Study to Evaluate the Safety and Efficacy of CTP-543 in Adults With Moderate to Severe Alopecia Areata

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata

This study will evaluate the safety and efficacy of CTP-543 on hair loss in adults with chronic, moderate to severe alopecia areata.

Study Overview

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

Detailed Description

This is a double-blind, randomized, placebo-controlled multi-center study consisting of 3 cohorts to assess the safety and efficacy of CTP-543. Each Cohort will be initiated sequentially in ascending dose order. Participants will be randomized to either an active dose of CTP-543 or placebo for a 24-week treatment period.

• Study Type: Interventional
• Enrollment (Actual): 149
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Irvine, California, United States, 92697
      • University of California, Irvine
    • Rancho Mirage, California, United States, 92270
      • Contour Dermatology & Cosmetic Surgery Center
    • Redwood City, California, United States, 94064
      • Stanford University School of Medicine
    • San Francisco, California, United States, 94118
      • Kaiser Permanente Northern California
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale School of Medicine
  • Florida
    • Boynton Beach, Florida, United States, 33472
      • Siperstein Dermatology Group
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Minnesota
    • Fridley, Minnesota, United States, 55432
      • Minnesota Clinical Study Center
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mt. Sinai
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27104
      • Wake Forest University Health Sciences
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Oregon
    • Portland, Oregon, United States, 97210
      • Northwest Dermatology
  • Texas
    • Houston, Texas, United States, 77056
      • Suzanne Bruce & Associates, PA

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 63 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: Sequential Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: CTP-543 4 mg BID; Participants will receive CTP-543 4 mg tablets, twice daily for up to 24 weeks.	Drug: CTP-543; Administered as tablets.
Experimental: Cohort 2: CTP-543 8 mg BID; Participants will receive CTP-543 8 mg tablets, twice daily for up to 24 weeks.	Drug: CTP-543; Administered as tablets.
Experimental: Cohort 3: CTP-543 12 mg BID; Participants will receive CTP-543 12 mg tablets, twice daily for up to 24 weeks.	Drug: CTP-543; Administered as tablets.
Placebo Comparator: Combined Placebo; Participants will receive CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3.	Drug: CTP-543 matching placebo; Administered as tablets.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline 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). Responders were defined as participants achieving at least a 50% relative reduction in SALT score from baseline at Week 24.	Week 24
Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE)	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. TEAE is defined as any adverse event that occurs after administration of the first dose of study drug.	From first dose of study drug up to safety follow up at Week 28

Collaborators and Investigators

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

Publications and helpful links

General Publications

• Cassella J, Hamilton C, von Hehn J, Braman V. JAK Inhibitor CTP-543 Achieves Primary Endpoint in Phase 2 Trial in Alopecia Areata. In: Proceedings from the 2019 American Academy of Dermatology Meeting; 01-05 March 2019; Washington, DC. Abstract 11291.
• Cassella J, Hamilton C, von Hehn J, Braman V. JAK Inhibitor CTP-543 Achieves Primary Endpoint in Phase 2 Trial in Alopecia Areata. In: Proceedings from the 11th World Congress Hair Research; 24-27 April 2019; Sitges, Barcelona.
• Cassella J, Hamilton C, von Hehn J, Braman V. CTP-543, an oral JAK inhibitor, achieves primary endpoint in Phase 2 randomized, placebo-controlled, dose ranging trial in patients with moderate to severe alopecia areata. In: Proceedings from the 28th EADV Congress; 09-13 October 2019, Madrid, Spain. D3T01.1: Late breaking news.

Study record dates

Study Major Dates

• Study Start (Actual): August 9, 2017
• Primary Completion (Actual): July 8, 2019
• Study Completion (Actual): July 8, 2019

Study Registration Dates

• First Submitted: April 28, 2017
• First Submitted That Met QC Criteria: May 1, 2017
• First Posted (Actual): May 2, 2017

Study Record Updates

• Last Update Posted (Actual): July 19, 2022
• Last Update Submitted That Met QC Criteria: June 27, 2022
• Last Verified: June 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: CP543.2001

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