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

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

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.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 57
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Irvine, California, United States, 92697
      • Investigative Site
    • San Francisco, California, United States, 94118
      • Investigative Site
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Investigative Site
  • Florida
    • Boynton Beach, Florida, United States, 33472
      • Investigative Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Investigative Site
    • Boston, Massachusetts, United States, 02114
      • Investigative Site
  • Minnesota
    • Fridley, Minnesota, United States, 55432
      • Investigative Site
  • New Hampshire
    • Portsmouth, New Hampshire, United States, 03801
      • Investigative Site
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27104
      • Investigative Site
  • Oregon
    • Portland, Oregon, United States, 97210
      • Investigative Site

Participation Criteria

Eligibility Criteria

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

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CTP-543 8 mg BID; 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; CTP-543 administered as 8 mg tablet.; Drug: CTP-543 Matching Placebo; Administered as tablets to aid treatment masking.
Experimental: CTP-543 16 mg QD; 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; CTP-543 administered as 8 mg tablet.; Drug: CTP-543 Matching Placebo; Administered as tablets to aid treatment masking.

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Adverse Events as a Measure of Safety	24 Weeks

Collaborators and Investigators

• Sponsor: Concert Pharmaceuticals
• Investigators: Study Director: Colleen E Hamilton, MS, Concert Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 21, 2019
• Primary Completion (Actual): November 21, 2019
• Study Completion (Actual): November 21, 2019

Study Registration Dates

• First Submitted: January 18, 2019
• First Submitted That Met QC Criteria: January 18, 2019
• First Posted (Actual): January 22, 2019

Study Record Updates

• Last Update Posted (Estimate): December 9, 2022
• Last Update Submitted That Met QC Criteria: November 15, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Pathological Conditions, Anatomical; Hypotrichosis; Hair Diseases; Alopecia; Alopecia Areata
• Other Study ID Numbers: CP543.2002

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