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

• Sponsor: Concert Pharmaceuticals (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT04784533
• Collaborator: (none)
• Enrollment: 300
• Locations: 39
• Arms: 8
• Anticipated Duration (Months): 25.1
• Patients Per Site: 7.7
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Alopecia Areata	Drug: CTP-543; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 300 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Study to Evaluate Maintenance of Hair Regrowth Following Dose Reduction of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata
• Actual Study Start Date: Feb 26, 2021
• Anticipated Primary Completion Date: Oct 1, 2022
• Anticipated Study Completion Date: Apr 1, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Percentage of patients achieving loss of maintenance criteria defined by SALT following dose reduction [24 weeks]

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

2. Percentage of patients achieving loss of maintenance criteria defined by SALT following drug discontinuation [24 weeks]

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

3. Proportion of patients by dose group achieving restoration of regrowth [24 weeks]

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

Secondary Outcome Measures

1. Percentage of patients achieving loss of maintenance criteria following dose reduction [Weeks 4, 8, 12, 16, and 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).

2. Percentage of patients achieving loss of maintenance criteria following drug discontinuation [Weeks 4, 8, 12, 16, and 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).

3. Part A, Period 1: Percentage of responders (defined as "satisfied" or "very satisfied") on the Hair Satisfaction Patient Reported Outcome (SPRO) scale [Weeks 12, 16, 20, and 24]

   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.

4. Part A, Period 1: Percentage of patients achieving an absolute SALT score ≤20 [Weeks 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).

5. Part A, Period 1: Relative change in SALT scores from Baseline [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).

6. Part A, Period 1: Percentage of responders (defined as "much improved" or "very much improved") using the CGI-I [Weeks 12, 16, 20, and 24]

   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.

7. Part A, Period 1: Percentage of responders (defined as "much improved" or "very much improved") using the PGI-I [Weeks 12, 16, 20, and 24]

   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.

8. Part A, Period 1: Change from Baseline in the CGI-S [Weeks 12, 16, 20, and 24]

   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.

9. Part A, Period 1: Change from Baseline in the PGI-S [Weeks 12, 16, 20, and 24]

   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.

10. Part A, Period 1: Change from Baseline on the individual items of the Hair Quality Patient Reported Outcome (QPRO) scale [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.

11. Part A, Period 1: Percentage of patients achieving the restoration of regrowth [Weeks 4, 8, 12, 16, and 20 of re-treatment]

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

12. Part A, Period 1: Relative change in SALT scores from Part B Baseline [Weeks 4, 8, 12, 16, 20, and 24 of re-treatment]

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

13. Number of Participants with Adverse Events (AEs) [72 weeks]

    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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Total Skin and Beauty Dermatology Center, PC	Birmingham	Alabama	United States	35205
2	Center for Dermatology and Plastic Surgery/CCT Research	Scottsdale	Arizona	United States	85260
3	Kern Research, Inc.	Bakersfield	California	United States	93301
4	Hope Clinical Research	Canoga Park	California	United States	91303
5	California Dermatology & Clinical Research Institute	Encinitas	California	United States	92024
6	University of California, Irvine	Irvine	California	United States	92697
7	Quest Dermatology Research	Northridge	California	United States	91324
8	Kaiser Permanente	Oakland	California	United States	94611
9	Palmtree Clinical Research, Inc.	Palm Springs	California	United States	92262
10	Kaiser Permanente	San Francisco	California	United States	94118
11	Colorado Center for Dermatology and Skin Surgery	Centennial	Colorado	United States	80111
12	Colorado Medical Research Center, Inc.	Denver	Colorado	United States	80210
13	Yale University Church Street Research Unit	New Haven	Connecticut	United States	06519
14	Skin Care Research, LLC	Boca Raton	Florida	United States	33486
15	Skin Care Research, LLC	Hollywood	Florida	United States	33021
16	DeNova Research	Chicago	Illinois	United States	60611
17	The Indiana Clinical Trials Center, PC	Plainfield	Indiana	United States	46168
18	DS Research	Louisville	Kentucky	United States	40241
19	Michigan Center for Research Company, LLC	Clarkston	Michigan	United States	48346
20	University of Minnesota Department of Dermatology	Minneapolis	Minnesota	United States	55455
21	Minnesota Clinical Study Center	New Brighton	Minnesota	United States	55112
22	Skin Specialists, PC	Omaha	Nebraska	United States	68144
23	Skin Laser and Surgery Specialists of New Jersey	Hackensack	New Jersey	United States	07601
24	Darst Dermatology	Charlotte	North Carolina	United States	28277
25	Dermatology Specialists of Charlotte	Charlotte	North Carolina	United States	28277
26	North Carolina Dermatology Associates, PLLC	Raleigh	North Carolina	United States	27617
27	Bexley Dermatology Research	Bexley	Ohio	United States	43209
28	Remington Davis	Columbus	Ohio	United States	43215
29	Vital Prospects Clinical Research Institute, PC	Tulsa	Oklahoma	United States	74136
30	Northwest Dermatology	Portland	Oregon	United States	97210
31	Dermatology Associates of Plymouth Meeting	Plymouth Meeting	Pennsylvania	United States	19462
32	PEAK Research, LLC	Upper Saint Clair	Pennsylvania	United States	15241
33	Rhode Island Hospital	Providence	Rhode Island	United States	02903
34	Clinical Research Center of the Carolinas	Charleston	South Carolina	United States	29407
35	Dermatology Treatment and Research Center, PA	Dallas	Texas	United States	75230
36	Austin Institute for Clinical Research	Houston	Texas	United States	77056
37	Austin Institute for Clinical Research, Inc.	Pflugerville	Texas	United States	78660
38	Springville Dermatology/CCT Research	Springville	Utah	United States	84663
39	West End Dermatology Associates	Richmond	Virginia	United States	23233

Sponsors and Collaborators

• Concert Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Concert Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT04784533
• Other Study ID Numbers: CP543.2004
• First Posted: Mar 5, 2021
• Last Update Posted: Jul 27, 2022
• Last Verified: Jul 1, 2022

• 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
• Keywords provided by Concert Pharmaceuticals: CTP-543
• Additional relevant MeSH terms: Alopecia; Alopecia Areata

Study Results

No Results Posted as of Jul 27, 2022