Efficacy and Tolerability Study of Two Dose Regimens of CTP-543 in Adults With Alopecia Areata

Study Overview
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Brief Summary
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This study will evaluate the efficacy and tolerability of once-daily versus twice-daily dosing of CTP-543, in adult patients with chronic, moderate to severe alopecia areata.

Official Title
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A Randomized Parallel-Group Study to Evaluate the Efficacy and Tolerability of Two Dosing Regimens 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: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CTP-543 8 mg BID<br>Participants received 1 x 8 mg CTP-543 tablet and 1 x CTP-543 matching placebo tablet, twice daily (BID) for 24 weeks. | Drug: CTP-543<br>* CTP-543 administered as 8 mg tablet.<br>Drug: CTP-543 Matching Placebo<br>* Administered as tablets to aid treatment masking.<br>|
| Experimental: CTP-543 16 mg QD<br>Participants received 16 mg (2 x 8 mg) CTP-543 tablets, once daily (QD) and after 12 hours, received 2 x CTP-543 matching placebo tablets, QD for 24 weeks. | Drug: CTP-543<br>* CTP-543 administered as 8 mg tablet.<br>Drug: CTP-543 Matching Placebo<br>* Administered as tablets to aid treatment masking.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Relative Change From Baseline in Severity of Alopecia Tool (SALT) Score 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). Relative change (percent change) to baseline is calculated as: 100 x ([baseline SALT score - follow-up SALT score]/baseline SALT score). | Baseline, Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Achieving at Least a 90%, 75%, and 50% Relative Reduction in SALT Score From Baseline | 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). Percentage of participants achieving at least a 50%, 75%, 90% relative reduction in SALT score from baseline at Weeks 4, 8, 12, 16, 20, and 24 are reported. | Baseline, Weeks 4, 8, 12, 16, 20, and 24 | 
| Absolute Change in SALT Scores From Baseline at Weeks 4, 8, 12, 16, 20, and 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). Absolute change equals the difference in SALT measurements (baseline SALT score - follow-up SALT score). | Baseline, Weeks 4, 8, 12, 16, 20, and 24 | 
| Relative Change in SALT Scores From Baseline at Weeks 4, 8, 12, 16 and 20 | 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). Relative change (percent change) to baseline is calculated as: 100 x ([baseline SALT score - follow-up SALT score]/baseline SALT score). | Baseline, Weeks 4, 8, 12, 16 and 20 | 
| Percentage of Participants With Change in Satisfaction of Hair Coverage | Participant satisfaction question was used to assess overall satisfaction with hair coverage, with responses ranging from 1 to 5, as follows: 1 (very dissatisfied), 2 (dissatisfied), 3 (somewhat satisfied), 4 (mostly satisfied), 5 (very satisfied). Higher scores indicate better satisfaction with hair coverage. The percentage of participants with change from Baseline to the Week 24 satisfaction level was reported as categories: satisfied to satisfied; satisfied to dissatisfied; dissatisfied to satisfied and dissatisfied to dissatisfied. Data is reported only for participants with change from Baseline in satisfaction level. | Baseline, Week 24 |