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    Clinical Trial Results:
    Evaluation of safety and gene expression with a single dose of pGM169/GL67A administered to the nose and lung of individuals with cystic fibrosis

    Summary
    EudraCT number
    		 2007-004050-85
    Trial protocol
    		 GB  
    Global end of trial date
    08 Jun 2011

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Jun 2019
    First version publication date
    07 Jun 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    cro851
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Imperial College London
    Sponsor organisation address
    1b Manresa road, London, United Kingdom, SW3 6LR
    Public contact
    Samia Soussi, Imperial College London, +44 2075947980, s.soussi@imperial.ac.uk
    Scientific contact
    Samia Soussi, Imperial College London, +44 2075947980, s.soussi@imperial.ac.uk
    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
    01 Dec 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Jun 2011
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Jun 2011
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the safety and gene transfer resulting from a single dose of pGM169/GL67A to the lungs and nose of patients with cystic fibrosis
    Protection of trial subjects
    Patients were fully informed at enrolment of all study procedures
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Feb 2009
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 35
    Worldwide total number of subjects
    35
    EEA total number of subjects
    35
    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)
    35
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Adults (>16 years) with cystic fibrosis attending the Royal Brompton Hospital fulfilling the inclusion/ exclusion criteria

    Pre-assignment period milestones
    Number of subjects started
    		 35
    Number of subjects completed
    		 35

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 20ml pGM169/GL67A
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    pGM169/GL67
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nasal/oromucosal spray, solution, Nebuliser solution
    Routes of administration
    Inhalation use, Nasal use
    Dosage and administration details
    Single nebulised and nasal dose pGM169/GL67A: Plasmid expressing the human CFTR gene complexed with cationic lipid 67 and helper lipids: 10 ml nebulised and 1 ml nasal administration (n=3) and 20 ml nebulised and 2 ml nasal (n=24)

    Arm title
    		 10ml pGM169/GL67A
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    pGM169/GL67
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution, Nasal/oromucosal spray, solution
    Routes of administration
    Nasal use, Inhalation use
    Dosage and administration details
    Single nebulised and nasal dose pGM169/GL67A: Plasmid expressing the human CFTR gene complexed with cationic lipid 67 and helper lipids: 10 ml nebulised and 1 ml nasal administration (n=3) and 20 ml nebulised and 2 ml nasal (n=24)

    Arm title
    		 5ml pGM169/GL67A
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    pGM169/GL67
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution, Nasal/oromucosal spray, solution
    Routes of administration
    Inhalation use, Nasal use
    Dosage and administration details
    Single nebulised and nasal dose pGM169/GL67A: Plasmid expressing the human CFTR gene complexed with cationic lipid 67 and helper lipids: 10 ml nebulised and 1 ml nasal administration (n=3) and 20 ml nebulised and 2 ml nasal (n=24)

    Number of subjects in period 1
    		20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Started
    17
    10
    8
    Completed
    17
    10
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    20ml pGM169/GL67A
    Reporting group description
    		 -

    Reporting group title
    10ml pGM169/GL67A
    Reporting group description
    		 -

    Reporting group title
    5ml pGM169/GL67A
    Reporting group description
    		 -

    Reporting group values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A Total
    Number of subjects
    		 17 10 8 35
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0
        Adolescents (12-17 years)
    		 1 0 1 2
        Adults (18-64 years)
    		 16 10 7 33
        From 65-84 years
    		 0 0 0 0
        85 years and over
    		 0 0 0 0
    Age continuous
    Units: years
        median (full range (min-max))
    		 26.7 (17.3 to 50.1) 33.2 (16.4 to 61.6) 32.6 (24.3 to 46.4) -
    Gender categorical
    Units: Subjects
        Female
    		 5 4 3 12
        Male
    		 12 6 5 23
    Subject analysis sets

    Subject analysis set title
    20ml pGM169/GL67A
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    20ml pGM169/GL67A

    Subject analysis set title
    10ml pGM169/GL67A
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    10ml pGM169/GL67A

    Subject analysis set title
    5ml pGM169/GL67A
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    5ml pGM169/GL67A

    Subject analysis sets values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects
    17
    10
    8
    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)
    0
    0
    0
        Adolescents (12-17 years)
    1
    0
    1
        Adults (18-64 years)
    16
    10
    7
        From 65-84 years
    0
    0
    0
        85 years and over
    0
    0
    0
    Age continuous
    Units: years
        median (full range (min-max))
    26.7 (17.3 to 50.1)
    33.2 (16.4 to 61.6)
    32.6 (24.3 to 46.4)
    Gender categorical
    Units: Subjects
        Female
        Male

    End points

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    End points reporting groups
    Reporting group title
    20ml pGM169/GL67A
    Reporting group description
    		 -

    Reporting group title
    10ml pGM169/GL67A
    Reporting group description
    		 -

    Reporting group title
    5ml pGM169/GL67A
    Reporting group description
    		 -

    Subject analysis set title
    20ml pGM169/GL67A
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    20ml pGM169/GL67A

    Subject analysis set title
    10ml pGM169/GL67A
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    10ml pGM169/GL67A

    Subject analysis set title
    5ml pGM169/GL67A
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    5ml pGM169/GL67A

    Primary: Fever Maximum temperature

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    End point title
    		 Fever Maximum temperature
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: celsius temperature
        arithmetic mean (standard deviation)
    38.6 ( 0.5 )
    38.0 ( 0.7 )
    37.4 ( 0.3 )
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data were compared with analysis of variance, Kruskall-Wallis, or Fisher's extract
    Comparison groups
    20ml pGM169/GL67A v 10ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    P-value
    		 = 0.0002
    Method
    		 Kruskal-wallis
    Confidence interval

    Primary: Blood leukocytes

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    End point title
    		 Blood leukocytes
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: x10E9/L
        arithmetic mean (standard deviation)
    15.8 ( 3.2 )
    14.1 ( 4.5 )
    12.8 ( 3.8 )
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data were compared with analysis of variance, Kruskall-Wallis, or Fisher's extract
    Comparison groups
    20ml pGM169/GL67A v 10ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.22
    Method
    		 Kruskal-wallis
    Confidence interval

    Primary: Blood neutrophils

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    End point title
    		 Blood neutrophils
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: x10E9/L
        arithmetic mean (standard deviation)
    13.9 ( 3.4 )
    11.3 ( 4.1 )
    9.8 ( 3.5 )
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data were compared with analysis of variance, Kruskall-Wallis, or Fisher's extract
    Comparison groups
    20ml pGM169/GL67A v 10ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.06
    Method
    		 Kruskal-wallis
    Confidence interval

    Primary: Serum C-reactive protein

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    End point title
    		 Serum C-reactive protein
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: mg/L
        median (full range (min-max))
    59 (36 to 73)
    51 (25 to 88)
    38 (5 to 78)
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data were compared with analysis of variance, Kruskall-Wallis, or Fisher's extract
    Comparison groups
    20ml pGM169/GL67A v 10ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.4
    Method
    		 Kruskal-wallis
    Confidence interval

    Primary: FEV1 relative % drop

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    End point title
    		 FEV1 relative % drop
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: percent
        arithmetic mean (standard deviation)
    24.6 ( 9.3 )
    17.5 ( 7.8 )
    16.8 ( 4.0 )
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data were compared with analysis of variance, Kruskall-Wallis, or Fisher's extract
    Comparison groups
    10ml pGM169/GL67A v 20ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.08
    Method
    		 Kruskal-wallis
    Confidence interval

    Primary: FVC relative % drop

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    End point title
    		 FVC relative % drop
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: percent
        arithmetic mean (standard deviation)
    20.7 ( 2.9 )
    13.7 ( 2.2 )
    14.7 ( 2.2 )
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data analysis compared with analysis of variance, Kruskall-Wallis, or Fisher's extract tests
    Comparison groups
    20ml pGM169/GL67A v 10ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.22
    Method
    		 Kruskal-wallis
    Confidence interval

    Primary: LCI

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    End point title
    		 LCI
    End point description
    End point type
    Primary
    End point timeframe
    Post dose
    End point values
    		 20ml pGM169/GL67A 10ml pGM169/GL67A 5ml pGM169/GL67A
    Number of subjects analysed
    15
    6
    6
    Units: unit(s)
        arithmetic mean (standard deviation)
    0.75 ( 0.3 )
    0.32 ( 0.1 )
    0.32 ( 0.1 )
    Statistical analysis title
    		 Three group data analysis
    Statistical analysis description
    Three group data analysis compared with analysis of variance, Kruskall-Wallis, or Fisher's extract tests
    Comparison groups
    20ml pGM169/GL67A v 10ml pGM169/GL67A v 5ml pGM169/GL67A
    Number of subjects included in analysis
    27
    Analysis specification
    Post-hoc
    Analysis type
    		 other
    P-value
    		 = 0.03
    Method
    		 Kruskal-wallis
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Post dose
    Adverse event reporting additional description
    Mild self limiting influenza like systemic response
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    10
    Reporting groups
    Reporting group title
    pGM169/GL67A
    Reporting group description
    		 -

    Serious adverse events
    		 pGM169/GL67A
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 35 (5.71%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Respiratory, thoracic and mediastinal disorders
    Injury in luvula
    Additional description: Injury in luvula sustained from pressure of tube during bronchoscopy 03/04/2009
         subjects affected / exposed
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Endocrine disorders
    Acute pancreatitis
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 35 (2.86%)
         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
    		 pGM169/GL67A
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    33 / 35 (94.29%)
    Immune system disorders
    Mild self limiting influenza like systemic response
         subjects affected / exposed
    33 / 35 (94.29%)
         occurrences all number
    33

    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
    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.

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