Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    An Open-label, Exploratory Study to Establish the Safety and Efficacy of 3 Months Treatment With Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever

    Summary
    EudraCT number
    		 2015-003527-57
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    25 Oct 2011

    Results information
    Results version number
    		 v1(current)
    This version publication date
    29 Sep 2016
    First version publication date
    29 Sep 2016
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    CACZ885DTR01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01088880
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharmaceuticals AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharmaceuticals AG, +41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharmaceuticals 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
    25 Oct 2011
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Oct 2011
    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 subjects with at least 50% reduction in the attack frequency during 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
    31 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
    Turkey: 9
    Worldwide total number of subjects
    9
    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)
    0
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    8
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Patients who signed an informed consent and meet all inclusion and exclusion criteria in the screening period entered into a 30-day Run-in observation period.

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

    Arms
    Arm title
    		 Canakinumab
    Arm description
    		 150 mg sc canakinumab every 4 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    ACZ885
    Investigational medicinal product code
    Other name
    canakinumab
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects weighing less than 40 kg were administered canakinumab 2 mg/kg/month s.c., those weighting ≥ 40 kg were administered canakinumab 150 mg/month s.c in every 4 weeks during “Treatment Period”. The dose was doubled to 4 mg/kg/month for subjects weighing less than 40 kg and 300 mg/month for those weighing ≥ 40 kg at the second dosing if an attack occurs during the previous month. Canakinumab was supplied as a 180 mg white lyophilized powder for solution for subcutaneous injection.

    Number of subjects in period 1
    		Canakinumab
    Started
    9
    Completed
    9

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 150 mg sc canakinumab every 4 weeks

    Reporting group values
    		 Canakinumab Total
    Number of subjects
    		 9 9
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 1 1
        Adults (18-64 years)
    		 8 8
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 22.33 ( 6.34 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 7
        Male
    		 2 2

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Canakinumab
    Reporting group description
    		 150 mg sc canakinumab every 4 weeks

    Primary: Percentage of participants with at least 50% reduction in the attack frequency during 3 month treatment period

    		 Close Top of page
    End point title
    		 Percentage of participants with at least 50% reduction in the attack frequency during 3 month treatment period [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to 3 months (adjusted for 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 provided for this primary end point.
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: percent
    number (confidence interval 95%)
        Not observed
    0 (0 to 33.6)
        Observed
    100 (66.4 to 100)
    No statistical analyses for this end point

    Secondary: Number of attacks during treatment period

    		 Close Top of page
    End point title
    		 Number of attacks during treatment period
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through 3 months
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: percent
    number (confidence interval 95%)
        No attack
    88.9 (51.8 to 99.7)
        1 attack
    11.1 (0.3 to 48.2)
        ≥2 attack
    0 (0 to 33.6)
    No statistical analyses for this end point

    Secondary: Patient experienced attack during follow-up period

    		 Close Top of page
    End point title
    		 Patient experienced attack during follow-up period
    End point description
    End point type
    Secondary
    End point timeframe
    2-month follow-up period
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: percent
    number (not applicable)
        No
    44.4
        Yes
    55.6
    No statistical analyses for this end point

    Secondary: Time to attack during follow-up period

    		 Close Top of page
    End point title
    		 Time to attack during follow-up period
    End point description
    Time between last injection of canakinumab and Familial Mediterranean fever (FMF) attack.
    End point type
    Secondary
    End point timeframe
    Day 87-144 (follow-up)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: day
        arithmetic mean (standard deviation)
    58.8 ( 22.9 )
    No statistical analyses for this end point

    Secondary: Attack severity -Severity of FMF attack

    		 Close Top of page
    End point title
    		 Attack severity -Severity of FMF attack
    End point description
    End point type
    Secondary
    End point timeframe
    Pre-treatment (Visits 2-4); Post-Treatment (Visit 9/Day 86) and Follow-up period
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: percent
    number (not applicable)
        Pre-treatment: Very mild
    0
        Pre-treatment: Mild
    0
        Pre-treatment: Moderate
    0
        Pre-treatment: Severe
    44.4
        Pre-treatment: Very Severe
    55.6
        Post-treatment: Very mild
    0
        Post-treatment: Mild
    0
        Post-treatment: Moderate
    0
        Post-treatment: Severe
    0
        Post-treatment: Very Severe
    100
        Follow-up period: Very mild
    20
        Follow-up period: Mild
    0
        Follow-up period: Moderate
    40
        Follow-up period: Severe
    20
        Follow-up period: Very Severe
    20
    No statistical analyses for this end point

    Secondary: Time adjusted attack frequency comparison of attack history before 3 months from the study and pre and post treatment periods

    		 Close Top of page
    End point title
    		 Time adjusted attack frequency comparison of attack history before 3 months from the study and pre and post treatment periods
    End point description
    At each post-treatment time point, only patients with a value at both, pre-treatment and post-treatment time point, are included.
    End point type
    Secondary
    End point timeframe
    Pre-treatment (Visits 2-4) through Post-Treatment (Visit 9/Day 86)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: attack number
    arithmetic mean (standard deviation)
        Before Study
    4.46 ( 1.46 )
        Pre-treatment
    3.22 ( 0.7 )
        Post treatment
    0.11 ( 0.33 )
        Change from Before Study/Pre-treatment
    -1.24 ( 1.97 )
        Change from Before Study/Post-treatment
    -3.1 ( 0.6 )
    No statistical analyses for this end point

    Secondary: Attack Duration at baseline and post-treatment

    		 Close Top of page
    End point title
    		 Attack Duration at baseline and post-treatment
    End point description
    At each post-baseline time point, only patients with a value at both, baseline and post-baseline time point, are included.
    End point type
    Secondary
    End point timeframe
    Pre-treatment, Visit 7 (Day 29), Visit 8 (Day 57), Visit 9 (Day 86)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: days
    arithmetic mean (standard deviation)
        Pre-treatment period
    3.58 ( 3.11 )
        Visit 7
    0 ( 0 )
        Visit 8
    0 ( 0 )
        Visit 9
    3 ( 0 )
    No statistical analyses for this end point

    Secondary: Physician's Global Assessment of Familial Mediterranean fever (FMF) control

    		 Close Top of page
    End point title
    		 Physician's Global Assessment of Familial Mediterranean fever (FMF) control
    End point description
    End point type
    Secondary
    End point timeframe
    Visit 4 (Day 1), Visit 11 (Day 144), Visit 11 (Day 144) - [(RtT) Response to Treatment]
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: percent
    number (not applicable)
        Visit 4 - Very good
    0
        Visit 4 - Good
    0
        Visit 4 - Fair
    11.1
        Visit 4 - Poor
    88.9
        Visit 4 - Very poor
    0
        Visit 11 - Very good
    77.8
        Visit 11 - Good
    11.1
        Visit 11 - Fair
    11.1
        Visit 11 - Poor
    0
        Visit 11 - Very poor
    0
        Visit 11 RtT - Very good
    100
        Visit 11 RtT - Good
    0
        Visit 11 RtT - Fair
    0
        Visit 11 RtT - Poor
    0
        Visit 11 RtT - Very poor
    0
    No statistical analyses for this end point

    Secondary: Patient's Global Assessment of Familial Mediterranean fever (FMF) control

    		 Close Top of page
    End point title
    		 Patient's Global Assessment of Familial Mediterranean fever (FMF) control
    End point description
    End point type
    Secondary
    End point timeframe
    Visit 4 (Day 1), Visit 11 (Day 144), Visit 11 (Day 144) - [(RtT) Response to Treatment]
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: percent
    number (not applicable)
        Visit 4 - Very good
    0
        Visit 4 - Good
    11.1
        Visit 4 - Fair
    22.2
        Visit 4 - Poor
    22.2
        Visit 4 - Very poor
    44.4
        Visit 11 - Very good
    37.5
        Visit 11 - Good
    37.5
        Visit 11 - Fair
    12.5
        Visit 11 - Poor
    12.5
        Visit 11 - Very poor
    0
        Visit 11 RtT - Very good
    77.8
        Visit 11 RtT - Good
    22.2
        Visit 11 RtT - Fair
    0
        Visit 11 RtT - Poor
    0
        Visit 11 RtT - Very poor
    0
    No statistical analyses for this end point

    Secondary: SF-36 questionnaire results

    		 Close Top of page
    End point title
    		 SF-36 questionnaire results
    End point description
    At each post-baseline timepoint, only patients with a value at both, baseline and post-baseline timepoint, are included.
    End point type
    Secondary
    End point timeframe
    Visit 4 (Day 1) and Visit 11 (Day 144)
    End point values
    		 Canakinumab
    Number of subjects analysed
    9
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Physical Component - Summary Scores Visit 4
    38 ( 21.25 )
        Physical Component - Summary Scores Visit 11
    71.25 ( 17.1 )
        Physical Component - Difference from baseline
    29.88 ( 12.52 )
        Mental Component - Summary Scores Visit 4
    45.89 ( 22.53 )
        Mental Component - Summary Scores Visit 11
    69.88 ( 20.07 )
        Mental Component - Difference from baseline
    21.13 ( 20.54 )
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    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
    unk
    Reporting groups
    Reporting group title
    ACZ885
    Reporting group description
    		 -

    Serious adverse events
    		 ACZ885
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 9 (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
    		 ACZ885
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 9 (88.89%)
    Injury, poisoning and procedural complications
    Ankle Fracture
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    4
    Pregnancy, puerperium and perinatal conditions
    Pregnancy
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrointestinal disorders
    Vomitting
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Hidradenitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Pruritus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infections and infestations
    Tooth infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Apr 20 11:31:22 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA