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    Clinical Trial Results:
    A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects

    Summary
    EudraCT number
    		 2019-004881-16
    Trial protocol
    		 IE   DE   SE   GB  
    Global end of trial date
    04 May 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 May 2022
    First version publication date
    19 May 2022
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    VX19-864-101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04474197
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States,
    Public contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
    Scientific contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    01 Jun 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 May 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    04 May 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
    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
    24 Jul 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 22
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Ireland: 5
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Sweden: 3
    Worldwide total number of subjects
    44
    EEA total number of subjects
    11
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    37
    From 65 to 84 years
    7
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in subjects 18 through 80 of years of age, inclusive with the PiZZ genotype.

    Period 1
    Period 1 title
    Overall Trial (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 VX-864 in the treatment period for 28 days.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo (matched to VX-864)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to VX-864 in the morning and evening.

    Arm title
    		 VX-864 100 mg
    Arm description
    		 Subjects received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-864
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-864 100 mg in the morning and evening.

    Arm title
    		 VX-864 300 mg
    Arm description
    		 Subjects received VX-864 300 mg q12h in the treatment period for 28 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-864
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-864 300 mg in the morning and evening.

    Arm title
    		 VX-864 500 mg
    Arm description
    		 Subjects received VX-864 500 mg q12h in the treatment period for 28 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-864
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-864 500 mg in the morning and evening.

    Number of subjects in period 1
    		Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Started
    7
    10
    9
    18
    Completed
    7
    9
    9
    18
    Not completed
    0
    1
    0
    0
         Withdrawal of consent (not due to adverse event)
    -
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to VX-864 in the treatment period for 28 days.

    Reporting group title
    VX-864 100 mg
    Reporting group description
    		 Subjects received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.

    Reporting group title
    VX-864 300 mg
    Reporting group description
    		 Subjects received VX-864 300 mg q12h in the treatment period for 28 days.

    Reporting group title
    VX-864 500 mg
    Reporting group description
    		 Subjects received VX-864 500 mg q12h in the treatment period for 28 days.

    Reporting group values
    		 Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg Total
    Number of subjects
    		 7 10 9 18 44
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 63.4 ( 10.5 ) 55.1 ( 5.3 ) 53.2 ( 16.2 ) 57.4 ( 9.9 ) -
    Gender categorical
    Units: Subjects
        Female
    		 5 6 6 14 31
        Male
    		 2 4 3 4 13
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0 1 1
        Not Hispanic or Latino
    		 7 8 8 15 38
        Unknown or Not Reported
    		 0 2 1 2 5
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0
        Asian
    		 0 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0
        Black or African American
    		 0 0 0 0 0
        White
    		 7 10 9 18 44
        More than one race
    		 0 0 0 0 0
        Unknown or Not Reported
    		 0 0 0 0 0
    Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
    Units: micromole per liter
        arithmetic mean (standard deviation)
    		 4.7 ( 1.3 ) 4.0 ( 0.7 ) 3.8 ( 0.9 ) 4.1 ( 0.6 ) -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to VX-864 in the treatment period for 28 days.

    Reporting group title
    VX-864 100 mg
    Reporting group description
    		 Subjects received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.

    Reporting group title
    VX-864 300 mg
    Reporting group description
    		 Subjects received VX-864 300 mg q12h in the treatment period for 28 days.

    Reporting group title
    VX-864 500 mg
    Reporting group description
    		 Subjects received VX-864 500 mg q12h in the treatment period for 28 days.

    Primary: Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels

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    End point title
    		 Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
    End point description
    Full analysis set (FAS) included all randomized subjects who received at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    From Baseline at Day 28
    End point values
    		 Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Number of subjects analysed
    7
    10
    9
    18
    Units: micromole per liter
        least squares mean (standard error)
    -0.1 ( 0.3 )
    2.3 ( 0.3 )
    2.3 ( 0.2 )
    2.1 ( 0.2 )
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v VX-864 100 mg
    Number of subjects included in analysis
    17
    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
    2.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.5
         upper limit
    		 3.1
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    Placebo v VX-864 300 mg
    Number of subjects included in analysis
    16
    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
    2.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.6
         upper limit
    		 3.1
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    Placebo v VX-864 500 mg
    Number of subjects included in analysis
    25
    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
    2.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.5
         upper limit
    		 2.9

    Primary: Safety and Tolerability as Assessed by Number of Subjects With 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 Adverse Events (AEs) and Serious Adverse Events (SAEs) [1]
    End point description
    Safety set included all subjects who received at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    Day 1 up to 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 descriptive statistics were planned. No statistical comparisons were planned for this endpoint.
    End point values
    		 Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Number of subjects analysed
    7
    10
    9
    18
    Units: subjects
        Subjects with AEs
    4
    7
    7
    17
        Subjects with SAEs
    0
    0
    0
    2
    No statistical analyses for this end point

    Secondary: Change in Plasma Antigenic AAT Levels

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    End point title
    		 Change in Plasma Antigenic AAT Levels
    End point description
    FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline at Day 28
    End point values
    		 Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Number of subjects analysed
    7
    10
    9
    18
    Units: micromole per liter
        least squares mean (standard error)
    -0.1 ( 0.4 )
    3.4 ( 0.4 )
    2.9 ( 0.4 )
    2.6 ( 0.2 )
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Placebo v VX-864 100 mg
    Number of subjects included in analysis
    17
    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
    3.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.4
         upper limit
    		 4.6
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    Placebo v VX-864 300 mg
    Number of subjects included in analysis
    16
    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
    3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.9
         upper limit
    		 4
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    Placebo v VX-864 500 mg
    Number of subjects included in analysis
    25
    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
    2.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.8
         upper limit
    		 3.7

    Secondary: Observed Pre-dose Plasma Concentration (Ctrough) of VX-864

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    End point title
    		 Observed Pre-dose Plasma Concentration (Ctrough) of VX-864 [2]
    End point description
    PK set included subjects who received at least 1 dose of study drug. Here “n” signifies those subjects who were evaluable for this endpoint at specified time points for each reporting group respectively.
    End point type
    Secondary
    End point timeframe
    Pre-dose at Day 7, Day 14, Day 21 and Day 28
    Notes
    [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: Placebo group was not applicable for this endpoint.
    End point values
    		 VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Number of subjects analysed
    10
    9
    18
    Units: microgram per milliliter
    arithmetic mean (standard deviation)
        Day 7 (n=9,8,16)
    1.13 ( 0.743 )
    1.62 ( 0.665 )
    2.74 ( 2.89 )
        Day 14 (n=8,9,18)
    0.852 ( 0.340 )
    1.47 ( 0.847 )
    4.35 ( 5.36 )
        Day 21 (n=8,8,18)
    0.868 ( 0.503 )
    1.21 ( 0.770 )
    1.79 ( 0.922 )
        Day 28 (n=9,8,18)
    0.797 ( 0.327 )
    1.29 ( 0.646 )
    2.55 ( 2.40 )
    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 8
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to VX-864 in the treatment period for 28 days.

    Reporting group title
    VX-864 100 mg
    Reporting group description
    		 Subjects received VX-864 100 mg q12h in the treatment period for 28 days.

    Reporting group title
    VX-864 300 mg
    Reporting group description
    		 Subjects received VX-864 300 mg q12h in the treatment period for 28 days.

    Reporting group title
    VX-864 500 mg
    Reporting group description
    		 Subjects received VX-864 500 mg q12h in the treatment period for 28 days.

    Serious adverse events
    		 Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    2 / 18 (11.11%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pneumothorax spontaneous
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 7 (57.14%)
    7 / 10 (70.00%)
    7 / 9 (77.78%)
    17 / 18 (94.44%)
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Chills
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 10 (20.00%)
    0 / 9 (0.00%)
    3 / 18 (16.67%)
         occurrences all number
    0
    2
    0
    3
    Feeling abnormal
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Feeling cold
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    0
    1
    Malaise
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 10 (20.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Reproductive system and breast disorders
    Heavy menstrual bleeding
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Vaginal haemorrhage
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Cough
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 10 (20.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Dyspnoea
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Epistaxis
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Nasal congestion
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiration abnormal
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Sinus congestion
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sneezing
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Sputum increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Tachypnoea
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Poor quality sleep
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Blood bicarbonate decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood cholesterol increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    1 / 18 (5.56%)
         occurrences all number
    1
    0
    1
    1
    Blood potassium decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Blood pressure diastolic increased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Blood triglycerides increased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    1
    1
    Haemoglobin increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    0
    1
    Low density lipoprotein increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Lymphocyte count decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Oxygen saturation decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Protein total decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Red blood cell count increased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    White blood cells urine positive
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Injury, poisoning and procedural complications
    Back injury
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Muscle strain
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Post procedural contusion
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Post procedural haemorrhage
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Procedural pain
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Skin laceration
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Vaccination complication
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    1
    1
    Nervous system disorders
    Carpal tunnel syndrome
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Dizziness
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 10 (10.00%)
    2 / 9 (22.22%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    4
    1
    Dysaesthesia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Headache
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    2 / 9 (22.22%)
    2 / 18 (11.11%)
         occurrences all number
    0
    1
    2
    4
    Tremor
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Eye disorders
    Eye allergy
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Macular degeneration
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    1 / 9 (11.11%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Constipation
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    0
    0
    2
    Diarrhoea
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    1 / 9 (11.11%)
    4 / 18 (22.22%)
         occurrences all number
    0
    1
    1
    4
    Flatulence
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gingival pain
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    2 / 9 (22.22%)
    4 / 18 (22.22%)
         occurrences all number
    0
    0
    4
    4
    Toothache
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Photosensitivity reaction
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Polymorphic light eruption
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Rash
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Urticaria
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    0
    0
    2
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Muscle spasms
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Infections and infestations
    Infective exacerbation of bronchiectasis
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    1 / 9 (11.11%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Skin infection
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 10 (0.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 10 (10.00%)
    0 / 9 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    2
    1
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Jan 2021
    Amended to clarify that the monitoring procedures, consent and re-consent may be conducted through on-site or remote visits due to extenuating circumstances (e.g., events related to COVID-19). Exclusion criteria were updated.

    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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    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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