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    Clinical Trial Results:
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Effect of VX-770 on Lung Clearance Index in Subjects with Cystic Fibrosis, the G551D Mutation, and FEV1 >90% Predicted

    Summary
    EudraCT number
    2010-020546-96
    Trial protocol
    GB  
    Global end of trial date
    30 Nov 2011

    Results information
    Results version number
    v1(current)
    This version publication date
    28 Jun 2016
    First version publication date
    07 Aug 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX10-770-106
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01262352
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States, 022101862
    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)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-000335-PIP01-08
    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
    27 Jan 2012
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Nov 2011
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the effect of VX-770 on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D CFTR mutation on at least 1 allele.
    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
    14 Feb 2011
    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: 9
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    United States: 7
    Worldwide total number of subjects
    20
    EEA total number of subjects
    9
    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)
    8
    Adolescents (12-17 years)
    8
    Adults (18-64 years)
    4
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study started on 14 February 2011 (signing of first informed consent). After obtaining informed consent and assent (where applicable), screening evaluations were completed at any time during the period 10 to 18 days (Days -18 to -10) before first dose of study drug (Day 1).

    Pre-assignment
    Screening details
    A total of 21 subjects were randomized; 20 subjects received at least 1 dose of the study drug.

    Period 1
    Period 1 title
    Treatment Period 1
    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
    No

    Arm title
    Sequence 1: Ivacaftor Then Placebo
    Arm description
    Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Ivacaftor
    Investigational medicinal product code
    VX-770
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet of 150 mg of ivacaftor every 12 hours (q12h) for up to 28 days.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet q12h for up to 28 days.

    Arm title
    Sequence 2: Placebo Then Ivacaftor
    Arm description
    Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Ivacaftor
    Investigational medicinal product code
    VX-770
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet of 150 mg of ivacaftor q12h for up to 28 days.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet q12h for up to 28 days.

    Number of subjects in period 1
    Sequence 1: Ivacaftor Then Placebo Sequence 2: Placebo Then Ivacaftor
    Started
    10
    10
    Completed
    10
    10
    Period 2
    Period 2 title
    Treatment Period 2
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Sequence 1: Ivacaftor Then Placebo
    Arm description
    Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Ivacaftor
    Investigational medicinal product code
    VX-770
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet of 150 mg of ivacaftor q12h for up to 28 days.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet q12h for up to 28 days.

    Arm title
    Sequence 2: Placebo Then Ivacaftor
    Arm description
    Placebo administered in treatment period 1 and ivacaftor administered in treatment period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Ivacaftor
    Investigational medicinal product code
    VX-770
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet of 150 mg of ivacaftor q12h for up to 28 days.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet q12h for up to 28 days.

    Number of subjects in period 2
    Sequence 1: Ivacaftor Then Placebo Sequence 2: Placebo Then Ivacaftor
    Started
    9
    8
    Completed
    9
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sequence 1: Ivacaftor Then Placebo
    Reporting group description
    Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.

    Reporting group title
    Sequence 2: Placebo Then Ivacaftor
    Reporting group description
    Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Period 2.

    Reporting group values
    Sequence 1: Ivacaftor Then Placebo Sequence 2: Placebo Then Ivacaftor Total
    Number of subjects
    10 10 20
    Age categorical
    Units: Subjects
        <=18 years
    9 7 16
        Between 18 and 65 years
    1 3 4
        >=65 years
    0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    13.4 ± 7.12 19.8 ± 13.35 -
    Gender categorical
    Units: Subjects
        Female
    6 4 10
        Male
    4 6 10
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    0 0 0
        Not Hispanic or Latino
    10 10 20
        Unknown or Not Reported
    0 0 0
    Race (NIH/OMB)
    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
    10 10 20
        More than one race
    0 0 0
    Region of Enrollment
    Units: Subjects
        North America
    6 5 11
        Europe
    4 5 9
    Height
    Units: centimeters
        arithmetic mean (standard deviation)
    148.9 ± 19.54 156 ± 17.92 -
    Weight
    Units: kilograms
        arithmetic mean (standard deviation)
    45.06 ± 20.018 58.78 ± 30.576 -
    Body Mass Index
    Units: kilograms per square meter
        arithmetic mean (standard deviation)
    19.36 ± 3.707 22.66 ± 6.964 -
    Lung Clearance Index (LCI)
    Units: ratio
        arithmetic mean (standard deviation)
    9.17 ± 1.657 8.88 ± 1.462 -
    Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
    Units: percentage
        arithmetic mean (standard deviation)
    101.83 ± 11.587 92.58 ± 7.427 -
    Sweat Chloride
    Units: millimoles per liter
        arithmetic mean (standard deviation)
    97.1 ± 7.4 86.17 ± 19.219 -

    End points

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    End points reporting groups
    Reporting group title
    Sequence 1: Ivacaftor Then Placebo
    Reporting group description
    Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.

    Reporting group title
    Sequence 2: Placebo Then Ivacaftor
    Reporting group description
    Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Period 2.
    Reporting group title
    Sequence 1: Ivacaftor Then Placebo
    Reporting group description
    Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.

    Reporting group title
    Sequence 2: Placebo Then Ivacaftor
    Reporting group description
    Placebo administered in treatment period 1 and ivacaftor administered in treatment period 2.

    Subject analysis set title
    Placebo
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Oral tablet every 12 hours (q12h) for up to 28 days.

    Subject analysis set title
    Ivacaftor
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Oral tablet of 150 mg of ivacaftor q12h for up to 28 days.

    Primary: Absolute Change From Baseline in Lung Clearance Index (LCI) Through Day 29

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    End point title
    Absolute Change From Baseline in Lung Clearance Index (LCI) Through Day 29 [1]
    End point description
    Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal SF6 concentration to 1/40th of the starting value. Analysis was performed for all randomized subjects who received at least 1 dose of study drug (placebo or ivacaftor) and had available assessments during the time frame.
    End point type
    Primary
    End point timeframe
    Baseline through Day 29
    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 Analysis is provided in the attachment.
    End point values
    Placebo Ivacaftor
    Number of subjects analysed
    18
    17
    Units: ratio
        least squares mean (standard error)
    0.77 ± 0.291
    -1.3 ± 0.303
    Attachments
    Statistical Analysis
    No statistical analyses for this end point

    Secondary: Absolute Change From Baseline in ppFEV1 Through Day 29

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    End point title
    Absolute Change From Baseline in ppFEV1 Through Day 29
    End point description
    Spirometry (as measured by ppFEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies. Analysis was performed for all randomized subjects who received at least 1 dose of study drug (placebo or ivacaftor) and had available assessments during the time frame.
    End point type
    Secondary
    End point timeframe
    Baseline through Day 29
    End point values
    Placebo Ivacaftor
    Number of subjects analysed
    19
    18
    Units: percent
        least squares mean (standard error)
    0 ± 1.916
    7 ± 1.978
    Attachments
    Statistical Analysis
    No statistical analyses for this end point

    Secondary: Change From Baseline in Sweat Chloride Through Day 29

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    End point title
    Change From Baseline in Sweat Chloride Through Day 29
    End point description
    The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity. Analysis was performed for all randomized subjects who received at least 1 dose of study drug (placebo or ivacaftor) and had available assessments during the time frame.
    End point type
    Secondary
    End point timeframe
    Baseline through Day 29
    End point values
    Placebo Ivacaftor
    Number of subjects analysed
    18
    16
    Units: millimoles per liter
        least squares mean (standard error)
    0.11 ± 2.351
    -45.74 ± 2.632
    Attachments
    Statistical Analysis
    No statistical analyses for this end point

    Secondary: Change From Baseline in CF Questionnaire-Revised (CFQ-R) Score (Respiratory Domain Score, Pooled) Through Day 29

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    End point title
    Change From Baseline in CF Questionnaire-Revised (CFQ-R) Score (Respiratory Domain Score, Pooled) Through Day 29
    End point description
    The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID). Analysis was performed for all randomized subjects who received at least 1 dose of study drug (placebo or ivacaftor) and had available assessments during the time frame.
    End point type
    Secondary
    End point timeframe
    Baseline through Day 29
    End point values
    Placebo Ivacaftor
    Number of subjects analysed
    19
    18
    Units: score on a scale
        least squares mean (standard error)
    1.33 ± 3.067
    5.32 ± 3.166
    Attachments
    Statistical Analysis
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    For enrolled subjects, all adverse events were collected through the Follow-up Visit (4 weeks [+/-7 days] after the last dose of study drug).
    Adverse event reporting additional description
    For subjects who were screened but were not subsequently enrolled in the study, all adverse events were collected until the subject was deemed ineligible for the study. Treatment-emergent adverse events are reported here.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    12.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Oral tablet every 12 hours (q12h) for up to 28 days.

    Reporting group title
    Ivacaftor
    Reporting group description
    Oral tablet of 150 mg of ivacaftor q12h for up to 28 days.

    Serious adverse events
    Placebo Ivacaftor
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 19 (5.26%)
    2 / 18 (11.11%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Congenital, familial and genetic disorders
    Cystic fibrosis lung
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Distal ileal Obstruction syndrome
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Bronchopulmonary aspergillosis allergic
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Placebo Ivacaftor
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 19 (78.95%)
    13 / 18 (72.22%)
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 19 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 19 (15.79%)
    1 / 18 (5.56%)
         occurrences all number
    3
    1
    Fatigue
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Application site papules
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Nipple disorder
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Nipple pain
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Bite
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Fall
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Joint sprain
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Medical device complication
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Investigations
    Bacteria sputum identified
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Bacterial culture positive
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Lymph node palpable
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Prothrombin time prolonged
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Congenital, familial and genetic disorders
    Cystic fibrosis lung
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    7 / 19 (36.84%)
    5 / 18 (27.78%)
         occurrences all number
    10
    5
    Nasal congestion
         subjects affected / exposed
    2 / 19 (10.53%)
    1 / 18 (5.56%)
         occurrences all number
    2
    1
    Oropharyngeal pain
         subjects affected / exposed
    2 / 19 (10.53%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    Productive cough
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Epistaxis
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Nasal inflammation
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nasal oedema
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Rales
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Respiratory tract congestion
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Rhinorrhoea
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Sinus congestion
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    Sneezing
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 19 (5.26%)
    4 / 18 (22.22%)
         occurrences all number
    1
    4
    Dizziness
         subjects affected / exposed
    0 / 19 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    3
    Hypoaesthesia
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    4 / 19 (21.05%)
    1 / 18 (5.56%)
         occurrences all number
    4
    1
    Abdominal pain upper
         subjects affected / exposed
    2 / 19 (10.53%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    Constipation
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 18 (5.56%)
         occurrences all number
    1
    2
    Abdominal pain
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Abdominal tenderness
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Flatulence
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Hiatus hernia
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nausea
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Glycosuria
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Haematuria
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Rash macular
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Acne
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Blister
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Swelling face
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Back pain
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Glucose tolerance impaired
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Lower respiratory tract infection
         subjects affected / exposed
    2 / 19 (10.53%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal viral infection
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Mycobacterium abscessus infection
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Oral candidiasis
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Pneumococcal infection
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Sinusitis
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Staphylococcal infection
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Vulvovaginal mycotic infection
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 18 (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? Yes
    Date
    Amendment
    18 Jun 2010
    LCI assessments were updated; hypertonic saline restrictions were changed; discontinuation criteria related to liver tests and criteria for replacing subjects were updated.
    29 Jul 2010
    LCI measurements technique was clarified; minimum number of enrolled subjects was updated.
    07 Jan 2011
    ECG assessments were updated; interim analysis of the data was added.

    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.
    Statistical analysis is provided in attachment for individual endpoint.

    Online references

    http://www.ncbi.nlm.nih.gov/pubmed/24461666
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