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    Clinical Trial Results:
    A MULTICENTER, OPEN-LABEL, EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY AND EFFICACY OF CTP-543 IN ADULT PATIENTS WITH MODERATE TO SEVERE ALOPECIA AREATA

    Summary
    EudraCT number
    		 2021-002365-18
    Trial protocol
    		 DE   ES   HU  
    Global end of trial date
    18 Jul 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Aug 2025
    First version publication date
    02 Aug 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CP543.5002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05041803
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND: 131,423
    Sponsors
    Sponsor organisation name
    Sun Pharmaceutical Industries, Inc.
    Sponsor organisation address
    2 Independence Way, Princeton, NJ, United States, 08540
    Public contact
    Manager, Punit Patel, Clinical.Trial@sunpharma.com
    Scientific contact
    Associate Director, Aryany Sanchez, Clinical.Trial@sunpharma.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
    18 Jul 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    18 Jul 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Jul 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The overall objectives of the study are to evaluate long-term safety of CTP-543 and to assess long-term effects of CTP-543 on treating hair loss in adult patients with moderate to severe alopecia areata
    Protection of trial subjects
    Prior to performing any study-related activities under the study protocol, written informed consent with the approved Informed Consent Form was obtained from the subject. All study subjects were required to read and sign an Informed Consent Form.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    27 Sep 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Poland: 107
    Country: Number of subjects enrolled
    Spain: 72
    Country: Number of subjects enrolled
    France: 91
    Country: Number of subjects enrolled
    Germany: 124
    Country: Number of subjects enrolled
    Hungary: 13
    Worldwide total number of subjects
    407
    EEA total number of subjects
    407
    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)
    403
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Subjects who previously participated in a qualifying phase 3 study with CTP-543(CP543.3001 or CP543.3002), and who completed a 24-week Treatment Period on study drug (active or placebo), had the opportunity to enroll in this OLE study. A total of 407 subjects were enrolled: 114 subjects from CP543.3001 and 293 (72.0%) subjects from CP543.3002.

    Period 1
    Period 1 title
    Overall trial: Open label Extension (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    The study was not blinded. Initially, subjects were assigned to receive the same daily treatment at a dose of 8 mg or 12 mg CTP-543 BID as in the qualifying study. Subjects who received placebo in the qualifying study were randomized in a 1:1 ratio to CTP‑543 8 mg BID or CTP-543 12 mg BID.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 CTP-543 8 mg BID
    Arm description
    		 Participants received CTP-543 8 mg tablets, orally, twice a day
    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 8 mg tablets, orally, twice a day

    Arm title
    		 CTP-543 12 mg BID
    Arm description
    		 Participants received CTP-543 12 mg tablets, orally, twice a day
    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
    Deuruxolitinib (CTP-543) 12 mg tablets, orally, twice a day

    Arm title
    		 CTP-543 8 mg BID to CTP-543 12 mg BID
    Arm description
    		 Participants received CTP-543 12 mg tablets, orally, twice a day, after they received CTP-543 8 mg tablets, orally, twice a day
    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 8 mg tablets, orally, twice a day

    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
    Deuruxolitinib (CTP-543) 12 mg tablets, orally, twice a day

    Arm title
    		 CTP-543 12 mg BID to CTP-543 8 mg BID
    Arm description
    		 Participants received CTP-543 8 mg tablets, orally, twice a day, after they received CTP-543 12 mg tablets, orally, twice a day
    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 8 mg tablets, orally, twice a day

    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
    Deuruxolitinib (CTP-543) 12 mg tablets, orally, twice a day

    Number of subjects in period 1
    		CTP-543 8 mg BID CTP-543 12 mg BID CTP-543 8 mg BID to CTP-543 12 mg BID CTP-543 12 mg BID to CTP-543 8 mg BID
    Started
    137
    46
    115
    109
    Completed
    111
    27
    87
    103
    Not completed
    26
    19
    28
    6
         Consent withdrawn by subject
    15
    14
    18
    3
         Physician decision
    3
    -
    6
    1
         Other
    1
    -
    -
    1
         Pregnancy
    2
    1
    -
    -
         Lost to follow-up
    2
    1
    3
    1
         Treatment Emergement or Worsening Adverse Event
    3
    3
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    CTP-543 8 mg BID
    Reporting group description
    		 Participants received CTP-543 8 mg tablets, orally, twice a day

    Reporting group title
    CTP-543 12 mg BID
    Reporting group description
    		 Participants received CTP-543 12 mg tablets, orally, twice a day

    Reporting group title
    CTP-543 8 mg BID to CTP-543 12 mg BID
    Reporting group description
    		 Participants received CTP-543 12 mg tablets, orally, twice a day, after they received CTP-543 8 mg tablets, orally, twice a day

    Reporting group title
    CTP-543 12 mg BID to CTP-543 8 mg BID
    Reporting group description
    		 Participants received CTP-543 8 mg tablets, orally, twice a day, after they received CTP-543 12 mg tablets, orally, twice a day

    Reporting group values
    		 CTP-543 8 mg BID CTP-543 12 mg BID CTP-543 8 mg BID to CTP-543 12 mg BID CTP-543 12 mg BID to CTP-543 8 mg BID Total
    Number of subjects
    		 137 46 115 109 407
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 136 45 115 107 403
        From 65-84 years
    		 1 1 0 2 4
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.55 ( 12.588 ) 39.20 ( 14.784 ) 39.84 ( 12.351 ) 37.31 ( 11.794 ) -
    Gender categorical
    Units: Subjects
        Female
    		 96 21 83 70 270
        Male
    		 41 25 32 39 137

    End points

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    End points reporting groups
    Reporting group title
    CTP-543 8 mg BID
    Reporting group description
    		 Participants received CTP-543 8 mg tablets, orally, twice a day

    Reporting group title
    CTP-543 12 mg BID
    Reporting group description
    		 Participants received CTP-543 12 mg tablets, orally, twice a day

    Reporting group title
    CTP-543 8 mg BID to CTP-543 12 mg BID
    Reporting group description
    		 Participants received CTP-543 12 mg tablets, orally, twice a day, after they received CTP-543 8 mg tablets, orally, twice a day

    Reporting group title
    CTP-543 12 mg BID to CTP-543 8 mg BID
    Reporting group description
    		 Participants received CTP-543 8 mg tablets, orally, twice a day, after they received CTP-543 12 mg tablets, orally, twice a day

    Primary: Relative Change from Pre-zero Baseline (to 108 weeks) in Total SALT Scores

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    End point title
    		 Relative Change from Pre-zero Baseline (to 108 weeks) in Total SALT Scores [1]
    End point description
    SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). This outcome measures the relative Change from Pre-zero Baseline in Total SALT Scores. Relative change from baseline was defined as 100*(post-baseline value - baseline)/baseline.
    End point type
    Primary
    End point timeframe
    108 weeks
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary efficacy endpoint was presented with descriptive statistics with no statistical hypothesis testing. It was based on the Efficacy Population. Pre-zero baseline was defined as the last observation obtained prior to first dose of active drug in the study or a previous qualifying study.
    End point values
    		 CTP-543 8 mg BID CTP-543 12 mg BID CTP-543 8 mg BID to CTP-543 12 mg BID CTP-543 12 mg BID to CTP-543 8 mg BID
    Number of subjects analysed
    97
    0 [2]
    48
    99
    Units: score on a scale
        arithmetic mean (standard deviation)
    -96.38 ( 8.790 )
    ( )
    -88.57 ( 18.715 )
    -90.25 ( 18.860 )
    Notes
    [2] - 12 mg BID dose was discontinued
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    108 Weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    CTP-543 8 mg BID
    Reporting group description
    		 -

    Reporting group title
    CTP-543 12 mg BID
    Reporting group description
    		 -

    Serious adverse events
    		 CTP-543 8 mg BID CTP-543 12 mg BID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    16 / 367 (4.36%)
    5 / 273 (1.83%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Gastric cancer stage IV
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tongue neoplasm malignant stage unspecified
         subjects affected / exposed
    0 / 367 (0.00%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Abortion spontaneous
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Anaphylactic reaction
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Bartholin's cyst
         subjects affected / exposed
    0 / 367 (0.00%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Menorrhagia
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 367 (0.00%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Humerus fracture
         subjects affected / exposed
    0 / 367 (0.00%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Spinal fracture
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Tricuspid valve incompetence
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Intracranial hypotension
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 367 (0.27%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Osteoarthritis
         subjects affected / exposed
    2 / 367 (0.54%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Synovial cyst
         subjects affected / exposed
    0 / 367 (0.00%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Hepatitis E
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infectious mononucleosis
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tonsillitis
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Wound infection
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bartholin's abscess
         subjects affected / exposed
    0 / 367 (0.00%)
    1 / 273 (0.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Type 1 diabetes mellitus
         subjects affected / exposed
    1 / 367 (0.27%)
    0 / 273 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 CTP-543 8 mg BID CTP-543 12 mg BID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    176 / 367 (47.96%)
    127 / 273 (46.52%)
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    42 / 367 (11.44%)
    24 / 273 (8.79%)
         occurrences all number
    53
    27
    Weight increased
         subjects affected / exposed
    14 / 367 (3.81%)
    14 / 273 (5.13%)
         occurrences all number
    15
    14
    Nervous system disorders
    Headache
         subjects affected / exposed
    23 / 367 (6.27%)
    9 / 273 (3.30%)
         occurrences all number
    27
    10
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    13 / 367 (3.54%)
    14 / 273 (5.13%)
         occurrences all number
    16
    17
    Thrombocytosis
         subjects affected / exposed
    20 / 367 (5.45%)
    26 / 273 (9.52%)
         occurrences all number
    30
    28
    Infections and infestations
    Asymptomatic COVID-19
         subjects affected / exposed
    83 / 367 (22.62%)
    58 / 273 (21.25%)
         occurrences all number
    92
    65
    COVID-19
         subjects affected / exposed
    78 / 367 (21.25%)
    44 / 273 (16.12%)
         occurrences all number
    81
    46
    Upper respiratory tract infection
         subjects affected / exposed
    30 / 367 (8.17%)
    14 / 273 (5.13%)
         occurrences all number
    50
    16
    Folliculitis
         subjects affected / exposed
    4 / 367 (1.09%)
    14 / 273 (5.13%)
         occurrences all number
    4
    14
    Nasopharyngitis
         subjects affected / exposed
    76 / 367 (20.71%)
    48 / 273 (17.58%)
         occurrences all number
    118
    71
    Metabolism and nutrition disorders
    Hypercholesterolaemia
         subjects affected / exposed
    41 / 367 (11.17%)
    29 / 273 (10.62%)
         occurrences all number
    46
    32

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Sep 2021
    Protocol amendment included the following changes: 1. The addition of pregnancy testing on a monthly basis. 2. Amended vital sign collection to allow for flexibility with the type of assessment measure for temperature.
    21 Oct 2022
    1. Amended to include 1 additional year of treatment with CTP-543 for responders (up to 108 weeks)
    30 May 2023
    Following updates we made to the protocol: 1. Name change due to acquisition of Concert Pharmaceuticals by Sun Pharmaceutical Industries, Inc. 2. Due to an urgent Safety Measure, the 12 mg BID dose was discontinued and all patients previously assigned to the 12 mg BID dose were reduced to 8 mg BID dose, if they elected to continue in the trial

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    28 Apr 2023
    Dosing with 12 mg BID was interrupted due to an urgent safety measure
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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