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    Clinical Trial Results:
    A Randomized, Double-blind, Placebo-controlled, Crossover Study to Evaluate the Efficacy of Ivacaftor in Subjects with Cystic Fibrosis Who are 6 Years of Age and Older and Have Either a 3849 + 10KB C→T or D1152H-CFTR Mutation

    Summary
    EudraCT number
    		 2017-000457-39
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    18 Dec 2018

    Results information
    Results version number
    		 v2(current)
    This version publication date
    29 Feb 2020
    First version publication date
    04 Jul 2019
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Updated for Consistency with CT.gov Results

    Trial information

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    Trial identification
    Sponsor protocol code
    VX16-770-127
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03068312
    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
    22 Jan 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    18 Dec 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Dec 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of ivacaftor (IVA) treatment in subjects with cystic fibrosis (CF) 6 years of age and older who have a 3849 + 10KB C→T or D1152H CF transmembrane conductance regulator gene (CFTR) mutation.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Council on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    18 Jul 2017
    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
    Israel: 38
    Worldwide total number of subjects
    38
    EEA total number of subjects
    0
    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)
    4
    Adolescents (12-17 years)
    4
    Adults (18-64 years)
    30
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in subjects with CF.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Sequence 1: First IVA Then Placebo
    Arm description
    		 Subjects received IVA for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.
    Arm type
    		 Experimental

    Investigational medicinal product name
    IVA
    Investigational medicinal product code
    VX-770
    Other name
    Ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received IVA 150 milligram (mg) every 12 hours.

    Investigational medicinal product name
    Placebo (matched to IVA)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to IVA every 12 hours.

    Arm title
    		 Sequence 2: First Placebo Then IVA
    Arm description
    		 Subjects received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo (Matched to IVA)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to IVA every 12 hours.

    Investigational medicinal product name
    IVA
    Investigational medicinal product code
    VX-770
    Other name
    Ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received IVA 150 mg every 12 hours.

    Number of subjects in period 1
    		Sequence 1: First IVA Then Placebo Sequence 2: First Placebo Then IVA
    Started
    19
    19
    Completed
    19
    19

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sequence 1: First IVA Then Placebo
    Reporting group description
    		 Subjects received IVA for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

    Reporting group title
    Sequence 2: First Placebo Then IVA
    Reporting group description
    		 Subjects received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

    Reporting group values
    		 Sequence 1: First IVA Then Placebo Sequence 2: First Placebo Then IVA Total
    Number of subjects
    		 19 19 38
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 32.6 ( 15.3 ) 32.1 ( 15.6 ) -
    Gender categorical
    Units: Subjects
        Female
    		 10 10 20
        Male
    		 9 9 18
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 19 19 38
        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
    		 19 19 38
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0
    Lung Clearance Index 2.5 (LCI2.5)
    Measure 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.
    Units: Lung clearance index
        arithmetic mean (standard deviation)
    		 12.74 ( 4.04 ) 13.19 ( 5.45 ) -

    End points

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    End points reporting groups
    Reporting group title
    Sequence 1: First IVA Then Placebo
    Reporting group description
    		 Subjects received IVA for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

    Reporting group title
    Sequence 2: First Placebo Then IVA
    Reporting group description
    		 Subjects received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects who received placebo matched to IVA for 8 weeks in treatment period 1 or 2.

    Subject analysis set title
    IVA
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects who received IVA for 8 weeks in treatment period 1 or 2.

    Primary: Change in Lung Clearance Index 2.5

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    End point title
    		 Change in Lung Clearance Index 2.5
    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. The Full Analysis Set (FAS) included all randomized subjects who carried the intended CFTR allele mutation and received at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    From baseline through 8 weeks
    End point values
    		 Placebo IVA
    Number of subjects analysed
    37
    37
    Units: Lung clearance index
        least squares mean (standard error)
    0.20 ( 0.19 )
    -0.46 ( 0.19 )
    Statistical analysis title
    		 Placebo vs IVA
    Statistical analysis description
    The "number of subjects included in analysis" was 37 instead of 74 because this study has a cross-over design and same subjects received both the interventions. The number "74" is auto-calculated as the sum of numbers presented in 2 comparison groups and is appearing due to EudraCT database format constraints.
    Comparison groups
    Placebo v IVA
    Number of subjects included in analysis
    74
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    Method
    Parameter type
    		 Least squares mean difference
    Point estimate
    -0.66
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.1
         upper limit
    		 -0.21

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug up to safety follow-up visit (up to Week 28)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 All subjects who received placebo matched to IVA for 8 weeks in treatment period 1 or 2.

    Reporting group title
    Ivafactor
    Reporting group description
    		 All subjects who received IVA for 8 weeks in treatment period 1 or 2.

    Serious adverse events
    		 Placebo Ivafactor
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Pregnancy, puerperium and perinatal conditions
    Abortion spontaneous
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 38 (2.63%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Pancreatitis
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (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
    		 Placebo Ivafactor
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    19 / 38 (50.00%)
    12 / 38 (31.58%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 38 (5.26%)
         occurrences all number
    1
    2
    Malaise
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 38 (0.00%)
         occurrences all number
    3
    0
    Gastrointestinal disorders
    Aphthous ulcer
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 38 (0.00%)
         occurrences all number
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Haemoptysis
         subjects affected / exposed
    2 / 38 (5.26%)
    3 / 38 (7.89%)
         occurrences all number
    6
    4
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    2 / 38 (5.26%)
    5 / 38 (13.16%)
         occurrences all number
    2
    5
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 38 (15.79%)
    3 / 38 (7.89%)
         occurrences all number
    6
    3
    Viral upper respiratory tract infection
         subjects affected / exposed
    9 / 38 (23.68%)
    1 / 38 (2.63%)
         occurrences all number
    9
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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