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    Clinical Trial Results:
    A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

    Summary
    EudraCT number
    		 2018-002835-76
    Trial protocol
    		 GB   IE   DE   DK   FR   BE   NL   IT  
    Global end of trial date
    12 Jun 2020

    Results information
    Results version number
    		 v2(current)
    This version publication date
    22 Jul 2021
    First version publication date
    26 Dec 2020
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Addition of secondary endpoints

    Trial information

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    Trial identification
    Sponsor protocol code
    VX18-445-104
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04058353
    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
    30 Jun 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Jun 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Jun 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in cystic fibrosis (CF) subjects who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).
    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
    28 Aug 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 92
    Country: Number of subjects enrolled
    Australia: 27
    Country: Number of subjects enrolled
    France: 27
    Country: Number of subjects enrolled
    United Kingdom: 25
    Country: Number of subjects enrolled
    Italy: 20
    Country: Number of subjects enrolled
    Germany: 16
    Country: Number of subjects enrolled
    Netherlands: 14
    Country: Number of subjects enrolled
    Belgium: 13
    Country: Number of subjects enrolled
    Canada: 12
    Country: Number of subjects enrolled
    Spain: 12
    Country: Number of subjects enrolled
    Ireland: 11
    Country: Number of subjects enrolled
    Denmark: 2
    Worldwide total number of subjects
    271
    EEA total number of subjects
    140
    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)
    24
    Adults (18-64 years)
    242
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in cystic fibrosis (CF) subjects aged 12 years or older.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Control: IVA or TEZ/IVA
    Arm description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects either received IVA 150 milligrams (mg) every 12 hours (q12h) or TEZ 100 mg once daily (qd)/IVA 150 mg q12h in the treatment period for 8 weeks.
    Arm type
    		 Active comparator

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

    Investigational medicinal product name
    TEZ/IVA
    Investigational medicinal product code
    VX-661/VX-770
    Other name
    Tezacaftor/Ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received TEZ once daily and IVA every 12 hours.

    Arm title
    		 Triple Combination (TC): ELX/TEZ/IVA
    Arm description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 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 VX-445/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]
    		Control: IVA or TEZ/IVA Triple Combination (TC): ELX/TEZ/IVA
    Started
    126
    132
    Completed
    122
    131
    Not completed
    4
    1
         Physician decision
    1
    -
         Other
    1
    -
         Adverse event
    2
    -
         Adverse Events
    -
    1
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: A total of 271 subjects enrolled, 12 subjects discontinued in run-in period of the study. Of 259 subjects, 1 subject randomized but not dosed in the treatment period. Therefore, only 258 subjects are included in the subject disposition and baseline sections.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Control: IVA or TEZ/IVA
    Reporting group description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects either received IVA 150 milligrams (mg) every 12 hours (q12h) or TEZ 100 mg once daily (qd)/IVA 150 mg q12h in the treatment period for 8 weeks.

    Reporting group title
    Triple Combination (TC): ELX/TEZ/IVA
    Reporting group description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks.

    Reporting group values
    		 Control: IVA or TEZ/IVA Triple Combination (TC): ELX/TEZ/IVA Total
    Number of subjects
    		 126 132 258
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 37.6 ( 14.3 ) 37.7 ( 14.7 ) -
    Gender categorical
    Units: Subjects
        Female
    		 61 67 128
        Male
    		 65 65 130
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 4 5 9
        Not Hispanic or Latino
    		 114 117 231
        Unknown or Not Reported
    		 8 10 18
    Race/Ethnicity
    Units: Subjects
        Black or African American
    		 2 0 2
        Not Collected per Local Regulations
    		 9 9 18
        Aboriginal
    		 2 1 3
        Latin-American
    		 1 0 1
        Lebanese
    		 1 0 1
        White
    		 110 122 232
        White, American Indian or Alaska Native
    		 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: percentage points
        arithmetic mean (standard deviation)
    		 68.1 ( 16.4 ) 67.1 ( 15.7 ) -

    End points

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    End points reporting groups
    Reporting group title
    Control: IVA or TEZ/IVA
    Reporting group description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects either received IVA 150 milligrams (mg) every 12 hours (q12h) or TEZ 100 mg once daily (qd)/IVA 150 mg q12h in the treatment period for 8 weeks.

    Reporting group title
    Triple Combination (TC): ELX/TEZ/IVA
    Reporting group description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks.

    Primary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for the ELX/TEZ/IVA Group

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    End point title
    		 Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for the ELX/TEZ/IVA Group [1] [2]
    End point description
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Full analysis set (FAS) included all randomized subjects who have the intended CF transmembrane conductance regulator (CFTR) allele mutation and received at least 1 dose of study drug in the treatment period.
    End point type
    Primary
    End point timeframe
    From Baseline Through Week 8
    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 within treatment group comparison was planned for this endpoint. Individual within-group comparison cannot be reported in the EudraCT database. Therefore, no statistical comparisons were reported.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was applicable only for the triple combination arm.
    End point values
    		 Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    132
    Units: percentage points
        least squares mean (confidence interval 95%)
    3.7 (2.8 to 4.6)
    No statistical analyses for this end point

    Secondary: Absolute Change in Sweat Chloride (SwCl) for ELX/TEZ/IVA Group

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    End point title
    		 Absolute Change in Sweat Chloride (SwCl) for ELX/TEZ/IVA Group [3]
    End point description
    Sweat samples were collected using an approved collection device. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 8
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was applicable only for the triple combination arm.
    End point values
    		 Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    132
    Units: millimole per liter (mmol/L)
        least squares mean (confidence interval 95%)
    -22.3 (-24.5 to -20.2)
    No statistical analyses for this end point

    Secondary: Absolute Change in ppFEV1 for the ELX/TEZ/IVA Group Compared to the Control Group

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    End point title
    		 Absolute Change in ppFEV1 for the ELX/TEZ/IVA Group Compared to the Control Group
    End point description
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 8
    End point values
    		 Control: IVA or TEZ/IVA Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    126
    132
    Units: percentage points
        least squares mean (confidence interval 95%)
    0.2 (-0.7 to 1.1)
    3.7 (2.8 to 4.6)
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Control: IVA or TEZ/IVA v Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects included in analysis
    258
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed-effects model for repeated measure
    Parameter type
    		 Least Squares (LS) Mean Difference
    Point estimate
    3.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.2
         upper limit
    		 4.7

    Secondary: Absolute Change in SwCl for ELX/TEZ/IVA Group Compared to the Control Group

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    End point title
    		 Absolute Change in SwCl for ELX/TEZ/IVA Group Compared to the Control Group
    End point description
    Sweat samples were collected using an approved collection device. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 8
    End point values
    		 Control: IVA or TEZ/IVA Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    126
    132
    Units: mmol/L
        least squares mean (confidence interval 95%)
    0.7 (-1.4 to 2.8)
    -22.3 (-24.5 to -20.2)
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Control: IVA or TEZ/IVA v Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects included in analysis
    258
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed-effects model for repeated measure
    Parameter type
    		 LS Mean Difference
    Point estimate
    -23.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -26.1
         upper limit
    		 -20.1

    Secondary: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for ELX/TEZ/IVA Group

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    End point title
    		 Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for ELX/TEZ/IVA Group [4]
    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.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 8
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was applicable only for the triple combination arm.
    End point values
    		 Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    132
    Units: units on a scale
        least squares mean (confidence interval 95%)
    10.3 (8.0 to 12.7)
    No statistical analyses for this end point

    Secondary: Absolute Change in CFQ-R Respiratory Domain Score for ELX/TEZ/IVA Group Compared to the Control Group

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    End point title
    		 Absolute Change in CFQ-R Respiratory Domain Score for ELX/TEZ/IVA Group Compared to the Control Group
    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.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 8
    End point values
    		 Control: IVA or TEZ/IVA Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    126
    132
    Units: units on a scale
        least squares mean (confidence interval 95%)
    1.6 (-0.8 to 4.1)
    10.3 (8.0 to 12.7)
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    Control: IVA or TEZ/IVA v Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects included in analysis
    258
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed-effects model for repeated measure
    Parameter type
    		 LS Mean Difference
    Point estimate
    8.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 5.3
         upper limit
    		 12.1

    Secondary: Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) 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 (TEAEs) and Serious Adverse Events (SAEs)
    End point description
    Safety set included all subjects who received at least 1 dose of study drug in the treatment period.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Week 12
    End point values
    		 Control: IVA or TEZ/IVA Triple Combination (TC): ELX/TEZ/IVA
    Number of subjects analysed
    126
    132
    Units: subjects
        Subjects With TEAEs
    83
    88
        Subjects With SAEs
    11
    5
    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 12
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Control: IVA or TEZ/IVA
    Reporting group description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects either received IVA 150 mg q12h or TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks.

    Reporting group title
    TC: ELX/TEZ/IVA
    Reporting group description
    		 Following an IVA or TEZ/IVA run-in period of 4 weeks, subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks.

    Serious adverse events
    		 Control: IVA or TEZ/IVA TC: ELX/TEZ/IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    11 / 126 (8.73%)
    5 / 132 (3.79%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    0 / 126 (0.00%)
    1 / 132 (0.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    0 / 126 (0.00%)
    1 / 132 (0.76%)
         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
    1 / 126 (0.79%)
    1 / 132 (0.76%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 126 (0.79%)
    0 / 132 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Depression
         subjects affected / exposed
    1 / 126 (0.79%)
    0 / 132 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Endocrine disorders
    Hyperparathyroidism primary
         subjects affected / exposed
    1 / 126 (0.79%)
    0 / 132 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 126 (0.00%)
    1 / 132 (0.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    7 / 126 (5.56%)
    2 / 132 (1.52%)
         occurrences causally related to treatment / all
    0 / 7
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 126 (0.79%)
    0 / 132 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Control: IVA or TEZ/IVA TC: ELX/TEZ/IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    47 / 126 (37.30%)
    35 / 132 (26.52%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 126 (0.00%)
    8 / 132 (6.06%)
         occurrences all number
    0
    9
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 126 (0.00%)
    8 / 132 (6.06%)
         occurrences all number
    0
    8
    Nervous system disorders
    Headache
         subjects affected / exposed
    19 / 126 (15.08%)
    11 / 132 (8.33%)
         occurrences all number
    30
    11
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 126 (1.59%)
    7 / 132 (5.30%)
         occurrences all number
    2
    7
    Diarrhoea
         subjects affected / exposed
    8 / 126 (6.35%)
    5 / 132 (3.79%)
         occurrences all number
    13
    5
    Nausea
         subjects affected / exposed
    9 / 126 (7.14%)
    2 / 132 (1.52%)
         occurrences all number
    16
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    18 / 126 (14.29%)
    3 / 132 (2.27%)
         occurrences all number
    21
    3
    Sputum increased
         subjects affected / exposed
    8 / 126 (6.35%)
    6 / 132 (4.55%)
         occurrences all number
    8
    6
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    10 / 126 (7.94%)
    2 / 132 (1.52%)
         occurrences all number
    11
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Dec 2019
    Amended to add an exploratory endpoint.

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

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

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