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    Clinical Trial Results:
    A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF CTP-543 IN ADULT PATIENTS WITH MODERATE TO SEVERE ALOPECIA AREATA

    Summary
    EudraCT number
    2021-000387-30
    Trial protocol
    HU   ES  
    Global end of trial date
    29 Jun 2022

    Results information
    Results version number
    v1(current)
    This version publication date
    21 Jun 2023
    First version publication date
    21 Jun 2023
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    CP543.3002
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04797650
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Concert Pharmaceuticals, Inc.
    Sponsor organisation address
    65 Hayden Avenue, Lexington, MA, United States, 02421
    Public contact
    Medical Manager, Linical France SARL, +40 256207271, diana.chera@linical.com
    Scientific contact
    Medical Manager, Linical France SARL, +40 256207271, diana.chera@linical.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
    29 Jun 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Jun 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This study evaluates the safety and effectiveness of an investigational study drug (called CTP-543) in adults (18 years and older) who have 50% or greater scalp hair loss.
    Protection of trial subjects
    All study subjects were required to read and sign an Informed Consent Form.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    10 Jun 2021
    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
    France: 48
    Country: Number of subjects enrolled
    Poland: 61
    Country: Number of subjects enrolled
    Canada: 66
    Country: Number of subjects enrolled
    United States: 135
    Country: Number of subjects enrolled
    Germany: 135
    Country: Number of subjects enrolled
    Spain: 56
    Country: Number of subjects enrolled
    Hungary: 16
    Worldwide total number of subjects
    517
    EEA total number of subjects
    316
    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)
    515
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Subjects were enrolled at study centers in Canada, France, Germany, Hungary, Poland, Spain and the United States from 10 June 2021 to 29 June 2022.

    Pre-assignment
    Screening details
    671 subjects were screened, out of which 517 subjects who experienced moderate to severe hair loss due to alopecia areata were enrolled to receive CTP-543 or placebo.

    Period 1
    Period 1 title
    Overall Study (Overall Period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Subjects received CTP-543 matched placebo tablets, orally, twice daily (BID) for up to 24 weeks.
    Arm type
    Placebo

    Investigational medicinal product name
    CTP-543 matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    CTP-543 matched placebo administered BID for up to 24 weeks.

    Arm title
    CTP-543 8 mg BID
    Arm description
    Subjects received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    CTP-543
    Investigational medicinal product code
    Other name
    Deuruxolitinib
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    CTP-543 administered BID for up to 24 weeks.

    Arm title
    CTP-543 12 mg BID
    Arm description
    Subjects received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    CTP-543
    Investigational medicinal product code
    Other name
    Deuruxolitinib
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    CTP-543 administered BID for up to 24 weeks.

    Number of subjects in period 1
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Started
    130
    258
    129
    Completed
    119
    233
    120
    Not completed
    11
    25
    9
         Non-compliance with Study Drug
    -
    -
    2
         Lost to follow-up
    -
    -
    1
         Lost to follow-up
    4
    6
    -
         Reason not specified
    5
    12
    5
         Treatment Emergent or Worsening Adverse Event
    1
    7
    -
         Lack of efficacy
    1
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Subjects received CTP-543 matched placebo tablets, orally, twice daily (BID) for up to 24 weeks.

    Reporting group title
    CTP-543 8 mg BID
    Reporting group description
    Subjects received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.

    Reporting group title
    CTP-543 12 mg BID
    Reporting group description
    Subjects received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.

    Reporting group values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID Total
    Number of subjects
    130 258 129 517
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    39.7 ( 12.49 ) 38.4 ( 12.30 ) 39.7 ( 12.90 ) -
    Gender categorical
    Units: Subjects
        Female
    88 177 84 349
        Male
    42 81 45 168
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    11 23 9 43
        Not Hispanic or Latino
    108 205 111 424
        Unknown
    11 30 9 50
    Race
    Units: Subjects
        American Indian or Alaska Native
    1 5 0 6
        Asian
    7 4 4 15
        Black or African American
    10 17 7 34
        White
    100 203 109 412
        Other
    1 1 0 2
        Not reported
    11 28 9 48

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Subjects received CTP-543 matched placebo tablets, orally, twice daily (BID) for up to 24 weeks.

    Reporting group title
    CTP-543 8 mg BID
    Reporting group description
    Subjects received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.

    Reporting group title
    CTP-543 12 mg BID
    Reporting group description
    Subjects received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.

    Primary: Percentage of Subjects Achieving an Absolute Severity of Alopecia Tool (SALT) Score ≤20 at Week 24

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    End point title
    Percentage of Subjects Achieving an Absolute Severity of Alopecia Tool (SALT) Score ≤20 at Week 24
    End point description
    SALT is a quantitative assessment of scalp hair loss with scores ranging in severity from 0 (no scalp hair loss) to a maximum of 100 (complete scalp hair loss). Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Primary
    End point timeframe
    Week 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of subjects
        number (not applicable)
    0.8
    33.0
    38.3
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [1]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.31
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.25
         upper limit
    0.37
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.031
    Notes
    [1] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [2]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.36
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.27
         upper limit
    0.45
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.044
    Notes
    [2] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.

    Secondary: Percentage of Responders on the Hair Satisfaction Patient Reported Outcome (SPRO) Scale at Weeks 12, 16, 20, and 24

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    End point title
    Percentage of Responders on the Hair Satisfaction Patient Reported Outcome (SPRO) Scale at Weeks 12, 16, 20, and 24
    End point description
    SPRO is a questionnaire answered by the subject and designed to measure how satisfied alopecia areata subjects are with their hair at the time of the assessment. The responses range from 1 to 5: 1= very satisfied, 2= satisfied, 3= neither satisfied nor dissatisfied, 4= dissatisfied, and 5= very dissatisfied. Responders were defined as subjects with responses of "satisfied" or "very satisfied". Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of responders
    number (not applicable)
        Week 12
    4.9
    43.2
    41.0
        Week 16
    4.9
    41.0
    45.1
        Week 20
    1.7
    40.1
    52.5
        Week 24
    1.7
    46.5
    51.7
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [3]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.38
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.31
         upper limit
    0.46
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.038
    Notes
    [3] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [4]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.36
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.26
         upper limit
    0.45
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.049
    Notes
    [4] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [5]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.3
         upper limit
    0.45
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.037
    Notes
    [5] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [6]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.31
         upper limit
    0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.049
    Notes
    [6] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [7]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.39
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.32
         upper limit
    0.46
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.034
    Notes
    [7] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [8]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.41
         upper limit
    0.59
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.047
    Notes
    [8] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [9]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.45
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.38
         upper limit
    0.52
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.035
    Notes
    [9] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [10]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.49
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.4
         upper limit
    0.58
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.047
    Notes
    [10] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.

    Secondary: Percentage of Subjects Achieving an Absolute SALT Score of ≤20 at Weeks 4, 8, 12, 16, and 20

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    End point title
    Percentage of Subjects Achieving an Absolute SALT Score of ≤20 at Weeks 4, 8, 12, 16, and 20
    End point description
    SALT is a quantitative assessment of scalp hair loss with scores ranging in severity from 0 (no scalp hair loss) to a maximum of 100 (complete scalp hair loss). Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Weeks 4, 8, 12, 16, and 20
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of subjects
    number (not applicable)
        Week 4
    0
    0.4
    0
        Week 8
    0.8
    2.9
    2.4
        Week 12
    0.8
    10.5
    12.3
        Week 16
    0.8
    20.2
    21.1
        Week 20
    0.8
    24.8
    28.8
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 4
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.3175 [11]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0
         upper limit
    0.01
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.004
    Notes
    [11] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 8
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.102 [12]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0
         upper limit
    0.05
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.013
    Notes
    [12] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 8
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.3103 [13]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.01
         upper limit
    0.05
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.016
    Notes
    [13] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    CTP-543 8 mg BID v Placebo
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [14]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.06
         upper limit
    0.14
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.021
    Notes
    [14] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [15]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.12
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.06
         upper limit
    0.17
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.03
    Notes
    [15] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [16]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.19
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.14
         upper limit
    0.24
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.026
    Notes
    [16] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [17]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.13
         upper limit
    0.27
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.037
    Notes
    [17] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [18]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.18
         upper limit
    0.29
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.028
    Notes
    [18] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [19]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.27
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.19
         upper limit
    0.35
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.041
    Notes
    [19] - P-value was calculated by Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.

    Secondary: Relative Change in SALT Scores From Baseline at Weeks 4, 8, 12, 16, 20, and 24

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    End point title
    Relative Change in SALT Scores From Baseline at Weeks 4, 8, 12, 16, 20, and 24
    End point description
    SALT is a quantitative assessment of scalp hair loss with scores ranging in severity from 0 (no scalp hair loss) to a maximum of 100 (complete scalp hair loss). Relative change (percent change) from baseline (CFB) is calculated as: 100 x ([post-baseline SALT score – baseline SALT score]/baseline SALT score). Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8, 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    125
    242
    125
    Units: percent change
    arithmetic mean (standard deviation)
        Relative CFB at Week 4 (n=124, 242, 124)
    0.4 ( 5.40 )
    -2.6 ( 10.10 )
    -3.2 ( 9.45 )
        Relative CFB at Week 8 (n= 125, 239, 125)
    -2.0 ( 13.41 )
    -11.5 ( 20.63 )
    -16.4 ( 23.92 )
        Relative CFB at Week 12 (n= 123, 237, 122)
    -0.7 ( 14.72 )
    -23.9 ( 29.06 )
    -30.3 ( 31.85 )
        Relative CFB at Week 16 (n= 124, 238, 123)
    0.9 ( 13.19 )
    -34.3 ( 34.93 )
    -39.9 ( 35.43 )
        Relative CFB at Week 20 (n= 120, 234, 118)
    0.4 ( 15.49 )
    -38.9 ( 37.64 )
    -47.7 ( 37.62 )
        Relative CFB at Week 24 (n= 119, 233, 120)
    1.6 ( 16.54 )
    -44.6 ( 39.58 )
    -52.7 ( 38.44 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 4
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    367
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0039 [20]
    Method
    MMRM
    Parameter type
    Least Square (LS) Mean Difference
    Point estimate
    -2.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.7
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.97
    Notes
    [20] - P-value was calculated by mixed model repeated measures (MMRM) analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 4
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0027 [21]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -3.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.6
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.12
    Notes
    [21] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 8
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    367
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [22]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -9.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -13.6
         upper limit
    -5.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.15
    Notes
    [22] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 8
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [23]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -14.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -19
         upper limit
    -9.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.47
    Notes
    [23] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    367
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [24]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -28.8
         upper limit
    -17.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.92
    Notes
    [24] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [25]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -29.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -35.9
         upper limit
    -22.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.36
    Notes
    [25] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    367
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [26]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -34.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -41.4
         upper limit
    -28.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.37
    Notes
    [26] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [27]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -40.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -47.8
         upper limit
    -32.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.86
    Notes
    [27] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    367
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [28]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -38.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -45.9
         upper limit
    -31.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.63
    Notes
    [28] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [29]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -46.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -54.8
         upper limit
    -38.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    4.17
    Notes
    [29] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    367
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [30]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -45.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -53.4
         upper limit
    -38.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.8
    Notes
    [30] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [31]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -52.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -61.4
         upper limit
    -44.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    4.36
    Notes
    [31] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.

    Secondary: Percentage of Responders Assessed Using the Clinician Global Impression of Improvement (CGI-I) at Weeks 12, 16, 20, and 24

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    End point title
    Percentage of Responders Assessed Using the Clinician Global Impression of Improvement (CGI-I) at Weeks 12, 16, 20, and 24
    End point description
    The CGI-I is a questionnaire that asks the clinician to evaluate the improvement or worsening of the subject's alopecia areata as compared to the start of the study on a 7-point scale. Responses range from 1 (very much worse) to 7 (very much improved). Responders were defined as subjects with responses of 6 (much improved) or 7 (very much improved). Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of responders
    number (not applicable)
        Week 12
    3.3
    38.0
    44.3
        Week 16
    3.2
    45.0
    52.0
        Week 20
    1.7
    50.0
    59.0
        Week 24
    2.5
    54.1
    61.7
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [32]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [32] - P-value was calculated by cochran mantel haenszel (CMH) test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [33]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [33] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [34]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [34] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [35]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [35] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [36]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [36] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [37]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [37] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [38]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [38] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [39]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [39] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.

    Secondary: Percentage of Responders Assessed Using the Patient Global Impression of Improvement (PGI-I) at Weeks 12, 16, 20, and 24

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    End point title
    Percentage of Responders Assessed Using the Patient Global Impression of Improvement (PGI-I) at Weeks 12, 16, 20, and 24
    End point description
    The PGI-I is a self-administered questionnaire that asks the subject to evaluate the improvement or worsening of their alopecia areata as compared to the start of the study on a 7-point scale. Responses range from 1 (very much worse) to 7 (very much improved). Responders were defined as subjects with responses of 6 (much improved) or 7 (very much improved). Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of responders
    number (not applicable)
        Week 12
    3.3
    43.3
    49.2
        Week 16
    4.0
    46.2
    59.8
        Week 20
    5.8
    48.7
    64.4
        Week 24
    1.7
    50.9
    68.3
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [40]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [40] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [41]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [41] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [42]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [42] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [43]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [43] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [44]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [44] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [45]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [45] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [46]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [46] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [47]
    Method
    Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [47] - P-value was calculated by CMH test stratified by baseline scalp hair loss (partial vs complete/near-complete) for responders in each treatment group compared to placebo.

    Secondary: Change in the Clinician Global Impression of Severity (CGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24

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    End point title
    Change in the Clinician Global Impression of Severity (CGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24
    End point description
    The CGI-S is a questionnaire that asks the clinician to evaluate the symptom severity of the subject's alopecia areata at the time of assessment. The symptom severity was rated on a scale ranging from 1 to 7, where 1=normal, no hair loss; 2=borderline hair loss; 3=mild hair loss; 4=moderate hair loss; 5=marked hair loss; 6=severe hair loss; 7=among the most extreme hair loss. Higher scores indicate more hair loss. A negative CFB less hair loss. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed (n) indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    123
    236
    124
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Baseline
    6.4 ( 0.82 )
    6.5 ( 0.83 )
    6.4 ( 0.76 )
        CFB at Week 12 (n= 121, 232, 122)
    0.0 ( 0.75 )
    -1.3 ( 1.45 )
    -1.5 ( 1.52 )
        CFB at Week 16 (n= 122, 234, 123)
    0.1 ( 0.67 )
    -1.6 ( 1.61 )
    -1.8 ( 1.69 )
        CFB at Week 20 (n= 118, 231, 118)
    -0.0 ( 0.96 )
    -1.8 ( 1.80 )
    -2.2 ( 1.83 )
        CFB at Week 24 (n= 117, 229, 120)
    0.1 ( 0.79 )
    -2.1 ( 1.97 )
    -2.4 ( 1.96 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    359
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [48]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [48] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    247
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [49]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.7
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.16
    Notes
    [49] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    359
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [50]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.9
         upper limit
    -1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [50] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    247
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [51]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.2
         upper limit
    -1.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.18
    Notes
    [51] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    359
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [52]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2
         upper limit
    -1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.18
    Notes
    [52] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    247
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [53]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -2.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.5
         upper limit
    -1.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Notes
    [53] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    359
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [54]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -2.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.5
         upper limit
    -1.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.19
    Notes
    [54] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    247
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [55]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -2.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.8
         upper limit
    -2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.22
    Notes
    [55] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.

    Secondary: Change in the Patient Global Impression of Severity (PGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24

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    End point title
    Change in the Patient Global Impression of Severity (PGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24
    End point description
    The PGI-S is a self-administered questionnaire that asks the subject to evaluate the symptom severity of their alopecia areata at the time of assessment. Symptom severity was rated on a scale ranging from 1 to 7, where 1= normal, no hair loss; 2= borderline hair loss; 3= mild hair loss; 4= moderate hair loss; 5= marked hair loss; 6= severe hair loss; 7= among the most extreme hair loss. Higher scores indicate more hair loss. A negative CFB indicates less hair loss. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    124
    236
    124
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Baseline
    6.7 ( 0.64 )
    6.6 ( 0.79 )
    6.5 ( 0.78 )
        CFB at Week 12 (n= 122, 234, 122)
    -0.2 ( 0.97 )
    -1.6 ( 2.05 )
    -1.8 ( 1.88 )
        CFB at Week 16 (n= 123, 234, 122)
    -0.2 ( 1.06 )
    -1.8 ( 2.10 )
    -2.0 ( 1.90 )
        CFB at Week 20 (n= 119, 232, 118)
    -0.3 ( 1.26 )
    -2.0 ( 2.20 )
    -2.2 ( 1.91 )
        CFB at Week 24 (n= 118, 228, 120)
    -0.2 ( 1.04 )
    -2.3 ( 2.26 )
    -2.5 ( 1.98 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [56]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Notes
    [56] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [57]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.23
    Notes
    [57] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [58]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Notes
    [58] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [59]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.3
         upper limit
    -1.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.23
    Notes
    [59] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [60]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.2
         upper limit
    -1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.21
    Notes
    [60] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [61]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.4
         upper limit
    -1.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.24
    Notes
    [61] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [62]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -2.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.5
         upper limit
    -1.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.21
    Notes
    [62] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [63]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -2.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.8
         upper limit
    -1.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.24
    Notes
    [63] - P-value was calculated by MMRM with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.

    Secondary: Percentage of Subjects Achieving at Least a 75% and 90% Relative Reduction in SALT Score From Baseline at Weeks 12 and 24

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    End point title
    Percentage of Subjects Achieving at Least a 75% and 90% Relative Reduction in SALT Score From Baseline at Weeks 12 and 24
    End point description
    SALT is a quantitative assessment of scalp hair loss with scores ranging in severity from 0 (no scalp hair loss) to a maximum of 100 (complete scalp hair loss). Percentage of subjects achieving at least a 75% and 90% relative reduction in SALT score from baseline at Weeks 12 and 24 are reported. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of subjects
    number (not applicable)
        75% Relative Reduction: Week 12
    0
    8.9
    11.5
        75% Relative Reduction: Week 24
    0
    33.5
    38.3
        90% Relative Reduction: Week 12
    0
    1.7
    4.9
        90% Relative Reduction: Week 24
    0
    21.9
    25.0
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    75% Relative Reduction: Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [64]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.09
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.05
         upper limit
    0.12
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.018
    Notes
    [64] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    75% Relative Reduction: Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [65]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.12
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.06
         upper limit
    0.17
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.029
    Notes
    [65] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    75% Relative Reduction: Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [66]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.33
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.27
         upper limit
    0.38
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.03
    Notes
    [66] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    75% Relative Reduction: Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [67]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.28
         upper limit
    0.45
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.043
    Notes
    [67] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    90% Relative Reduction: Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0409 [68]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0
         upper limit
    0.03
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.009
    Notes
    [68] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    90% Relative Reduction: Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.012 [69]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.01
         upper limit
    0.09
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.019
    Notes
    [69] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    90% Relative Reduction: Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [70]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.21
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.16
         upper limit
    0.26
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.026
    Notes
    [70] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    90% Relative Reduction: Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [71]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.24
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.16
         upper limit
    0.31
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.038
    Notes
    [71] - P-value was calculated by mantel-haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.

    Secondary: Change in the Brigham Eyebrow Tool for Alopecia (BETA) Scores From Baseline at Weeks 12 and 24

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    End point title
    Change in the Brigham Eyebrow Tool for Alopecia (BETA) Scores From Baseline at Weeks 12 and 24
    End point description
    BETA is a clinician-rated scale that assesses the total eyebrow hair present. The BETA score is calculated based on hair density and surface area of each individual eyebrow of the subject, ranging from 0 to 3, where 0= no eyebrow, 1= minimal eyebrow, 2= moderate eyebrow, and 3= normal eyebrow. The BETA score is the sum of the right and left eyebrow scores, ranging from 0 to 6. Higher scores indicate less hair loss of eyebrows. A positive CFB indicates less hair loss of eyebrows. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed (n) indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    95
    173
    90
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Baseline
    0.9 ( 1.64 )
    0.7 ( 1.44 )
    1.1 ( 1.65 )
        CFB at Week 12 (n= 88, 157, 80)
    -0.3 ( 0.93 )
    0.8 ( 1.53 )
    0.7 ( 1.66 )
        CFB at Week 24 (n= 86, 156, 83)
    -0.3 ( 1.10 )
    1.2 ( 1.76 )
    1.2 ( 1.81 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    268
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [72]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.7
         upper limit
    1.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.17
    Notes
    [72] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    185
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [73]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.7
         upper limit
    1.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Notes
    [73] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    268
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [74]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.1
         upper limit
    1.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Notes
    [74] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    185
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [75]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.3
         upper limit
    2.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.23
    Notes
    [75] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.

    Secondary: Change in the Brigham Eyelash Tool for Alopecia (BELA) Scores From Baseline at Weeks 12 and 24

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    End point title
    Change in the Brigham Eyelash Tool for Alopecia (BELA) Scores From Baseline at Weeks 12 and 24
    End point description
    BELA is a clinician-rated scale that assesses the total eyelash hair present. The BELA is calculated based on distribution and grade values, ranging from 0 (no eyelashes)to 3 (full eyelashes). The BELA score is the sum of the individual scores for the left and right eyes, ranging from 0 to 6. Higher scores indicate less hair loss of eyelashes. A positive CFB indicates less hair loss of eyelashes. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed (n) indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    85
    165
    82
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Baseline
    0.7 ( 1.38 )
    0.9 ( 1.68 )
    1.1 ( 1.83 )
        CFB at Week 12 (n= 84, 156, 81)
    -0.1 ( 0.91 )
    0.9 ( 1.60 )
    1.0 ( 1.76 )
        CFB at Week 24 (n= 78, 153, 69)
    -0.0 ( 0.93 )
    1.4 ( 2.09 )
    1.3 ( 1.97 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [76]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.7
         upper limit
    1.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Notes
    [76] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    167
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [77]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.7
         upper limit
    1.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.23
    Notes
    [77] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    250
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [78]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1
         upper limit
    2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.24
    Notes
    [78] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    167
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [79]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1
         upper limit
    2.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.29
    Notes
    [79] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.

    Secondary: Change in the SPRO Scale From Baseline at Weeks 12, 16, 20, and 24

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    End point title
    Change in the SPRO Scale From Baseline at Weeks 12, 16, 20, and 24
    End point description
    SPRO is a questionnaire answered by the subject and designed to measure how satisfied alopecia areata subjects are with their hair at the time of the assessment. The responses range from 1 to 5: 1= very satisfied, 2= satisfied, 3= neither satisfied nor dissatisfied, 4= dissatisfied, and 5= very dissatisfied. Higher scores indicate the greater hair dissatisfaction. A negative CFB indicate the greater hair satisfaction. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed (n) indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    124
    236
    124
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Baseline
    4.5 ( 0.85 )
    4.6 ( 0.71 )
    4.6 ( 0.80 )
        CFB at Week 12 (n=122, 234, 122)
    -0.4 ( 1.14 )
    -1.6 ( 1.39 )
    -1.7 ( 1.33 )
        CFB at Week 16 (n= 123, 234, 122)
    -0.1 ( 1.10 )
    -1.6 ( 1.31 )
    -1.7 ( 1.36 )
        CFB at Week 20 (n= 119, 232, 118)
    -0.1 ( 0.97 )
    -1.5 ( 1.40 )
    -1.9 ( 1.38 )
        CFB at Week 24 (n= 118, 228, 120)
    -0.1 ( 0.93 )
    -1.6 ( 1.47 )
    -1.9 ( 1.41 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [80]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [80] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [81]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [81] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [82]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [82] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [83]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [83] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [84]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [84] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [85]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2
         upper limit
    -1.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [85] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [86]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    -1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [86] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [87]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.1
         upper limit
    -1.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.16
    Notes
    [87] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, and baseline value.

    Secondary: Percentage of Subjects Achieving a ≥2-point Change From Baseline in the SPRO Scale at Weeks 12, 16, 20, and 24

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    End point title
    Percentage of Subjects Achieving a ≥2-point Change From Baseline in the SPRO Scale at Weeks 12, 16, 20, and 24
    End point description
    SPRO is a questionnaire answered by the subject and designed to measure how satisfied alopecia areata subjects are with their hair at the time of the assessment. The responses range from 1 to 5: 1= very satisfied, 2= satisfied, 3= neither satisfied nor dissatisfied, 4= dissatisfied, and 5= very dissatisfied. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of subjects
    number (not applicable)
        Week 12
    15.6
    50.4
    59.0
        Week 16
    9.8
    52.6
    57.4
        Week 20
    9.2
    50.4
    63.6
        Week 24
    8.5
    52.6
    61.7
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [88]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.34
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.25
         upper limit
    0.43
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.046
    Notes
    [88] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [89]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.43
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.33
         upper limit
    0.54
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.055
    Notes
    [89] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [90]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.43
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.35
         upper limit
    0.51
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.042
    Notes
    [90] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [91]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.37
         upper limit
    0.57
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.052
    Notes
    [91] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [92]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.42
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.34
         upper limit
    0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.041
    Notes
    [92] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [93]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.54
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.44
         upper limit
    0.63
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.05
    Notes
    [93] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [94]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.44
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.36
         upper limit
    0.52
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.041
    Notes
    [94] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [95]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.52
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.42
         upper limit
    0.62
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.051
    Notes
    [95] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.

    Secondary: Change in the Individual Items of the Hair Quality Patient Reported Outcome (QPRO) Scale From Baseline at Weeks 12, 16, 20, and 24

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    End point title
    Change in the Individual Items of the Hair Quality Patient Reported Outcome (QPRO) Scale From Baseline at Weeks 12, 16, 20, and 24
    End point description
    The QPRO questionnaire provides additional details on key attributes of hair and helps provide context to the SPRO response. The individual items of QPRO are: Satisfied thickness hair coverage (STHC); Satisfied evenness hair coverage (SEHC); How satisfied with your eyebrows (HSWYE); How satisfied with your eyelashes (HSWYEl), scored on a scale ranging from 1 to 5 where 1=very satisfied, 2=satisfied, 3=neither satisfied nor dissatisfied, 4=dissatisfied, 5=very dissatisfied. Higher scores indicate the greater dissatisfaction on hair quality. A negative CFB indicate the greater satisfaction on hair quality. Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed indicates the number of subjects with data available for analysis of this end point. Number of subjects analysed indicates the number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 16, 20, and 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    124
    236
    124
    Units: Score on a scale
    arithmetic mean (standard deviation)
        STHC: Baseline
    4.6 ( 0.76 )
    4.6 ( 0.66 )
    4.6 ( 0.83 )
        STHC: CFB at Week 12 (n=122, 234, 122)
    -0.3 ( 0.95 )
    -1.3 ( 1.20 )
    -1.4 ( 1.35 )
        STHC: CFB at Week 16 (n= 123, 234, 122)
    -0.1 ( 0.91 )
    -1.3 ( 1.23 )
    -1.5 ( 1.30 )
        STHC: CFB at Week 20 (n= 119, 232, 118)
    -0.1 ( 0.85 )
    -1.3 ( 1.35 )
    -1.7 ( 1.36 )
        SHTC: CFB at Week 24 (n=118, 228, 120)
    -0.1 ( 0.89 )
    -1.4 ( 1.46 )
    -1.6 ( 1.41 )
        SEHC: Baseline
    4.7 ( 0.61 )
    4.7 ( 0.58 )
    4.7 ( 0.74 )
        SEHC: CFB at Week 12 (n= 122, 234, 122)
    -0.3 ( 0.78 )
    -1.2 ( 1.10 )
    -1.3 ( 1.26 )
        SEHC: CFB at Week 16 (n= 123, 234, 122)
    -0.2 ( 0.82 )
    -1.1 ( 1.12 )
    -1.3 ( 1.20 )
        SEHC: CFB at Week 20 (n= 119, 232, 118)
    -0.1 ( 0.76 )
    -1.3 ( 1.27 )
    -1.5 ( 1.29 )
        SEHC: CFB at Week 24 (n= 118, 228, 120)
    -0.1 ( 0.71 )
    -1.4 ( 1.36 )
    -1.6 ( 1.37 )
        HSWYE: Baseline
    4.3 ( 1.07 )
    4.3 ( 1.16 )
    4.1 ( 1.32 )
        HSWYE: CFB at Week 12 (n= 122, 234, 122)
    -0.2 ( 0.84 )
    -1.3 ( 1.31 )
    -1.3 ( 1.42 )
        HSWYE: CFB at Week 16 (n= 123, 234, 122)
    -0.2 ( 0.78 )
    -1.2 ( 1.28 )
    -1.4 ( 1.54 )
        HSWYE: CFB at Week 20 (n= 119, 232, 118)
    -0.1 ( 0.78 )
    -1.4 ( 1.40 )
    -1.4 ( 1.47 )
        HSWYE: CFB at Week 24 (n= 118, 228, 120)
    -0.1 ( 0.81 )
    -1.4 ( 1.43 )
    -1.6 ( 1.50 )
        HSWYEI: Baseline
    4.1 ( 1.23 )
    4.1 ( 1.32 )
    3.8 ( 1.42 )
        HSWYEl: CFB at Week 12 (n= 122, 234, 122)
    -0.2 ( 0.72 )
    -1.1 ( 1.34 )
    -1.0 ( 1.29 )
        HSWYEl: CFB at Week 16 (n= 123, 234, 122)
    -0.2 ( 0.82 )
    -1.1 ( 1.29 )
    -1.1 ( 1.41 )
        HSWYEI: CFB at Week 20 (n= 119, 232, 118)
    -0.1 ( 0.83 )
    -1.2 ( 1.39 )
    -1.2 ( 1.31 )
        HSWYEl: CFB at Week 24 (n= 118, 228, 120)
    -0.1 ( 0.73 )
    -1.3 ( 1.41 )
    -1.3 ( 1.45 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    STHC: Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [96]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.2
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [96] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    STHC: Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [97]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [97] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    STHC: Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [98]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [98] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    STHC: Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [99]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [99] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    STHC: Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [100]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [100] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    STHC: Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [101]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.9
         upper limit
    -1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [101] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    STHC: Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [102]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [102] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    STHC: Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [103]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [103] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    SEHC: Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [104]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.1
         upper limit
    -0.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    Notes
    [104] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    SEHC: Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [105]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [105] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    SEHC: Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [106]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.2
         upper limit
    -0.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    Notes
    [106] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    SEHC: Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [107]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [107] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    SEHC: Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [108]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [108] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    SEHC: Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [109]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [109] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    SEHC: Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [110]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [110] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    SEHC: Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [111]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [111] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWYE: Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [112]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [112] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWYE: Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [113]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [113] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWYE: Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [114]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [114] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWYE: Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [115]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [115] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWYE: Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [116]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [116] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWYE: Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [117]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.7
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [117] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWYE: Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [118]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    Notes
    [118] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWYE: Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [119]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.15
    Notes
    [119] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWYEl: Week 12
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [120]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.1
         upper limit
    -0.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [120] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWYEl: Week 12
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [121]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.2
         upper limit
    -0.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [121] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWEl: Week 16
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [122]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.2
         upper limit
    -0.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [122] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWEl: Week 16
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [123]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [123] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWEl: Week 20
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [124]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [124] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWEl: Week 20
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [125]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.5
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [125] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    HSWEl: Week 24
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    360
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [126]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    Notes
    [126] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    HSWEl: Week 24
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    248
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [127]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    Notes
    [127] - P-value was calculated by MMRM analysis with effects for treatment, visit, treatment-by-visit interaction, baseline value, and baseline SALT score.

    Secondary: Change in the Anxiety and Depression Scale Scores of the Hospital Anxiety and Depression Scale (HADS) From Baseline at Week 24

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    End point title
    Change in the Anxiety and Depression Scale Scores of the Hospital Anxiety and Depression Scale (HADS) From Baseline at Week 24
    End point description
    HADS is questionnaire designed to assess anxiety and depression symptoms which is completed by subjects. The questionnaire is comprised of two separate scales with a total of 14 items: A 7-item scale related to anxiety and 7-item scale related to depression. Each item within both scales is scored using a 4-point scale, ranging from 0 to 3 and the total scores in each scale can range from 0 to 21. Separate scores were created for anxiety and depression. A score between 0-7 is considered normal, 8-10 is mild, 11-14 is moderate, and >14 is severe anxiety or depression. Higher scores indicate greater severity. A negative CFB indicates less severity. Efficacy population= all subjects who were randomised in the study and dispensed study drug during the treatment period. Overall number of subjects analysed=number of subjects with data available for analysis of this end point. Number of subjects analysed (n) = number of subjects with data available for analysis at the specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    118
    228
    120
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Anxiety: Baseline
    6.2 ( 3.99 )
    6.4 ( 4.21 )
    6.2 ( 3.79 )
        Anxiety: CFB at Week 24
    -0.6 ( 2.74 )
    -1.1 ( 3.00 )
    -0.8 ( 2.85 )
        Depression: Baseline
    4.7 ( 3.96 )
    4.2 ( 3.66 )
    4.3 ( 3.56 )
        Depression: CFB at Week 24
    -0.6 ( 2.97 )
    -1.2 ( 3.16 )
    -1.5 ( 2.60 )
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Anxiety
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.1206 [128]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.1
         upper limit
    1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.29
    Notes
    [128] - P-value was calculated by analysis of covariance (ANCOVA) analysis with effects for treatment, visit, baseline SALT score, and baseline HADS value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Anxiety
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.5367 [129]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.5
         upper limit
    0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.34
    Notes
    [129] - P-value was calculated by ANCOVA analysis with effects for treatment, visit, baseline SALT score, and baseline HADS value.
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Statistical analysis description
    Depression
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0064 [130]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.2
         upper limit
    1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.29
    Notes
    [130] - P-value was calculated by ANCOVA analysis with effects for treatment, visit, baseline SALT score, and baseline HADS value.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Statistical analysis description
    Depression
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0011 [131]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.4
         upper limit
    1.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.33
    Notes
    [131] - P-value was calculated by ANCOVA analysis with effects for treatment, visit, baseline SALT score, and baseline HADS value.

    Secondary: Percentage of Subjects Achieving an Absolute SALT Score of ≤10 at Week 24

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    End point title
    Percentage of Subjects Achieving an Absolute SALT Score of ≤10 at Week 24
    End point description
    SALT is a quantitative assessment of scalp hair loss with scores ranging in severity from 0 (no scalp hair loss) to a maximum of 100 (complete scalp hair loss). Efficacy population included all subjects who were randomised in the study and dispensed study drug during the treatment period.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Number of subjects analysed
    127
    249
    127
    Units: Percentage of subjects
        number (not applicable)
    0
    24.9
    26.7
    Statistical analysis title
    Placebo vs CTP-543 8 mg BID
    Comparison groups
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    376
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [132]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.24
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.19
         upper limit
    0.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.027
    Notes
    [132] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.
    Statistical analysis title
    Placebo vs CTP-543 12 mg BID
    Comparison groups
    Placebo v CTP-543 12 mg BID
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [133]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.25
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.18
         upper limit
    0.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.039
    Notes
    [133] - P-value was calculated by the Mantel-Haenszel estimate stratified by baseline scalp hair loss (partial vs complete/near-complete) for each treatment group compared to placebo.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All-cause mortality: Randomisation up to Week 28; Adverse events: From first dose of study drug up to last follow up visit (Week 28)
    Adverse event reporting additional description
    All-cause mortality: All randomised subjects included all subjects who were randomised in the study (N= 130, 258, 129). Adverse events: Safety Population included all subjects who received study drug during the treatment period.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Subjects received CTP-543 matched placebo tablets, orally, BID for up to 24 weeks.

    Reporting group title
    CTP-543 8 mg BID
    Reporting group description
    Subjects received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.

    Reporting group title
    CTP-543 12 mg BID
    Reporting group description
    Subjects received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.

    Serious adverse events
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 130 (0.00%)
    3 / 256 (1.17%)
    2 / 129 (1.55%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Radius fracture
         subjects affected / exposed
    0 / 130 (0.00%)
    0 / 256 (0.00%)
    1 / 129 (0.78%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Migraine with aura
         subjects affected / exposed
    0 / 130 (0.00%)
    1 / 256 (0.39%)
    0 / 129 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Osteoarthritis
         subjects affected / exposed
    0 / 130 (0.00%)
    0 / 256 (0.00%)
    1 / 129 (0.78%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 130 (0.00%)
    1 / 256 (0.39%)
    0 / 129 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia influenzal
         subjects affected / exposed
    0 / 130 (0.00%)
    1 / 256 (0.39%)
    0 / 129 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Placebo CTP-543 8 mg BID CTP-543 12 mg BID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    60 / 130 (46.15%)
    137 / 256 (53.52%)
    67 / 129 (51.94%)
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    4 / 130 (3.08%)
    13 / 256 (5.08%)
    15 / 129 (11.63%)
         occurrences all number
    4
    13
    15
    Nervous system disorders
    Headache
         subjects affected / exposed
    19 / 130 (14.62%)
    32 / 256 (12.50%)
    13 / 129 (10.08%)
         occurrences all number
    19
    32
    13
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    3 / 130 (2.31%)
    23 / 256 (8.98%)
    13 / 129 (10.08%)
         occurrences all number
    3
    23
    13
    Infections and infestations
    COVID-19
         subjects affected / exposed
    22 / 130 (16.92%)
    60 / 256 (23.44%)
    24 / 129 (18.60%)
         occurrences all number
    22
    60
    24
    Asymptomatic COVID-19
         subjects affected / exposed
    22 / 130 (16.92%)
    33 / 256 (12.89%)
    21 / 129 (16.28%)
         occurrences all number
    22
    33
    21
    Nasopharyngitis
         subjects affected / exposed
    16 / 130 (12.31%)
    33 / 256 (12.89%)
    16 / 129 (12.40%)
         occurrences all number
    16
    33
    16

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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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