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    Clinical Trial Results:
    Pseudomonas aeruginosa eradication in patients with cystic fibrosis: a randomised multicentre study comparing classic treatment protocols with classic treatment together with antibiotic treatment of upper airways.

    Summary
    EudraCT number
    		 2015-003881-96
    Trial protocol
    		 IT  
    Global end of trial date
    08 Jan 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Aug 2021
    First version publication date
    04 Aug 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    EPAFC
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    AZIENDA OSPEDALIERO UNIVERSITARIA MEYER
    Sponsor organisation address
    VIALE PIERACCINI 24, FIRENZE, Italy, 50139
    Public contact
    FIBROSI CISTICA, AZIENDA OSPEDALIERO UNIVERSITARIA MEYER, +39 0555662474, giovanni.taccetti@meyer.it
    Scientific contact
    FIBROSI CISTICA, AZIENDA OSPEDALIERO UNIVERSITARIA MEYER, +39 0555662474, giovanni.taccetti@meyer.it
    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 Sep 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Jan 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Jan 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy (P. aeruginosa eradication from the lower airways) of two types of treatment in Cystic Fibrosis (CF) patients: the classic eradication protocol used until now (treatment A), versus the classic protocol together with nasal lavage with colistin (treatment B).
    Protection of trial subjects
    No specific risks, pain or distress were expected for the trial subjects. Moreover, risks were comparable to that observed in clinical practice. No specific measures were put in place for trial subjects.
    Background therapy
    		 The following treatments were approved as efficacious standard therapy for the eradication of Pseudomonas Aeruginosa in the lower airways of CF patients: a) Inhalation of colistin (2.000.000 UI or 1.000.000 UI if I-NEB® is used) b.i.d. (total daily dose 4.000.000 IU) and 30 mg/kg/day of oral ciprofloxacin divided into 2 doses for 4 weeks (maximum daily dose 750 mg x 2); b) Inhalation of tobramycin, 300 mg twice daily (600 mg total daily dose) for 4 weeks with appropriate nebulizers; c) Aztreonam lysine for inhalation, 75 mg three times daily (total daily dose 225 mg) for 4 weeks with appropriate nebulizers
    Evidence for comparator
    		 No active comparator or placebo were used in this trial.
    Actual start date of recruitment
    01 May 2016
    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
    Italy: 51
    Worldwide total number of subjects
    51
    EEA total number of subjects
    51
    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)
    32
    Adolescents (12-17 years)
    8
    Adults (18-64 years)
    11
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The recruitment period lasted 2 years.

    Pre-assignment
    Screening details
    		 All the screened patients (n=51) were enrolled in the study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Standard of care - A
    Arm description
    		 Enrolled patients received the classic eradication therapy as per standard of care
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    		 Experimental - B
    Arm description
    		 Enrolled patients received the classic eradication therapy as per standard of care with nasal lavage with colistin.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Colistin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for injection
    Routes of administration
    External use
    Dosage and administration details
    Nasal irrigation with colistin 2.000.000 U b.i.d (total daily dose 4.000.000 IU) for a period of 4 weeks.

    Number of subjects in period 1
    		Standard of care - A Experimental - B
    Started
    25
    26
    Completed
    21
    21
    Not completed
    4
    5
         Consent withdrawn by subject
    1
    3
         Physician decision
    3
    1
         Adverse event, non-fatal
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Standard of care - A
    Reporting group description
    		 Enrolled patients received the classic eradication therapy as per standard of care

    Reporting group title
    Experimental - B
    Reporting group description
    		 Enrolled patients received the classic eradication therapy as per standard of care with nasal lavage with colistin.

    Reporting group values
    		 Standard of care - A Experimental - B Total
    Number of subjects
    		 25 26 51
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 14 18 32
        Adolescents (12-17 years)
    		 6 2 8
        Adults (18-64 years)
    		 5 6 11
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 12 14 26
        Male
    		 13 12 25

    End points

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    End points reporting groups
    Reporting group title
    Standard of care - A
    Reporting group description
    		 Enrolled patients received the classic eradication therapy as per standard of care

    Reporting group title
    Experimental - B
    Reporting group description
    		 Enrolled patients received the classic eradication therapy as per standard of care with nasal lavage with colistin.

    Primary: Difference in P. aeruginosa eradication from the lower airways in the two treatment groups (A and B). Eradication is defined as 3 negative, successive P. aeruginosa cultures within 6 months

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    End point title
    		 Difference in P. aeruginosa eradication from the lower airways in the two treatment groups (A and B). Eradication is defined as 3 negative, successive P. aeruginosa cultures within 6 months
    End point description
    Difference in P. aeruginosa eradication from the lower airways in the two treatment groups (A and B). Eradication is defined as 3 negative, successive P. aeruginosa cultures within 6 months
    End point type
    Primary
    End point timeframe
    6 months
    End point values
    		 Standard of care - A Experimental - B
    Number of subjects analysed
    25 [1]
    26 [2]
    Units: Number of patients
        Success
    16
    14
        No success
    9
    12
    Notes
    [1] - According to the intention to treat analysis dropped-out patients were included
    [2] - According to the intention to treat analysis dropped-out patients were included
    Statistical analysis title
    		 Primary endpoint
    Comparison groups
    Standard of care - A v Experimental - B
    Number of subjects included in analysis
    51
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [3]
    Method
    Parameter type
    		 Risk difference (RD)
    Point estimate
    10.15
    Confidence interval
         level
    		 95%
         sides
    1-sided
         lower limit
    		 -16.7
         upper limit
    		 -
    Notes
    [3] - Intention to treat analysis was conducted and patients dropped-out were considered as treatment failure

    Primary: P. Aeruginosa recurrence risk

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    End point title
    		 P. Aeruginosa recurrence risk
    End point description
    We estimated the P. aeruginosa recurrence risk in the two groups and relative risk
    End point type
    Primary
    End point timeframe
    6 months
    End point values
    		 Standard of care - A Experimental - B
    Number of subjects analysed
    25 [4]
    26 [5]
    Units: Number of patients
        Success
    16
    14
        No success
    9
    12
    Notes
    [4] - According to the intention to treat analysis dropped-out patients were included
    [5] - According to the intention to treat analysis dropped-out patients were included
    Statistical analysis title
    		 Primary endpoint
    Statistical analysis description
    We estimated the risk of recurrence in the Group B and the risk of recurrence in the Group A.
    Comparison groups
    Standard of care - A v Experimental - B
    Number of subjects included in analysis
    51
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Risk ratio (RR)
    Point estimate
    1.28
    Confidence interval
         level
    		 95%
         sides
    1-sided
         lower limit
    		 0.7
         upper limit
    		 -

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment period (28 days) and 6-months follow-up
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Experimental group - B
    Reporting group description
    		 Subjects in the experimental group received the classic eradication therapy as per standard of care with nasal lavage with colistin.

    Reporting group title
    Standard of care group - A
    Reporting group description
    		 Subjects in the standard of care group received the classic eradication therapy.

    Serious adverse events
    		 Experimental group - B Standard of care group - A
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 22 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Haemoptysis
    Additional description: Patient experienced haemoptysis (related to cystic fibrosis) and hospitalization occurred in the reporting period.
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 22 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Experimental group - B Standard of care group - A
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 22 (0.00%)
    Respiratory, thoracic and mediastinal disorders
    Haemoptysis
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 22 (0.00%)
         occurrences all number
    1
    0

    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.
    This study is under-powered as the trial did not enrol the initial estimated sample.
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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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