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

Concert Pharmaceuticals317 enrolled

Overview

This study is designed 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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

317

Conditions

Alopecia Areata

Interventions

CTP-543DRUG

Oral dosing

PlaceboDRUG

Oral dosing

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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 severity of alopecia tool (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.

Locations (39)

Outcomes

Primary Outcomes

Part A, Period 2: Percentage of Participants Achieving Loss of Regrowth Maintenance (LOM) Criteria Defined by Severity of Alopecia Tool (SALT) Score > 20 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). LOM is defined as an absolute SALT score \>20. The percentage of participants achieving LOM criteria (SALT \>20) was compared to the percentage of participants maintaining treatment success (SALT ≤ 20) for each of the following dose reduction conditions. Due to the variable time on study for each participant within Part A Period 2, the primary analysis visit was the end of Part A Period 2, where the last observed non-missing SALT value was selected for each participant.

Time frame: From Week 24 to Week 48

Part A, Period 2: Percentage of Participants Achieving LOM Criteria Defined by SALT Score > 20 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). LOM is defined as an absolute SALT score \>20. The percentage of participants achieving LOM criteria (SALT \>20) was compared to the percentage of participants maintaining treatment success (SALT ≤ 20) for each of the following dose discontinuation conditions. Due to the variable time on study for each participant within Part A Period 2, the primary analysis visit was at the end of Part A Period 2, where the last observed non-missing SALT value was selected for each participant.

Time frame: From Week 24 to Week 48

Part B: Percentage of Participants by Dose Group Who Achieved Restoration of Regrowth (ROR) at Week 24

SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). ROR is defined as the participant's attainment of an absolute SALT score of ≤20 at Week 24 of re-treatment.

Time frame: Week 24 of re-treatment

Secondary Outcomes

Part A, Period 2: Percentage of Participants Who Achieved LOM Criteria Defined by SALT Score > 20 Following Dose Reduction Conditions at Weeks 28, 32, 36, 40, 44, and 48

Data from ClinicalTrials.gov

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Total Skin and Beauty Dermatology Center, PC

Birmingham, Alabama, United States

Center for Dermatology and Plastic Surgery/CCT Research

Scottsdale, Arizona, United States

Kern Research, Inc.

Bakersfield, California, United States

Hope Clinical Research

Canoga Park, California, United States

California Dermatology & Clinical Research Institute

Encinitas, California, United States

University of California, Irvine

Irvine, California, United States

Quest Dermatology Research

Northridge, California, United States

Kaiser Permanente

Oakland, California, United States

Palmtree Clinical Research, Inc.

Palm Springs, California, United States

Kaiser Permanente

San Francisco, California, United States

...and 29 more locations

SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). LOM is defined as an absolute SALT score \>20.

Time frame: Weeks 28, 32, 36, 40, 44, and 48

Part A, Period 2: Percentage of Participants Who Achieved LOM Criteria Defined by SALT > 20 Following Drug Discontinuation Conditions at Weeks 28, 32, 36, 40, 44, and 48

SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss). LOM is defined as an absolute SALT score \>20.

Time frame: Weeks 28, 32, 36, 40, 44, and 48

Part A, Period 1: Percentage of Responders as Assessed on the Hair Satisfaction Patient Reported Outcome (SPRO) Scale at Weeks 12, 16, 20, and 24

SPRO is a questionnaire answered by the participant and designed to measure how satisfied alopecia areata participants are with their hair at the time of the assessment. The responses range from 1 to 5: 1= very satisfied, 2= satisfied, 3= neither satisfied nor dissatisfied, 4= dissatisfied, and 5= very dissatisfied. SPRO responder is defined as a post-baseline response of 'very satisfied' or 'satisfied'.

Time frame: Weeks 12, 16, 20, and 24

Part A, Period 1: Percentage of Participants Who Achieved an Absolute SALT Score ≤20 at Weeks 4, 8, 12, 16, 20, and 24

SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss).

Time frame: Weeks 4, 8, 12, 16, 20, and 24

Part A, Period 1: Relative Change in SALT Scores From Baseline at Weeks 4, 8, 12, 16, 20, and 24

SALT is a quantitative assessment of scalp hair loss with scores ranging in severity from 0 (no scalp hair loss) to a maximum of 100 (complete scalp hair loss). Relative change (percent change) to baseline is calculated as: 100 x (\[post-baseline SALT score - baseline SALT score\]/baseline SALT score). Negative change indicates no hair loss.

Time frame: Baseline, Weeks 4, 8, 12, 16, 20, and 24

Part A, Period 1: Percentage of Responders Assessed Using the Clinician Global Impression of Improvement (CGI-I) at Weeks 12, 16, 20 and 24

The CGI-I is a questionnaire that asks the clinician to evaluate the improvement or worsening of the participant's alopecia areata as compared to the start of the study on a 7-point scale. Responses range from 1 (very much worse) to 7 (very much improved). Responders were defined as participants with responses of 6 (much improved) or 7 (very much improved).

Time frame: Weeks 12, 16, 20, and 24

Part A, Period 1: Percentage of Responders Assessed Using the Patient Global Impression of Improvement (PGI-I) at Weeks 12, 16, 20, and 24

The PGI-I is a self-administered questionnaire that asks the participant to evaluate the improvement or worsening of their alopecia areata as compared to the start of the study on a 7-point scale. Responses range from 1 (very much worse) to 7 (very much improved). Responders were defined as participants with responses of 6 (much improved) or 7 (very much improved). PGI responder is a response of 'very much improved' or 'much improved'.

Time frame: Weeks 12, 16, 20, and 24

Part A, Period 1: Change in the Clinician Global Impression of Severity (CGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24

The CGI-S is a questionnaire that asks the clinician to evaluate the symptom severity of the participant's alopecia areata at the time of assessment. The symptom severity was rated on a scale ranging from 1 to 7, where 1=normal, no hair loss; 2=borderline hair loss; 3=mild hair loss; 4=moderate hair loss; 5=marked hair loss; 6=severe hair loss; 7=among the most extreme hair loss. Higher scores indicate more hair loss. A negative change from baseline indicates less hair loss.

Time frame: Baseline, Weeks 12, 16, 20, and 24

Part A, Period 1: Change in the Patient Global Impression of Severity (PGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24

The PGI-S is a self-administered questionnaire that asks the participant to evaluate the symptom severity of their alopecia areata at the time of assessment. Symptom severity was rated on a scale ranging from 1 to 7, where 1=normal, no hair loss; 2=borderline hair loss; 3=mild hair loss; 4=moderate hair loss; 5=marked hair loss; 6=severe hair loss; 7=among the most extreme hair loss. Higher scores indicate more hair loss. A negative change from baseline indicates less hair loss.

Time frame: Baseline, Weeks 12, 16, 20, and 24

Part A, Period 1: Change in the Individual Items of the Hair Quality Patient Reported Outcome (QPRO) Scale From Baseline at Weeks 12, 16, 20, and 24

The QPRO questionnaire provides additional details on key attributes of hair and helps provide context to the SPRO response. The individual items of QPRO are: Satisfied thickness hair coverage; Satisfied evenness hair coverage; How satisfied with your eyebrows; How satisfied with your eyelashes, scored on a scale ranging from 1 to 5 where 1=very satisfied, 2=satisfied, 3=neither satisfied nor dissatisfied, 4=dissatisfied, 5=very dissatisfied. Higher scores indicates the greater dissatisfaction on hair quality. A negative change from baseline indicate the greater satisfaction on hair quality.

Time frame: Baseline, Weeks 12, 16, 20, and 24

Part B: Percentage of Participants Who Achieved Restoration of Regrowth Criteria at Weeks 4, 8, 12, 16, 20, and 24

Time frame: Weeks 4, 8, 12, 16, 20, and 24 of re-treatment

Part B: Relative Change in SALT Scores From Part B Baseline at Weeks 4, 8, 12, 16, 20, and 24

Time frame: Baseline, Weeks 4, 8, 12, 16, 20, and 24 of re-treatment

Number of Participants Experiencing at Least One Treatment-Emergent Adverse Events (TEAEs)

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. TEAEs were defined as any adverse event that occurs after administration of the first dose of study drug in each Part/Period (ie, on or after the day of the first dose in each Part/Period). TEAEs included both serious and non-serious TEAEs.

Time frame: From first dose of study drug up to last follow up visit (Week 76)