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    Clinical Trial Results:
    A Phase 2a, Two-part, Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation With and Without Oral Ivacaftor in Subjects With Primary Ciliary Dyskinesia

    Summary
    EudraCT number
    		 2015-004917-26
    Trial protocol
    		 DE   DK   NL   PL   GB   IT  
    Global end of trial date
    20 Nov 2018

    Results information
    Results version number
    		 v1
    This version publication date
    02 Jun 2019
    First version publication date
    02 Jun 2019
    Other versions
    		 v2

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    PS-G202
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02871778
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Parion Sciences Inc.
    Sponsor organisation address
    2800 Meridian Parkway, Durham, NC, United States,
    Public contact
    Parion Sciences Inc., Clinical Operations, +1 919 313 1210, nchurch@parion.com
    Scientific contact
    Parion Sciences Inc., Clinical Operations, +1 919 313 1210, nchurch@parion.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
    06 Feb 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Nov 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Nov 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and efficacy of treatment with VX-371 in Part A and ivacaftor (IVA) with VX-371 in Part B, administered with and without 4.2% hypertonic saline (HS) in subjects with primary ciliary dyskinesia (PCD) who are >=12 years of age.
    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
    23 Aug 2016
    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
    Netherlands: 7
    Country: Number of subjects enrolled
    Poland: 17
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Denmark: 14
    Country: Number of subjects enrolled
    Germany: 14
    Country: Number of subjects enrolled
    Canada: 9
    Country: Number of subjects enrolled
    Italy: 11
    Country: Number of subjects enrolled
    United States: 43
    Worldwide total number of subjects
    123
    EEA total number of subjects
    71
    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)
    36
    Adults (18-64 years)
    87
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Subjects with PCD greater than 12 years of age were enrolled in the study. Subjects with cystic fibrosis (CF) or only 1 mutation in the CF transmembrane regulator (CFTR) gene and a sweat chloride test>=60 millimoles by quantitative pilocarpine inotophresis were excluded.

    Pre-assignment
    Screening details
    		 This study included 2 parts (Part A and B). Part A consisted of 2 treatment periods (Treatment Period 1 and 2) separated by a 28-day Washout Period. Part B (optional) consisted of one treatment period (Treatment Period 3). Subjects who enrolled in Part B received IVA in addition to the treatment they were receiving in Treatment Period 2 of Part A.

    Period 1
    Period 1 title
    Part A (Treatment period 1)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part A: VX-371 in HS
    Arm description
    		 Subjects received VX-371 in 4.2% HS in treatment period 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-371
    Investigational medicinal product code
    P-1037
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received VX-371 in 4.2% HS twice daily from Day 1 through Day 29 in treatment period 1.

    Arm title
    		 Part A: HS
    Arm description
    		 Subjects received 4.2% HS in treatment period 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    HS
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received 4.2% HS twice daily from Day 1 through Day 29 in treatment period 1.

    Arm title
    		 Part A: VX-371
    Arm description
    		 Subjects received VX-371 in 0.17% saline in treatment period 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-371
    Investigational medicinal product code
    P-1037
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received VX-371 in 0.17% saline twice daily from Day 1 through Day 29 in treatment period 1.

    Arm title
    		 Part A: Placebo
    Arm description
    		 Subjects received Placebo (0.17% saline) in treatment period 1.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received Placebo (0.17% saline) twice daily from Day 1 through Day 29 in treatment period 1.

    Number of subjects in period 1
    		Part A: VX-371 in HS Part A: HS Part A: VX-371 Part A: Placebo
    Started
    43
    41
    21
    18
    Completed
    40
    37
    18
    16
    Not completed
    3
    4
    3
    2
         Non-Compliance with Study Drug
    -
    -
    1
    -
         Other
    -
    1
    1
    -
         Adverse event
    1
    1
    1
    -
         Withdrawal of Consent (not due to AE)
    2
    2
    -
    2
    Period 2
    Period 2 title
    Part A (Treatment period 2)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part A: HS
    Arm description
    		 Subjects who received VX-371 in 4.2% HS in treatment period 1, followed by a washout period, received 4.2% HS in treatment period 2.
    Arm type
    		 Experimental

    Investigational medicinal product name
    HS
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received 4.2% HS twice daily from Day 57 through Day 85 in treatment period 2.

    Arm title
    		 Part A: VX-371 in HS
    Arm description
    		 Subjects who received 4.2% HS in treatment period 1, followed by a washout period, received VX-371 in 4.2% HS in treatment period 2.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-371
    Investigational medicinal product code
    P-1037
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received VX-371 in 4.2% HS twice daily from Day 57 through Day 85 in treatment period 2.

    Arm title
    		 Part A: Placebo
    Arm description
    		 Subjects who received VX-371 in 0.17% saline in treatment period 1, followed by a washout period, received Placebo (0.17% saline) in treatment period 2.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received Placebo (0.17% saline) twice daily from Day 57 through Day 85 in treatment period 2.

    Arm title
    		 Part A: VX-371
    Arm description
    		 Subjects who received Placebo (0.17% saline) in treatment period 1, followed by a washout period, received VX-371 in 0.17% saline in treatment period 2.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-371
    Investigational medicinal product code
    P-1037
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received VX-371 in 0.17% saline twice daily from Day 57 through Day 85 in treatment period 2.

    Number of subjects in period 2
    		Part A: HS Part A: VX-371 in HS Part A: Placebo Part A: VX-371
    Started
    40
    37
    18
    16
    Completed
    37
    35
    17
    15
    Not completed
    3
    2
    1
    1
         Non-Compliance with Study Drug
    1
    -
    1
    -
         Adverse event
    2
    2
    -
    -
         Lost to follow-up
    -
    -
    -
    1
    Period 3
    Period 3 title
    Part B (Treatment period 3)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Assessor, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part B: VX-371 in HS + IVA
    Arm description
    		 Subjects who received VX-371 in 4.2% HS in treatment period 2, received VX-371 in 4.2% HS and IVA in optional treatment period 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-371
    Investigational medicinal product code
    P-1037
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received VX-371 in 4.2% HS twice daily from Day 85 through Day 113 in treatment period 3.

    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 tablet orally twice daily from Day 85 through Day 113 in treatment period 3.

    Arm title
    		 Part B: HS + IVA
    Arm description
    		 Subjects who received 4.2% HS in treatment period 2, received 4.2% HS and IVA in optional treatment period 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    HS
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received 4.2% HS twice daily from Day 85 through Day 113 in treatment period 3.

    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 twice daily from Day 85 through Day 113 in treatment period 3.

    Arm title
    		 Part B: VX-371 + IVA
    Arm description
    		 Subjects who received VX-371 in 0.17% saline in treatment period 2, received VX-371 in 0.17% saline and IVA in optional treatment period 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-371
    Investigational medicinal product code
    P-1037
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received VX-371 in 0.17% saline twice daily from Day 85 through Day 113 in treatment period 3.

    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 twice daily from Day 85 through Day 113 in treatment period 3.

    Arm title
    		 Part B: Placebo + IVA
    Arm description
    		 Subjects who received Placebo (0.17% saline) in treatment period 2, received Placebo (0.17% saline) and IVA in optional treatment period 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Subjects received Placebo (0.17% saline) twice daily from Day 85 through Day 113 in treatment period 3.

    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 twice daily from Day 85 through Day 113 in treatment period 3.

    Number of subjects in period 3 [1]
    		Part B: VX-371 in HS + IVA Part B: HS + IVA Part B: VX-371 + IVA Part B: Placebo + IVA
    Started
    11
    27
    7
    12
    Completed
    10
    26
    6
    12
    Not completed
    1
    1
    1
    0
         Adverse event
    1
    1
    1
    -
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Out of 104 subjects who completed Part A, 57 subjects rolled-over to optional Part B.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Part A (Treatment period 1)
    Reporting group description
    		 All subjects who received at least 1 dose of study drug during Part A were included for presenting Baseline Characteristics.

    Reporting group values
    		 Part A (Treatment period 1) Total
    Number of subjects
    		 123 123
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 27.80 ( 12.954 ) -
    Gender categorical
    Units: Subjects
        Female
    		 78 78
        Male
    		 45 45

    End points

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    End points reporting groups
    Reporting group title
    Part A: VX-371 in HS
    Reporting group description
    		 Subjects received VX-371 in 4.2% HS in treatment period 1.

    Reporting group title
    Part A: HS
    Reporting group description
    		 Subjects received 4.2% HS in treatment period 1.

    Reporting group title
    Part A: VX-371
    Reporting group description
    		 Subjects received VX-371 in 0.17% saline in treatment period 1.

    Reporting group title
    Part A: Placebo
    Reporting group description
    		 Subjects received Placebo (0.17% saline) in treatment period 1.
    Reporting group title
    Part A: HS
    Reporting group description
    		 Subjects who received VX-371 in 4.2% HS in treatment period 1, followed by a washout period, received 4.2% HS in treatment period 2.

    Reporting group title
    Part A: VX-371 in HS
    Reporting group description
    		 Subjects who received 4.2% HS in treatment period 1, followed by a washout period, received VX-371 in 4.2% HS in treatment period 2.

    Reporting group title
    Part A: Placebo
    Reporting group description
    		 Subjects who received VX-371 in 0.17% saline in treatment period 1, followed by a washout period, received Placebo (0.17% saline) in treatment period 2.

    Reporting group title
    Part A: VX-371
    Reporting group description
    		 Subjects who received Placebo (0.17% saline) in treatment period 1, followed by a washout period, received VX-371 in 0.17% saline in treatment period 2.
    Reporting group title
    Part B: VX-371 in HS + IVA
    Reporting group description
    		 Subjects who received VX-371 in 4.2% HS in treatment period 2, received VX-371 in 4.2% HS and IVA in optional treatment period 3.

    Reporting group title
    Part B: HS + IVA
    Reporting group description
    		 Subjects who received 4.2% HS in treatment period 2, received 4.2% HS and IVA in optional treatment period 3.

    Reporting group title
    Part B: VX-371 + IVA
    Reporting group description
    		 Subjects who received VX-371 in 0.17% saline in treatment period 2, received VX-371 in 0.17% saline and IVA in optional treatment period 3.

    Reporting group title
    Part B: Placebo + IVA
    Reporting group description
    		 Subjects who received Placebo (0.17% saline) in treatment period 2, received Placebo (0.17% saline) and IVA in optional treatment period 3.

    Subject analysis set title
    Part A: VX-371 in HS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects who received VX-371 in 4.2% HS in treatment period 1 or in treatment period 2.

    Subject analysis set title
    Part A: HS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects who received 4.2% HS in treatment period 1 or in treatment period 2.

    Subject analysis set title
    Part A: VX-371
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects who received VX-371 in 0.17% saline in treatment period 1 or in treatment period 2.

    Subject analysis set title
    Part A: Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects who received Placebo (0.17% saline) in treatment period 1 or in treatment period 2.

    Primary: Part A: Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Part A: Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs) [1]
    End point description
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 28 days after last dose of study drug
    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: Only descriptive statistics were planned. No statistical comparisons were planned for primary safety endpoint.
    End point values
    		 Part A: VX-371 in HS Part A: HS Part A: VX-371 Part A: Placebo
    Number of subjects analysed
    81
    80
    37
    36
    Units: Subjects
        Subjects with AEs
    52
    46
    22
    23
        Subjects with SAEs
    1
    1
    1
    1
    No statistical analyses for this end point

    Primary: Part B: Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Part B: Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs) [2]
    End point description
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 28 days after last dose of study drug
    Notes
    [2] - 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: Only descriptive statistics were planned. No statistical comparisons were planned for primary safety endpoint.
    End point values
    		 Part B: VX-371 in HS + IVA Part B: HS + IVA Part B: VX-371 + IVA Part B: Placebo + IVA
    Number of subjects analysed
    11
    27
    7
    12
    Units: Subjects
        Subjects with AEs
    5
    17
    5
    9
        Subjects with SAEs
    0
    0
    1
    0
    No statistical analyses for this end point

    Primary: Part A : Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

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    End point title
    		 Part A : Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
    End point description
    End point type
    Primary
    End point timeframe
    Study Baseline, 28 Days Post-dose in each treatment period
    End point values
    		 Part A: VX-371 in HS Part A: HS Part A: VX-371 Part A: Placebo
    Number of subjects analysed
    78
    75
    34
    34
    Units: percent predicted of FEV1
        least squares mean (standard error)
    0.99 ( 0.71 )
    -0.53 ( 0.72 )
    -0.49 ( 1.07 )
    -1.33 ( 1.07 )
    Statistical analysis title
    		 Statistical Analysis
    Statistical analysis description
    For ‘number of subjects included in the analysis’ field: total number of subjects analysed were 78 for VX-371 in HS arm and 75 for HS arm instead of 153 subjects because this study is a cross-over design and same subjects may have received both the interventions.
    Comparison groups
    Part A: VX-371 in HS v Part A: HS
    Number of subjects included in analysis
    153
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0437
    Method
    		 Mixed-effects Model
    Parameter type
    		 Least Square (LS) Mean difference
    Point estimate
    1.519
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.044
         upper limit
    		 2.995
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Part A: HS v Part A: VX-371
    Number of subjects included in analysis
    109
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.9755
    Method
    		 Mixed-effects Model
    Parameter type
    		 LS Mean difference
    Point estimate
    0.04
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.509
         upper limit
    		 2.589
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Part A: VX-371 in HS v Part A: Placebo
    Number of subjects included in analysis
    112
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0731
    Method
    		 Mixed-effects Model
    Parameter type
    		 LS Mean difference
    Point estimate
    2.318
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.22
         upper limit
    		 4.856
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Part A: HS v Part A: Placebo
    Number of subjects included in analysis
    109
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.5373
    Method
    		 Mixed-effects Model
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.799
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.751
         upper limit
    		 3.348
    Statistical analysis title
    		 Statistical Analysis
    Statistical analysis description
    For ‘number of subjects included in the analysis’ field: total number of subjects analysed were 34 for VX-371 arm and Placebo arm instead of 68 subjects because this study is a cross-over design and same subjects may have received both the interventions.
    Comparison groups
    Part A: VX-371 v Part A: Placebo
    Number of subjects included in analysis
    68
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.453
    Method
    		 Mixed-effects Model
    Parameter type
    		 LS Mean difference
    Point estimate
    0.838
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.368
         upper limit
    		 3.045

    Primary: Part B : Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

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    End point title
    		 Part B : Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [3]
    End point description
    End point type
    Primary
    End point timeframe
    Study Baseline, Part B Baseline and 28 Days post IVA dose in Part B
    Notes
    [3] - 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 endpoint for Part B was designed to perform within group statistical comparison. Because only between treatment statistical comparisons can be reported in the EudraCT database, no statistical analyses are reported for Part B endpoint.
    End point values
    		 Part B: VX-371 in HS + IVA Part B: HS + IVA Part B: VX-371 + IVA Part B: Placebo + IVA
    Number of subjects analysed
    10
    26
    6
    12
    Units: percent predicted of FEV1
    least squares mean (standard error)
        Change from study baseline
    4.72 ( 1.93 )
    1.72 ( 1.20 )
    -0.59 ( 2.50 )
    -0.97 ( 1.84 )
        Change from part B baseline
    2.53 ( 1.86 )
    1.68 ( 1.14 )
    -1.02 ( 2.38 )
    -2.04 ( 1.74 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug up to 28 days after last dose of study drug
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Part A: VX-371 in HS
    Reporting group description
    		 All subjects who received VX-371 in 4.2% HS in treatment period 1 or treatment period 2.

    Reporting group title
    Part A: HS
    Reporting group description
    		 All subjects who received 4.2% HS in treatment period 1 or treatment period 2.

    Reporting group title
    Part A: VX-371
    Reporting group description
    		 All subjects who received VX-371 in 0.17% saline in treatment period 1 or treatment period 2.

    Reporting group title
    Part A: Placebo
    Reporting group description
    		 All subjects who received Placebo (0.17% saline) in treatment period 1 or treatment period 2.

    Reporting group title
    Part B: VX-371 in HS + IVA
    Reporting group description
    		 All subjects who received VX-371 in 4.2% HS and IVA in treatment period 3.

    Reporting group title
    Part B: HS + IVA
    Reporting group description
    		 All subjects who received 4.2% HS and IVA in treatment period 3.

    Reporting group title
    Part B: VX-371 + IVA
    Reporting group description
    		 All subjects who received VX-371 in 0.17% saline and IVA in treatment period 3.

    Reporting group title
    Part B: Placebo + IVA
    Reporting group description
    		 All subjects who received Placebo (0.17% saline) and IVA in treatment period 3.

    Serious adverse events
    		 Part A: VX-371 in HS Part A: HS Part A: VX-371 Part A: Placebo Part B: VX-371 in HS + IVA Part B: HS + IVA Part B: VX-371 + IVA Part B: Placebo + IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 81 (1.23%)
    1 / 80 (1.25%)
    1 / 37 (2.70%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Respiratory, thoracic and mediastinal disorders
    Bronchiectasis
         subjects affected / exposed
    1 / 81 (1.23%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Haemoptysis
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Lung disorder
         subjects affected / exposed
    0 / 81 (0.00%)
    1 / 80 (1.25%)
    1 / 37 (2.70%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Part A: VX-371 in HS Part A: HS Part A: VX-371 Part A: Placebo Part B: VX-371 in HS + IVA Part B: HS + IVA Part B: VX-371 + IVA Part B: Placebo + IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    52 / 81 (64.20%)
    45 / 80 (56.25%)
    22 / 37 (59.46%)
    23 / 36 (63.89%)
    5 / 11 (45.45%)
    17 / 27 (62.96%)
    4 / 7 (57.14%)
    9 / 12 (75.00%)
    Investigations
    Haemophilus test positive
         subjects affected / exposed
    1 / 81 (1.23%)
    1 / 80 (1.25%)
    3 / 37 (8.11%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    3
    1
    0
    0
    0
    0
    Moraxella test positive
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    3 / 37 (8.11%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    3
    0
    0
    0
    0
    0
    Lipase increased
         subjects affected / exposed
    1 / 81 (1.23%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    0
    2
    Amylase increased
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    1
    Forced expiratory volume decreased
         subjects affected / exposed
    1 / 81 (1.23%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Hepatic enzyme increased
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    1 / 11 (9.09%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    0
    Streptococcus test positive
         subjects affected / exposed
    1 / 81 (1.23%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Weight decreased
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Vascular disorders
    Hot flush
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    2 / 27 (7.41%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    0
    0
    Nervous system disorders
    Dysgeusia
         subjects affected / exposed
    8 / 81 (9.88%)
    3 / 80 (3.75%)
    3 / 37 (8.11%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    9
    3
    4
    1
    0
    0
    0
    0
    Headache
         subjects affected / exposed
    8 / 81 (9.88%)
    7 / 80 (8.75%)
    2 / 37 (5.41%)
    6 / 36 (16.67%)
    3 / 11 (27.27%)
    4 / 27 (14.81%)
    0 / 7 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    10
    8
    3
    6
    5
    9
    0
    3
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    5 / 81 (6.17%)
    6 / 80 (7.50%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    3 / 27 (11.11%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    5
    6
    0
    1
    0
    3
    0
    0
    Pyrexia
         subjects affected / exposed
    4 / 81 (4.94%)
    1 / 80 (1.25%)
    2 / 37 (5.41%)
    2 / 36 (5.56%)
    0 / 11 (0.00%)
    3 / 27 (11.11%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    5
    1
    2
    2
    0
    4
    2
    0
    Fatigue
         subjects affected / exposed
    2 / 81 (2.47%)
    4 / 80 (5.00%)
    2 / 37 (5.41%)
    2 / 36 (5.56%)
    2 / 11 (18.18%)
    1 / 27 (3.70%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    2
    4
    3
    2
    2
    1
    1
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 81 (0.00%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Eye disorders
    Eye pruritus
         subjects affected / exposed
    1 / 81 (1.23%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    1
    0
    Vision blurred
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    1 / 37 (2.70%)
    2 / 36 (5.56%)
    0 / 11 (0.00%)
    1 / 27 (3.70%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    1 / 37 (2.70%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    5 / 27 (18.52%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    6
    3
    0
    Abdominal pain
         subjects affected / exposed
    0 / 81 (0.00%)
    2 / 80 (2.50%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    2 / 27 (7.41%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    0
    0
    0
    3
    0
    1
    Constipation
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    1 / 37 (2.70%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    2 / 27 (7.41%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    2
    1
    0
    Abdominal distension
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    4 / 81 (4.94%)
    0 / 80 (0.00%)
    1 / 37 (2.70%)
    1 / 36 (2.78%)
    1 / 11 (9.09%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    1
    1
    1
    0
    0
    0
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    1 / 37 (2.70%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    11 / 81 (13.58%)
    8 / 80 (10.00%)
    4 / 37 (10.81%)
    3 / 36 (8.33%)
    3 / 11 (27.27%)
    2 / 27 (7.41%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    12
    8
    5
    4
    3
    2
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    8 / 81 (9.88%)
    3 / 80 (3.75%)
    3 / 37 (8.11%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    2 / 27 (7.41%)
    2 / 7 (28.57%)
    1 / 12 (8.33%)
         occurrences all number
    8
    3
    3
    0
    0
    2
    2
    1
    Nasal congestion
         subjects affected / exposed
    5 / 81 (6.17%)
    3 / 80 (3.75%)
    1 / 37 (2.70%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    1 / 27 (3.70%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    5
    3
    1
    1
    0
    1
    0
    0
    Sputum increased
         subjects affected / exposed
    5 / 81 (6.17%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    1 / 11 (9.09%)
    1 / 27 (3.70%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    5
    1
    0
    0
    1
    1
    0
    0
    Rhinorrhoea
         subjects affected / exposed
    1 / 81 (1.23%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    3 / 27 (11.11%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    1
    0
    3
    0
    0
    Dyspnoea
         subjects affected / exposed
    2 / 81 (2.47%)
    2 / 80 (2.50%)
    1 / 37 (2.70%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    1 / 27 (3.70%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    2
    2
    1
    1
    0
    0
    0
    0
    Dysphonia
         subjects affected / exposed
    1 / 81 (1.23%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    1
    0
    Paranasal sinus hypersecretion
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    Sinus congestion
         subjects affected / exposed
    1 / 81 (1.23%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    0
    1
    0
    0
    0
    1
    Wheezing
         subjects affected / exposed
    2 / 81 (2.47%)
    2 / 80 (2.50%)
    1 / 37 (2.70%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    3
    2
    1
    0
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    Rash
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    2
    0
    Skin disorder
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    0
    Rash pruritic
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    1 / 11 (9.09%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 81 (0.00%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    2 / 36 (5.56%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    2
    0
    0
    0
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    4 / 81 (4.94%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    3 / 36 (8.33%)
    0 / 11 (0.00%)
    3 / 27 (11.11%)
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    4
    1
    0
    3
    0
    3
    1
    1
    Pseudomonas infection
         subjects affected / exposed
    1 / 81 (1.23%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    0 / 36 (0.00%)
    0 / 11 (0.00%)
    0 / 27 (0.00%)
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    1
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 81 (0.00%)
    1 / 80 (1.25%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    1 / 11 (9.09%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    1
    1
    0
    0
    0
    Sinusitis
         subjects affected / exposed
    0 / 81 (0.00%)
    0 / 80 (0.00%)
    0 / 37 (0.00%)
    1 / 36 (2.78%)
    1 / 11 (9.09%)
    0 / 27 (0.00%)
    0 / 7 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    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
    24 Feb 2016
    - Moved PCD genotype collection from Day 1 to Screening Visit - Added new exclusion criterion to exclude pregnant and/or nursing females
    06 Jun 2016
    - Modified contraception language
    15 Aug 2016
    - Modified contraception language

    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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