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    Clinical Trial Results:
    A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

    Summary
    EudraCT number
    		 2021-005320-38
    Trial protocol
    		 ES   DE   FR   BE   AT   IT   PT   NL   SE   CZ   NO   HU   PL  
    Global end of trial date
    05 Jul 2023

    Results information
    Results version number
    		 v2(current)
    This version publication date
    18 Aug 2024
    First version publication date
    18 Jan 2024
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Secondary end points information to be updated.

    Trial information

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    Trial identification
    Sponsor protocol code
    VX21-445-124
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05274269
    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
    25 Jul 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    05 Jul 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Jul 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).
    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
    09 May 2022
    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
    Canada: 24
    Country: Number of subjects enrolled
    Switzerland: 4
    Country: Number of subjects enrolled
    Netherlands: 21
    Country: Number of subjects enrolled
    Norway: 2
    Country: Number of subjects enrolled
    Poland: 6
    Country: Number of subjects enrolled
    Portugal: 5
    Country: Number of subjects enrolled
    Spain: 52
    Country: Number of subjects enrolled
    Sweden: 4
    Country: Number of subjects enrolled
    Austria: 2
    Country: Number of subjects enrolled
    Belgium: 19
    Country: Number of subjects enrolled
    Czechia: 6
    Country: Number of subjects enrolled
    France: 41
    Country: Number of subjects enrolled
    Germany: 38
    Country: Number of subjects enrolled
    Hungary: 2
    Country: Number of subjects enrolled
    Italy: 81
    Worldwide total number of subjects
    307
    EEA total number of subjects
    279
    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)
    32
    Adolescents (12-17 years)
    32
    Adults (18-64 years)
    237
    From 65 to 84 years
    5
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in CF subjects aged 6 years and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.

    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
    		 Placebo
    Arm description
    		 Subjects received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo (matched to ELX/TEZ/IVA)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to ELX/TEZ/IVA once daily in the morning.

    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 once daily in the evening.

    Arm title
    		 ELX/TEZ/IVA
    Arm description
    		 Subjects 6 to less than (<) 12 years of age and weighing <30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Subjects ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ELX/TEZ/IVA
    Investigational medicinal product code
    VX-445/VX-661/VX-770
    Other name
    elexacaftor/tezacaftor/ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received ELX/TEZ/IVA FDC once daily in the morning.

    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 once daily in the evening.

    Number of subjects in period 1
    		Placebo ELX/TEZ/IVA
    Started
    102
    205
    Completed
    102
    197
    Not completed
    0
    8
         Adverse Event
    -
    3
         Death
    -
    1
         Other
    -
    2
         Withdrawal of consent (not due to AE)
    -
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.

    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Subjects 6 to less than (<) 12 years of age and weighing <30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Subjects ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.

    Reporting group values
    		 Placebo ELX/TEZ/IVA Total
    Number of subjects
    		 102 205 307
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 33.9 ( 16.4 ) 33.3 ( 15.9 ) -
    Gender categorical
    Units: Subjects
        Female
    		 52 113 165
        Male
    		 50 92 142
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 3 8 11
        Not Hispanic or Latino
    		 88 171 259
        Not Collected per Local Regulations
    		 11 26 37
    Race
    Units: Subjects
        White
    		 86 172 258
        Asian
    		 2 4 6
        Other
    		 1 3 4
        Not Collected per Local Regulations
    		 12 26 38
        White, Asian
    		 1 0 1
    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: percent predicted FEV1
        arithmetic mean (standard deviation)
    		 68.1 ( 18.1 ) 67.5 ( 17.6 ) -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.

    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Subjects 6 to less than (<) 12 years of age and weighing <30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Subjects ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 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)
    End point description
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The Full Analysis Set (FAS) included all randomized subjects who carry the intended mutation and received at least 1 dose of study drug. Here “ Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Primary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    102
    205
    Units: percent predicted FEV1
        least squares mean (confidence interval 95%)
    -0.4 (-2.0 to 1.3)
    8.9 (7.7 to 10.0)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v ELX/TEZ/IVA
    Number of subjects included in analysis
    307
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Models for Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    9.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 7.2
         upper limit
    		 11.3

    Secondary: Absolute Change in Sweat Chloride (SwCl)

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    End point title
    		 Absolute Change in Sweat Chloride (SwCl)
    End point description
    Sweat samples were collected using an approved collection device. FAS. Here “ Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    100
    200
    Units: millimole per liter (mmol/L)
        least squares mean (confidence interval 95%)
    0.5 (-2.6 to 3.6)
    -27.8 (-30.0 to -25.6)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v ELX/TEZ/IVA
    Number of subjects included in analysis
    300
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Models for Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    -28.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -32.1
         upper limit
    		 -24.5

    Secondary: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

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    End point title
    		 Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
    End point description
    The CFQ-R is a validated subject-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. FAS. Here “ Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    102
    202
    Units: units on a scale
        least squares mean (confidence interval 95%)
    -2.0 (-5.2 to 1.3)
    17.5 (15.2 to 19.8)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v ELX/TEZ/IVA
    Number of subjects included in analysis
    304
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    19.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 15.5
         upper limit
    		 23.5

    Secondary: Absolute Change in Body Mass Index (BMI)

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    End point title
    		 Absolute Change in Body Mass Index (BMI)
    End point description
    BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). FAS. Here “ Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 24
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    102
    196
    Units: kg/m^2
        least squares mean (confidence interval 95%)
    0.35 (0.16 to 0.53)
    0.81 (0.68 to 0.94)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v ELX/TEZ/IVA
    Number of subjects included in analysis
    298
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Models for Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    0.47
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.24
         upper limit
    		 0.69

    Secondary: Absolute Change in Weight

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    End point title
    		 Absolute Change in Weight
    End point description
    FAS. Here “Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 24
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    102
    196
    Units: kilogram (kg)
        least squares mean (confidence interval 95%)
    1.2 (0.6 to 1.7)
    2.4 (2.1 to 2.8)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v ELX/TEZ/IVA
    Number of subjects included in analysis
    298
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Model for Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    1.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.6
         upper limit
    		 1.9

    Secondary: Number of Pulmonary Exacerbations (PEx)

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    End point title
    		 Number of Pulmonary Exacerbations (PEx)
    End point description
    Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. FAS. Here “Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    102
    205
    Units: PEx events
        number (not applicable)
    40
    21
    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 was defined as all subjects who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Week 28
    End point values
    		 Placebo ELX/TEZ/IVA
    Number of subjects analysed
    102
    205
    Units: Subjects
        Subjects with TEAEs
    97
    193
        Subjects with SAEs
    15
    18
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Week 28
    Adverse event reporting additional description
    Safety Set was defined as all subjects who received at least 1 dose of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.

    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Subjects 6 to <12 years of age and weighing <30 kg at Day 1 received ELX 100mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Subjects ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.

    Serious adverse events
    		 Placebo ELX/TEZ/IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    15 / 102 (14.71%)
    18 / 205 (8.78%)
         number of deaths (all causes)
    0
    1
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lung adenocarcinoma
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Cardiac disorders
    Stress cardiomyopathy
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 205 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Drug intolerance
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 205 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Subileus
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis acute
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Haemoptysis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Rash maculo-papular
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Anxiety disorder
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Post-traumatic stress disorder
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal colic
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 205 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Polyarthritis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Bronchopulmonary aspergillosis allergic
         subjects affected / exposed
    0 / 102 (0.00%)
    2 / 205 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatitis viral
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    13 / 102 (12.75%)
    5 / 205 (2.44%)
         occurrences causally related to treatment / all
    0 / 19
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vestibular neuronitis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo ELX/TEZ/IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    86 / 102 (84.31%)
    164 / 205 (80.00%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    13 / 102 (12.75%)
    37 / 205 (18.05%)
         occurrences all number
    23
    58
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    14 / 102 (13.73%)
    27 / 205 (13.17%)
         occurrences all number
    17
    34
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    13 / 102 (12.75%)
    17 / 205 (8.29%)
         occurrences all number
    16
    21
    Abdominal pain upper
         subjects affected / exposed
    7 / 102 (6.86%)
    10 / 205 (4.88%)
         occurrences all number
    8
    11
    Constipation
         subjects affected / exposed
    4 / 102 (3.92%)
    15 / 205 (7.32%)
         occurrences all number
    4
    22
    Diarrhoea
         subjects affected / exposed
    10 / 102 (9.80%)
    26 / 205 (12.68%)
         occurrences all number
    16
    49
    Vomiting
         subjects affected / exposed
    7 / 102 (6.86%)
    15 / 205 (7.32%)
         occurrences all number
    10
    16
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    26 / 102 (25.49%)
    36 / 205 (17.56%)
         occurrences all number
    39
    46
    Haemoptysis
         subjects affected / exposed
    6 / 102 (5.88%)
    12 / 205 (5.85%)
         occurrences all number
    8
    16
    Nasal congestion
         subjects affected / exposed
    8 / 102 (7.84%)
    6 / 205 (2.93%)
         occurrences all number
    8
    6
    Oropharyngeal pain
         subjects affected / exposed
    10 / 102 (9.80%)
    17 / 205 (8.29%)
         occurrences all number
    11
    18
    Productive cough
         subjects affected / exposed
    6 / 102 (5.88%)
    6 / 205 (2.93%)
         occurrences all number
    6
    6
    Sputum increased
         subjects affected / exposed
    13 / 102 (12.75%)
    20 / 205 (9.76%)
         occurrences all number
    20
    25
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 102 (0.98%)
    45 / 205 (21.95%)
         occurrences all number
    1
    51
    Infections and infestations
    COVID-19
         subjects affected / exposed
    10 / 102 (9.80%)
    19 / 205 (9.27%)
         occurrences all number
    10
    19
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    36 / 102 (35.29%)
    28 / 205 (13.66%)
         occurrences all number
    52
    36
    Influenza
         subjects affected / exposed
    2 / 102 (1.96%)
    18 / 205 (8.78%)
         occurrences all number
    2
    20
    Nasopharyngitis
         subjects affected / exposed
    20 / 102 (19.61%)
    42 / 205 (20.49%)
         occurrences all number
    30
    58
    Rhinitis
         subjects affected / exposed
    6 / 102 (5.88%)
    20 / 205 (9.76%)
         occurrences all number
    8
    22
    Upper respiratory tract infection
         subjects affected / exposed
    10 / 102 (9.80%)
    17 / 205 (8.29%)
         occurrences all number
    12
    17

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Jan 2022
    Amended to add ELX/TEZ/IVA-responsive mutations to the list of qualifying CFTR mutations and adjust for the increased number of qualifying CFTR mutations, the maximum number of subjects with a given CFTR mutation that can be enrolled was decreased.
    21 Apr 2022
    Amended to maximum qualifying ppFEV1 value was expanded from 90% to 100%, specified that up to 10% of subjects may be enrolled with a screening ppFEV1 value >90% and ≤100% (approximately 27 subjects) and including history of hypersensitivity exclusion criterion and appendix to specify blood volumes.

    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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