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    Clinical Trial Results:
    A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

    Summary
    EudraCT number
    		 2018-003970-28
    Trial protocol
    		 GB   BE   NL   DE  
    Global end of trial date
    20 Aug 2020

    Results information
    Results version number
    		 v2(current)
    This version publication date
    19 Feb 2022
    First version publication date
    25 Nov 2021
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Updates based on NIH comment addressal

    Trial information

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    Trial identification
    Sponsor protocol code
    VX18-561-101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03911713
    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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    03 Sep 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Aug 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Aug 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of VX-561 in cystic fibrosis (CF) subjects.
    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
    17 Apr 2019
    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 States: 54
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Australia: 5
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Ireland: 6
    Country: Number of subjects enrolled
    United Kingdom: 3
    Worldwide total number of subjects
    77
    EEA total number of subjects
    15
    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)
    76
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in CF subjects aged 18 years or older who have a gating mutation and were previously taking stable dose of ivacaftor (IVA).

    Period 1
    Period 1 title
    Overall Period
    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
    		 Ivacaftor
    Arm description
    		 Subjects received IVA 150 milligrams (mg) in the treatment period for 12 weeks.
    Arm type
    		 Active comparator

    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 (q12h).

    Arm title
    		 VX-561: 25 mg
    Arm description
    		 Subjects received VX-561 25 mg in the treatment period for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-561
    Investigational medicinal product code
    CTP-656
    Other name
    Deutivacaftor (D-IVA)
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-561 25 mg once daily (qd).

    Arm title
    		 VX-561: 50 mg
    Arm description
    		 Subjects received VX-561 50 mg in the treatment period for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-561
    Investigational medicinal product code
    CTP-656
    Other name
    D-IVA
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-561 50 mg qd.

    Arm title
    		 VX-561: 150 mg
    Arm description
    		 Subjects received VX-561 150 mg in the treatment period for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-561
    Investigational medicinal product code
    CTP-656
    Other name
    D-IVA
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-561 150 mg qd.

    Arm title
    		 VX-561: 250 mg
    Arm description
    		 Subjects received VX-561 250 mg in the treatment period for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-561
    Investigational medicinal product code
    CTP-656
    Other name
    D-IVA
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-561 250 mg qd.

    Number of subjects in period 1 [1]
    		Ivacaftor VX-561: 25 mg VX-561: 50 mg VX-561: 150 mg VX-561: 250 mg
    Started
    11
    6
    11
    23
    24
    Completed
    11
    4
    11
    22
    24
    Not completed
    0
    2
    0
    1
    0
         Other
    -
    -
    -
    1
    -
         Lost to follow-up
    -
    2
    -
    -
    -
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: A total of 77 subjects were enrolled in the study, out of those 77 subjects, 2 subjects were randomized but not dosed in the treatment period. Therefore, only 75 subjects were included in the subject disposition and baseline sections.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ivacaftor
    Reporting group description
    		 Subjects received IVA 150 milligrams (mg) in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 25 mg
    Reporting group description
    		 Subjects received VX-561 25 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 50 mg
    Reporting group description
    		 Subjects received VX-561 50 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 150 mg
    Reporting group description
    		 Subjects received VX-561 150 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 250 mg
    Reporting group description
    		 Subjects received VX-561 250 mg in the treatment period for 12 weeks.

    Reporting group values
    		 Ivacaftor VX-561: 25 mg VX-561: 50 mg VX-561: 150 mg VX-561: 250 mg Total
    Number of subjects
    		 11 6 11 23 24 75
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 33.3 ± 11.7 33.0 ± 10.6 27.8 ± 9.0 32.5 ± 8.5 37.4 ± 11.4 -
    Gender categorical
    Units: Subjects
        Female
    		 4 2 3 8 9 26
        Male
    		 7 4 8 15 15 49
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0 1 1 0 2
        Not Hispanic or Latino
    		 11 6 10 22 24 73
        Unknown or Not Reported
    		 0 0 0 0 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0 0
        Asian
    		 1 0 0 0 0 1
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0 0
        Black or African American
    		 0 0 0 0 0 0
        White
    		 10 6 11 23 24 74
        More than one race
    		 0 0 0 0 0 0
        Unknown or Not Reported
    		 0 0 0 0 0 0
    Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
    Units: percentage points
        arithmetic mean (standard deviation)
    		 74.0 ± 21.2 63.6 ± 22.4 66.8 ± 17.4 72.6 ± 17.3 73.9 ± 17.0 -

    End points

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    End points reporting groups
    Reporting group title
    Ivacaftor
    Reporting group description
    		 Subjects received IVA 150 milligrams (mg) in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 25 mg
    Reporting group description
    		 Subjects received VX-561 25 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 50 mg
    Reporting group description
    		 Subjects received VX-561 50 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 150 mg
    Reporting group description
    		 Subjects received VX-561 150 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 250 mg
    Reporting group description
    		 Subjects received VX-561 250 mg in the treatment period for 12 weeks.

    Primary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

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    End point title
    		 Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [1] [2]
    End point description
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Full analysis set (FAS) included all randomized subjects who have intended CF transmembrane conductance regulator gene (CFTR) genotype and received at least 1 dose of study drug in treatment period. VX-561:25 mg and VX-561:50 mg arms were discontinued at sponsor's discretion and it was specified in statistical plan that data will be reported for only IVA, VX-561:150 mg and VX-561:250 mg arms for this end point.
    End point type
    Primary
    End point timeframe
    From Baseline at Week 12
    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: The results for the primary endpoint are the within group change from baseline at Week 12, for each treatment group. No between group comparison are reported.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: VX-561:25 mg and VX-561:50 mg arms were discontinued at sponsor’s discretion and were not included in mixed-effects model for repeated measures (MMRM) analysis as pre-specified in analysis plan. Therefore data are reported for IVA, VX-561:150 mg and VX-561:250 mg arms for this outcome measure.
    End point values
    		 Ivacaftor VX-561: 150 mg VX-561: 250 mg
    Number of subjects analysed
    11
    23
    24
    Units: percentage points
        least squares mean (confidence interval 95%)
    -0.8 (-6.2 to 4.7)
    3.1 (-0.8 to 7.0)
    2.7 (-1.0 to 6.5)
    No statistical analyses for this end point

    Secondary: Absolute Change in Sweat Chloride (SwCl)

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    End point title
    		 Absolute Change in Sweat Chloride (SwCl) [3]
    End point description
    Sweat samples were collected using an approved collection device. FAS. VX-561:25 mg and VX-561:50 mg arms were discontinued at sponsor's discretion and it was specified in statistical plan that data will be reported for only IVA, VX-561:150 mg and VX-561:250 mg arms for this end point.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 12
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: VX-561:25 mg and VX-561:50 mg arms were discontinued at sponsor’s discretion and were not included in MMRM analysis as pre-specified in analysis plan. Therefore data are reported for IVA, VX-561:150 mg and VX-561:250 mg arms for this outcome measure.
    End point values
    		 Ivacaftor VX-561: 150 mg VX-561: 250 mg
    Number of subjects analysed
    11
    23
    24
    Units: millimole per Liter (mmol/L)
        least squares mean (confidence interval 95%)
    0.9 (-9.5 to 11.3)
    3.3 (-4.6 to 11.2)
    -6.5 (-14.2 to 1.2)
    No statistical analyses for this end point

    Secondary: Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)

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    End point title
    		 Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
    End point description
    Pharmacokinetic (PK) set included all subjects who received at least 1 dose of study drug and for whom the primary PK data were considered to be sufficient and interpretable. Here "Subjects Analysed" signifies those participants who were evaluable for this end point and “n” signifies those subjects those who were evaluable for the specific category. Here, "99999" (penta nine) indicates that summary statistics were not reported as the number of observations below the limit of quantification was greater than 50% of the total number of observations at the specified time points and "999999"(hexa nine) indicates "not applicable" category for respective Ctrough assessment.
    End point type
    Secondary
    End point timeframe
    At Week 4
    End point values
    		 Ivacaftor VX-561: 25 mg VX-561: 50 mg VX-561: 150 mg VX-561: 250 mg
    Number of subjects analysed
    9
    4
    7
    20
    20
    Units: nanogram per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        VX-561: Week 4 (n = 0, 4, 7, 20, 20)
    999999 ± 999999
    26.1 ± 24.7
    123 ± 61.6
    458 ± 273
    1100 ± 856
        M1-VX-561: Week 4 (n = 0, 4, 7, 20, 20)
    999999 ± 999999
    18.1 ± 17.7
    108 ± 58.6
    378 ± 213
    739 ± 407
        M6-VX-561: Week 4 (n= 0, 3, 7, 20, 20)
    999999 ± 999999
    99999 ± 99999
    59.8 ± 34.0
    211 ± 189
    370 ± 233
        IVA: Week 4 (n= 9, 0, 0, 0, 0)
    952 ± 766
    999999 ± 999999
    999999 ± 999999
    999999 ± 999999
    999999 ± 999999
        M1-IVA: Week 4 (n = 9, 0, 0, 0, 0)
    1330 ± 774
    999999 ± 999999
    999999 ± 999999
    999999 ± 999999
    999999 ± 999999
        M6-IVA: Week 4 (n = 9, 0, 0, 0, 0)
    662 ± 398
    999999 ± 999999
    999999 ± 999999
    999999 ± 999999
    999999 ± 999999
    No statistical analyses for this end point

    Secondary: Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
    End point description
    Safety Set included all subjects who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 16
    End point values
    		 Ivacaftor VX-561: 25 mg VX-561: 50 mg VX-561: 150 mg VX-561: 250 mg
    Number of subjects analysed
    11
    6
    11
    23
    24
    Units: subjects
    number (not applicable)
        Subjects with AEs
    8
    4
    8
    21
    23
        Subjects with SAEs
    1
    2
    2
    2
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 16
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    VX-561: 250 mg
    Reporting group description
    		 Subjects received VX-561 250 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 25 mg
    Reporting group description
    		 Subjects received VX-561 25 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 150 mg
    Reporting group description
    		 Subjects received VX-561 150 mg in the treatment period for 12 weeks.

    Reporting group title
    Ivacaftor
    Reporting group description
    		 Subjects received IVA 150 mg in the treatment period for 12 weeks.

    Reporting group title
    VX-561: 50 mg
    Reporting group description
    		 Subjects received VX-561 50 mg in the treatment period for 12 weeks.

    Serious adverse events
    		 VX-561: 250 mg VX-561: 25 mg VX-561: 150 mg Ivacaftor VX-561: 50 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 24 (4.17%)
    2 / 6 (33.33%)
    2 / 23 (8.70%)
    1 / 11 (9.09%)
    2 / 11 (18.18%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Forced expiratory volume decreased
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Distal intestinal obstruction syndrome
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    2 / 23 (8.70%)
    1 / 11 (9.09%)
    2 / 11 (18.18%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    1 / 2
    0 / 1
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 VX-561: 250 mg VX-561: 25 mg VX-561: 150 mg Ivacaftor VX-561: 50 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    20 / 24 (83.33%)
    4 / 6 (66.67%)
    17 / 23 (73.91%)
    8 / 11 (72.73%)
    8 / 11 (72.73%)
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    Fatigue
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Malaise
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    6 / 23 (26.09%)
    2 / 11 (18.18%)
    1 / 11 (9.09%)
         occurrences all number
    1
    2
    9
    2
    1
    Dyspnoea
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 6 (33.33%)
    2 / 23 (8.70%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    2
    2
    0
    0
    Haemoptysis
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 6 (0.00%)
    3 / 23 (13.04%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    3
    0
    0
    Increased viscosity of bronchial secretion
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Lower respiratory tract congestion
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    2
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    4 / 23 (17.39%)
    2 / 11 (18.18%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    4
    2
    0
    Nasal congestion
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    2 / 23 (8.70%)
    2 / 11 (18.18%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    2
    2
    1
    Paranasal sinus discomfort
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    1 / 23 (4.35%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    0
    1
    Pulmonary pain
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Rales
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    1 / 23 (4.35%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    1
    0
    1
    Sputum increased
         subjects affected / exposed
    3 / 24 (12.50%)
    0 / 6 (0.00%)
    4 / 23 (17.39%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    3
    0
    6
    0
    1
    Respiration abnormal
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    4 / 23 (17.39%)
    2 / 11 (18.18%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    4
    2
    1
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    1 / 23 (4.35%)
    0 / 11 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    1
    1
    1
    0
    2
    Atypical mycobacterium test positive
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    0
    0
    1
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    2 / 11 (18.18%)
         occurrences all number
    1
    0
    0
    1
    2
    Bacterial test positive
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Blood glucose increased
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Coronavirus test positive
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Forced expiratory volume decreased
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    0
    0
    2
    Protein urine present
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Pulmonary function test decreased
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Urine ketone body present
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Weight decreased
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    Sinus headache
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Headache
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    1 / 23 (4.35%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    Migraine
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    2
    0
    0
    Blood and lymphatic system disorders
    Leukocytosis
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Tinnitus
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Eye disorders
    Glaucoma
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    2
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    Abdominal pain lower
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    Constipation
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    2
    0
    1
    0
    Duodenitis
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    0
    1
    1
    Skin and subcutaneous tissue disorders
    Hyperhidrosis
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    2
    0
    0
    0
    1
    Dermatitis contact
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Rash
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    Back pain
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Infections and infestations
    Bronchopulmonary aspergillosis allergic
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Epididymitis
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    0
    1
    Fungal skin infection
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    4 / 24 (16.67%)
    0 / 6 (0.00%)
    2 / 23 (8.70%)
    2 / 11 (18.18%)
    2 / 11 (18.18%)
         occurrences all number
    5
    0
    3
    2
    2
    Nasopharyngitis
         subjects affected / exposed
    3 / 24 (12.50%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    2 / 11 (18.18%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    0
    4
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 6 (0.00%)
    1 / 23 (4.35%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    1
    1
    0
    Oral candidiasis
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    1
    1
    0
    1
    0
    Sinusitis
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    1
    0
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 6 (0.00%)
    0 / 23 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Hyperkalaemia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 6 (16.67%)
    0 / 23 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Oct 2019
    Amended to remove VX-561: 25 mg and VX-561: 50 mg arms and updated planned efficacy and pharmacodynamic analyses. Revised the sample size and power calculation to account for increased enrollment to the VX-561: 150 mg, VX-561: 250 mg and Ivacaftor arms.

    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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