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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 Subjects with Moderate to Severe Alopecia Areata (THRIVE-AA1)

    Summary
    EudraCT number
    2020-002704-40
    Trial protocol
    FR  
    Global end of trial date
    19 Apr 2022

    Results information
    Results version number
    v1(current)
    This version publication date
    23 Apr 2023
    First version publication date
    23 Apr 2023
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    CP543.3001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04518995
    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
    19 Apr 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Apr 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary purpose of this study was to assess the safety and efficacy of CTP-543 on regrowth of hair following 24 weeks of treatment.
    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
    23 Nov 2020
    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: 50
    Country: Number of subjects enrolled
    Poland: 50
    Country: Number of subjects enrolled
    Canada: 212
    Country: Number of subjects enrolled
    United States: 375
    Country: Number of subjects enrolled
    Spain: 19
    Worldwide total number of subjects
    706
    EEA total number of subjects
    119
    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)
    703
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Subjects were enrolled at study centers in Canada, France, Poland, Spain, and the United States from 23 November 2020 to 19 April 2022.

    Pre-assignment
    Screening details
    946 subjects were screened, out of which 706 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

    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
    140
    351
    215
    Completed
    129
    318
    199
    Not completed
    11
    33
    16
         Non-compliance with Study Drug
    -
    3
    2
         Protocol violation
    2
    -
    1
         Pregnancy
    -
    -
    1
         Reason not specified
    4
    11
    2
         Lost to follow-up
    1
    10
    6
         Treatment Emergent or Worsening Adverse Event
    2
    8
    4
         Lack of efficacy
    2
    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
    140 351 215 706
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    38.7 ( 13.81 ) 38.9 ( 13.32 ) 38.2 ( 12.80 ) -
    Gender categorical
    Units: Subjects
        Female
    89 217 131 437
        Male
    51 134 84 269
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    11 30 13 54
        Not Hispanic or Latino
    119 292 188 599
        Unknown
    10 29 14 53
    Race
    Units: Subjects
        American Indian or Alaska Native
    0 2 1 3
        Asian
    10 22 21 53
        Black or African American
    16 40 27 83
        Native Hawaiian or other Pacific Islander
    1 3 1 5
        White
    98 241 145 484
        Other
    5 17 6 28
        Not reported
    10 26 14 50

    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 of ≤20 at Week 24

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    End point title
    Percentage of Subjects Achieving an Absolute Severity of Alopecia Tool (SALT) Score of ≤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
    140
    351
    215
    Units: percentage of subjects
        number (not applicable)
    0.8
    29.6
    41.5
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [1]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.28
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.23
         upper limit
    0.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.026
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [2]
    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
    [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
    140
    351
    215
    Units: percentage of responders
    number (not applicable)
        Week 12
    11.9
    35.3
    46.4
        Week 16
    8.3
    38.2
    51.5
        Week 20
    6.1
    37.4
    54.0
        Week 24
    4.7
    42.1
    53.0
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [3]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.25
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.17
         upper limit
    0.32
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [4]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.35
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.26
         upper limit
    0.43
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.044
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [5]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.31
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.24
         upper limit
    0.38
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.035
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [6]
    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
    0.041
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [7]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.32
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.26
         upper limit
    0.39
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.033
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [8]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.39
         upper limit
    0.55
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.04
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [9]
    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
    0.44
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.033
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [10]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.39
         upper limit
    0.55
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.039
    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
    140
    351
    215
    Units: percentage of subjects
    number (not applicable)
        Week 4
    0
    0.9
    0
        Week 8
    0
    3.3
    6.1
        Week 12
    0.7
    10.4
    18.2
        Week 16
    2.3
    16.5
    29.6
        Week 20
    0.8
    24.1
    37.0
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0816 [11]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0
         upper limit
    0.02
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.005
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0005 [12]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.01
         upper limit
    0.05
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.01
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0002 [13]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.06
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.03
         upper limit
    0.09
    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
    Placebo v CTP-543 8 mg BID
    Number of subjects included in analysis
    491
    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
    0.018
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [15]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.17
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.12
         upper limit
    0.22
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.027
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [16]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.14
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.1
         upper limit
    0.19
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.024
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [17]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.27
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.2
         upper limit
    0.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.033
    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
    491
    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
    0.19
         upper limit
    0.28
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.024
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [19]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.35
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.28
         upper limit
    0.41
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.034
    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) to 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
    139
    345
    212
    Units: percent change
    arithmetic mean (standard deviation)
        Relative CFB at Week 4 (n= 139, 345, 211)
    -0.2 ( 5.59 )
    -2.6 ( 12.79 )
    -4.0 ( 12.03 )
        Relative CFB at Week 8 (n= 138, 336, 212)
    -2.3 ( 11.41 )
    -10.9 ( 21.00 )
    -17.5 ( 27.16 )
        Relative CFB at Week 12 (n= 136, 328, 209)
    -0.6 ( 16.63 )
    -22.2 ( 29.45 )
    -31.1 ( 35.53 )
        Relative CFB at Week 16 (n= 131, 322, 203)
    -1.8 ( 21.06 )
    -30.3 ( 34.20 )
    -41.2 ( 38.95 )
        Relative CFB at Week 20 (n= 131, 316, 200)
    -1.3 ( 22.14 )
    -36.0 ( 37.82 )
    -46.8 ( 41.32 )
        Relative CFB at Week 24 (n= 128, 318, 200)
    -1.5 ( 23.30 )
    -41.2 ( 39.37 )
    -50.4 ( 41.52 )
    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
    484
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0564 [20]
    Method
    MMRM
    Parameter type
    Least Square (LS) Mean Difference
    Point estimate
    -2.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.4
         upper limit
    0.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.13
    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
    351
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0054 [21]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -3.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.9
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.23
    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
    484
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [22]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -8.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -12.7
         upper limit
    -4.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.16
    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
    351
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [23]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -14.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -19.5
         upper limit
    -10.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.34
    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
    484
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [24]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -21.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -27.5
         upper limit
    -16
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.94
    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
    351
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [25]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -30
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -36.3
         upper limit
    -23.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.19
    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
    484
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [26]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -28.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -35.1
         upper limit
    -21.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.38
    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
    351
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [27]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -38.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -45.7
         upper limit
    -31.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.66
    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
    484
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [28]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -34.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -42.1
         upper limit
    -27.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.66
    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
    351
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [29]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -44.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -52.3
         upper limit
    -36.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.96
    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
    484
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [30]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -39.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -46.7
         upper limit
    -31.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.79
    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
    351
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [31]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -55.1
         upper limit
    -39
    Variability estimate
    Standard error of the mean
    Dispersion value
    4.1
    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
    140
    351
    215
    Units: percentage of responders
    number (not applicable)
        Weeks 12
    3.7
    36.3
    43.5
        Weeks 16
    7.7
    44.2
    54.7
        Weeks 20
    8.4
    49.1
    57.1
        Weeks 24
    9.4
    53.8
    59.8
    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
    491
    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
    355
    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
    CTP-543 8 mg BID v Placebo
    Number of subjects included in analysis
    491
    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
    355
    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
    491
    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
    355
    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
    491
    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
    355
    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
    140
    351
    215
    Units: percentage of responders
    number (not applicable)
        Week 12
    6.7
    38.9
    49.8
        Week 16
    9.0
    49.1
    58.3
        Week 20
    10.0
    50.6
    60.0
        Week 24
    10.2
    56.6
    65.5
    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
    491
    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
    355
    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
    491
    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
    355
    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
    491
    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
    355
    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
    491
    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
    355
    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 change from baseline indicates (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 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
    136
    328
    209
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline
    6.5 ( 0.74 )
    6.4 ( 0.86 )
    6.4 ( 0.76 )
        CFB at Week 12 (n= 136, 327, 209)
    -0.1 ( 0.74 )
    -1.0 ( 1.38 )
    -1.4 ( 1.53 )
        CFB at Week 16 (n= 131, 321, 203)
    -0.2 ( 0.99 )
    -1.3 ( 1.55 )
    -1.9 ( 1.77 )
        CFB at Week 20 (n= 131, 314, 200)
    -0.1 ( 0.83 )
    -1.6 ( 1.77 )
    -2.2 ( 1.92 )
        CFB at Week 24 (n= 127, 317, 200)
    -0.2 ( 0.90 )
    -1.9 ( 1.89 )
    -2.4 ( 2.03 )
    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
    464
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [48]
    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
    0.13
    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
    345
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [49]
    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
    0.14
    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
    464
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [50]
    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
    0.15
    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
    345
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [51]
    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
    0.16
    Notes
    [51] - CFB in the CGI-S score was analysed using mixed model repeated measures 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
    464
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [52]
    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
    0.16
    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
    345
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [53]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.3
         upper limit
    -1.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.18
    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
    464
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [54]
    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
    [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
    345
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [55]
    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
    0.19
    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
    137
    329
    209
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline
    6.5 ( 0.76 )
    6.5 ( 0.80 )
    6.4 ( 0.86 )
        CFB at Week 12 (n= 135, 329, 209)
    -0.4 ( 1.08 )
    -1.1 ( 1.64 )
    -1.4 ( 1.79 )
        CFB at Week 16 (n= 133, 322, 204)
    -0.2 ( 0.78 )
    -1.5 ( 1.83 )
    -2.0 ( 1.95 )
        CFB at Week 20 (n= 130, 318, 200)
    -0.3 ( 1.01 )
    -1.8 ( 2.01 )
    -2.2 ( 2.07 )
        CFB at Week 24 (n= 128, 318, 200)
    -0.1 ( 0.74 )
    -1.9 ( 2.05 )
    -2.4 ( 2.14 )
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [56]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    -0.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.16
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [57]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.3
         upper limit
    -0.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.17
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [58]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.6
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.17
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [59]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.1
         upper limit
    -1.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.18
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [60]
    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
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.18
    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
    346
    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
    -2.3
         upper limit
    -1.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [62]
    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
    0.19
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [63]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -2.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.6
         upper limit
    -1.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    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
    140
    351
    215
    Units: percentage of subjects
    number (not applicable)
        75% Relative Reduction: Week 12
    0
    8.8
    17.7
        75% Relative Reduction: Week 24
    0
    28.6
    40.0
        90% Relative Reduction: Week 12
    0
    3.4
    10.0
        90% Relative Reduction: Week 24
    0
    19.2
    32.0
    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
    491
    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
    0.06
         upper limit
    0.12
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.016
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [65]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.17
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.12
         upper limit
    0.22
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.026
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [66]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.28
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.23
         upper limit
    0.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.025
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [67]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.38
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.32
         upper limit
    0.45
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.033
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0006 [68]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.04
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.02
         upper limit
    0.06
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.01
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [69]
    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
    0.02
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [70]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.19
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.15
         upper limit
    0.23
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.022
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [71]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.24
         upper limit
    0.37
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.032
    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 analyzed 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
    83
    204
    132
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline
    1.7 ( 2.05 )
    1.2 ( 1.77 )
    1.4 ( 1.91 )
        CFB at Week 12 (n= 76, 187, 123)
    -0.2 ( 1.38 )
    0.8 ( 1.70 )
    1.1 ( 2.05 )
        CFB at Week 24 (n= 72, 192, 118)
    -0.2 ( 1.68 )
    1.6 ( 1.96 )
    1.8 ( 2.17 )
    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
    287
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.001 [72]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.3
         upper limit
    1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.22
    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
    215
    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
    0.6
         upper limit
    1.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.23
    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
    287
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [74]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.2
         upper limit
    2.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.24
    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
    215
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [75]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.4
         upper limit
    2.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.26
    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 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
    86
    219
    150
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline
    1.5 ( 2.12 )
    1.4 ( 2.10 )
    1.7 ( 2.12 )
        CFB at Week 12 (n= 84, 215, 145)
    -0.1 ( 1.18 )
    0.9 ( 1.50 )
    1.3 ( 2.08 )
        CFB at Week 24 (n= 78, 209, 138)
    0.3 ( 1.30 )
    1.7 ( 1.99 )
    2.0 ( 2.28 )
    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
    305
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [76]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.5
         upper limit
    1.3
    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
    236
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [77]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1
         upper limit
    1.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.22
    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
    305
    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
    236
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [79]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    1.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.4
         upper limit
    2.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.26
    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 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
    137
    329
    209
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline
    4.5 ( 0.86 )
    4.5 ( 0.89 )
    4.5 ( 0.85 )
        CFB at Week 12 (n=135, 329, 209)
    -0.6 ( 1.15 )
    -1.3 ( 1.42 )
    -1.7 ( 1.36 )
        CFB at Week 16 (n=133, 322, 204)
    -0.4 ( 1.18 )
    -1.4 ( 1.44 )
    -1.8 ( 1.43 )
        CFB at Week 20 (n=131, 318, 200)
    -0.4 ( 1.14 )
    -1.4 ( 1.52 )
    -1.8 ( 1.52 )
        CFB at Week 24 (n=128, 318, 200)
    -0.3 ( 1.09 )
    -1.5 ( 1.54 )
    -1.9 ( 1.64 )
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [80]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1
         upper limit
    -0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [81]
    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
    0.13
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [82]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [83]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [84]
    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
    0.13
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [85]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.7
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [86]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [87]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.6
    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
    [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
    140
    351
    215
    Units: percentage of subjects
    number (not applicable)
        Week 12
    20.7
    44.7
    53.1
        Week 16
    14.3
    48.1
    53.9
        Week 20
    13.7
    47.5
    57.5
        Week 24
    12.5
    49.1
    58.0
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [88]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.25
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.17
         upper limit
    0.34
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.044
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [89]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.32
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.23
         upper limit
    0.42
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.049
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [90]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.35
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.27
         upper limit
    0.42
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.04
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [91]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.39
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.3
         upper limit
    0.48
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.045
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [92]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.34
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.26
         upper limit
    0.42
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.04
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [93]
    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
    0.51
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.045
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [94]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.29
         upper limit
    0.44
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.039
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [95]
    Method
    Mantel-Haenszel
    Parameter type
    Risk difference (RD)
    Point estimate
    0.44
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.35
         upper limit
    0.53
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.045
    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
    137
    329
    209
    Units: score on a scale
    arithmetic mean (standard deviation)
        STHC: Baseline
    4.5 ( 0.79 )
    4.6 ( 0.79 )
    4.5 ( 0.76 )
        STHC: CFB at Week 12 (n=135, 329, 209)
    -0.4 ( 0.98 )
    -1.1 ( 1.23 )
    -1.3 ( 1.17 )
        STHC: CFB at Week 16 (n=133, 322, 204)
    -0.3 ( 1.03 )
    -1.2 ( 1.28 )
    -1.5 ( 1.33 )
        STHC: CFB at Week 20 (n=131, 318, 200)
    -0.3 ( 0.98 )
    -1.3 ( 1.35 )
    -1.6 ( 1.36 )
        STHC: CFB at Week 24 (n=128, 318, 200)
    -0.2 ( 1.00 )
    -1.4 ( 1.43 )
    -1.7 ( 1.45 )
        SEHC: Baseline
    4.6 ( 0.67 )
    4.6 ( 0.76 )
    4.6 ( 0.68 )
        SEHC: CFB at Week 12 (n=135, 329, 209)
    -0.4 ( 0.83 )
    -1.0 ( 1.19 )
    -1.3 ( 1.16 )
        SEHC: CFB at Week 16 (n=133, 322, 204)
    -0.3 ( 0.84 )
    -1.1 ( 1.31 )
    -1.4 ( 1.34 )
        SEHC: CFB at Week 20 (n=131, 318, 200)
    -0.3 ( 0.90 )
    -1.2 ( 1.36 )
    -1.5 ( 1.40 )
        SEHC: CFB at Week 24 (n=128, 318, 200)
    -0.2 ( 0.84 )
    -1.2 ( 1.41 )
    -1.6 ( 1.52 )
        HSWYE: Baseline
    3.8 ( 1.39 )
    4.0 ( 1.28 )
    3.9 ( 1.35 )
        HSWYE: CFB at Week 12 (n=135, 329, 209)
    -0.2 ( 0.88 )
    -1.0 ( 1.20 )
    -1.1 ( 1.34 )
        HSWYE: CFB at Week 16 (n=133, 322, 204)
    -0.1 ( 0.90 )
    -1.1 ( 1.24 )
    -1.3 ( 1.27 )
        HSWYE: CFB at Week 20 (n=131, 318, 200)
    -0.2 ( 0.88 )
    -1.2 ( 1.25 )
    -1.4 ( 1.32 )
        HSWYE: CFB at Week 24 (n=128, 318, 200)
    -0.2 ( 0.87 )
    -1.2 ( 1.26 )
    -1.4 ( 1.36 )
        HSWYEl: Baseline
    3.7 ( 1.44 )
    3.9 ( 1.37 )
    3.7 ( 1.44 )
        HSWYEl: CFB at Week 12 (n=135, 329, 209)
    -0.3 ( 0.79 )
    -0.9 ( 1.21 )
    -0.9 ( 1.24 )
        HSWYEl: CFB at Week 16 (n=133, 322, 204)
    -0.2 ( 0.97 )
    -1.0 ( 1.24 )
    -1.2 ( 1.30 )
        HSWYEl: CFB at Week 20 (n=131, 318, 200)
    -0.3 ( 0.99 )
    -1.1 ( 1.30 )
    -1.1 ( 1.33 )
        HSWYEl: CFB at Week 24 (n=128, 318, 200)
    -0.2 ( 0.95 )
    -1.1 ( 1.37 )
    -1.2 ( 1.37 )
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [96]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    -0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [97]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [98]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.1
         upper limit
    -0.6
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [99]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.13
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [100]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [101]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [102]
    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
    0.13
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [103]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.7
         upper limit
    -1.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.14
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [104]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.8
         upper limit
    -0.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.1
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [105]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [106]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    -0.5
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [107]
    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
    0.12
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [108]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [109]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [110]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [111]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [112]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    -0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [113]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.1
         upper limit
    -0.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [114]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [115]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.4
         upper limit
    -0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [116]
    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
    [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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [117]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [118]
    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
    0.11
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [119]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [120]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.7
         upper limit
    -0.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.1
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [121]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.8
         upper limit
    -0.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [122]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    -0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [123]
    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
    [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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [124]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    -0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.11
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [125]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.8
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1
         upper limit
    -0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12
    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
    466
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [126]
    Method
    MMRM
    Parameter type
    LS Mean Difference
    Point estimate
    -0.9
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.1
         upper limit
    -0.6
    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
    346
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [127]
    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
    0.13
    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= 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
    128
    318
    200
    Units: score on a scale
    arithmetic mean (standard deviation)
        Anxiety: Baseline
    5.5 ( 3.92 )
    6.2 ( 4.06 )
    6.0 ( 3.92 )
        Anxiety: CFB at Week 24
    -0.4 ( 2.98 )
    -1.0 ( 3.26 )
    -1.1 ( 3.24 )
        Depression: Baseline
    3.3 ( 3.06 )
    3.7 ( 3.31 )
    3.6 ( 3.39 )
        Depression: CFB at Week 24
    -0.7 ( 2.67 )
    -0.9 ( 2.93 )
    -1.1 ( 2.88 )
    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
    446
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.2489 [128]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.2
         upper limit
    0.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.3
    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
    328
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.1235 [129]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.1
         upper limit
    1.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.32
    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
    446
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.9765 [130]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.5
         upper limit
    0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.25
    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
    328
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.3559 [131]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference
    Point estimate
    0.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.3
         upper limit
    0.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.27
    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
    140
    351
    215
    Units: percentage of subjects
        number (not applicable)
    0
    20.8
    34.5
    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
    491
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [132]
    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
    0.25
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.022
    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
    355
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001 [133]
    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
    0.39
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.032
    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. 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
    4 / 140 (2.86%)
    4 / 350 (1.14%)
    1 / 215 (0.47%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Nervous system disorders
    Epilepsy
         subjects affected / exposed
    1 / 140 (0.71%)
    0 / 350 (0.00%)
    0 / 215 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Abortion spontaneous
         subjects affected / exposed
    1 / 140 (0.71%)
    0 / 350 (0.00%)
    0 / 215 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 140 (0.00%)
    1 / 350 (0.29%)
    0 / 215 (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
    Pyrexia
         subjects affected / exposed
    0 / 140 (0.00%)
    1 / 350 (0.29%)
    0 / 215 (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
    Gastrointestinal disorders
    Ileus
         subjects affected / exposed
    0 / 140 (0.00%)
    0 / 350 (0.00%)
    1 / 215 (0.47%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 140 (0.00%)
    1 / 350 (0.29%)
    0 / 215 (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
    Psychiatric disorders
    Adjustment disorder
         subjects affected / exposed
    1 / 140 (0.71%)
    0 / 350 (0.00%)
    0 / 215 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    1 / 140 (0.71%)
    1 / 350 (0.29%)
    1 / 215 (0.47%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    0 / 140 (0.00%)
    1 / 350 (0.29%)
    0 / 215 (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
    Meningitis
         subjects affected / exposed
    0 / 140 (0.00%)
    1 / 350 (0.29%)
    0 / 215 (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
    31 / 140 (22.14%)
    117 / 350 (33.43%)
    75 / 215 (34.88%)
    Investigations
    Blood creatine phosphokinase (increased)
         subjects affected / exposed
    2 / 140 (1.43%)
    21 / 350 (6.00%)
    11 / 215 (5.12%)
         occurrences all number
    2
    21
    11
    Nervous system disorders
    Headache
         subjects affected / exposed
    8 / 140 (5.71%)
    41 / 350 (11.71%)
    24 / 215 (11.16%)
         occurrences all number
    8
    41
    24
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    7 / 140 (5.00%)
    31 / 350 (8.86%)
    26 / 215 (12.09%)
         occurrences all number
    7
    31
    26
    Infections and infestations
    COVID-19
         subjects affected / exposed
    8 / 140 (5.71%)
    19 / 350 (5.43%)
    15 / 215 (6.98%)
         occurrences all number
    8
    19
    15
    Nasopharyngitis
         subjects affected / exposed
    5 / 140 (3.57%)
    18 / 350 (5.14%)
    8 / 215 (3.72%)
         occurrences all number
    5
    18
    8
    Upper respiratory tract infection
         subjects affected / exposed
    9 / 140 (6.43%)
    9 / 350 (2.57%)
    8 / 215 (3.72%)
         occurrences all number
    9
    9
    8

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Aug 2020
    • Clarified contraception language to state “confirmed infertility”. • Clarified inclusion of subjects previously treated with a JAK inhibitor was at the discretion of the principal investigator, with consultation with the medical monitor if tolerability questions arose. • Amended vital sign collection to allow for contactless monitoring of subject temperature. • Removed the word “coverage” from question 1 and question 2 of the QPRO.
    15 Oct 2020
    • Removed stratification restriction to enable the study to cover the overall eligible subject population. • Adjusted the original randomization ratio (3:3:1) to 3:5:2 due to re-evaluation of power calculations. • Distinguished treatment withdrawal from study withdrawal. • Removed the exclusion criterion “Atypical nevi or cutaneous lesions that are suspicious for malignancy”. • Adjusted the efficacy evaluation by adding Week 8 to the key secondary endpoints for SALT score assessment and adding Week 16 and 20 to secondary endpoints for CGI/PGI. • Added collection time points for ECG (added Week 12), PGI-I/CGI-I (added Week 16 and Week 20), and PGI-S/CGI-S (added Week 16 and Week 20). • Added evaluation for progressive multifocal leukoencephalopathy (PML) and presence or absence of nasal hairs during physical exams. • Updated statistical analyses to reflect modifications to primary endpoint analysis, missing data, adjustment for multiplicity, and tipping point analysis.
    08 Oct 2021
    • Coordinating investigator contact information updated.

    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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