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    Clinical Trial Results:
    A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects

    Summary
    EudraCT number
    2019-003650-92
    Trial protocol
    DE   SE   GB   IE  
    Global end of trial date
    14 Nov 2020

    Results information
    Results version number
    v1(current)
    This version publication date
    23 Jan 2022
    First version publication date
    23 Jan 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX19-814-101
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04167345
    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
    22 Dec 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    14 Nov 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Nov 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 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 Council on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    13 Jan 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
    Canada: 3
    Country: Number of subjects enrolled
    United States: 30
    Country: Number of subjects enrolled
    Germany: 9
    Country: Number of subjects enrolled
    Ireland: 6
    Worldwide total number of subjects
    48
    EEA total number of subjects
    15
    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)
    38
    From 65 to 84 years
    10
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    There were 3 parts in study: Parts A1, A2 and B. As specified in SAP, data from placebo group for Parts A1, A2 and B were pooled and reported as a combined arm (Parts A1, A2 and B Combined: Placebo) and data from VX-814 400 mg for Parts A1 and A2 were pooled and reported as a combined arm (Parts A1 and A2 Combined: VX-814 400 mg) in below results.

    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
    Carer, Assessor, Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Parts A1, A2 and B Combined: Placebo
    Arm description
    Subjects received placebo matched to VX-814 in the treatment period for 28 days.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo (matched to VX-814)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to VX-814 daily in morning and evening.

    Arm title
    Part A1: VX-814 100 milligrams (mg)
    Arm description
    Subjects received VX-814 100 mg once every 12 hours (q12h) in the treatment period for 28 days.
    Arm type
    Experimental

    Investigational medicinal product name
    VX-814
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-814 100 mg daily in the morning and evening.

    Arm title
    Part A1: VX-814 200 mg
    Arm description
    Subjects received VX-814 200 mg q12h in the treatment period for 28 days.
    Arm type
    Experimental

    Investigational medicinal product name
    VX-814
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-814 200 mg daily in the morning and evening.

    Arm title
    Parts A1 and A2 Combined: VX-814 400 mg
    Arm description
    Subjects received VX-814 400 mg q12h in the treatment period for 28 days.
    Arm type
    Experimental

    Investigational medicinal product name
    VX-814
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-814 400 mg daily in the morning and evening.

    Arm title
    Part B: VX-814 600 mg
    Arm description
    Subjects received VX-814 600 mg q12h in the treatment period for 28 days.
    Arm type
    Experimental

    Investigational medicinal product name
    VX-814
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-814 600 mg daily in the morning and evening.

    Number of subjects in period 1
    Parts A1, A2 and B Combined: Placebo Part A1: VX-814 100 milligrams (mg) Part A1: VX-814 200 mg Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg
    Started
    10
    4
    3
    13
    18
    Completed
    9
    3
    3
    13
    18
    Not completed
    1
    1
    0
    0
    0
         Other
    1
    1
    -
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Parts A1, A2 and B Combined: Placebo
    Reporting group description
    Subjects received placebo matched to VX-814 in the treatment period for 28 days.

    Reporting group title
    Part A1: VX-814 100 milligrams (mg)
    Reporting group description
    Subjects received VX-814 100 mg once every 12 hours (q12h) in the treatment period for 28 days.

    Reporting group title
    Part A1: VX-814 200 mg
    Reporting group description
    Subjects received VX-814 200 mg q12h in the treatment period for 28 days.

    Reporting group title
    Parts A1 and A2 Combined: VX-814 400 mg
    Reporting group description
    Subjects received VX-814 400 mg q12h in the treatment period for 28 days.

    Reporting group title
    Part B: VX-814 600 mg
    Reporting group description
    Subjects received VX-814 600 mg q12h in the treatment period for 28 days.

    Reporting group values
    Parts A1, A2 and B Combined: Placebo Part A1: VX-814 100 milligrams (mg) Part A1: VX-814 200 mg Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg Total
    Number of subjects
    10 4 3 13 18 48
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    54.6 ± 11.3 51.8 ± 14.8 55.5 ± 7.8 57.9 ± 13.2 57.9 ± 9.1 -
    Gender categorical
    Units: Subjects
        Female
    5 3 0 7 12 27
        Male
    5 1 3 6 6 21
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    1 1 0 0 0 2
        Not Hispanic or Latino
    9 3 3 12 14 41
        Unknown or Not Reported
    0 0 0 1 4 5
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0 0 0 0
        Asian
    0 0 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    0 0 0 0 0 0
        Black or African American
    0 0 0 0 0 0
        White
    10 4 3 13 18 48
        More than one race
    0 0 0 0 0 0
        Unknown or Not Reported
    0 0 0 0 0 0
    Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
    Units: micromole per liter
        arithmetic mean (standard deviation)
    4.2 ± 1.2 5.1 ± 2.3 3.9 ± 0.4 3.9 ± 0.7 4.5 ± 1.0 -

    End points

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    End points reporting groups
    Reporting group title
    Parts A1, A2 and B Combined: Placebo
    Reporting group description
    Subjects received placebo matched to VX-814 in the treatment period for 28 days.

    Reporting group title
    Part A1: VX-814 100 milligrams (mg)
    Reporting group description
    Subjects received VX-814 100 mg once every 12 hours (q12h) in the treatment period for 28 days.

    Reporting group title
    Part A1: VX-814 200 mg
    Reporting group description
    Subjects received VX-814 200 mg q12h in the treatment period for 28 days.

    Reporting group title
    Parts A1 and A2 Combined: VX-814 400 mg
    Reporting group description
    Subjects received VX-814 400 mg q12h in the treatment period for 28 days.

    Reporting group title
    Part B: VX-814 600 mg
    Reporting group description
    Subjects received VX-814 600 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. The "number of subjects analysed" signifies subjects who were evaluable at the specified time point. As pre-specified in SAP, statistical comparisons with placebo were planned only for VX-814 400 mg and 600 mg treatment arms.
    End point type
    Primary
    End point timeframe
    From Baseline at Day 28
    End point values
    Parts A1, A2 and B Combined: Placebo Part A1: VX-814 100 milligrams (mg) Part A1: VX-814 200 mg Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg
    Number of subjects analysed
    6
    2
    3
    8
    15
    Units: micromole per liter
        arithmetic mean (standard deviation)
    -0.4 ± 0.3
    0.2 ± 0.1
    0.3 ± 0.5
    1.4 ± 0.6
    1.6 ± 1.0
    Statistical analysis title
    Statistical analysis VX-814 400 mg versus placebo
    Comparison groups
    Parts A1 and A2 Combined: VX-814 400 mg v Parts A1, A2 and B Combined: Placebo
    Number of subjects included in analysis
    14
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    t-test, 2-sided
    Parameter type
    Mean Difference
    Point estimate
    1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.1
         upper limit
    2.3
    Statistical analysis title
    Statistical analysis VX-814 600 mg versus placebo
    Comparison groups
    Part B: VX-814 600 mg v Parts A1, A2 and B Combined: Placebo
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    t-test, 2-sided
    Parameter type
    Mean Difference
    Point estimate
    2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.1
         upper limit
    2.9

    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
    The 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 data were planned for this primary safety endpoint. No statistical comparison were planned.
    End point values
    Parts A1, A2 and B Combined: Placebo Part A1: VX-814 100 milligrams (mg) Part A1: VX-814 200 mg Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg
    Number of subjects analysed
    10
    4
    3
    13
    18
    Units: subjects
        Subjects With AEs
    4
    3
    1
    10
    14
        Subjects With SAEs
    0
    2
    1
    1
    0
    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. The "number of subjects analysed" signifies subjects who were evaluable at the specified time point. As pre-specified in SAP, statistical comparisons with placebo were planned only for VX-814 400 mg and 600 mg treatment arms.
    End point type
    Secondary
    End point timeframe
    From Baseline at Day 28
    End point values
    Parts A1, A2 and B Combined: Placebo Part A1: VX-814 100 milligrams (mg) Part A1: VX-814 200 mg Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg
    Number of subjects analysed
    6
    2
    3
    8
    15
    Units: micromole per liter
        arithmetic mean (standard deviation)
    0.4 ± 1.3
    0.1 ± 0.2
    0.7 ± 0.7
    2.4 ± 1.2
    2.6 ± 1.1
    Statistical analysis title
    Statistical analysis VX-814 400 mg versus placebo
    Comparison groups
    Parts A1 and A2 Combined: VX-814 400 mg v Parts A1, A2 and B Combined: Placebo
    Number of subjects included in analysis
    14
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0114
    Method
    t-test, 2-sided
    Parameter type
    Mean Difference
    Point estimate
    2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.5
         upper limit
    3.4
    Statistical analysis title
    Statistical analysis VX-814 600 mg versus placebo
    Comparison groups
    Part B: VX-814 600 mg v Parts A1, A2 and B Combined: Placebo
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0009
    Method
    t-test, 2-sided
    Parameter type
    Mean Difference
    Point estimate
    2.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.1
         upper limit
    3.5

    Secondary: Observed Pre-dose Plasma Concentration of VX-814

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    End point title
    Observed Pre-dose Plasma Concentration of VX-814 [2]
    End point description
    Pharmacokinetic analysis included all randomized subjects who received at least 1 dose of study drug. Here "n" signifies subjects who were evaluable at the specified time point and "99999" represents "not reported" because the number evaluable subject was 1 at the specified time point.
    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: Pharmacokinetic analysis was not applicable for Placebo arm.
    End point values
    Part A1: VX-814 100 milligrams (mg) Part A1: VX-814 200 mg Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg
    Number of subjects analysed
    4
    3
    13
    18
    Units: micrograms per milliliter
    arithmetic mean (standard deviation)
        Day 7 (n=2, 3, 9, 14)
    0.476 ± 0.375
    0.948 ± 0.512
    3.53 ± 1.72
    10.3 ± 6.55
        Day 14 (n=2, 3, 8, 17)
    0.244 ± 0.0919
    1.07 ± 0.152
    3.25 ± 2.23
    8.53 ± 11.8
        Day 21 (n=1, 1, 6, 16)
    0.700 ± 99999
    1.45 ± 99999
    3.68 ± 3.40
    7.20 ± 6.05
        Day 28 (n=3, 3, 7, 15)
    0.326 ± 0.0282
    0.993 ± 0.0300
    3.23 ± 2.82
    5.80 ± 3.73
    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
    23.1
    Reporting groups
    Reporting group title
    Part A1: VX-814 100 mg
    Reporting group description
    Subjects received VX-814 100 mg q12h in the treatment period for 28 days.

    Reporting group title
    Parts A1, A2 and B Combined: Placebo
    Reporting group description
    Subjects received placebo matched to VX-814 in the treatment period for 28 days.

    Reporting group title
    Parts A1 and A2 Combined: VX-814 400 mg
    Reporting group description
    Subjects received VX-814 400 mg q12h in the treatment period for 28 days.

    Reporting group title
    Part B: VX-814 600 mg
    Reporting group description
    Subjects received VX-814 600 mg q12h in the treatment period for 28 days.

    Reporting group title
    Part A1: VX-814 200 mg
    Reporting group description
    Subjects received VX-814 200 mg q12h in the treatment period for 28 days.

    Serious adverse events
    Part A1: VX-814 100 mg Parts A1, A2 and B Combined: Placebo Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg Part A1: VX-814 200 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 4 (50.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    1 / 3 (33.33%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Rash erythematous
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Gastrointestinal infection
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Part A1: VX-814 100 mg Parts A1, A2 and B Combined: Placebo Parts A1 and A2 Combined: VX-814 400 mg Part B: VX-814 600 mg Part A1: VX-814 200 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 4 (25.00%)
    4 / 10 (40.00%)
    9 / 13 (69.23%)
    14 / 18 (77.78%)
    0 / 3 (0.00%)
    Vascular disorders
    Hot flush
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    2 / 18 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    Psychiatric disorders
    Nightmare
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Injury, poisoning and procedural complications
    Dental restoration failure
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    2 / 13 (15.38%)
    6 / 18 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    2
    6
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    2 / 13 (15.38%)
    6 / 18 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    2
    6
    0
    Blood cholesterol increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Blood pressure increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    2 / 18 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    Eosinophil count increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Crystal urine present
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    International normalised ratio increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Protein urine present
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Prothrombin time prolonged
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 10 (10.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Dyspnoea exertional
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    Epistaxis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Respiration abnormal
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    2 / 13 (15.38%)
    2 / 18 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    2
    2
    0
    Restless legs syndrome
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 10 (10.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    3 / 18 (16.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    Abdominal distension
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 10 (10.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 10 (20.00%)
    1 / 13 (7.69%)
    3 / 18 (16.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    1
    3
    0
    Frequent bowel movements
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Dyspepsia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 10 (10.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    2 / 18 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    4
    0
    Vomiting
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Rash pruritic
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Bone pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 10 (10.00%)
    0 / 13 (0.00%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Arthralgia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Infections and infestations
    Ear infection
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Tooth abscess
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Rhinitis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    1 / 13 (7.69%)
    0 / 18 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    2 / 18 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    Vulvovaginal candidiasis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 10 (0.00%)
    0 / 13 (0.00%)
    1 / 18 (5.56%)
    0 / 3 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Mar 2020
    Amended to update inclusion criteria.
    21 May 2020
    Amended to add Parts A2 and B.

    Interruptions (globally)

    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    20 Mar 2020
    Vertex Pharmaceuticals temporarily paused screening and enrollment in the VX19-814-101 study due to the outbreak of the COVID-19 pandemic.
    28 Apr 2020

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Based on elevated AST/ALT and low achieved VX-814 exposures, Vertex concluded to terminate study early as it was not feasible to safely reach VX-814 targeted exposure level. Therefore, data are incomplete and result should be interpreted with caution
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