Study to Evaluate the Safety and Efficacy of CTP-543 in Adults With Moderate to Severe Alopecia Areata

Study Overview
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Brief Summary
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This study will evaluate the safety and efficacy of CTP-543 on hair loss in adults with chronic, moderate to severe alopecia areata.

Detailed Description
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This is a double-blind, randomized, placebo-controlled multi-center study consisting of 3 cohorts to assess the safety and efficacy of CTP-543. Each Cohort will be initiated sequentially in ascending dose order. Participants will be randomized to either an active dose of CTP-543 or placebo for a 24-week treatment period.

Official Title
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A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata

Conditions
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Alopecia Areata

Intervention / Treatment
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* Drug: CTP-543
* Drug: CTP-543 matching placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Definitive diagnosis of alopecia areata with a current episode lasting at least 6 months and not exceeding 10 years at the time of Screening. Total disease duration greater than 10 years is permitted. At least 50% scalp hair loss, as defined by a Severity of Alopecia Tool (SALT) score ≥50, at Screening and Baseline. Clinical lab results within the normal range Exclusion Criteria: Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or untreated actinic keratosis on the scalp. Treatment with systemic immunosuppressive medications or biologics. Vaccination with herpes zoster vaccine or any live virus within 6 weeks of screening or during the study

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1: CTP-543 4 mg BID<br>Participants will receive CTP-543 4 mg tablets, twice daily for up to 24 weeks. | Drug: CTP-543<br>* Administered as tablets.<br>|
| Experimental: Cohort 2: CTP-543 8 mg BID<br>Participants will receive CTP-543 8 mg tablets, twice daily for up to 24 weeks. | Drug: CTP-543<br>* Administered as tablets.<br>|
| Experimental: Cohort 3: CTP-543 12 mg BID<br>Participants will receive CTP-543 12 mg tablets, twice daily for up to 24 weeks. | Drug: CTP-543<br>* Administered as tablets.<br>|
| Placebo Comparator: Combined Placebo<br>Participants will receive CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | Drug: CTP-543 matching placebo<br>* Administered as tablets.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 | The SALT is a quantitative assessment of scalp hair loss. SALT scores range in severity from 0 (no hair loss) to a maximum of 100 (complete hair loss). Responders were defined as participants achieving at least a 50% relative reduction in SALT score from baseline at Week 24. | Week 24 | 
| Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) | An adverse event is any untoward medical occurrence that may appear or worsen in a participant during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the patient's health, including laboratory test values, regardless of etiology. Any worsening (ie, any clinically significant adverse change in the frequency or intensity of a pre-existing condition) should be considered an adverse event. TEAE is defined as any adverse event that occurs after administration of the first dose of study drug. | From first dose of study drug up to safety follow up at Week 28 |