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    Clinical Trial Results:
    A Phase 2, Randomized, Double-blind, Placebo-controlled, 6-Week, Parallel-design Study of the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy

    Summary
    EudraCT number
    		 2017-001042-10
    Trial protocol
    		 DE   NL   IT  
    Global end of trial date
    08 Nov 2018

    Results information
    Results version number
    		 v2(current)
    This version publication date
    13 Dec 2021
    First version publication date
    27 Nov 2019
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Secondary endpoints data will be added

    Trial information

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    Trial identification
    Sponsor protocol code
    VX16-150-102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03304522
    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
    05 Dec 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Oct 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Nov 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of VX-150 for the treatment of pain caused by small fiber neuropathy.
    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
    20 Sep 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Italy: 10
    Country: Number of subjects enrolled
    Netherlands: 6
    Country: Number of subjects enrolled
    United States: 71
    Country: Number of subjects enrolled
    Germany: 2
    Worldwide total number of subjects
    89
    EEA total number of subjects
    18
    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)
    66
    From 65 to 84 years
    23
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 89 subjects were enrolled and randomized in the study.

    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
    		 VX-150
    Arm description
    		 Subjects received VX-150 once daily for 6 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-150
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-150 1250 milligrams (mg) once daily.

    Arm title
    		 Placebo
    Arm description
    		 Subjects received placebo matched to VX-150 once daily for 6 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo (matched to VX-150)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received placebo matched to VX-150 once daily.

    Number of subjects in period 1
    		VX-150 Placebo
    Started
    46
    43
    Completed
    45
    35
    Not completed
    1
    8
         Other
    -
    1
         Withdrawal of consent (for other reason)
    -
    2
         Adverse event
    -
    4
         Withdrawal of consent (due to lack of efficacy)
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    VX-150
    Reporting group description
    		 Subjects received VX-150 once daily for 6 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to VX-150 once daily for 6 weeks.

    Reporting group values
    		 VX-150 Placebo Total
    Number of subjects
    		 46 43 89
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 55.1 ( 12.34 ) 58.1 ( 11.86 ) -
    Gender categorical
    Units: Subjects
        Female
    		 22 21 43
        Male
    		 24 22 46
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 3 5 8
        Not Hispanic or Latino
    		 43 38 81
        Unknown or Not Reported
    		 0 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 1 1
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 6 4 10
        White
    		 39 34 73
        More than one race
    		 1 2 3
        Unknown or Not Reported
    		 0 2 2
    Pain Intensity at Baseline on 11-point Numeric Rating Scale (NRS)
    Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain). Higher score indicates greater level of pain. Baseline score was average of daily pain scores from Day -7 to Day -1.
    Units: units on a scale
        arithmetic mean (standard deviation)
    		 6.433 ( 1.440 ) 5.990 ( 1.413 ) -

    End points

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    End points reporting groups
    Reporting group title
    VX-150
    Reporting group description
    		 Subjects received VX-150 once daily for 6 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to VX-150 once daily for 6 weeks.

    Primary: Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS

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    End point title
    		 Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS
    End point description
    Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. 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 Week 6
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    46
    43
    Units: units on a scale
        least squares mean (standard error)
    -2.018 ( 0.274 )
    -0.933 ( 0.287 )
    Statistical analysis title
    		 Statistical Analysis
    Comparison groups
    VX-150 v Placebo
    Number of subjects included in analysis
    89
    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
    -1.085
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.876
         upper limit
    		 -0.293

    Secondary: Percentage of Subjects With Greater Than or Equal to (>=) 30 Percent (%) Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS

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    End point title
    		 Percentage of Subjects With Greater Than or Equal to (>=) 30 Percent (%) Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS
    End point description
    Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. Percentage of subjects >= 30% reduction in the weekly average of daily pain intensity on the 11-Point NRS were reported. FAS. Here, 'number of subjects analysed' signifies subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 6
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    40
    34
    Units: percentage of subjects
        number (not applicable)
    45.0
    26.5
    No statistical analyses for this end point

    Secondary: Percentage of Subjects With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS

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    End point title
    		 Percentage of Subjects With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS
    End point description
    Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. Percentage of subjects >= 50% reduction in the weekly average of daily pain intensity on the 11-Point NRS were reported. FAS. Here, 'number of subjects analysed' signifies subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 6
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    40
    34
    Units: percentage of subjects
        number (not applicable)
    32.5
    17.6
    No statistical analyses for this end point

    Secondary: Change in the Daily Sleep Interference Scale (DSIS)

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    End point title
    		 Change in the Daily Sleep Interference Scale (DSIS)
    End point description
    Pain-associated sleep interference was assessed using DSIS, based on an 11-point scale (where 0 signified none: pain does not interfere with sleep and 10 signified severe: pain completely interferes with sleep, unable to sleep). Higher score indicates greater pain associated sleep interference. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 6
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    46
    43
    Units: units on a scale
        least squares mean (standard error)
    -1.777 ( 0.276 )
    -0.665 ( 0.289 )
    Statistical analysis title
    		 VX-150 vs Placebo
    Comparison groups
    VX-150 v Placebo
    Number of subjects included in analysis
    89
    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
    -1.111
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.911
         upper limit
    		 -0.312

    Secondary: Percentage of Subjects Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale

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    End point title
    		 Percentage of Subjects Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale
    End point description
    PGIC scale evaluated the change in activity limitations, symptoms, emotions, and overall quality of life (QoL) related to the subjects painful condition on 7-point scale from 1 (improved) to 7 (worse). Subjects were categorized as following: scale from 1 - 2 were categorized as "improved", scale from 3 - 4 as "no change" and scale from 5 - 7 were categorized as "worse". Percentage of subjects categorized as improved on PGIC scale at week 6 were reported for this outcome measure. FAS. Here, 'number of subjects analysed' signifies subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    At Week 6
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    46
    43
    Units: percentage of subjects
        number (not applicable)
    39.5
    13.5
    No statistical analyses for this end point

    Secondary: Change in Pain Intensity on the 11-Point NRS

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    End point title
    		 Change in Pain Intensity on the 11-Point NRS
    End point description
    Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 6
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    46
    43
    Units: units on a scale
        least squares mean (standard error)
    -18.4 ( 3.1 )
    -5.0 ( 3.1 )
    Statistical analysis title
    		 VX-150 vs Placebo
    Comparison groups
    Placebo v VX-150
    Number of subjects included in analysis
    89
    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
    -0.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.5
         upper limit
    		 0.3

    Secondary: Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114

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    End point title
    		 Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 [1]
    End point description
    Pharmacokinetic (PK) set included subjects who received at least 1 dose of study drug and for whom the primary PK data was considered to be sufficient and interpretable.
    End point type
    Secondary
    End point timeframe
    Pre-dose at Day 7
    Notes
    [1] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was applicable for VX-150 arm. Therefore, data are reported for this arm only.
    End point values
    		 VX-150
    Number of subjects analysed
    12
    Units: microgram per milliliter (mcg/mL)
    arithmetic mean (standard deviation)
        VRT-1207355
    3.89 ( 2.73 )
        VRT-1268114
    1.35 ( 0.752 )
    No statistical analyses for this end point

    Secondary: Number of Subjects With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses

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    End point title
    		 Number of Subjects With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
    End point description
    The C-SSRS is an interview-based rating scale was evaluated through a series of questions about suicidal thoughts and behaviors with the possible answers yes or no. Yes represents a worse outcome. Clinically Meaningfulness of C-SSRS responses were judged by investigator based on answers received from subjects. Here "99999" represents that no subject had any clinically meaningful findings in C-SSRS responses. Safety Set included all subjects who have received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Week 10
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    46
    43
    Units: subjects
    99999
    99999
    No statistical analyses for this end point

    Secondary: 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)
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 up to Week 10
    End point values
    		 VX-150 Placebo
    Number of subjects analysed
    46
    43
    Units: subjects
        Subjects with AEs
    29
    24
        Subjects with SAEs
    0
    3
    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 10
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Subjects received placebo matched to VX-150 once daily for 6 weeks.

    Reporting group title
    VX-150
    Reporting group description
    		 Subjects received VX-150 once daily for 6 weeks.

    Serious adverse events
    		 Placebo VX-150
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 43 (6.98%)
    0 / 46 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Tendon rupture
         subjects affected / exposed
    1 / 43 (2.33%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 43 (2.33%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Diverticulitis
         subjects affected / exposed
    1 / 43 (2.33%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo VX-150
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 43 (16.28%)
    16 / 46 (34.78%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 43 (11.63%)
    11 / 46 (23.91%)
         occurrences all number
    6
    14
    Gastrointestinal disorders
    Dry mouth
         subjects affected / exposed
    1 / 43 (2.33%)
    3 / 46 (6.52%)
         occurrences all number
    1
    3
    Nausea
         subjects affected / exposed
    1 / 43 (2.33%)
    3 / 46 (6.52%)
         occurrences all number
    1
    3
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 43 (2.33%)
    3 / 46 (6.52%)
         occurrences all number
    1
    3
    Muscle spasms
         subjects affected / exposed
    0 / 43 (0.00%)
    3 / 46 (6.52%)
         occurrences all number
    0
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Apr 2017
    Amended inclusion criteria to clarify the study population and Body Mass Index (BMI)
    19 May 2017
    Amended the dose limit of VX-150
    11 Aug 2017
    Allowed inclusion of women of childbearing potential; updated the upper limit of BMI and the visit requirements
    13 Apr 2018
    Updated inclusion and exclusion criteria to facilitate subject recruitment
    25 May 2018
    The protocol was amended to enable timely assessment of study data to support continued development

    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
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