        Follow-up period, Absent
    0
        Follow-up period, Minimal
    0
        Follow-up period, Mild
    0
        Follow-up period, Moderate
    2
        Follow-up period, Severe
    0
        12-month long-term treatment period, Absent
    0
        12-month long-term treatment period, Minimal
    1
        12-month long-term treatment period, Mild
    1
        12-month long-term treatment period, Moderate
    2
        12-month long-term treatment period, Severe
    0
        24-month long-term treatment period, Absent
    0
        24-month long-term treatment period, Minimal
    1
        24-month long-term treatment period, Mild
    0
        24-month long-term treatment period, Moderate
    0
        24-month long-term treatment period, Severe
    0
    No statistical analyses for this end point

    Secondary: Percentage of subjects with defined grades of subjects assessed symptom control

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    End point title
    		 Percentage of subjects with defined grades of subjects assessed symptom control
    End point description
    Subjects were assessed by subject/parent (subjects aged 6-18 years) for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
    number (not applicable)
        Treatment period, No control (n=9)
    0
        Treatment period, Poor control (n=9)
    0
        Treatment period, Somewhat control (n=9)
    0
        Treatment period, Good control (n=9)
    44.44
        Treatment period, Excellent control (n=9)
    55.56
        Follow-up period, No control (n=9)
    0
        Follow-up period, Poor control (n=9)
    0
        Follow-up period, Somewhat control (n=9)
    11.11
        Follow-up period, Good control (n=9)
    33.33
        Follow-up period, Excellent control (n=9)
    55.56
        Long-term 12 month period, No control (n=8)
    0
        Long-term 12 month period, Poor control (n=8)
    0
        Long-term 12 month period, Somewhat control (n=8)
    12.5
        Long-term 12 month period, Good control (n=8)
    25
        Long-term 12 month period, Excellent control (n=8
    62.5
        Long-term 24 month period, No control (n=8)
    0
        Long-term 24 month period, Poor control (n=8)
    0
        Long-term 24 month period, Somewhat control (n=8)
    0
        Long-term 24 month period, Good control (n=8)
    12.5
        Long-term 24 month period, Excellent control (n=8
    87.5
    No statistical analyses for this end point

    Secondary: Percentage of subjects with defined grades of physician assessed symptom control

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    End point title
    		 Percentage of subjects with defined grades of physician assessed symptom control
    End point description
    Subjects were assessed by physician for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
    number (not applicable)
        Treatment period, No control (n=9)
    0
        Treatment period, Poor control (n=9)
    0
        Treatment period, Somewhat control (n=9)
    0
        Treatment period, Good control (n=9)
    44.44
        Treatment period, Excellent control (n=9)
    55.56
        Follow-up period, No control (n=9)
    0
        Follow-up period, Poor control (n=9)
    0
        Follow-up period, Somewhat control (n=9)
    0
        Follow-up period, Good control (n=9)
    33.33
        Follow-up period, Excellent control (n=9)
    66.67
        Long-term 12 month period, No control (n=8)
    0
        Long-term 12 month period, Poor control (n=8)
    0
        Long-term 12 month period, Somewhat control (n=8)
    12.5
        Long-term 12 month period, Good control (n=8)
    25
        Long-term 12 month period, Excellent control (n=8
    62.5
        Long-term 24 month period, No control (n=8)
    0
        Long-term 24 month period, Poor control (n=8)
    0
        Long-term 24 month period, Somewhat control (n=8)
    0
        Long-term 24 month period, Good control (n=8)
    0
        Long-term 24 month period, Excellent control (n=8
    100
    No statistical analyses for this end point

    Secondary: Percentage of subjects experiencing fever as assessed by physician's global assessment

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    End point title
    		 Percentage of subjects experiencing fever as assessed by physician's global assessment
    End point description
    Fever severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
    number (not applicable)
        Treatment period, Absent (n=9)
    88.89
        Treatment period, Minimal (n=9)
    11.11
        Treatment period, Mild (n=9)
    0
        Treatment period, Moderate (n=9)
    0
        Treatment period, Severe (n=9)
    0
        Follow-up period, Absent (n=9)
    100
        Follow-up period, Minimal (n=9)
    0
        Follow-up period, Mild (n=9)
    0
        Follow-up period, Moderate (n=9)
    0
        Follow-up period, Severe (n=9)
    0
        Long-term 12 month period, Absent (n=8)
    75
        Long-term 12 month period, Minimal (n=8)
    12.5
        Long-term 12 month period, Mild (n=8)
    12.5
        Long-term 12 month period, Moderate (n=8)
    0
        Long-term 12 month period, Severe (n=8)
    0
        Long-term 24 month period, Absent (n=8)
    100
        Long-term 24 month period, Minimal (n=8)
    0
        Long-term 24 month period, Mild (n=8)
    0
        Long-term 24 month period, Moderate (n=8)
    0
        Long-term 24 month period, Severe (n=8)
    0
    No statistical analyses for this end point

    Secondary: Percentage of subjects experiencing apthus ulcers as assessed by physician's global assessment

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    End point title
    		 Percentage of subjects experiencing apthus ulcers as assessed by physician's global assessment
    End point description
    Apthus ulcers were assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
    number (not applicable)
        Treatment period, Absent (n=9)
    88.89
        Treatment period, Minimal (n=9)
    0
        Treatment period, Mild (n=9)
    11.11
        Treatment period, Moderate (n=9)
    0
        Treatment period, Severe (n=9)
    0
        Follow-up period, Absent (n=9)
    100
        Follow-up period, Minimal (n=9)
    0
        Follow-up period, Mild (n=9)
    0
        Follow-up period, Moderate (n=9)
    0
        Follow-up period, Severe (n=9)
    0
        Long-term 12 month period, Absent (n=8)
    100
        Long-term 12 month period, Minimal (n=8)
    0
        Long-term 12 month period, Mild (n=8)
    0
        Long-term 12 month period, Moderate (n=8)
    0
        Long-term 12 month period, Severe (n=8)
    0
        Long-term 24 month period, Absent (n=8)
    100
        Long-term 24 month period, Minimal (n=8)
    0
        Long-term 24 month period, Mild (n=8)
    0
        Long-term 24 month period, Moderate (n=8)
    0
        Long-term 24 month period, Severe (n=8)
    0
    No statistical analyses for this end point

    Secondary: Percentage of subjects experiencing lymphadenopathy as assessed by physician's global assessment

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    End point title
    		 Percentage of subjects experiencing lymphadenopathy as assessed by physician's global assessment
    End point description
    Lymphadenopathy severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
    number (not applicable)
        Treatment period, Absent (n=9)
    88.89
        Treatment period, Minimal (n=9)
    11.11
        Treatment period, Mild (n=9)
    0
        Treatment period, Moderate (n=9)
    0
        Treatment period, Severe (n=9)
    0
        Follow-up period, Absent (n=9)
    77.78
        Follow-up period, Minimal (n=9)
    22.22
        Follow-up period, Mild (n=9)
    0
        Follow-up period, Moderate (n=9)
    0
        Follow-up period, Severe (n=9)
    0
        Long-term 12 month period, Absent (n=8)
    87.5
        Long-term 12 month period, Minimal (n=8)
    12.5
        Long-term 12 month period, Mild (n=8)
    0
        Long-term 12 month period, Moderate (n=8)
    0
        Long-term 12 month period, Severe (n=8)
    0
        Long-term 24 month period, Absent (n=8)
    100
        Long-term 24 month period, Minimal (n=8)
    0
        Long-term 24 month period, Mild (n=8)
    0
        Long-term 24 month period, Moderate (n=8)
    0
        Long-term 24 month period, Severe (n=8)
    0
    No statistical analyses for this end point

    Secondary: Percentage of subjects experiencing abdominal pain as assessed by physician's global assessment

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    End point title
    		 Percentage of subjects experiencing abdominal pain as assessed by physician's global assessment
    End point description
    Abdominal pain was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (End of treatment period), Month 6 (End of follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
    number (not applicable)
        Treatment period, Absent (n=9)
    100
        Treatment period, Minimal (n=9)
    0
        Treatment period, Mild (n=9)
    0
        Treatment period, Moderate (n=9)
    0
        Treatment period, Severe (n=9)
    0
        Follow-up period, Absent (n=9)
    100
        Follow-up period, Minimal (n=9)
    0
        Follow-up period, Mild (n=9)
    0
        Follow-up period, Moderate (n=9)
    0
        Follow-up period, Severe (n=9)
    0
        Long-term 12 month period, Absent (n=8)
    87.5
        Long-term 12 month period, Minimal (n=8)
    12.5
        Long-term 12 month period, Mild (n=8)
    0
        Long-term 12 month period, Moderate (n=8)
    0
        Long-term 12 month period, Severe (n=8)
    0
        Long-term 24 month period, Absent (n=8)
    100
        Long-term 24 month period, Minimal (n=8)
    0
        Long-term 24 month period, Mild (n=8)
    0
        Long-term 24 month period, Moderate (n=8)
    0
        Long-term 24 month period, Severe (n=8)
    0
    No statistical analyses for this end point

    Secondary: Time to resolution of the initial flare after first canakinumab treatment

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    End point title
    		 Time to resolution of the initial flare after first canakinumab treatment
    End point description
    Time to resolution of the initial flare after first dose of canakinumab was determined. The analysis was performed in the FAS population.
    End point type
    Secondary
    End point timeframe
    Day 1 (Baseline), Day 28
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Days
        median (full range (min-max))
    3 (1 to 5)
    No statistical analyses for this end point

    Secondary: Change from baseline in inflammation markers over time up to Month 24

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    End point title
    		 Change from baseline in inflammation markers over time up to Month 24
    End point description
    The C-­reactive Protein (CRP) and/or Serum amyloid A protein (SAA) were used as inflammatory markers. The normal range of CRP was 0-10 mg/L. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group respectively.
    End point type
    Secondary
    End point timeframe
    Day 1 (Baseline), Month 6 (End to treatment period), Month 6 (End to follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Milligram(s)/litre
    median (full range (min-max))
        CRP- 6 month (Treatment period), (n=6)
    -120.5 (-164 to -23)
        CRP- 6 month (Follow-up period), (n=9)
    -111 (-164 to -23)
        CRP- 12-month (Long-term period), (n=8)
    -113.55 (-159.5 to -49.9)
        CRP- 24 month (End of study), (n=8)
    -125.2 (-164 to -34.1)
        SAA- 6 month (Treatment period), (n=1)
    -624.2 (-624.2 to -624.2)
        SAA- 6 month (Follow-up period), (n=2)
    -685.95 (-748.2 to -623.7)
        SAA- 12-month (Long-term period), (n=2)
    -684.1 (-749.2 to -619)
        SAA- 24 month (End of study), (n=2)
    -686.7 (-747.7 to -625.7)
    No statistical analyses for this end point

    Secondary: Health Assessment Questionnaire (HAQ) global score in adults over time

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    End point title
    		 Health Assessment Questionnaire (HAQ) global score in adults over time
    End point description
    Subjects were assessed for health-related quality of life (HRQoL) based on Health Assessment Questionnaire (HAQ). HAQ was an eight 8 categories questionnaire representing all activities related to physical function. Each category has various sub-categories, which were rated by the subject on a 4-point difficulty scale: 0 = any difficulty; 1 = some difficulty; 2 = much difficulty; 3 = unable to do. The total score was the mean of the 8 scores, and ranged from 0 (no disability) to 3 (completely disabled). The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group respectively. Here "Number of subjects analysed" signifies the subjects assessed for HAQ during study.
    End point type
    Secondary
    End point timeframe
    Day 1 (Baseline), Month 6 (End to treatment period), Month 6 (End to follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    6
    Units: Score on a scale
    median (full range (min-max))
        Treatment period, (n=3)
    0 (0 to 0)
        Follow-up period, (n=4)
    0 (0 to 0)
        12-month (Long-term period), (n=5)
    0 (0 to 1)
        24 month (End of study), (n=4)
    0 (0 to 0)
    No statistical analyses for this end point

    Secondary: Childhood Health Assessment Questionnaire (CHAQ) global score in children over time

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    End point title
    		 Childhood Health Assessment Questionnaire (CHAQ) global score in children over time
    End point description
    Subjects or their parents (subjects aged 6 to 17 years) were assessed for HRQoL based on Childhood Health Assessment Questionnaire (CHAQ). CHAQ was an eight domain questionnaire representing functional capacity and independence, evaluated for previous week. Each domain was rated on a 4-point difficulty scale: 0 = any difficulty; 1 = some difficulty; 2 = much difficulty; 3 = unable to do. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group respectively. Here "Number of subjects analysed" signifies the subjects assessed for CHAQ during study. Here, the value 99999.9 in the field represents not estimable data.
    End point type
    Secondary
    End point timeframe
    Day 1 (Baseline), Month 6 (End to treatment period), Month 6 (End to follow-up period), Month 12 (Long term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    6
    Units: Score on a scale
    median (full range (min-max))
        End to treatment period (n= 2)
    0 (0 to 0)
        End to follow-up period (n=4)
    0.1 (0 to 1)
        12 month- Long term period, (n=2)
    0.1 (0 to 1)
        24 month- End of study (n=1)
    0.1 (0 to 1)
    No statistical analyses for this end point

    Secondary: Percentage of subjects who received dose up-titration during 6-month treatment period

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    End point title
    		 Percentage of subjects who received dose up-titration during 6-month treatment period
    End point description
    Subjects who experienced a new HIDS flare between baseline and Week 4 and received an escalated dose of 450 mg of canakinumab every 6 weeks thereafter starting at Week 6 were determined. The analysis was performed in the FAS population.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Day 167 (End of follow-up)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
        number (not applicable)
    22.22
    No statistical analyses for this end point

    Secondary: Duration of flares experienced during the study

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    End point title
    		 Duration of flares experienced during the study
    End point description
    Flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L. The change in post canakinumab treatment flare duration during the study were assessed as compared to historical period. The analysis was performed in the FAS population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group respectively.
    End point type
    Secondary
    End point timeframe
    Month 6 (Treatment period), Month 6 (Follow-up period), Month 12 (Long-term period) and Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Days
    median (full range (min-max))
        Treatment period, (n=3)
    3 (2 to 4)
        Follow-up period, (n=7)
    4 (2 to 10)
        12-month (Long-term period), (n=6)
    3.5 (2 to 8)
        24 month (End of study), (n=2)
    8.5 (6 to 11)
    No statistical analyses for this end point

    Secondary: Time to flare after the last dose of canakinumab during the follow-up period

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    End point title
    		 Time to flare after the last dose of canakinumab during the follow-up period
    End point description
    The median time to flare by the subject after administration of the last dose of canakinumab during the follow-up period was analysed using Kaplan-Meier method.The analysis was performed on the FAS population. Here "Number of subjects analysed" signifies the subjects assessed for time to flare after the last dose of canakinumab during follow-up period.
    End point type
    Secondary
    End point timeframe
    Last dose of canakinumab treatment in follow-up period to end of follow-up period (Day 337)
    End point values
    		 Canakinumab
    Number of subjects analysed
    7
    Units: Days
        median (full range (min-max))
    110 (62 to 196)
    No statistical analyses for this end point

    Secondary: Number of subjects with adverse events (AEs) and serious adverse events (SAEs)

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    End point title
    		 Number of subjects with adverse events (AEs) and serious adverse events (SAEs)
    End point description
    Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. The analysis was performed on Safety Set (SAF) population defined as all subjects who received at least one application of study treatment and had at least one post-baseline safety assessment.
    End point type
    Secondary
    End point timeframe
    Day 1 (Start of study treatment) up to 24 Month (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of subjects
        Adverse events
    9
        Serious adverse events
    4
    No statistical analyses for this end point

    Secondary: Subjects who received rescue treatment

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    End point title
    		 Subjects who received rescue treatment
    End point description
    Subjects who experienced flares were treated with corticosteroids and NSAIDs as rescue medication. The analysis was performed on the FAS population.
    End point type
    Secondary
    End point timeframe
    Day 1 (Baseline) up to 24 Month (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Percentage of subjects
        number (not applicable)
    11.11
    No statistical analyses for this end point

    Secondary: Serum concentration-time profile of canakinumab

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    End point title
    		 Serum concentration-time profile of canakinumab
    End point description
    Canakinumab concentrations in serum were assessed for evaluating pharmacokinetics (PK) of the drug. The analysis was performed on the FAS population.
    End point type
    Secondary
    End point timeframe
    Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Microgram(s)/millilitre
    arithmetic mean (standard deviation)
        Day 4
    28.8 ( 7.9 )
        Day 15
    26.9 ( 8 )
        Day 43
    12.7 ( 4.6 )
        Day 85
    19.4 ( 9.6 )
        Day 127
    23.8 ( 12.8 )
        Day 169
    24 ( 13.4 )
        Day 197
    12.6 ( 13.7 )
        Day 225
    11.5 ( 7.9 )
        Day 253
    6.5 ( 4.8 )
        Day 281
    1.6 ( 1.6 )
        Day 309
    0.9 ( 1 )
        Day 337
    32.6 ( 15.9 )
    No statistical analyses for this end point

    Secondary: Serum concentration of total Interleukin-1β antibody (IL-1β)

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    End point title
    		 Serum concentration of total Interleukin-1β antibody (IL-1β)
    End point description
    Pharmacodynamics of canakinumab was assessed by total IL-1β (sum of free and bound canakinumab) concentration, determined in serum by means of sandwich ELISA assay with limit of detection at 0.1 picogram/millilitre. The analysis was performed on the FAS population.
    End point type
    Secondary
    End point timeframe
    Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169, Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Picogram(s)/millilitre
    arithmetic mean (standard deviation)
        Day 4
    27.3 ( 25.2 )
        Day 15
    22.2 ( 14.7 )
        Day 43
    21.8 ( 13.9 )
        Day 85
    27.3 ( 14 )
        Day 127
    24.3 ( 12.1 )
        Day 169
    32.5 ( 17.9 )
        Day 197
    16.3 ( 5.9 )
        Day 225
    17 ( 8.3 )
        Day 253
    10.2 ( 5.8 )
        Day 281
    4.9 ( 3.3 )
        Day 309
    3.7 ( 2 )
        Day 337
    49.1 ( 60.2 )
    No statistical analyses for this end point

    Secondary: Number of subjects exhibiting anti-canakinumab antibodies at any visit

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    End point title
    		 Number of subjects exhibiting anti-canakinumab antibodies at any visit
    End point description
    Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using bridging ECLIA assay. The analysis was performed on the FAS population.
    End point type
    Secondary
    End point timeframe
    Day 1 to Month 24 (End of study)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: Number of subjects
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    15.1
    Reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 Canakinumab

    Serious adverse events
    		 Canakinumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 9 (44.44%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Vascular disorders
    Hypertensive crisis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Disease progression
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Hidradenitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Temporomandibular joint syndrome
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Peritonitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Streptococcal bacteraemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Fluid overload
         subjects affected / exposed
    1 / 9 (11.11%)
         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
    		 Canakinumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 9 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Bone neoplasm
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Upper limb fracture
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Surgical and medical procedures
    Tooth extraction
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    General disorders and administration site conditions
    Impaired healing
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Influenza like illness
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Pyrexia
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    11
    Eye disorders
    Conjunctival hyperaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    4
    Abdominal pain upper
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Aphthous stomatitis
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Diarrhoea
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Dyspepsia
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Toothache
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Reproductive system and breast disorders
    Vaginal ulceration
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Oropharyngeal pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Eczema
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Erythema nodosum
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hidradenitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Back pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Neck pain
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Scoliosis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Candidiasis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Cellulitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Folliculitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Impetigo
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Nasopharyngitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    4
    Pharyngotonsillitis
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    6
    Respiratory tract infection
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Sinusitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Tonsillitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Tonsillitis streptococcal
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Tooth infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Urinary tract infection
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Vulvovaginal mycotic infection
         subjects affected / exposed
    1 / 9 (11.11%)
         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
    23 Nov 2010
    Subjects with indeterminate QuantiFERON test result at investigator’s discretion and subjects who received any investigational drug during the 30 days before enrolment were excluded from the study.. Assessment parameters for the clinical assessment of auto-inflammatory disease activity by the investigator and subject were reduced from 8 global HIDS-specific signs and symptoms to 4: fever, lymphadenopathy, aphthous ulcers and abdominal pain.
    03 Feb 2011
    Exclusion criterion on the use of Etanercept treatment was modified from four weeks prior to this visit to two weeks prior to the baseline visit (wash-out period).
    30 Sep 2011
    Exclusion criteria was extended to: Subjects included in the screening were stopped for biologic treatments during the screening period (Etanercept, Adalimumab, Infliximab, Kineret, other investigational biologics or other drugs as NSAID, corticosteroids). No subject received first dose of study medication without complying described timelines. Subjects received a dose of canakinumab at day 169 (Visit 8), the visit was considered as follow up period after flare occurrence at this visit (+/- 5 days). The length of follow-up treatment was increased from 6 to 30 months, to include an assessment of the long-term (24-month) efficacy, safety and tolerability of ACZ885 in the treatment of Hyper-IgD with periodic fever syndrome was added.
    21 Dec 2011
    The visit schedule was modified to follow the schedule of drug dosing frequency. Time frame of data analysis for the primary efficacy analysis was modified to the end of the treatment period.
    01 Mar 2012
    Two interim analyses were added: one interim analysis after the end of the follow-up period (maximum 6 months after the end of the subjects treatment period), and second analysis after the first year of the extension period.

    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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