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    Summary
    EudraCT Number:2020-002704-40
    Sponsor's Protocol Code Number:CP543.3001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-30
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2020-002704-40
    A.3Full title of the trial
    A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of CTP- 543 in Adult Patients with Moderate to Severe Alopecia Areata
    ÉTUDE EN DOUBLE AVEUGLE, RANDOMISÉE, CONTRÔLÉE PAR PLACEBO,
    VISANT À ÉVALUER L'EFFICACITÉ ET LA SÉCURITÉ D'EMPLOI DU CTP-543
    CHEZ DES PATIENTS ADULTES ATTEINTS DE PELADE MODÉRÉE À SÉVÈRE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess the efficacy and safety following administration of CTP-543 in adult patients with moderate to severe alopecia areata
    A.4.1Sponsor's protocol code numberCP543.3001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04518995
    A.5.4Other Identifiers
    Name:IND NUMBERNumber:131,423
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorConcert Pharmaceuticals, Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportConcert Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLinical France SARL
    B.5.2Functional name of contact pointMedical Manager
    B.5.3 Address:
    B.5.3.1Street Address52 Take Ionescu Boulevard
    B.5.3.2Town/ cityTimisoara
    B.5.3.3Post code300073
    B.5.3.4CountryRomania
    B.5.4Telephone number+40256207271
    B.5.5Fax number+40256207273
    B.5.6E-maildiana.chera@linical.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namedeuruxolitinib (Tablet 8 mg every 12 hours)
    D.3.2Product code CTP-543
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeuruxolitinib phosphate
    D.3.9.1CAS number 2147706-60-1
    D.3.9.2Current sponsor codeCTP-543 phosphate
    D.3.9.3Other descriptive nameC-21543; D8-ruxolitinib
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namedeuruxolitinib (Tablet 12 mg every 12 hours)
    D.3.2Product code CTP-543
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeuruxolitinib phosphate
    D.3.9.1CAS number 2147706-60-1
    D.3.9.2Current sponsor codeCTP-543 phosphate
    D.3.9.3Other descriptive nameC-21543; D8-ruxolitinib
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Moderate to severe alopecia areata in adult patients
    E.1.1.1Medical condition in easily understood language
    Episode of hair loss associated with alopecia areata in adult patients
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10001761
    E.1.2Term Alopecia areata
    E.1.2System Organ Class 10040785 - Skin and subcutaneous tissue disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The overall objectives of the study are to assess the efficacy and safety following administration of CTP-543 in adult patients with moderate to severe alopecia areata.
    The primary objectives of the study are to assess:
    -The efficacy of CTP-543 on regrowth of hair following 24 weeks of treatment
    -The safety of CTP-543 following 24 weeks of treatment.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to assess:
    - Clinician- and patient-reported impression of the severity and improvement of alopecia areata
    - Patient-reported satisfaction with their scalp hair
    - Patient-reported levels of anxiety and depression
    - Changes in eyebrows and eyelashes.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Written informed consent, and authorization for release and use of protected health information.
    2.Between 18 and 65 years of age, inclusive, at the time of informed consent.
    3.Definitive diagnosis of alopecia areata with a current episode of scalp hair loss lasting at least 6 months and not exceeding 10 years at the time of Screening. Total disease duration greater than 10 years is permitted.
    4.At least 50% scalp hair loss, as defined by a SALT score ≥50, at Screening and Baseline.
    5. Female subjects are eligible to participate if at least one of the following conditions applies:
    a) Is a woman of childbearing potential (WOCBP) and using a medically highly effective form of birth control with a failure rate less than 1% per year from at least 4 weeks prior to Baseline until at least 30 days following last dose of study drug. Examples of medically highly effective birth control methods include:
    i. Combined (estrogen and progestogen containing) hormonal contraception (oral, patch, vaginal ring)
    ii. Progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
    iii. Intrauterine device or intrauterine hormone-releasing system
    iv. Bilateral tubal occlusion
    v. Vasectomized partner (confirmed as the sole sexual partner and confirmed medical assessment of the surgical success)
    vi. Sexual abstinence (reliable as refraining from heterosexual intercourse during the above-mentioned period)
    b) Is not a WOCBP:
    i. Premenopausal with one of the following:
    a. Documented hysterectomy;
    b. Documented bilateral salpingectomy;
    c. Documented bilateral oophorectomy.
    ii. Postmenopausal (cessation of menses for at least 12 months prior to screening)
    Postmenopausal is defined as no menses for 12 months without an alternative medical cause. In addition, a high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm postmenopausal in women under 60 years old and not using hormonal contraception or hormone replacement therapy (HRT). However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. Females on HRT and whose menopausal status is in doubt will be required to use one of the nonestrogen hormonal highly effective contraception methods if they wish to continue their HRT during the study.
    Otherwise, they must discontinue HRT to allow confirmation of
    postmenopausal status before study enrollment
    6. Male participants must:
    a) Agree to use, with their partners, male contraception (condom) and one of the highly effective contraceptive methods listed in Inclusion Criterion 5, from Baseline until at least 90 days following last dose of study drug.
    b) Refrain from donating sperm during the study and for at least 90 days after the end of the study.
    7. Willing to comply with the study visits and requirements of the study protocol.
    E.4Principal exclusion criteria
    1.History or presence of hair transplants.
    2.Treatment with other medications or agents within 1 month of Baseline or during the study that may affect hair regrowth or immune response, including but not limited to: corticosteroids administered orally, by injection, or applied to areas of skin affected by alopecia (intranasal and inhaled corticosteroids are allowed, eye and ear drops containing corticosteroids are also allowed); oral retinoids, oral cyclines (minocin, tetracycline); platelet-rich plasma injections; topical application to affected areas of retinoids, anthralin, squaric acid, diphenylcyclopropenone, or minoxidil.
    3. Treatment with systemic immunosuppressive medications including but not limited to methotrexate, cyclosporine, azathioprine, chloroquine derivatives, etanercept, JAK inhibitors within 3 months of Screening or during the study.
    NOTE: Patients previously treated with a Janus Kinase inhibitor more than 3 months prior to Screening may be allowed to enter the study at the discretion of the PI, if their previous treatment with a JAK inhibitor was well tolerated, and subsequent JAK treatment would not be thought to put the subject at risk. Consultation with the Medical Monitor may be warranted should questions arise with regard to prior tolerability.
    4.Treatment with biologics (e.g., adalimumab, atlizumab, canakinumab, certolizumab, fontolizumab, golimumab, infliximab, mepolizumab, rituximab, secukinumab, tocilizumab, ustekinumab) within 6 months of Screening or during the study.
    5.Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or other scalp condition that may interfere with the SALT assessment, or untreated actinic keratosis anywhere on the body at Screening and/or Baseline.
    6.Known history of moderate to severe androgenic alopecia or female pattern hair loss prior to alopecia areata.
    7.Unwilling to maintain a consistent hair style, including shampoo and hair products (including hair dye, process, and timing to hair appointments), and to refrain from weaves or extensions throughout the course of the study, or shaving of scalp hair for 2 weeks preceding a SALT assessment.
    8.Use of adhesive wigs, other than banded perimeter wigs, during the study.
    9.History of a lymphoproliferative disease or malignancy, other than non-melanoma skin cancer or cervical carcinoma. Patients with 3 or more basal or squamous cell carcinomas diagnosed in the past 2 years are excluded.
    10.History of solid organ or hematological transplantation.
    11.Fever, inflammation, or systemic signs of illness suggestive of systemic or invasive infection within 2 weeks prior to first dose of study drug.
    12.Abnormal levels of thyroid stimulating hormone at Screening, defined as <0.9 x the lower limit of normal (LLN) and >1.5 x the upper limit of normal (ULN).
    13.Screening labs outside the normal range for parameters associated with potential risk for treatment under investigation. This will include but is not limited to:
    a.Platelets ≤100 x 109/L or ≥ 600 x 109/L
    b.Absolute neutrophil count ≤1.5 x 109/L
    c.Hemoglobin levels ≤10.5 g/dL for females, or hemoglobin levels ≤12.0 g/dL for males
    14.Screening blood level of hemoglobin A1c ≥7% (53 mmol/mol, 8.6 mmol/L).
    15.Abnormal liver function at Screening, defined as ≥2 × ULN of serum alanine transaminase, serum aspartate transaminase, and serum alkaline phosphatase, or ≥1.5 x ULN total bilirubin (unless isolated Gilbert's syndrome).
    16.Abnormal renal function (estimated glomerular filtration rate <60 mL/min/1.73 m2 using the CKD-EPI 2009 equation) at Screening.
    17.Patient has positive results for hepatitis B surface antigens (HBsAg), antibodies to hepatitis B core antigens (anti-HBc), or hepatitis C virus (HCV) at Screening, or known history of human immunodeficiency virus infection.
    18.Vaccination with herpes zoster vaccine or plans to receive a live attenuated vaccine during the study and up to 6 weeks or 5 half-lives prior to randomization, whichever is longer.
    19.Positive TB test, or history of incompletely treated or untreated tuberculosis.
    Note: TB test may be performed via a QuantiFERON-TB Gold test or a Tuberculin Skin Test (TST). If the TST is negative, patients can be randomized into the study, assuming they meet all other inclusion and exclusion criteria. If results are equivocal, or there is reason to believe the result is a false positive, a QuantiFERON-TB Gold must be performed before a patient is eligible for randomization.
    20.History of prolonged QT syndrome or a Screening QTc interval with Fridericia's correction (QTcF) > 450 msec for males or QTcF > 470 msec for females.
    21.History of alcohol, medication, or illicit drug abuse within 1 year before the first dose of study drug.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint
    -The primary efficacy endpoint will be the percentage of patients achieving an absolute SALT score of ≤20 at Week 24.

    E.5.1.1Timepoint(s) of evaluation of this end point
    At Week 24
    E.5.2Secondary end point(s)
    Key Secondary Endpoints
    1.Percentage of responders (defined as “satisfied” or “very satisfied”) on the Hair Satisfaction Patient Reported Outcome (SPRO) scale at Week 24
    2.Percentage of patients achieving an absolute SALT score of ≤20 at Week 20, 16, 12 and 8.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At Week 24, 20, 16, 12 and 8.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    United States
    France
    Poland
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 690
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state45
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 700
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Following the 24-week Treatment Period, patients will have the opportunity to continue receiving treatment in an Open-Label Extension study. If a patient does not wish to continue into the Open-Label Extension study, they will complete treatment at Week 24 and return in 4 weeks for the Post-Treatment Safety Follow-up to assess safety following treatment completion.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-03-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-04-19
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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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