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    Clinical Trial Results:
    A Phase 3, Double-Blind, Randomized, Vehicle-Controlled, Efficacy and Safety Study of Ruxolitinib Cream Followed by an Extension Period in Participants With Vitiligo

    Summary
    EudraCT number
    		 2019-000846-37
    Trial protocol
    		 FR   PL   DE   BG   ES   IT  
    Global end of trial date
    21 Oct 2021

    Results information
    Results version number
    		 v2(current)
    This version publication date
    11 Oct 2022
    First version publication date
    05 May 2022
    Other versions
    		 v1
    Version creation reason

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    INCB 18424-306
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04052425
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Incyte Corporation
    Sponsor organisation address
    1801 Augustine Cutoff Drive, Wilmington, United States, 19803
    Public contact
    Study Director, Incyte Corporation, 1 18554633463, medinfo@incyte.com
    Scientific contact
    Study Director, Incyte Corporation, 1 18554633463, medinfo@incyte.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002618-PIP02-20
    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
    21 Oct 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The purpose of this study was to evaluate the efficacy and safety of ruxolitinib cream in adolescent and adult participants with non-segmental vitiligo with facial involvement for whom total body involved vitiligo area (facial and nonfacial) did not exceed 10% body surface area (BSA).
    Protection of trial subjects
    This study was performed in accordance with ethical principles that have their origin in the Declaration of Helsinki and conducted in adherence to the study Protocol, applicable Good Clinical Practices, and applicable laws and country-specific regulations in which the study was being conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Sep 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 204
    Country: Number of subjects enrolled
    Bulgaria: 17
    Country: Number of subjects enrolled
    Canada: 16
    Country: Number of subjects enrolled
    France: 19
    Country: Number of subjects enrolled
    Germany: 6
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Poland: 63
    Country: Number of subjects enrolled
    Spain: 4
    Worldwide total number of subjects
    330
    EEA total number of subjects
    110
    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)
    36
    Adults (18-64 years)
    265
    From 65 to 84 years
    29
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was conducted at 45 study centers in North America and Europe.

    Pre-assignment
    Screening details
    		 A total of 330 participants were randomized into the study. All randomized participants (Intent-to-Treat Population) applied study drug at least once (Safety Population), and 283 participants applied ruxolitinib cream at least once during the Treatment-Extension (TE) Period (TE Evaluable Population).

    Period 1
    Period 1 title
    24-Week Double-blind Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID
    Arm description
    		 Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ruxolitinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cream
    Routes of administration
    Topical use
    Dosage and administration details
    1.5% cream twice daily

    Arm title
    		 Double-Blind Period: Vehicle cream BID
    Arm description
    		 Participants applied matching vehicle cream BID for 24 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Vehicle
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cream
    Routes of administration
    Topical use
    Dosage and administration details
    twice daily

    Number of subjects in period 1
    		Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Started
    221
    109
    Completed
    193
    90
    Not completed
    28
    19
         Physician decision
    1
    -
         Consent withdrawn by subject
    9
    10
         Adverse event, non-fatal
    -
    1
         Discontinued Treatment Due to COVID-19 Pandemic
    3
    -
         Lost to follow-up
    14
    7
         Protocol deviation
    1
    -
         Lack of efficacy
    -
    1
    Period 2
    Period 2 title
    28-Week Treatment-Extension Period
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID
    Arm description
    		 Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ruxolitinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cream
    Routes of administration
    Topical use
    Dosage and administration details
    1.5% cream twice daily

    Arm title
    		 TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Arm description
    		 Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5% BID for 28 weeks in the Treatment-Extension Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vehicle
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cream
    Routes of administration
    Topical use
    Dosage and administration details
    twice daily

    Investigational medicinal product name
    ruxolitinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cream
    Routes of administration
    Topical use
    Dosage and administration details
    1.5% cream twice daily

    Number of subjects in period 2
    		Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Started
    193
    90
    Completed
    174
    80
    Not completed
    19
    10
         Consent withdrawn by subject
    10
    7
         Physician decision
    -
    1
         Adverse event, non-fatal
    1
    -
         Participant Moved
    1
    1
         Lost to follow-up
    5
    1
         Sponsor Opinion Due to Safety Reason
    1
    -
         Lack of efficacy
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Double-Blind Period: Ruxolitinib cream 1.5% BID
    Reporting group description
    		 Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.

    Reporting group title
    Double-Blind Period: Vehicle cream BID
    Reporting group description
    		 Participants applied matching vehicle cream BID for 24 weeks.

    Reporting group values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID Total
    Number of subjects
    		 221 109 330
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 25 11 36
        Adults (18-64 years)
    		 180 85 265
        From 65-84 years
    		 16 13 29
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 40.5 ( 15.44 ) 39.7 ( 16.71 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 136 50 186
        Male
    		 85 59 144
    Race, Customized
    Units: Subjects
        White
    		 180 96 276
        Black/African American
    		 11 4 15
        Asian
    		 5 4 9
        American Indian/Alaska Native
    		 1 0 1
        Not Reported
    		 16 3 19
        Latino
    		 3 1 4
        Hispanic or Latino
    		 1 0 1
        Iranian
    		 1 0 1
        Indian
    		 1 1 2
        Hispanic
    		 1 0 1
        Black-Hispanic
    		 1 0 1
    Ethnicity, Customized
    Units: Subjects
        Hispanic or Latino
    		 53 20 73
        Not Hispanic or Latino
    		 151 86 237
        Not Reported
    		 15 3 18
        Unknown
    		 1 0 1
        Captured as "Other"
    		 1 0 1
    Face Vitiligo Area Scoring Index (F-VASI)
    F-VASI was measured by the percentage of vitiligo involvement (percentage of body surface area [BSA]; assessed by the Investigator) and the degree of depigmentation: 0% (no depigmentation), 10% (specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), 100% (no pigment). F-VASI was derived by multiplying the vitiligo involvement values by the percentage of affected skin for each facial site and summing all values (range: 0-3; higher values=worse outcome).
    Units: scores on a scale
        arithmetic mean (standard deviation)
    		 0.932 ( 0.5813 ) 0.999 ( 0.5942 ) -
    Facial Body Surface Area (F-BSA) Involvement
    F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area "Face" was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area "Face" did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids.
    Units: percentage of facial surface area
        arithmetic mean (standard deviation)
    		 1.05 ( 0.692 ) 1.15 ( 0.710 ) -
    Total Body Vitiligo Area Scoring Index (T-VASI)
    T-VASI was measured by the percentage of vitiligo involvement from all body regions (percentage of BSA; Investigator assessed) and the degree of depigmentation: 0% (no depigmentation), 10% (specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), 100% (no pigment). T-VASI was derived by multiplying the vitiligo involvement values by the percentage of affected skin for each site and summing all values (possible range: 0-100; higher values=worse outcome).
    Units: scores on a scale
        arithmetic mean (standard deviation)
    		 6.489 ( 2.0228 ) 6.424 ( 1.9241 ) -
    Total Body Surface Area (T-BSA) Involvement
    T-BSA involvement was the proportion of the body surface area with vitiligo. The body was divided into the following 6 separate and mutually exclusive sites: (1) head/neck, (2) hands, (3) upper extremities (excluding hands), (4) trunk, (5) lower extremities (excluding feet), and (6) feet.
    Units: percentage of total body surface area
        arithmetic mean (standard deviation)
    		 7.28 ( 2.033 ) 7.22 ( 2.008 ) -

    End points

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    End points reporting groups
    Reporting group title
    Double-Blind Period: Ruxolitinib cream 1.5% BID
    Reporting group description
    		 Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.

    Reporting group title
    Double-Blind Period: Vehicle cream BID
    Reporting group description
    		 Participants applied matching vehicle cream BID for 24 weeks.
    Reporting group title
    Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID
    Reporting group description
    		 Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.

    Reporting group title
    TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Reporting group description
    		 Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5% BID for 28 weeks in the Treatment-Extension Period.

    Subject analysis set title
    Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.

    Subject analysis set title
    TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5% BID for 28 weeks in the Treatment-Extension Period.

    Primary: Percentage of participants achieving a ≥ 75% Improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24

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    End point title
    		 Percentage of participants achieving a ≥ 75% Improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24
    End point description
    An F-VASI75 responder achieved at least 75% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area [BSA]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
    End point type
    Primary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
        number (not applicable)
    29.8
    7.4
    Statistical analysis title
    		 exact logistic regression
    Comparison groups
    Double-Blind Period: Vehicle cream BID v Double-Blind Period: Ruxolitinib cream 1.5% BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001 [1]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    5.28
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.341
         upper limit
    		 11.903
    Notes
    [1] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).

    Secondary: Percentage of participants achieving a ≥ 50% Improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24

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    End point title
    		 Percentage of participants achieving a ≥ 50% Improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24
    End point description
    An F-VASI50 responder achieved at least 50% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
        number (not applicable)
    51.2
    16.9
    Statistical analysis title
    		 exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001 [2]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    5.18
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.831
         upper limit
    		 9.482
    Notes
    [2] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).

    Secondary: Percentage of participants achieving a ≥ 90% Improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24

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    End point title
    		 Percentage of participants achieving a ≥ 90% Improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24
    End point description
    An F-VASI90 responder achieved at least 90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
        number (not applicable)
    15.3
    2.2
    Statistical analysis title
    		 exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0038 [3]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    8.49
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.997
         upper limit
    		 36.048
    Notes
    [3] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).

    Secondary: Percentage of participants achieving a ≥ 50% Improvement from Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24

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    End point title
    		 Percentage of participants achieving a ≥ 50% Improvement from Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24
    End point description
    A T-VASI50 responder achieved at least 50% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
        number (not applicable)
    20.6
    5.1
    Statistical analysis title
    		 exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.002 [4]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    4.93
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.795
         upper limit
    		 13.566
    Notes
    [4] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).

    Secondary: Percentage of participants achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24

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    End point title
    		 Percentage of participants achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24
    End point description
    The VNS is a patient‐reported measure of vitiligo treatment success that is rated on a 5-point scale. The Baseline facial photograph was shown to the participants for reference, and a mirror was provided for the participants to assess the vitiligo on their face. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
        number (not applicable)
    24.5
    3.3
    Statistical analysis title
    		 exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0002 [5]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    9.53
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.9
         upper limit
    		 31.29
    Notes
    [5] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).

    Secondary: Percentage change from Baseline in Facial Body Surface Area (F-BSA) at Week 24

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    End point title
    		 Percentage change from Baseline in Facial Body Surface Area (F-BSA) at Week 24
    End point description
    F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area "Face" was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area "Face" did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant’s entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant’s thumb was considered as 0.1% BSA. Percentage change = ([post-Baseline (BL) value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage change
        least squares mean (standard error)
    -28.9 ( 2.22 )
    -9.5 ( 3.25 )
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001 [6]
    Method
    		 ANCOVA
    Parameter type
    		 least squares mean difference
    Point estimate
    -19.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -27.05
         upper limit
    		 -11.64
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.93
    Notes
    [6] - Response Variable = Treatment + Stratification Factors (Skin Type Fitzpatrick scale Type I, II versus Type III, IV, V, and VI, Region North America/Europe) + Baseline

    Secondary: Number of participants with treatment-emergent adverse events (TEAEs) during the Double-Blind Period

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    End point title
    		 Number of participants with treatment-emergent adverse events (TEAEs) during the Double-Blind Period
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE reported for the first time or the worsening of a pre-existing event after the first application of study drug.
    End point type
    Secondary
    End point timeframe
    from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 24)
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: participants
    101
    42
    No statistical analyses for this end point

    Secondary: Number of participants with treatment-emergent adverse events (TEAEs) during the Treatment-Extension Period

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    End point title
    		 Number of participants with treatment-emergent adverse events (TEAEs) during the Treatment-Extension Period
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE reported for the first time or the worsening of a pre-existing event after the first application of study drug.
    End point type
    Secondary
    End point timeframe
    from the completion of the Week 24 assessments until at least 30 days after the last application of study drug (up to Week 52 + 30 days)
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    193
    90
    Units: participants
    65
    31
    No statistical analyses for this end point

    Secondary: Percentage of participants achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24

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    End point title
    		 Percentage of participants achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24
    End point description
    An F-VASI25 responder achieved at least 25% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
        number (not applicable)
    69.8
    30.0
    Statistical analysis title
    		 exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001 [7]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    5.56
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 3.226
         upper limit
    		 9.578
    Notes
    [7] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).

    Secondary: Percentage of participants achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52

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    End point title
    		 Percentage of participants achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52
    End point description
    An F-VASI25/50/75/90 responder achieved at least 25/50/75/90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    173
    82
    Units: percentage of participants
    number (not applicable)
        F-VASI25
    89.6
    74.4
        F-VASI50
    75.1
    56.1
        F-VASI75
    52.6
    26.8
        F-VASI90
    32.9
    12.2
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in F-VASI at Week 24

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    End point title
    		 Percentage change from Baseline in F-VASI at Week 24
    End point description
    F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    195
    90
    Units: percentage change
        least squares mean (standard error)
    -47.79 ( 2.43 )
    -17.18 ( 3.53 )
    Statistical analysis title
    		 mixed-effect model; repeated measurement
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    285
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001
    Method
    		 mixed-effect model; repeated measurement
    Parameter type
    		 least squares mean difference
    Point estimate
    -30.61
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -39.03
         upper limit
    		 -22.19
    Variability estimate
    Standard error of the mean
    Dispersion value
    4.28

    Secondary: Percentage change from Baseline in F-VASI at Week 52

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    End point title
    		 Percentage change from Baseline in F-VASI at Week 52
    End point description
    F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    173
    82
    Units: percentage change
        arithmetic mean (standard deviation)
    -67.24 ( 33.660 )
    -52.98 ( 30.174 )
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in F-BSA at Week 52

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    End point title
    		 Percentage change from Baseline in F-BSA at Week 52
    End point description
    F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area "Face" was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area "Face" did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant’s entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant’s thumb was considered as 0.1% BSA. Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    173
    82
    Units: percentage change
        arithmetic mean (standard deviation)
    -44.87 ( 43.954 )
    -32.40 ( 30.068 )
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in T-VASI at Week 24

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    End point title
    		 Percentage change from Baseline in T-VASI at Week 24
    End point description
    T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    195
    90
    Units: percentage change
        least squares mean (standard error)
    -27.60 ( 1.81 )
    -10.62 ( 2.64 )
    Statistical analysis title
    		 mixed-effect model; repeated measurement
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    285
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001
    Method
    		 mixed-effect model; repeated measurement
    Parameter type
    		 least squares mean difference
    Point estimate
    -16.98
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -23.28
         upper limit
    		 -10.68
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.2

    Secondary: Percentage change from Baseline in T-VASI at Week 52

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    End point title
    		 Percentage change from Baseline in T-VASI at Week 52
    End point description
    T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    173
    82
    Units: percentage change
        arithmetic mean (standard deviation)
    -49.23 ( 26.366 )
    -29.85 ( 37.832 )
    No statistical analyses for this end point

    Secondary: Percentage change from Baseline in T-BSA at Week 24

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    End point title
    		 Percentage change from Baseline in T-BSA at Week 24
    End point description
    T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant’s entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant’s thumb was considered as 0.1% BSA. Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    195
    90
    Units: percentage change
        least squares mean (standard error)
    -13.08 ( 1.40 )
    -4.02 ( 2.05 )
    Statistical analysis title
    		 mixed-effect model; repeated measurement
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    285
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0003
    Method
    		 mixed-effect model; repeated measurement
    Parameter type
    		 least squares mean difference
    Point estimate
    -9.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -13.96
         upper limit
    		 -4.18
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.49

    Secondary: Percentage change from Baseline in T-BSA at Week 52

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    End point title
    		 Percentage change from Baseline in T-BSA at Week 52
    End point description
    T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant’s entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant’s thumb was considered as 0.1% BSA. Percentage change = ([post-BL value minus BL value]/BL value) X 100.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    173
    82
    Units: percentage change
        arithmetic mean (standard deviation)
    -27.39 ( 25.705 )
    -11.83 ( 34.654 )
    No statistical analyses for this end point

    Secondary: Percentage of participants achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24

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    End point title
    		 Percentage of participants achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24
    End point description
    A T-VASI25/75/90 responder achieved at least 25/75/90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    221
    109
    Units: percentage of participants
    number (not applicable)
        T-VASI25
    48.8
    23.8
        T-VASI75
    4.1
    1.8
        T-VASI90
    0.5
    0.0
    Statistical analysis title
    		 T-VASI25; exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001 [8]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    3.04
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.746
         upper limit
    		 5.307
    Notes
    [8] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).
    Statistical analysis title
    		 T-VASI75; exact logistic regression
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.2921 [9]
    Method
    		 exact logistic regression
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.489
         upper limit
    		 10.823
    Notes
    [9] - The model included the treatment group (1.5% BID and vehicle) and stratification factors (skin type and region).
    Statistical analysis title
    		 T-VASI90
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    330
    Analysis specification
    Pre-specified
    Analysis type
    		 [10]
    Method
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.49
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -9999
         upper limit
    		 9999
    Notes
    [10] - The confidence interval was not calculated for the estimated parameter. Values reported (-9999, 9999) are placeholders only, not actual data.

    Secondary: Percentage of participants achieving a ≥ 25%, ≥ 50%, 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52

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    End point title
    		 Percentage of participants achieving a ≥ 25%, ≥ 50%, 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52
    End point description
    A T-VASI25/50/75/90 responder achieved ≥25/50/75/90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant’s hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    173
    82
    Units: percentage of participants
    number (not applicable)
        T-VASI25
    77.5
    56.1
        T-VASI50
    53.2
    31.7
        T-VASI75
    20.2
    9.8
        T-VASI90
    3.5
    2.4
    No statistical analyses for this end point

    Secondary: Percentage of participants in each category of VNS during the treatment period (Double-Blind and Treatment-Extension Periods)

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    End point title
    		 Percentage of participants in each category of VNS during the treatment period (Double-Blind and Treatment-Extension Periods)
    End point description
    The VNS is a patient‐reported measure of vitiligo treatment success that is rated on a 5-point scale. The Baseline facial photograph was shown to the participants for reference, and a mirror was provided for the participants to assess the vitiligo on their face. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24 and Week 52
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    195 [11]
    90 [12]
    173 [13]
    82 [14]
    Units: percentage of participants
    number (not applicable)
        Week 24, more noticeable
    6.2
    14.4
    9999
    9999
        Week 24, as noticeable
    17.4
    46.7
    9999
    9999
        Week 24, slightly less noticeable
    51.3
    35.6
    9999
    9999
        Week 24, a lot less noticeable
    24.1
    3.3
    9999
    9999
        Week 24, no longer noticeable
    1.0
    0.0
    9999
    9999
        Week 52, more noticeable
    9999
    9999
    4.0
    4.9
        Week 52, as noticeable
    9999
    9999
    9.2
    15.9
        Week 52, slightly less noticeable
    9999
    9999
    46.8
    59.8
        Week 52, a lot less noticeable
    9999
    9999
    39.3
    19.5
        Week 52, no longer noticeable
    9999
    9999
    0.6
    0.0
    Notes
    [11] - 9999=participants in this treatment group weren't analyzed at this time point.
    [12] - 9999=participants in this treatment group weren't analyzed at this time point.
    [13] - 9999=participants in this treatment group weren't analyzed at this time point.
    [14] - 9999=participants in this treatment group weren't analyzed at this time point.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Dermatology Life Quality Index (DLQI) at Week 24

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    End point title
    		 Change from Baseline in Dermatology Life Quality Index (DLQI) at Week 24
    End point description
    The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. Each question is scored as: very much = 3; a lot = 2; a little = 1; not at all = 0; not relevant = 0. For Question 7, “Prevented work or studying” = 3. The DLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID
    Number of subjects analysed
    178
    87
    Units: scores on a scale
        least squares mean (standard error)
    -1.17 ( 0.27 )
    -0.85 ( 0.39 )
    Statistical analysis title
    		 mixed-effect model; repeated measurement
    Comparison groups
    Double-Blind Period: Ruxolitinib cream 1.5% BID v Double-Blind Period: Vehicle cream BID
    Number of subjects included in analysis
    265
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.497
    Method
    		 mixed-effect model; repeated measurement
    Parameter type
    		 least squares mean difference
    Point estimate
    -0.32
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.26
         upper limit
    		 0.62
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.48

    Secondary: Change from Baseline in DLQI at Week 52

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    End point title
    		 Change from Baseline in DLQI at Week 52
    End point description
    The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. Each question is scored as: very much = 3; a lot = 2; a little = 1; not at all = 0; not relevant = 0. For Question 7, “Prevented work or studying” = 3. The DLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 52
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    204 [15]
    105 [16]
    204 [17]
    105 [18]
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline, n=204, 105
    4.63 ( 4.446 )
    4.59 ( 4.871 )
    9999 ( 9999 )
    9999 ( 9999 )
        Week 52, n=157, 79
    9999 ( 9999 )
    9999 ( 9999 )
    -1.40 ( 4.087 )
    -1.37 ( 3.617 )
    Notes
    [15] - 9999=participants in this treatment group weren't analyzed at this time point.
    [16] - 9999=participants in this treatment group weren't analyzed at this time point.
    [17] - 9999=participants in this treatment group weren't analyzed at this time point.
    [18] - 9999=participants in this treatment group weren't analyzed at this time point.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Children’s Dermatology Life Quality Index (CDLQI) during the treatment period (Double-Blind and Treatment-Extension Periods)

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    End point title
    		 Change from Baseline in Children’s Dermatology Life Quality Index (CDLQI) during the treatment period (Double-Blind and Treatment-Extension Periods)
    End point description
    The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. The CDLQI is the youth/children’s version of the DLQI and was completed by adolescents aged ≥ 12 years to < 16 years. Each question is scored as: very much = 3; quite a lot = 2; only a little = 1; not at all = 0; question unanswered = 0. For Question 7: “Prevented school” = 3. The CDLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
    End point type
    Secondary
    End point timeframe
    Baseline; Week 24 and Week 52
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Double-Blind Period: Vehicle cream BID Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    17 [19]
    4 [20]
    17 [21]
    4 [22]
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline, n=16, 4
    2.50 ( 2.805 )
    1.25 ( 1.893 )
    9999 ( 9999 )
    9999 ( 9999 )
        Week 24, n=16, 3
    -0.25 ( 2.113 )
    0.00 ( 0.000 )
    9999 ( 9999 )
    9999 ( 9999 )
        Week 52, n=15, 3
    9999 ( 9999 )
    9999 ( 9999 )
    -1.00 ( 2.507 )
    0.00 ( 1.000 )
    Notes
    [19] - 9999=participants in this treatment group weren't analyzed at this time point.
    [20] - 9999=participants in this treatment group weren't analyzed at this time point.
    [21] - 9999=participants in this treatment group weren't analyzed at this time point.
    [22] - 9999=participants in this treatment group weren't analyzed at this time point.
    No statistical analyses for this end point

    Secondary: Trough plasma concentrations of ruxolitinib at Weeks 4, 24, and 40

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    End point title
    		 Trough plasma concentrations of ruxolitinib at Weeks 4, 24, and 40 [23]
    End point description
    Trough plasma concentration was defined as the measurement of the plasma concentration of ruxolitinib before drug application.
    End point type
    Secondary
    End point timeframe
    pre-dose at Weeks 4, 24, and 40
    Notes
    [23] - 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: Pharmacokinetics was assessed for ruxolitinib only; thus, data are not reported for the following arm: Double-Blind Period: Vehicle cream BID. Furthermore, no statistical analysis was conducted for this endpoint.
    End point values
    		 Double-Blind Period: Ruxolitinib cream 1.5% BID Treatment-Extension (TE) Period: Ruxolitinib cream 1.5% BID TE Period: Vehicle cream to Ruxolitinib cream 1.5% BID
    Number of subjects analysed
    206 [24]
    173 [25]
    80 [26]
    Units: nanomoles
    arithmetic mean (standard deviation)
        Week 4, n=206, 0, 0
    57.1 ( 61.4 )
    9999 ( 9999 )
    9999 ( 9999 )
        Week 24, n=191, 0, 0
    56.3 ( 69.4 )
    9999 ( 9999 )
    9999 ( 9999 )
        Week 40, n=0, 173, 80
    9999 ( 9999 )
    55.5 ( 63.6 )
    50.1 ( 55.8 )
    Notes
    [24] - 9999=participants in this treatment group weren't analyzed at this time point.
    [25] - 9999=participants in this treatment group weren't analyzed at this time point.
    [26] - 9999=participants in this treatment group weren't analyzed at this time point.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days)
    Adverse event reporting additional description
    Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Vehicle cream BID
    Reporting group description
    		 Participants applied matching vehicle cream twice a day (BID) for 24 weeks in the Double-Blind Period.

    Reporting group title
    Ruxolitinib cream 1.5% BID
    Reporting group description
    		 Participants applied ruxolitinib cream during the Double-Blind Treatment Period and the Treatment-Extension Period. Participants applied ruxolitinib 1.5% cream BID for 24 weeks. Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5% BID for 28 weeks in the Treatment-Extension Period.

    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: No non-serious adverse events occurred in at least 5% of participants in either treatment arm.
    Serious adverse events
    		 Vehicle cream BID Ruxolitinib cream 1.5% BID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 109 (0.92%)
    8 / 311 (2.57%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Prostate cancer
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Concussion
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Kidney contusion
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    1 / 109 (0.92%)
    0 / 311 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Myocarditis
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Subacute combined cord degeneration
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Anal fistula
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatitis infectious mononucleosis
         subjects affected / exposed
    0 / 109 (0.00%)
    1 / 311 (0.32%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Vehicle cream BID Ruxolitinib cream 1.5% BID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 109 (0.00%)
    0 / 311 (0.00%)

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Dec 2019
    The primary purpose of this amendment was to incorporate revisions requested by the Voluntary Harmonisation Procedure (VHP). - Added language to exclude participant who had current and/or history of tuberculosis - Added language to exclude participants who lived with anyone participating in any current Incyte-sponsored ruxolitinib cream study - Added language to instruct that German participants whose hemoglobin was between 10 grams per deciliter (g/dL) and 10.5 g/dL during the screening visit should have been further evaluated per local guidelines before enrolling into the study
    21 Feb 2020
    The primary purpose of this amendment was to incorporate revisions requested by the German and French Ethics Committees (ECs) and FDA. - Reordered and revised the key secondary endpoints and updated the analysis plan - Added 1 key exclusion criteria (exclude other forms of vitiligo) to the Population section, added information about the exit interview in the Study Design section, and added a Data and Safety Monitoring Board (DSMB) section to indicate that a DSMB was not required in this study - Added language that targeted physical examination should have been conducted as indicated by symptoms reported by the participant, adverse events (AEs), or other findings. Abnormalities that were considered clinically significant in the judgment of the investigator were to be reported as AEs. - Added language to instruct that adolescent participants screened in Germany whose hemoglobin was between 10 g/dL and 12 g/dL during the screening visit should have been further evaluated per local guidelines before being enrolled into the study

    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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