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

NCT04784533 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CP543.2004
• Study Type: interventional
• Target: 317
• Locations: 1 country
• Sites: 39

Brief Summary

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.

Conditions

Alopecia Areata

Keywords

CTP-543

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (13)

• 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

  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

  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

  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

  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

  Baseline, Weeks 4, 8, 12, 16, 20, and 24

Study Arms (10)

Part A: Period 1 - CTP-543 8 mg BID

EXPERIMENTAL

Participants received CTP-543 8 milligrams (mg) tablets, orally, twice daily (BID) for up to 24 weeks.

Drug: CTP-543

Part A: Period 1 - CTP-543 12 mg BID

EXPERIMENTAL

Participants received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.

Drug: CTP-543

Part A: Period 2 - CTP-543 8 mg BID to 4 mg BID

EXPERIMENTAL

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

Part A: Period 2 - CTP-543 12 mg BID to 8 mg BID

EXPERIMENTAL

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

Part A: Period 2 - CTP-543 8 mg BID to Placebo

PLACEBO COMPARATOR

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

Part A: Period 2 - CTP-543 12 mg BID to Placebo

PLACEBO COMPARATOR

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

Part B: CTP-543 8 mg BID to 4 mg BID to 8 mg BID

EXPERIMENTAL

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

Part B: CTP-543 12 mg BID to 8 mg BID to 12 mg BID

EXPERIMENTAL

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

Part B: CTP-543 8 mg BID to Placebo to 8 mg BID

EXPERIMENTAL

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

Part B: CTP-543 12 mg BID to Placebo to 12 mg BID

EXPERIMENTAL

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

Interventions

CTP-543 DRUG

Oral dosing

Part A: Period 1 - CTP-543 12 mg BID; Part A: Period 1 - CTP-543 8 mg BID; Part A: Period 2 - CTP-543 12 mg BID to 8 mg BID; Part A: Period 2 - CTP-543 8 mg BID to 4 mg BID; Part B: CTP-543 12 mg BID to 8 mg BID to 12 mg BID; Part B: CTP-543 12 mg BID to Placebo to 12 mg BID; Part B: CTP-543 8 mg BID to 4 mg BID to 8 mg BID; Part B: CTP-543 8 mg BID to Placebo to 8 mg BID

Placebo DRUG

Oral dosing

Part A: Period 2 - CTP-543 12 mg BID to Placebo; Part A: Period 2 - CTP-543 8 mg BID to Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Concert Pharmaceuticals: lead

Study Sites (39)

Total Skin and Beauty Dermatology Center, PC

Birmingham, Alabama, 35205, United States

Location

Center for Dermatology and Plastic Surgery/CCT Research

Scottsdale, Arizona, 85260, United States

Location

Kern Research, Inc.

Bakersfield, California, 93301, United States

Location

Hope Clinical Research

Canoga Park, California, 91303, United States

Location

California Dermatology & Clinical Research Institute

Encinitas, California, 92024, United States

Location

University of California, Irvine

Irvine, California, 92697, United States

Location

Quest Dermatology Research

Northridge, California, 91324, United States

Location

Kaiser Permanente

Oakland, California, 94611, United States

Location

Palmtree Clinical Research, Inc.

Palm Springs, California, 92262, United States

Location

Kaiser Permanente

San Francisco, California, 94118, United States

Location

Colorado Center for Dermatology and Skin Surgery

Centennial, Colorado, 80111, United States

Location

Colorado Medical Research Center, Inc.

Denver, Colorado, 80210, United States

Location

Yale University Church Street Research Unit

New Haven, Connecticut, 06519, United States

Location

Skin Care Research, LLC

Boca Raton, Florida, 33486, United States

Location

Skin Care Research, LLC

Hollywood, Florida, 33021, United States

Location

DeNova Research

Chicago, Illinois, 60611, United States

Location

The Indiana Clinical Trials Center, PC

Plainfield, Indiana, 46168, United States

Location

DS Research

Louisville, Kentucky, 40241, United States

Location

Michigan Center for Research Company, LLC

Clarkston, Michigan, 48346, United States

Location

University of Minnesota Department of Dermatology

Minneapolis, Minnesota, 55455, United States

Location

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

Location

Skin Specialists, PC

Omaha, Nebraska, 68144, United States

Location

Skin Laser and Surgery Specialists of New Jersey

Hackensack, New Jersey, 07601, United States

Location

Darst Dermatology

Charlotte, North Carolina, 28277, United States

Location

Dermatology Specialists of Charlotte

Charlotte, North Carolina, 28277, United States

Location

North Carolina Dermatology Associates, PLLC

Raleigh, North Carolina, 27617, United States

Location

Bexley Dermatology Research

Bexley, Ohio, 43209, United States

Location

Remington Davis

Columbus, Ohio, 43215, United States

Location

Vital Prospects Clinical Research Institute, PC

Tulsa, Oklahoma, 74136, United States

Location

Northwest Dermatology

Portland, Oregon, 97210, United States

Location

Dermatology Associates of Plymouth Meeting

Plymouth Meeting, Pennsylvania, 19462, United States

Location

PEAK Research, LLC

Upper Saint Clair, Pennsylvania, 15241, United States

Location

Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Clinical Research Center of the Carolinas

Charleston, South Carolina, 29407, United States

Location

Dermatology Treatment and Research Center, PA

Dallas, Texas, 75230, United States

Location

Austin Institute for Clinical Research

Houston, Texas, 77056, United States

Location

Austin Institute for Clinical Research, Inc.

Pflugerville, Texas, 78660, United States

Location

Springville Dermatology/CCT Research

Springville, Utah, 84663, United States

Location

West End Dermatology Associates

Richmond, Virginia, 23233, United States

Location

MeSH Terms

Conditions

Alopecia Areata

Condition Hierarchy (Ancestors)

Alopecia; Hypotrichosis; Hair Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Colleen E. Hamilton
• Organization: Concert Pharmaceuticals, Inc.

AAclinicaltrial_inquiries@concertpharma.com

781-860-0045

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2021
• First Posted: March 5, 2021
• Study Start: February 26, 2021
• Primary Completion: May 16, 2023
• Study Completion: May 16, 2023
• Last Updated: October 9, 2024
• Results First Posted: October 9, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (39)