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    Clinical Trial Results:
    A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

    Summary
    EudraCT number
    2017-004134-29
    Trial protocol
    GB   DE   IE   ES   DK   PL  
    Global end of trial date
    09 Sep 2020

    Results information
    Results version number
    v1(current)
    This version publication date
    25 Mar 2021
    First version publication date
    25 Mar 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX17-659-105
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03447262
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States,
    Public contact
    Vertex Pharmaceuticals Incorporated, Medical Monitor, +1 617-341-6777, medicalinfo@vrtx.com
    Scientific contact
    Vertex Pharmaceuticals Incorporated, Medical Monitor, +1 617-341-6777, medicalinfo@vrtx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-002191-PIP02-17
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    27 Oct 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Sep 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Sep 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Council on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    13 Jul 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety
    Long term follow-up duration
    1 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 49
    Country: Number of subjects enrolled
    Canada: 9
    Country: Number of subjects enrolled
    Israel: 19
    Country: Number of subjects enrolled
    United States: 262
    Country: Number of subjects enrolled
    Denmark: 4
    Country: Number of subjects enrolled
    Germany: 57
    Country: Number of subjects enrolled
    Ireland: 23
    Country: Number of subjects enrolled
    Poland: 2
    Country: Number of subjects enrolled
    Spain: 19
    Country: Number of subjects enrolled
    Switzerland: 7
    Country: Number of subjects enrolled
    United Kingdom: 33
    Worldwide total number of subjects
    484
    EEA total number of subjects
    105
    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)
    119
    Adults (18-64 years)
    365
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    This study was conducted in CF subjects aged 12 years or older who participated in parent studies VX17-659-102 (NCT03447249) and VX17-659-103 (NCT03460990). Eligible subjects from parent studies were enrolled in this study.

    Period 1
    Period 1 title
    Triple Combination Treatment Period (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    VX-659/TEZ/IVA TC
    Arm description
    Subjects received VX-659/TEZ/IVA for 96 weeks in TC treatment period.
    Arm type
    Experimental

    Investigational medicinal product name
    VX-659/TEZ/IVA
    Investigational medicinal product code
    VX-659/VX-661/VX-770
    Other name
    VX-659/Tezacaftor/Ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-659/TEZ/IVA fixed-dose combination 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 [1]
    VX-659/TEZ/IVA TC
    Started
    481
    Completed
    2
    Not completed
    479
         Death
    2
         Other
    4
         Commercial drug is available for subject
    9
         Adverse event
    7
         Withdrawal of consent (not due to AE)
    2
         Study termination by sponsor
    455
    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: The total of 484 subjects were enrolled from the parent studies. Out of which, 3 subjects were enrolled but never dosed in this study. Therefore, 481 subjects are included in subject disposition and baseline sections.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    VX-659/TEZ/IVA TC
    Reporting group description
    Subjects received VX-659/TEZ/IVA for 96 weeks in TC treatment period.

    Reporting group values
    VX-659/TEZ/IVA TC Total
    Number of subjects
    481 481
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    26.9 ± 9.7 -
    Gender categorical
    Units: Subjects
        Female
    219 219
        Male
    262 262

    End points

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    End points reporting groups
    Reporting group title
    VX-659/TEZ/IVA TC
    Reporting group description
    Subjects received VX-659/TEZ/IVA for 96 weeks in TC treatment period.

    Primary: 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) [1]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 100)
    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 this endpoint.
    End point values
    VX-659/TEZ/IVA TC
    Number of subjects analysed
    481
    Units: subjects
        Subjects with TEAEs
    470
        Subjects with Serious TEAEs
    99
    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 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 100)
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    VX-659/TEZ/IVA TC
    Reporting group description
    Subjects received VX-659/TEZ/IVA for 96 weeks in TC treatment period.

    Serious adverse events
    VX-659/TEZ/IVA TC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    99 / 481 (20.58%)
         number of deaths (all causes)
    2
         number of deaths resulting from adverse events
    Immune system disorders
    Anaphylactic reaction
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Generalised oedema
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Systemic inflammatory response syndrome
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Delirium
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Breathing-related sleep disorder
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Anxiety
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Suicidal ideation
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Alcohol poisoning
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Contusion
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Craniocerebral injury
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Forearm fracture
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Toxicity to various agents
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Stoma site extravasation
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Intentional overdose
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    4 / 481 (0.83%)
         occurrences causally related to treatment / all
    4 / 5
         deaths causally related to treatment / all
    0 / 0
    Alanine aminotransferase increased
         subjects affected / exposed
    4 / 481 (0.83%)
         occurrences causally related to treatment / all
    4 / 5
         deaths causally related to treatment / all
    0 / 0
    Pulmonary function test decreased
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Restrictive cardiomyopathy
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac failure acute
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Congenital, familial and genetic disorders
    Cystic fibrosis related diabetes
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Granulomatous pneumonitis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Haemoptysis
         subjects affected / exposed
    5 / 481 (1.04%)
         occurrences causally related to treatment / all
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    Increased bronchial secretion
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pleuritic pain
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Syncope
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Seizure
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Abdominal pain
         subjects affected / exposed
    3 / 481 (0.62%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Constipation
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Distal intestinal obstruction syndrome
         subjects affected / exposed
    6 / 481 (1.25%)
         occurrences causally related to treatment / all
    0 / 7
         deaths causally related to treatment / all
    0 / 0
    Duodenitis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastritis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Intestinal obstruction
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Mechanical ileus
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Hydronephrosis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    3 / 481 (0.62%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Pelvi-ureteric obstruction
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholangitis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Biliary colic
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bile duct stone
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Autoimmune hepatitis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cholecystitis
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Cholecystitis chronic
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cholelithiasis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Product issues
    Device leakage
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Compartment syndrome
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Metabolism and nutrition disorders
    Diabetes mellitus inadequate control
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diabetes mellitus
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypoglycaemia
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bacterial disease carrier
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bronchitis bacterial
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    H1N1 influenza
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Mastoiditis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Lower respiratory tract infection bacterial
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Influenza
         subjects affected / exposed
    4 / 481 (0.83%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    40 / 481 (8.32%)
         occurrences causally related to treatment / all
    0 / 53
         deaths causally related to treatment / all
    0 / 0
    Medical device site cellulitis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Pneumonia pseudomonal
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tonsillitis
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 481 (0.21%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular device infection
         subjects affected / exposed
    2 / 481 (0.42%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    VX-659/TEZ/IVA TC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    446 / 481 (92.72%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    43 / 481 (8.94%)
         occurrences all number
    55
    Blood creatine phosphokinase increased
         subjects affected / exposed
    37 / 481 (7.69%)
         occurrences all number
    43
    Aspartate aminotransferase increased
         subjects affected / exposed
    42 / 481 (8.73%)
         occurrences all number
    51
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    126 / 481 (26.20%)
         occurrences all number
    205
    Sinus congestion
         subjects affected / exposed
    27 / 481 (5.61%)
         occurrences all number
    39
    Rhinorrhoea
         subjects affected / exposed
    40 / 481 (8.32%)
         occurrences all number
    50
    Respiration abnormal
         subjects affected / exposed
    28 / 481 (5.82%)
         occurrences all number
    30
    Oropharyngeal pain
         subjects affected / exposed
    80 / 481 (16.63%)
         occurrences all number
    106
    Nasal congestion
         subjects affected / exposed
    53 / 481 (11.02%)
         occurrences all number
    66
    Sputum increased
         subjects affected / exposed
    69 / 481 (14.35%)
         occurrences all number
    90
    Haemoptysis
         subjects affected / exposed
    39 / 481 (8.11%)
         occurrences all number
    57
    Nervous system disorders
    Headache
         subjects affected / exposed
    51 / 481 (10.60%)
         occurrences all number
    69
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    31 / 481 (6.44%)
         occurrences all number
    34
    Pyrexia
         subjects affected / exposed
    56 / 481 (11.64%)
         occurrences all number
    64
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    42 / 481 (8.73%)
         occurrences all number
    51
    Vomiting
         subjects affected / exposed
    27 / 481 (5.61%)
         occurrences all number
    33
    Nausea
         subjects affected / exposed
    36 / 481 (7.48%)
         occurrences all number
    46
    Diarrhoea
         subjects affected / exposed
    42 / 481 (8.73%)
         occurrences all number
    47
    Abdominal pain upper
         subjects affected / exposed
    26 / 481 (5.41%)
         occurrences all number
    36
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    28 / 481 (5.82%)
         occurrences all number
    37
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    149 / 481 (30.98%)
         occurrences all number
    264
    Influenza
         subjects affected / exposed
    40 / 481 (8.32%)
         occurrences all number
    45
    Upper respiratory tract infection
         subjects affected / exposed
    104 / 481 (21.62%)
         occurrences all number
    178
    Sinusitis
         subjects affected / exposed
    30 / 481 (6.24%)
         occurrences all number
    45
    Nasopharyngitis
         subjects affected / exposed
    97 / 481 (20.17%)
         occurrences all number
    163
    Viral upper respiratory tract infection
         subjects affected / exposed
    38 / 481 (7.90%)
         occurrences all number
    53

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Jan 2018
    Amended to add study drug dose and tablet strength. Clarified completion of study participation and study drug interruptions and discontinuation scenarios.
    23 Oct 2018
    Amended to update stopping rules and add clarifications on descriptions for efficacy and pharmacodynamics variables.

    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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