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    Clinical Trial Results:
    Anakinra (Kineret®) for a hereditary autoinflammatory disease with MEFV mutation and inflammasome activation.

    Summary
    EudraCT number
    		 2015-004292-69
    Trial protocol
    		 BE  
    Global end of trial date
    30 Aug 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Jul 2023
    First version publication date
    28 Jul 2023
    Other versions
    Summary report(s)
    		 Anakinra in PAAND

    Trial information

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    Trial identification
    Sponsor protocol code
    MEFV01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    UZ Leuven
    Sponsor organisation address
    Herestraat 49, Leuven, Belgium, 3000
    Public contact
    Wouters Carine, UZ Leuven, 32 1634 39 74, carine.wouters@uzleuven.be
    Scientific contact
    Wouters Carine, UZ Leuven, 32 1634 39 74, carine.wouters@uzleuven.be
    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
    30 Aug 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Aug 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Primary: 1. Control of inflammatory symptoms (cutaneous, articular, muscular) and of systemic inflammation (anemia, acute phase reactants). 2. Proof of concept that the novel MEFV mutation identified in this family, causes inflammasome and caspase-1activation with increased release of IL-1.
    Protection of trial subjects
    Alll patients fulfilled inclusion criteria, gave informed consent for enrollement and treatment with Anakinra. The trial protocol lwas approved by the ethical comittee of UZ Leuven.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Dec 2015
    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
    Belgium: 3
    Worldwide total number of subjects
    3
    EEA total number of subjects
    3
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    2
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Mutation in MEFV gene confirmed

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

    Arms
    Arm title
    		 Anakinra treatment
    Arm description
    		 -
    Arm type
    		 active treatment

    Investigational medicinal product name
    Anakinra
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    100 mg daily via sc injection

    Number of subjects in period 1
    		Anakinra treatment
    Started
    3
    Completed
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    whole study
    Reporting group description
    		 -

    Reporting group values
    		 whole study Total
    Number of subjects
    		 3 3
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 2 2
        From 65-84 years
    		 1 1
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 61.3 ( 12.7 ) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 3 3
    disease duration
    Units: years
        arithmetic mean (standard deviation)
    		 34.0 ( 20.8 ) -

    End points

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    End points reporting groups
    Reporting group title
    Anakinra treatment
    Reporting group description
    		 -

    Primary: remission of systemic inflammatory markers (CRP)

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    End point title
    		 remission of systemic inflammatory markers (CRP) [1]
    End point description
    End point type
    Primary
    End point timeframe
    at week 12
    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: this is a pilot open label study to evaluate the efficacy and safety of anakinra in PAAND patients. Only evaluation of 3 subjects to report, only descriptive statistics possible.
    End point values
    		 Anakinra treatment
    Number of subjects analysed
    3
    Units: ≤ 5.0 mg/L
        yes
    2
        no
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    01-12-2015 until 30-08-2022.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    2.1
    Reporting groups
    Reporting group title
    whole study
    Reporting group description
    		 -

    Serious adverse events
    		 whole study
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 whole study
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
    Skin and subcutaneous tissue disorders
    local site reaction
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2

    More information

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    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
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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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