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    Clinical Trial Results:
    A 6 Month Phase 2, Multi-Center, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Treatment With Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever

    Summary
    EudraCT number
    		 2015-003522-13
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    26 Feb 2012

    Results information
    Results version number
    		 v1(current)
    This version publication date
    12 Nov 2016
    First version publication date
    12 Nov 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CACZ885D2204
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01148797
    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
    26 Feb 2012
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Feb 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To measure the effect of canakinumab on the frequency of FMF attacks defined as percentage of patients with at least 50% reduction in the attack frequency during a 3 month Treatment period.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    29 Dec 2010
    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
    Israel: 7
    Worldwide total number of subjects
    7
    EEA total number of subjects
    0
    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)
    5
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Informed consent was obtained from each patient in writing before screening. The study was described by the investigator, who answered any questions, and written information was also provided.

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

    Arms
    Arm title
    		 Canakinumab
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Canakinumab 150 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects weighing <40 kg received 2 mg/kg canakinumab monthly administered s.c. ( 150 mg for patients ≥ 40 kg). The dose could be increased to 4 mg/kg for patients < 40 kg at the second dosing (300 mg for patients ≥ 40 kg) in case an attack occurred between Baseline and visit 7 on Day 29.

    Number of subjects in period 1
    		Canakinumab
    Started
    7
    Completed
    7

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 -

    Reporting group values
    		 Canakinumab Total
    Number of subjects
    		 7 7
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 5 5
        Adolescents (12-17 years)
    		 2 2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 9.9 ± 3 -
    Gender categorical
    Units: Subjects
        Female
    		 2 2
        Male
    		 5 5

    End points

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    End points reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 -

    Primary: Percentage of patients with more than 50% reduction in attack frequency

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    End point title
    		 Percentage of patients with more than 50% reduction in attack frequency [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline up to 84 days
    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: Statistical analyses have not been reported for this primary end point.
    End point values
    		 Canakinumab
    Number of subjects analysed
    7
    Units: percent
    number (not applicable)
        Reduction<50%
    14.3
        Reduction>=50%
    85.7
    No statistical analyses for this end point

    Secondary: Attack frequency comparison at pre-treatment and treatment period

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    End point title
    		 Attack frequency comparison at pre-treatment and treatment period
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through treatment (84 days).
    End point values
    		 Canakinumab
    Number of subjects analysed
    7
    Units: attack rate
    arithmetic mean (standard deviation)
        Pre-treatment
    7.98 ± 3.65
        Treatment
    1.11 ± 1.45
        Absolute change from pre-treat - end of treatment
    -6.86 ± 4.31
        Percent change from pre-treat to end of treatment
    -80.48 ± 31.88
    No statistical analyses for this end point

    Secondary: Percentage of patients with Familial Mediterranean Fever (FMF) attacks during the post-treatment period

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    End point title
    		 Percentage of patients with Familial Mediterranean Fever (FMF) attacks during the post-treatment period
    End point description
    End point type
    Secondary
    End point timeframe
    Day 57 to End of Study (EOS)
    End point values
    		 Canakinumab
    Number of subjects analysed
    7
    Units: percent
    number (not applicable)
        No. of attacks = 0
    28.6
        No. of attacks = 1
    57.1
        No. of attacks = 3
    14.3
    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
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    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
    4 / 7 (57.14%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Injection site pain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Eye disorders
    Laceration and eyelid edema
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    3
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Infections and infestations
    Hordeolum
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Streptococcal 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
    10 Feb 2011
    1. Visits and Assessments Schedule – there were a few discrepancies between the Table and the protocol text. 2. Study Design Figure: There were some details missing from the schema. 3. The limitation of a minimum of 14 day-attack free interval between attacks was deleted, due to nonconformity with the study population. 4. SAE follow-up period was extended from 4 to 8 weeks after EOS visit, due to long half life of canakinumab. 5. There were a few abbreviations used without detailing. 6. There were a few typing errors and spelling mistakes.

    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
    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.

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