Study to assess the efficacy and safety following administration of CTP-543 in adult patients with moderate to severe alopecia areata

Phase 1

• Conditions: Moderate to severe alopecia areata in adult patients; MedDRA version: 20.0Level: PTClassification code 10001761Term: Alopecia areataSystem Organ Class: 10040785 - Skin and subcutaneous tissue disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2020-002704-40-FR
• Lead Sponsor: Concert Pharmaceuticals, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-30
• Last Update Posted: 23 May 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 700

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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 690
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

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 Gilber

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified