A Phase 2 Durability of Response Study of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata

Study Overview
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Brief Summary
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This is a two part, double-blind, randomized, multicenter study to evaluate the regrowth of hair with CTP-543 and subsequent durability of that regrowth following dose reduction in adult patients with moderate to severe alopecia areata.

Official Title
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A Study to Evaluate Maintenance of Hair Regrowth Following Dose Reduction 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: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. At least 50% scalp hair loss, as defined by a SALT score ≥50, at Screening and Baseline. Willing to comply with the study visits and requirements of the study protocol. Exclusion Criteria: Treatment with other medications or agents within 1 month of Baseline or during the study that may affect hair regrowth or immune response. 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. Treatment with systemic immunosuppressive medications within 3 months of Screening or during the study, or biologics within 6 months of Screening or during the study. Females who are nursing, pregnant, or planning to become pregnant while in the study, and for 30 days after last dose of study drug. Clinically significant medical condition, psychiatric disease, or social condition, as determined by the Investigator, that may unfavorably alter the risk-benefit of study participation, adversely affect study compliance, or confound interpretation of study results.

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: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Part A, Period 1 - Low dose<br>8 mg BID CTP-543 for 24 weeks | Drug: CTP-543<br>* Oral dosing<br>|
| Experimental: Part A, Period 1 - High dose<br>12 mg BID CTP-543 for 24 weeks | Drug: CTP-543<br>* Oral dosing<br>|
| Experimental: Part A, Period 2 - Dose reduction from low dose<br>Dose reduction for a maximum of 24 weeks for those previously receiving 8 mg BID | Drug: CTP-543<br>* Oral dosing<br>|
| Placebo Comparator: Part A, Period 2 - Drug discontinuation from low dose<br>Placebo for a maximum of 24 weeks for those previously receiving 8 mg BID | Drug: Placebo<br>* Oral dosing<br>|
| Experimental: Part A, Period 2 - Dose reduction from high dose<br>Dose reduction for a maximum of 24 weeks for those previously receiving 12 mg BID | Drug: CTP-543<br>* Oral dosing<br>|
| Placebo Comparator: Part A, Period 2 - Drug discontinuation from high dose<br>Placebo for a maximum of 24 weeks for those previously receiving 12 mg BID | Drug: Placebo<br>* Oral dosing<br>|
| Experimental: Part B - Low dose<br>8 mg BID CTP-543 for a maximum of 24 weeks for those meeting loss of maintenance criteria | Drug: CTP-543<br>* Oral dosing<br>|
| Experimental: Part B - High dose<br>12 mg BID CTP-543 for a maximum of 24 weeks for those meeting loss of maintenance criteria | Drug: CTP-543<br>* Oral dosing<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of patients achieving loss of maintenance criteria defined by SALT following dose reduction | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | 24 weeks | 
| Percentage of patients achieving loss of maintenance criteria defined by SALT following drug discontinuation | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | 24 weeks | 
| Proportion of patients by dose group achieving restoration of regrowth | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of patients achieving loss of maintenance criteria following dose reduction | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | Weeks 4, 8, 12, 16, and 20 | 
| Percentage of patients achieving loss of maintenance criteria following drug discontinuation | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | Weeks 4, 8, 12, 16, and 20 | 
| Part A, Period 1: Percentage of responders (defined as satisfied or very satisfied) on the Hair Satisfaction Patient Reported Outcome (SPRO) scale | SPRO is a questionnaire answered by the patient designed to measure how satisfied alopecia areata patients are with their hair at the time of the assessment. | Weeks 12, 16, 20, and 24 | 
| Part A, Period 1: Percentage of patients achieving an absolute SALT score ≤20 | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | Weeks 8, 12, 16, 20, and 24 | 
| Part A, Period 1: Relative change in SALT scores from Baseline | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | Weeks 4, 8, 12, 16, 20, and 24 | 
| Part A, Period 1: Percentage of responders (defined as much improved or very much improved) using the CGI-I | The Clinician Global Impression of Improvement (CGI-I) is a questionnaire that asks the clinician to evaluate the improvement or worsening of the patient's alopecia areata as compared to the start of the study. | Weeks 12, 16, 20, and 24 | 
| Part A, Period 1: Percentage of responders (defined as much improved or very much improved) using the PGI-I | The Patient Global Impression of Improvement (PGI-I) is a self-administered questionnaire that asks the patient to evaluate the improvement or worsening of their alopecia areata as compared to the start of the study. | Weeks 12, 16, 20, and 24 | 
| Part A, Period 1: Change from Baseline in the CGI-S | The Clinician Global Impression of Severity (CGI-S) is a questionnaire that asks the clinician to evaluate the severity of the patient's alopecia areata at the time of assessment. | Weeks 12, 16, 20, and 24 | 
| Part A, Period 1: Change from Baseline in the PGI-S | The Patient Global Impression of Severity (PGI-S) is a self-administered questionnaire that asks the patient to evaluate the severity of their alopecia areata at the time of assessment. | Weeks 12, 16, 20, and 24 | 
| Part A, Period 1: Change from Baseline on the individual items of the Hair Quality Patient Reported Outcome (QPRO) scale | The QPRO questionnaire provides additional details on key attributes of hair and helps provide context to the SPRO response. | Weeks 12, 16, 20, and 24 | 
| Part A, Period 1: Percentage of patients achieving the restoration of regrowth | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | Weeks 4, 8, 12, 16, and 20 of re-treatment | 
| Part A, Period 1: Relative change in SALT scores from Part B Baseline | SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). | Weeks 4, 8, 12, 16, 20, and 24 of re-treatment | 
| Number of Participants with Adverse Events (AEs) | An adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention | 72 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CTP-543