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

A Study to Evaluate Maintenance of Hair Regrowth Following Dose Reduction of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata

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 Overview

• Status: Completed
• Conditions: Alopecia Areata
• Intervention / Treatment: Drug: CTP-543; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 317
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35205
      • Total Skin and Beauty Dermatology Center, PC
  • Arizona
    • Scottsdale, Arizona, United States, 85260
      • Center for Dermatology and Plastic Surgery/CCT Research
  • California
    • Bakersfield, California, United States, 93301
      • Kern Research, Inc.
    • Canoga Park, California, United States, 91303
      • HOPE Clinical Research
    • Encinitas, California, United States, 92024
      • California Dermatology & Clinical Research Institute
    • Irvine, California, United States, 92697
      • University of California, Irvine
    • Northridge, California, United States, 91324
      • Quest Dermatology Research
    • Oakland, California, United States, 94611
      • Kaiser Permanente
    • Palm Springs, California, United States, 92262
      • Palmtree Clinical Research, Inc.
    • San Francisco, California, United States, 94118
      • Kaiser Permanente
  • Colorado
    • Centennial, Colorado, United States, 80111
      • Colorado Center for Dermatology and Skin Surgery
    • Denver, Colorado, United States, 80210
      • Colorado Medical Research Center, Inc.
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University Church Street Research Unit
  • Florida
    • Boca Raton, Florida, United States, 33486
      • Skin Care Research, LLC
    • Hollywood, Florida, United States, 33021
      • Skin Care Research, LLC
  • Illinois
    • Chicago, Illinois, United States, 60611
      • DeNova Research
  • Indiana
    • Plainfield, Indiana, United States, 46168
      • The Indiana Clinical Trials Center, PC
  • Kentucky
    • Louisville, Kentucky, United States, 40241
      • DS Research
  • Michigan
    • Clarkston, Michigan, United States, 48346
      • Michigan Center for Research Company, LLC
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota Department of Dermatology
    • New Brighton, Minnesota, United States, 55112
      • Minnesota Clinical Study Center
  • Nebraska
    • Omaha, Nebraska, United States, 68144
      • Skin Specialists, PC
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Skin Laser and Surgery Specialists of New Jersey
  • North Carolina
    • Charlotte, North Carolina, United States, 28277
      • Darst Dermatology
    • Charlotte, North Carolina, United States, 28277
      • Dermatology Specialists of Charlotte
    • Raleigh, North Carolina, United States, 27617
      • North Carolina Dermatology Associates, PLLC
  • Ohio
    • Bexley, Ohio, United States, 43209
      • Bexley Dermatology Research
    • Columbus, Ohio, United States, 43215
      • Remington Davis
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Vital Prospects Clinical Research Institute, PC
  • Oregon
    • Portland, Oregon, United States, 97210
      • Northwest Dermatology
  • Pennsylvania
    • Plymouth Meeting, Pennsylvania, United States, 19462
      • Dermatology Associates of Plymouth Meeting
    • Upper Saint Clair, Pennsylvania, United States, 15241
      • PEAK Research, LLC
  • Rhode Island
    • Providence, Rhode Island, United States, 02903
      • Rhode Island Hospital
  • South Carolina
    • Charleston, South Carolina, United States, 29407
      • Clinical Research Center of the Carolinas
  • Texas
    • Dallas, Texas, United States, 75230
      • Dermatology Treatment and Research Center, PA
    • Houston, Texas, United States, 77056
      • Austin Institute for Clinical Research
    • Pflugerville, Texas, United States, 78660
      • Austin Institute for Clinical Research, Inc.
  • Utah
    • Springville, Utah, United States, 84663
      • Springville Dermatology/CCT Research
  • Virginia
    • Richmond, Virginia, United States, 23233
      • West End Dermatology Associates

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

10

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A: Period 1 - CTP-543 8 mg BID; Participants received CTP-543 8 milligrams (mg) tablets, orally, twice daily (BID) for up to 24 weeks.	Drug: CTP-543; Oral dosing
Experimental: Part A: Period 1 - CTP-543 12 mg BID; Participants received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.	Drug: CTP-543; Oral dosing
Experimental: Part A: Period 2 - CTP-543 8 mg BID to 4 mg BID; Participants who received CTP-543 8 mg during Period 1 of Part A and achieved an absolute severity of alopecia tool (SALT) score of ≤20 at Week 24, received CTP-543 4 mg tablets, orally, BID, for up to 24 weeks.	Drug: CTP-543; Oral dosing
Experimental: Part A: Period 2 - CTP-543 12 mg BID to 8 mg BID; Participants who received CTP-543 12 mg during Period 1 of Part A and achieved an absolute SALT score of ≤20 at Week 24, received CTP-543 8 mg tablets, orally, BID, for up to 24 weeks.	Drug: CTP-543; Oral dosing
Placebo Comparator: Part A: Period 2 - CTP-543 8 mg BID to Placebo; Participants who received CTP-543 8 mg during Period 1 of Part A and achieved an absolute SALT score of ≤20 at Week 24, received CTP-543 matched placebo tablets, orally, BID, for up to 24 weeks.	Drug: Placebo; Oral dosing
Placebo Comparator: Part A: Period 2 - CTP-543 12 mg BID to Placebo; Participants who received CTP-543 12 mg during Period 1 of Part A and achieved an absolute SALT score of ≤20 at Week 24, received CTP-543 matched placebo tablets, orally, BID, for up to 24 weeks.	Drug: Placebo; Oral dosing
Experimental: Part B: CTP-543 8 mg BID to 4 mg BID to 8 mg BID; Participants who received CTP-543 8 mg during Period 1, followed by CTP-543 4 mg during Period 2 of Part A and met the criteria for loss of regrowth maintenance (LOM) (absolute SALT score of > 20), received re-treatment with CTP-543 8 mg tablets, orally, BID, for up to 24 weeks.	Drug: CTP-543; Oral dosing
Experimental: Part B: CTP-543 12 mg BID to 8 mg BID to 12 mg BID; Participants who received CTP-543 12 mg during Period 1, followed by CTP-543 8 mg during Period 2 of Part A and met the criteria for LOM (absolute SALT score of > 20), received re-treatment with CTP-543 12 mg tablets, orally, BID, for up to 24 weeks.	Drug: CTP-543; Oral dosing
Experimental: Part B: CTP-543 8 mg BID to Placebo to 8 mg BID; Participants who received CTP-543 8 mg during Period 1, followed by CTP-543 matched placebo during Period 2 of Part A and met the criteria for LOM (absolute SALT score of > 20), received re-treatment with CTP-543 8 mg tablets, orally, BID, for up to 24 weeks.	Drug: CTP-543; Oral dosing
Experimental: Part B: CTP-543 12 mg BID to Placebo to 12 mg BID; Participants who received CTP-543 12 mg during Period 1, followed by CTP-543 matched placebo during Period 2 of Part A and met the criteria for LOM (absolute SALT score of > 20), received re-treatment with CTP-543 12 mg tablets, orally, BID, for up to 24 weeks.	Drug: CTP-543; Oral dosing

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	Week 24 of re-treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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.	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.	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'.	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).	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.	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).	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'.	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.	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.	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.	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	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 during re-treatment.	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	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).	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.	From first dose of study drug up to last follow up visit (Week 76)

Collaborators and Investigators

• Sponsor: Concert Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 26, 2021
• Primary Completion (Actual): May 16, 2023
• Study Completion (Actual): May 16, 2023

Study Registration Dates

• First Submitted: March 3, 2021
• First Submitted That Met QC Criteria: March 3, 2021
• First Posted (Actual): March 5, 2021

Study Record Updates

• Last Update Posted (Estimated): October 9, 2024
• Last Update Submitted That Met QC Criteria: October 3, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: CTP-543
• Additional Relevant MeSH Terms: Skin Diseases; Pathological Conditions, Anatomical; Hypotrichosis; Hair Diseases; Alopecia; Alopecia Areata
• Other Study ID Numbers: CP543.2004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No