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    Clinical Trial Results:
    A Clinical Study of Two Doses of a Selective p38 MAP Kinase Inhibitor, VX-745, to Evaluate the Effects of 12-Week Oral Twice-Daily Dosing on Amyloid Plaque Load as Assessed by Quantitative Dynamic 11C-PiB Positive Emission Tomography (PET) Amyloid Scanning

    Summary
    EudraCT number
    		 2014-002855-25
    Trial protocol
    		 NL  
    Global end of trial date
    14 Sep 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    14 Oct 2017
    First version publication date
    14 Oct 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    EIP-VX00-745-302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02423122
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    EIP Pharma, LLC
    Sponsor organisation address
    11 Channing Street, Cambridge, MA, United States, 02138
    Public contact
    Clinical Trial Operations, Voisin Consulting, clinicaltrialinformation@voisinconsulting.com
    Scientific contact
    Clinical Trial Operations, Voisin Consulting, clinicaltrialinformation@voisinconsulting.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
    10 Mar 2017
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Sep 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the effects on amyloid plaque burden of administration of VX-745 for 12-weeks, as assessed by Dynamic 11C-PiB PET amyloid scanning in patients with MCI due to AD or mild AD
    Protection of trial subjects
    Dosimetry for the neuroimaging studies were prospectively defined and the radiation burden were assured to be within International Committee for Radiation Protection (ICRP) and World Health Organization (WHO) guidelines. Otherwise, the usual and customary safety measures for an early phase 2 clinical trial were conducted, and, as the only invasive assessments were routine blood tests, no specific additional measures to protect trial subjects needed to be taken.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    04 May 2015
    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
    Netherlands: 16
    Worldwide total number of subjects
    16
    EEA total number of subjects
    16
    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)
    5
    From 65 to 84 years
    11
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 21 [1]
    Number of subjects completed
    		 16

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Screen failure: 4
    Reason: Number of subjects
    		 Consent withdrawn by subject: 1
    Notes
    [1] - The number of subjects reported to have started the pre-assignment 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 worldwide number corresponds to the number of patients randomized (16) and not to the number of patients screened (21).
    Period 1
    Period 1 title
    Treatment period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 VX-745 40 mg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-745
    Investigational medicinal product code
    VX-745
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VX-745 capsule(s) will be administered orally, twice daily with food for 12 weeks. Doses should be taken within 30 minutes following a meal or snack. Doses should be taken approximately 12 hours apart at the same times each day throughout the study.

    Arm title
    		 VX-745 125 mg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-745
    Investigational medicinal product code
    VX-745
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VX-745 capsule(s) will be administered orally, twice daily with food for 12 weeks. Doses should be taken within 30 minutes following a meal or snack. Doses should be taken approximately 12 hours apart at the same times each day throughout the study.

    Number of subjects in period 1
    		VX-745 40 mg VX-745 125 mg
    Started
    9
    7
    Completed
    8
    7
    Not completed
    1
    0
         PET-scan failure
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    VX-745 40 mg
    Reporting group description
    		 -

    Reporting group title
    VX-745 125 mg
    Reporting group description
    		 -

    Reporting group values
    		 VX-745 40 mg VX-745 125 mg Total
    Number of subjects
    		 9 7 16
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 2 3 5
        From 65-84 years
    		 7 4 11
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 68.33 (60 to 76) 65.29 (60 to 75) -
    Gender categorical
    Units: Subjects
        Female
    		 2 4 6
        Male
    		 7 3 10

    End points

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    End points reporting groups
    Reporting group title
    VX-745 40 mg
    Reporting group description
    		 -

    Reporting group title
    VX-745 125 mg
    Reporting group description
    		 -

    Primary: PET Metrics - Global (change from baseline)

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    End point title
    		 PET Metrics - Global (change from baseline)
    End point description
    End point type
    Primary
    End point timeframe
    Dynamic PET scanning with full quantitative analysis will be performed at baseline and at the end of treatment
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    8
    7
    Units: N/A
        arithmetic mean (full range (min-max))
    -0.02 (-0.27 to 0.17)
    0.03 (-0.03 to 0.09)
    Statistical analysis title
    		 Wilcoxon Signed Rank Test
    Comparison groups
    VX-745 40 mg v VX-745 125 mg
    Number of subjects included in analysis
    15
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.619
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: PET Metrics - Global (number of responders)

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    End point title
    		 PET Metrics - Global (number of responders) [1]
    End point description
    End point type
    Primary
    End point timeframe
    Dynamic PET scanning with full quantitative analysis will be performed at baseline and at the end of treatment
    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: Descriptive statistics, i.e. number and percentage of responders by dose group were tabulated.
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    8
    7
    Units: Number of responders
    3
    1
    No statistical analyses for this end point

    Secondary: WMS - Overall Score (change from baseline)

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    End point title
    		 WMS - Overall Score (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    WMS (immediate and delayed recall) were performed at Days 1, 28 and 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    8
    7
    Units: N/A
        arithmetic mean (full range (min-max))
    9.5 (-11 to 19)
    37.57 (-7 to 76)
    Statistical analysis title
    		 Wilcoxon signed rank test
    Statistical analysis description
    P-value is based on Wilcoxon signed rank test that tested for improvement (1-sided)
    Comparison groups
    VX-745 40 mg v VX-745 125 mg
    Number of subjects included in analysis
    15
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.002
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: WMS - Overall Composite (number of responders)

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    End point title
    		 WMS - Overall Composite (number of responders)
    End point description
    End point type
    Secondary
    End point timeframe
    WMS (immediate and delayed recall) were performed at Days 1, 28 and 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    8
    7
    Units: Number of responders
    6
    6
    No statistical analyses for this end point

    Secondary: MMSE - Composite Score (Day 7)

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    End point title
    		 MMSE - Composite Score (Day 7)
    End point description
    End point type
    Secondary
    End point timeframe
    MMSE scores were evaluated during 6 visits, including screening, Days 1, 28, 56, 84 and follow-up visits
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    8
    7
    Units: N/A
        arithmetic mean (full range (min-max))
    23.88 (18 to 28)
    24.29 (20 to 30)
    No statistical analyses for this end point

    Secondary: VX-745 Plasma Concentration - <3 hours

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    End point title
    		 VX-745 Plasma Concentration - <3 hours
    End point description
    End point type
    Secondary
    End point timeframe
    On Days 14, 28, 56 and 84, a 5 mL sample of whole blood will be collected.
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: ng.hr/mL
        arithmetic mean (full range (min-max))
    9.77 (5.43 to 19.1)
    24.97 (9.88 to 50.1)
    No statistical analyses for this end point

    Secondary: VX-745 Plasma Concentration - 3 to 4 hours

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    End point title
    		 VX-745 Plasma Concentration - 3 to 4 hours
    End point description
    End point type
    Secondary
    End point timeframe
    On Days 14, 28, 56 and 84, a 5 mL sample of whole blood will be collected.
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: ng.hr/mL
        arithmetic mean (full range (min-max))
    11.62 (6.18 to 22.1)
    24.16 (17.7 to 36.2)
    No statistical analyses for this end point

    Secondary: VX-745 Plasma Concentration - >4 to <6 hours

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    End point title
    		 VX-745 Plasma Concentration - >4 to <6 hours
    End point description
    End point type
    Secondary
    End point timeframe
    On Days 14, 28, 56 and 84, a 5 mL sample of whole blood will be collected.
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: ng.hr/mL
        arithmetic mean (full range (min-max))
    7.59 (2.35 to 18.2)
    15.37 (1.84 to 35.1)
    No statistical analyses for this end point

    Secondary: VX-745 Plasma Concentration - ≥6 hours

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    End point title
    		 VX-745 Plasma Concentration - ≥6 hours
    End point description
    End point type
    Secondary
    End point timeframe
    On Days 14, 28, 56 and 84, a 5 mL sample of whole blood will be collected.
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: ng.hr/mL
        arithmetic mean (full range (min-max))
    4.95 (3.01 to 8.38)
    12.02 (5.96 to 20.8)
    No statistical analyses for this end point

    Secondary: Summary of Adverse Events (AE)

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    End point title
    		 Summary of Adverse Events (AE)
    End point description
    End point type
    Secondary
    End point timeframe
    Adverse events that occur from the first dose of study drug on Day 1 through the Follow-up Visit will be considered treatment-emergent adverse events
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: Subjects with any AE
    19
    9
    No statistical analyses for this end point

    Secondary: Absolute Basophils (change from baseline)

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    End point title
    		 Absolute Basophils (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    0.01 (-0.01 to 0.03)
    0 (-0.04 to 0.06)
    No statistical analyses for this end point

    Secondary: Absolute Eosinophils (change from baseline)

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    End point title
    		 Absolute Eosinophils (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    0.04 (-0.01 to 0.09)
    0.01 (-0.11 to 0.07)
    No statistical analyses for this end point

    Secondary: Absolute Lymphocytes (change from baseline)

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    End point title
    		 Absolute Lymphocytes (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    0.29 (-0.23 to 0.75)
    0.11 (-0.39 to 0.49)
    No statistical analyses for this end point

    Secondary: Absolute Monocytes (change from baseline)

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    End point title
    		 Absolute Monocytes (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    -0.03 (-0.45 to 0.12)
    0.02 (-0.42 to 0.26)
    No statistical analyses for this end point

    Secondary: Absolute Neutrophils (change from baseline)

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    End point title
    		 Absolute Neutrophils (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    -0.11 (-3.61 to 1.41)
    -0.66 (-2.07 to 0.41)
    No statistical analyses for this end point

    Secondary: Basophils Percentage (change from baseline)

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    End point title
    		 Basophils Percentage (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: percent
        arithmetic mean (full range (min-max))
    0.08 (-0.5 to 0.7)
    -0.03 (-0.8 to 0.8)
    No statistical analyses for this end point

    Secondary: Eosinophils Percentage (change from baseline)

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    End point title
    		 Eosinophils Percentage (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: percent
        arithmetic mean (full range (min-max))
    0.3 (-0.7 to 1.6)
    0.15 (-1.5 to 1.4)
    No statistical analyses for this end point

    Secondary: Lymphocytes Percentage (change from baseline)

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    End point title
    		 Lymphocytes Percentage (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: percent
        arithmetic mean (full range (min-max))
    3.68 (-2.6 to 19.6)
    2.03 (-5.3 to 9.6)
    No statistical analyses for this end point

    Secondary: Monocytes Percentage (change from baseline)

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    End point title
    		 Monocytes Percentage (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: percent
        arithmetic mean (full range (min-max))
    -0.91 (-4.8 to 0.6)
    0.48 (-6.2 to 3.3)
    No statistical analyses for this end point

    Secondary: Neutrophils Percentage (change from baseline)

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    End point title
    		 Neutrophils Percentage (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    6
    Units: percent
        arithmetic mean (full range (min-max))
    -3.1 (-20.1 to 2.7)
    -4.43 (-13.6 to 5.1)
    No statistical analyses for this end point

    Secondary: Erythrocytes (change from baseline)

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    End point title
    		 Erythrocytes (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: X10^12/L
        arithmetic mean (full range (min-max))
    -0.05 (-0.39 to 0.37)
    0.03 (-0.31 to 0.33)
    No statistical analyses for this end point

    Secondary: MCH (change from baseline)

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    End point title
    		 MCH (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: AMOL/CEL
        arithmetic mean (full range (min-max))
    51.67 (-81 to 211)
    30 (-43 to 130)
    No statistical analyses for this end point

    Secondary: MCHC (change from baseline)

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    End point title
    		 MCHC (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.52 (-0.9 to 2.4)
    0 (-0.8 to 1.1)
    No statistical analyses for this end point

    Secondary: MCV (change from baseline)

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    End point title
    		 MCV (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: FL
        arithmetic mean (full range (min-max))
    0.22 (-1 to 1)
    1.43 (0 to 3)
    No statistical analyses for this end point

    Secondary: Hemoglobin (change from baseline)

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    End point title
    		 Hemoglobin (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.14 (-0.8 to 0.9)
    0.21 (-0.4 to 0.5)
    No statistical analyses for this end point

    Secondary: Leukocytes (change from baseline)

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    End point title
    		 Leukocytes (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    0.18 (-3.2 to 1.8)
    -0.27 (-1.5 to 1)
    No statistical analyses for this end point

    Secondary: Platelets (change from baseline)

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    End point title
    		 Platelets (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: X10^9/L
        arithmetic mean (full range (min-max))
    10.67 (-9 to 40)
    -24.71 (-145 to 33)
    No statistical analyses for this end point

    Secondary: Albumin (change from baseline)

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    End point title
    		 Albumin (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: G/L
        arithmetic mean (full range (min-max))
    0.31 (-2.6 to 3.7)
    -1.14 (-4.7 to 1.2)
    No statistical analyses for this end point

    Secondary: Alkaline Phosphatase (change from baseline)

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    End point title
    		 Alkaline Phosphatase (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: U/L
        arithmetic mean (full range (min-max))
    -1.08 (-21.6 to 9.1)
    0.39 (-10.2 to 14.1)
    No statistical analyses for this end point

    Secondary: ALT (change from baseline)

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    End point title
    		 ALT (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: U/L
        arithmetic mean (full range (min-max))
    2.52 (-4.8 to 10.1)
    1.6 (-7 to 6.5)
    No statistical analyses for this end point

    Secondary: AST (change from baseline)

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    End point title
    		 AST (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: U/L
        arithmetic mean (full range (min-max))
    0.3 (-4.6 to 6.7)
    0.93 (-8.5 to 6.1)
    No statistical analyses for this end point

    Secondary: Total Bilirubin (change from baseline)

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    End point title
    		 Total Bilirubin (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MICROMOL/L
        arithmetic mean (full range (min-max))
    -0.18 (-1.8 to 1.7)
    -0.91 (-8.2 to 2.9)
    No statistical analyses for this end point

    Secondary: Glucose (change from baseline)

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    End point title
    		 Glucose (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.69 (-2.33 to 4.1)
    -1.2 (-5.18 to 2.23)
    No statistical analyses for this end point

    Secondary: Blood Urea Nitrogen (change from baseline)

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    End point title
    		 Blood Urea Nitrogen (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.22 (-1.2 to 2.2)
    0.06 (-1 to 1.1)
    No statistical analyses for this end point

    Secondary: Calcium (change from baseline)

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    End point title
    		 Calcium (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.01 (-0.1 to 0.08)
    -0.02 (-0.1 to 0.07)
    No statistical analyses for this end point

    Secondary: Bicarbonate (change from baseline)

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    End point title
    		 Bicarbonate (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    -0.6 (-3.9 to 1.7)
    -0.2 (-2.6 to 2.3)
    No statistical analyses for this end point

    Secondary: Chloride (change from baseline)

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    End point title
    		 Chloride (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    8
    6
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    -0.7 (-3.6 to 3.6)
    1.45 (-2 to 4.8)
    No statistical analyses for this end point

    Secondary: Total cholesterol (change from baseline)

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    End point title
    		 Total cholesterol (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.03 (-1.02 to 1.32)
    0.4 (-0.19 to 0.79)
    No statistical analyses for this end point

    Secondary: Triglycerides (change from baseline)

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    End point title
    		 Triglycerides (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    -0.2 (-15.6 to 17.3)
    4 (0.7 to 6.6)
    No statistical analyses for this end point

    Secondary: Creatinine (change from baseline)

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    End point title
    		 Creatinine (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MICROMOL/L
        arithmetic mean (full range (min-max))
    -0.2 (-15.6 to 17.3)
    4 (0.7 to 6.6)
    No statistical analyses for this end point

    Secondary: GGT (change from baseline)

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    End point title
    		 GGT (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: U/L
        arithmetic mean (full range (min-max))
    4.63 (-7.4 to 22.2)
    10.07 (0.4 to 37)
    No statistical analyses for this end point

    Secondary: LDH (change from baseline)

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    End point title
    		 LDH (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    7
    5
    Units: U/L
        arithmetic mean (full range (min-max))
    -3.84 (-18.6 to 30)
    -0.26 (-14.7 to 14.3)
    No statistical analyses for this end point

    Secondary: Phosphate (change from baseline)

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    End point title
    		 Phosphate (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    -0.02 (-0.37 to 0.49)
    0.07 (-0.08 to 0.19)
    No statistical analyses for this end point

    Secondary: Potassium (change from baseline)

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    End point title
    		 Potassium (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.11 (-0.33 to 0.69)
    -0.05 (-1.19 to 0.44)
    No statistical analyses for this end point

    Secondary: Sodium (change from baseline)

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    End point title
    		 Sodium (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    -0.73 (-4.8 to 3.2)
    1.01 (-0.6 to 2.6)
    No statistical analyses for this end point

    Secondary: Total protein (change from baseline)

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    End point title
    		 Total protein (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: G/L
        arithmetic mean (full range (min-max))
    1.28 (-3.6 to 5.2)
    -0.8 (-6.2 to 4.3)
    No statistical analyses for this end point

    Secondary: Uric acid (change from baseline)

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    End point title
    		 Uric acid (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: MICROMOL/L
        arithmetic mean (full range (min-max))
    -13.86 (-50.3 to 22.6)
    -11.34 (-20.2 to -6.1)
    No statistical analyses for this end point

    Secondary: HDL (change from baseline)

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    End point title
    		 HDL (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    2
    1
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    -0.05 (-0.16 to 0.06)
    -0.09 (-0.09 to -0.09)
    No statistical analyses for this end point

    Secondary: LDL (change from baseline)

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    End point title
    		 LDL (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Clinical laboratory sampling performed at screening, at each treatment period study visits (from Day 1 to 84) and at the follow-up visit
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    2
    1
    Units: MMOL/L
        arithmetic mean (full range (min-max))
    0.1 (0 to 0.2)
    0.3 (0.3 to 0.3)
    No statistical analyses for this end point

    Secondary: Systolic Blood Pressure (change from baseline)

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    End point title
    		 Systolic Blood Pressure (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Vital signs measurements performed at screening and at Day 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: mmHg
        arithmetic mean (full range (min-max))
    -4.3 (-28 to 36)
    -6.6 (-26 to 30)
    No statistical analyses for this end point

    Secondary: Diastolic Blood Pressure (change from baseline)

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    End point title
    		 Diastolic Blood Pressure (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Vital signs measurements performed at screening and at Day 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: mmHg
        arithmetic mean (full range (min-max))
    -1.1 (-15 to 17)
    -5.3 (-15 to 2)
    No statistical analyses for this end point

    Secondary: Heart Rate (change from baseline)

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    End point title
    		 Heart Rate (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Vital signs measurements performed at screening and at Day 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: beats/min
        arithmetic mean (full range (min-max))
    7.1 (-6 to 23)
    4.3 (-8 to 19)
    No statistical analyses for this end point

    Secondary: Weight (change from baseline)

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    End point title
    		 Weight (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Vital signs measurements performed at screening and at Day 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: kg
        arithmetic mean (full range (min-max))
    1.5 (0 to 5)
    0.5 (-0.4 to 1.4)
    No statistical analyses for this end point

    Secondary: Temperature (change from baseline)

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    End point title
    		 Temperature (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Vital signs measurements performed at screening and at Day 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: celsius
        arithmetic mean (full range (min-max))
    -0.2 (-1.5 to 0.7)
    0.2 (-0.4 to 0.9)
    No statistical analyses for this end point

    Secondary: BMI (change from baseline)

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    End point title
    		 BMI (change from baseline)
    End point description
    End point type
    Secondary
    End point timeframe
    Vital signs measurements performed at screening and at Day 84
    End point values
    		 VX-745 40 mg VX-745 125 mg
    Number of subjects analysed
    9
    7
    Units: kg/m^2
        arithmetic mean (full range (min-max))
    0.5 (0 to 1.79)
    0.2 (-0.12 to 0.59)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events that occur from the first dose of study drug on Day 1 through the Follow-up Visit will be considered treatment-emergent adverse events.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    VX-745 40 mg
    Reporting group description
    		 -

    Reporting group title
    VX-745 125 mg
    Reporting group description
    		 -

    Serious adverse events
    		 VX-745 40 mg VX-745 125 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 VX-745 40 mg VX-745 125 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 9 (100.00%)
    5 / 7 (71.43%)
    Vascular disorders
    VASCULAR DISORDERS NEC
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    19
    9
    VASCULAR HYPERTENSIVE DISORDERS
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    19
    9
    General disorders and administration site conditions
    GENERAL SYSTEM DISORDERS NEC
         subjects affected / exposed
    2 / 9 (22.22%)
    1 / 7 (14.29%)
         occurrences all number
    19
    9
    Respiratory, thoracic and mediastinal disorders
    RESPIRATORY DISORDERS NEC
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Psychiatric disorders
    ANXIETY DISORDERS AND SYMPTOMS
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    PERSONALITY DISORDERS AND DISTURBANCES IN BEHAVIOUR
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Investigations
    WATER, ELECTROLYTE AND MINERAL INVESTIGATIONS
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Injury, poisoning and procedural complications
    BONE AND JOINT INJURIES
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Nervous system disorders
    NEUROLOGICAL DISORDERS NEC
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 7 (14.29%)
         occurrences all number
    19
    9
    SPINAL CORD AND NERVE ROOT DISORDERS
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Blood and lymphatic system disorders
    ANAEMIAS NONHAEMOLYTIC AND MARROW DEPRESSION
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Ear and labyrinth disorders
    INNER EAR AND VIIITH CRANIAL NERVE DISORDERS
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Gastrointestinal disorders
    GASTROINTESTINAL MOTILITY AND DEFAECATION CONDITIONS
         subjects affected / exposed
    4 / 9 (44.44%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    GASTROINTESTINAL SIGNS AND SYMPTOMS
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 7 (14.29%)
         occurrences all number
    19
    9
    Skin and subcutaneous tissue disorders
    EPIDERMAL AND DERMAL CONDITIONS
         subjects affected / exposed
    0 / 9 (0.00%)
    3 / 7 (42.86%)
         occurrences all number
    19
    9
    Endocrine disorders
    HYPOTHALAMUS AND PITUITARY GLAND DISORDERS
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    THYROID GLAND DISORDERS
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    19
    9
    Infections and infestations
    INFECTIONS - PATHOGEN UNSPECIFIED
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 7 (14.29%)
         occurrences all number
    19
    9

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Dec 2015
    Amendment 2.0 dated 22 December 2015
    15 Mar 2016
    Amendment 3.0 dated 15 March 2016

    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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