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    Clinical Trial Results:
    An Exploratory Phase 2, 2-part, Randomized, Double blind, Placebo controlled Study With a Long term, Open label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

    Summary
    EudraCT number
    		 2017-003761-99
    Trial protocol
    		 DE  
    Global end of trial date
    07 Oct 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    22 Apr 2022
    First version publication date
    22 Apr 2022
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    VX16-809-121
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03625466
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States,
    Public contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
    Scientific contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    03 Nov 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Oct 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To explore the impact of lumacaftor (LUM)/ivacaftor (IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Conference on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    10 Aug 2018
    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
    Germany: 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)
    51
    Adolescents (12-17 years)
    0
    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
    		 This study was planned in 2 parts: Part 1 (Placebo-controlled Period) and Part 2 (Open-label Period). The primary and secondary efficacy analyses were planned for only Part 1.

    Pre-assignment
    Screening details
    		 This study was conducted in subjects with CF aged 2 to 5 years.

    Period 1
    Period 1 title
    Placebo-controlled Period (48 Weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part 1: Placebo
    Arm description
    		 Subjects received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo (matched to LUM/IVA)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to LUM/IVA twice daily.

    Arm title
    		 Part 1: LUM/IVA
    Arm description
    		 Subjects weighing less than (<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Subjects weighing greater than or equals to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/VX-770
    Other name
    Lumacaftor/Ivacaftor
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received LUM/IVA FDC twice daily.

    Number of subjects in period 1
    		Part 1: Placebo Part 1: LUM/IVA
    Started
    16
    35
    Completed
    16
    33
    Not completed
    0
    2
         Adverse Event
    -
    1
         Withdrawal of Consent (not due to AE)
    -
    1
    Period 2
    Period 2 title
    Open-label Period (48 Weeks)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Part 2: Overall LUM/IVA
    Arm description
    		 Subjects who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for subjects <6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for subjects >=6 years of age at week 48) in open-label period for 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received LUM/IVA FDC twice daily.

    Number of subjects in period 2
    		Part 2: Overall LUM/IVA
    Started
    49
    Completed
    48
    Not completed
    1
         Withdrawal of Consent (not due to AE)
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Part 1: Placebo
    Reporting group description
    		 Subjects received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.

    Reporting group title
    Part 1: LUM/IVA
    Reporting group description
    		 Subjects weighing less than (<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Subjects weighing greater than or equals to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.

    Reporting group values
    		 Part 1: Placebo Part 1: LUM/IVA Total
    Number of subjects
    		 16 35 51
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 4.2 ( 1.0 ) 4.2 ( 1.0 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 11 18
        Male
    		 9 24 33
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 16 35 51
        Unknown or Not Reported
    		 0 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 16 35 51
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Part 1: Placebo
    Reporting group description
    		 Subjects received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.

    Reporting group title
    Part 1: LUM/IVA
    Reporting group description
    		 Subjects weighing less than (<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Subjects weighing greater than or equals to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
    Reporting group title
    Part 2: Overall LUM/IVA
    Reporting group description
    		 Subjects who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for subjects <6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for subjects >=6 years of age at week 48) in open-label period for 48 weeks.

    Primary: Part 1: Absolute Change From Baseline in Magnetic Resonance Imaging (MRI) Global Chest Score at Week 48

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    End point title
    		 Part 1: Absolute Change From Baseline in Magnetic Resonance Imaging (MRI) Global Chest Score at Week 48
    End point description
    MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each subject had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = <50% of lobe involved and 2 = >=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement. Full Analysis Set (FAS) included all randomized subjects who carried the intended CF transmembrane conductance regulator gene (CFTR) allele mutation and received at least 1 dose of study drug in Part 1.Here "n" signifies those subjects who were evaluable at specified time points for each reporting group respectively.
    End point type
    Primary
    End point timeframe
    From Baseline at Week 48
    End point values
    		 Part 1: Placebo Part 1: LUM/IVA
    Number of subjects analysed
    16
    35
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline (n=15,34)
    21.4 ( 9.3 )
    17.6 ( 9.7 )
        Change at Week 48 (n=15,32)
    -0.3 ( 6.1 )
    -1.7 ( 6.6 )
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Part 1: Placebo v Part 1: LUM/IVA
    Number of subjects included in analysis
    51
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5.5
         upper limit
    		 2.6

    Secondary: Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48

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    End point title
    		 Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48
    End point description
    LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 48
    End point values
    		 Part 1: Placebo Part 1: LUM/IVA
    Number of subjects analysed
    16
    35
    Units: lung clearance index
        arithmetic mean (confidence interval 95%)
    0.32 (-0.20 to 0.84)
    -0.37 (-0.85 to 0.10)
    No statistical analyses for this end point

    Secondary: Part 1: Absolute Change in Weight-for-age Z-score at Week 48

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    End point title
    		 Part 1: Absolute Change in Weight-for-age Z-score at Week 48
    End point description
    The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 48
    End point values
    		 Part 1: Placebo Part 1: LUM/IVA
    Number of subjects analysed
    16
    35
    Units: z-score
        arithmetic mean (confidence interval 95%)
    -0.07 (-0.24 to 0.11)
    0.13 (-0.01 to 0.27)
    No statistical analyses for this end point

    Secondary: Part 1: Absolute Change in Stature-for-age Z-score at Week 48

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    End point title
    		 Part 1: Absolute Change in Stature-for-age Z-score at Week 48
    End point description
    The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 48
    End point values
    		 Part 1: Placebo Part 1: LUM/IVA
    Number of subjects analysed
    16
    35
    Units: z-score
        arithmetic mean (confidence interval 95%)
    0.10 (-0.04 to 0.24)
    0.09 (-0.05 to 0.22)
    No statistical analyses for this end point

    Secondary: Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48

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    End point title
    		 Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48
    End point description
    BMI was defined as weight in kilogram (kg) divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 48
    End point values
    		 Part 1: Placebo Part 1: LUM/IVA
    Number of subjects analysed
    16
    35
    Units: z-score
        arithmetic mean (confidence interval 95%)
    -0.24 (-0.55 to 0.07)
    0.20 (-0.02 to 0.41)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
    Adverse event reporting additional description
    MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1, 24.0
    Reporting groups
    Reporting group title
    Part 1: Placebo
    Reporting group description
    		 Subjects received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.

    Reporting group title
    Part 1: LUM/IVA
    Reporting group description
    		 Subjects weighing <14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.

    Reporting group title
    Part 2: Overall LUM/IVA
    Reporting group description
    		 Subjects who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for subjects <6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for subjects >=6 years of age at week 48) in open-label period for 48 weeks.

    Serious adverse events
    		 Part 1: Placebo Part 1: LUM/IVA Part 2: Overall LUM/IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 16 (12.50%)
    7 / 35 (20.00%)
    12 / 49 (24.49%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Sedation complication
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    2 / 49 (4.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haematemesis
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Intussusception
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Coeliac disease
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Distal intestinal obstruction syndrome
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Lung infiltration
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    1 / 16 (6.25%)
    3 / 35 (8.57%)
    3 / 49 (6.12%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 3
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Bronchopulmonary aspergillosis allergic
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Chronic sinusitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Otitis media
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pseudomonas bronchitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tonsillitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Part 1: Placebo Part 1: LUM/IVA Part 2: Overall LUM/IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    16 / 16 (100.00%)
    33 / 35 (94.29%)
    41 / 49 (83.67%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    3 / 35 (8.57%)
    4 / 49 (8.16%)
         occurrences all number
    0
    3
    6
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    2 / 49 (4.08%)
         occurrences all number
    0
    2
    3
    Staphylococcus test positive
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    3 / 49 (6.12%)
         occurrences all number
    1
    1
    4
    Pseudomonas test positive
         subjects affected / exposed
    2 / 16 (12.50%)
    1 / 35 (2.86%)
    1 / 49 (2.04%)
         occurrences all number
    2
    1
    1
    Injury, poisoning and procedural complications
    Skin laceration
         subjects affected / exposed
    1 / 16 (6.25%)
    2 / 35 (5.71%)
    0 / 49 (0.00%)
         occurrences all number
    2
    2
    0
    Skin abrasion
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Traumatic haematoma
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    1
    0
    1
    Arthropod bite
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 35 (0.00%)
    3 / 49 (6.12%)
         occurrences all number
    0
    0
    3
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 16 (12.50%)
    3 / 35 (8.57%)
    3 / 49 (6.12%)
         occurrences all number
    3
    5
    6
    General disorders and administration site conditions
    Peripheral swelling
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    3 / 16 (18.75%)
    6 / 35 (17.14%)
    2 / 49 (4.08%)
         occurrences all number
    4
    6
    2
    Ear and labyrinth disorders
    Otorrhoea
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Eye disorders
    Astigmatism
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    1 / 49 (2.04%)
         occurrences all number
    0
    2
    1
    Blepharospasm
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    2 / 16 (12.50%)
    1 / 35 (2.86%)
    2 / 49 (4.08%)
         occurrences all number
    2
    1
    2
    Abdominal pain
         subjects affected / exposed
    2 / 16 (12.50%)
    7 / 35 (20.00%)
    6 / 49 (12.24%)
         occurrences all number
    3
    7
    8
    Diarrhoea
         subjects affected / exposed
    1 / 16 (6.25%)
    4 / 35 (11.43%)
    3 / 49 (6.12%)
         occurrences all number
    1
    4
    3
    Faeces pale
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    2
    0
    0
    Constipation
         subjects affected / exposed
    0 / 16 (0.00%)
    4 / 35 (11.43%)
    2 / 49 (4.08%)
         occurrences all number
    0
    4
    5
    Abnormal faeces
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    1
    0
    1
    Vomiting
         subjects affected / exposed
    2 / 16 (12.50%)
    2 / 35 (5.71%)
    4 / 49 (8.16%)
         occurrences all number
    2
    2
    6
    Frequent bowel movements
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences all number
    1
    1
    0
    Post-tussive vomiting
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    1 / 49 (2.04%)
         occurrences all number
    1
    2
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    5 / 16 (31.25%)
    10 / 35 (28.57%)
    11 / 49 (22.45%)
         occurrences all number
    7
    12
    19
    Dyspnoea
         subjects affected / exposed
    2 / 16 (12.50%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences all number
    2
    1
    0
    Epistaxis
         subjects affected / exposed
    2 / 16 (12.50%)
    2 / 35 (5.71%)
    2 / 49 (4.08%)
         occurrences all number
    2
    2
    2
    Nasal polyps
         subjects affected / exposed
    2 / 16 (12.50%)
    1 / 35 (2.86%)
    1 / 49 (2.04%)
         occurrences all number
    2
    1
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 16 (0.00%)
    3 / 35 (8.57%)
    0 / 49 (0.00%)
         occurrences all number
    0
    3
    0
    Nasal obstruction
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    1
    0
    1
    Nasal congestion
         subjects affected / exposed
    4 / 16 (25.00%)
    0 / 35 (0.00%)
    8 / 49 (16.33%)
         occurrences all number
    4
    0
    10
    Lung infiltration
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Productive cough
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    4
    0
    0
    Wheezing
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinorrhoea
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    2 / 49 (4.08%)
         occurrences all number
    4
    0
    2
    Skin and subcutaneous tissue disorders
    Dry skin
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    0 / 49 (0.00%)
         occurrences all number
    0
    2
    0
    Rash
         subjects affected / exposed
    1 / 16 (6.25%)
    3 / 35 (8.57%)
    2 / 49 (4.08%)
         occurrences all number
    2
    3
    2
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Conjunctivitis
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    1 / 49 (2.04%)
         occurrences all number
    1
    1
    1
    Bronchitis
         subjects affected / exposed
    1 / 16 (6.25%)
    2 / 35 (5.71%)
    6 / 49 (12.24%)
         occurrences all number
    1
    2
    6
    Bacterial disease carrier
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 35 (0.00%)
    3 / 49 (6.12%)
         occurrences all number
    2
    0
    3
    Enterobiasis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    1
    0
    1
    Diarrhoea infectious
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Gastroenteritis
         subjects affected / exposed
    2 / 16 (12.50%)
    3 / 35 (8.57%)
    2 / 49 (4.08%)
         occurrences all number
    3
    3
    2
    Febrile infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    9 / 16 (56.25%)
    15 / 35 (42.86%)
    11 / 49 (22.45%)
         occurrences all number
    14
    25
    12
    Nasopharyngitis
         subjects affected / exposed
    8 / 16 (50.00%)
    22 / 35 (62.86%)
    19 / 49 (38.78%)
         occurrences all number
    16
    45
    31
    Otitis media acute
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    0
    0
    Otitis media
         subjects affected / exposed
    1 / 16 (6.25%)
    3 / 35 (8.57%)
    2 / 49 (4.08%)
         occurrences all number
    2
    4
    2
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    2
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 35 (5.71%)
    0 / 49 (0.00%)
         occurrences all number
    0
    2
    0
    Sinusitis
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    4 / 49 (8.16%)
         occurrences all number
    1
    2
    5
    Rhinitis
         subjects affected / exposed
    6 / 16 (37.50%)
    9 / 35 (25.71%)
    8 / 49 (16.33%)
         occurrences all number
    8
    10
    11
    Tonsillitis
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    1 / 49 (2.04%)
         occurrences all number
    1
    3
    1
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 16 (18.75%)
    1 / 35 (2.86%)
    2 / 49 (4.08%)
         occurrences all number
    5
    2
    2
    Viral infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 35 (0.00%)
    2 / 49 (4.08%)
         occurrences all number
    1
    0
    2
    Pneumonia pseudomonal
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 35 (2.86%)
    0 / 49 (0.00%)
         occurrences all number
    1
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    29 May 2018
    Amended to include safety data analysis by IDMC and additional flexibility was added to the schedule of assessments to reduce the burden on subjects.
    11 Jun 2018
    Amended to update study phase from Phase 3b to Phase 2.
    24 Jul 2019
    Amended to allow for a delay in the Week 48 and/or Week 96 MRI assessment.

    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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