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Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA1)

Phase 3
Completed
Conditions
Alopecia Areata
Interventions
Drug: CTP-543 matching placebo
Registration Number
NCT04518995
Lead Sponsor
Concert Pharmaceuticals
Brief Summary

This study evaluates the safety and effectiveness of an investigational study drug (called CTP-543) in adults (18 years and older) who have 50% or greater scalp hair loss.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
706
Inclusion Criteria
  • Clinical presentation compatible with 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.
  • 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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboCTP-543 matching placeboParticipants received CTP-543 matched placebo tablets, orally, twice daily (BID) for up to 24 weeks.
CTP-543 8 mg BIDCTP-543Participants received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.
CTP-543 12 mg BIDCTP-543Participants received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants Achieving an Absolute Severity of Alopecia Tool (SALT) Score of ≤20 at Week 24Week 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).

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants Achieving an Absolute SALT Score of ≤20 at Weeks 4, 8, 12, 16, and 20Weeks 4, 8, 12, 16, and 20

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 in SALT Scores From Baseline at Weeks 4, 8, 12, 16, 20, and 24Baseline, 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).

Percentage of Responders Assessed Using the Patient Global Impression of Improvement (PGI-I) at Weeks 12, 16, 20, and 24Weeks 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).

Change in the Clinician Global Impression of Severity (CGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24Baseline, 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.

Percentage of Responders on the Hair Satisfaction Patient Reported Outcome (SPRO) Scale at Weeks 12, 16, 20, and 24Weeks 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. Responders were defined as participants with responses of "satisfied" or "very satisfied".

Percentage of Responders Assessed Using the Clinician Global Impression of Improvement (CGI-I) at Weeks 12, 16, 20, and 24Weeks 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).

Change in the Brigham Eyebrow Tool for Alopecia (BETA) Scores From Baseline at Weeks 12 and 24Baseline, Weeks 12, and 24

BETA is a clinician-rated scale that assesses the total eyebrow hair present. The BETA score is calculated based on hair density and surface area of each individual eyebrow of the participant, ranging from 0 to 3, where 0 = no eyebrow, 1 = minimal eyebrow, 2 = moderate eyebrow, 3 = normal eyebrow. The BETA score is the sum of the right and left eyebrow scores, ranging from 0 to 6. Higher scores indicate less hair loss of eyebrows. A positive change from baseline indicates less hair loss of eyebrows.

Change in the Patient Global Impression of Severity (PGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24Baseline, 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.

Percentage of Participants Achieving at Least a 75% and 90% Relative Reduction in SALT Score From Baseline at Weeks 12 and 24Baseline, Weeks 12, 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). Percentage of participants achieving at least a 75% and 90% relative reduction in SALT score from baseline at Weeks 12 and 24 are reported.

Percentage of Participants Achieving a ≥2-point Change From Baseline in the SPRO Scale at Weeks 12, 16, 20, and 24Weeks 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.

Percentage of Participants Achieving an Absolute SALT Score of ≤10 at Week 24Week 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).

Change in the Brigham Eyelash Tool for Alopecia (BELA) Scores From Baseline at Weeks 12 and 24Baseline, Weeks 12, and 24

BELA is a clinician-rated scale that assesses the total eyelash hair present. The BELA is calculated based on distribution and grade values, ranging from 0 (no eyelashes) to 3 (full eyelashes). The BELA score is the sum of the individual scores for the left and right eyes, ranging from 0 to 6. Higher scores indicate less hair loss of eyelashes. A positive change from baseline indicates less hair loss of eyelashes.

Change in the SPRO Scale From Baseline at Weeks 12, 16, 20, and 24Baseline, 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. Higher scores indicate the greater hair dissatisfaction. A negative change from baseline indicate the greater hair satisfaction.

Change in the Individual Items of the Hair Quality Patient Reported Outcome (QPRO) Scale From Baseline at Weeks 12, 16, 20, and 24Baseline, 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 indicate the greater dissatisfaction on hair quality. A negative change from baseline indicate the greater satisfaction on hair quality.

Change in the Anxiety and Depression Scale Scores of the Hospital Anxiety and Depression Scale (HADS) From Baseline at Week 24Baseline and Week 24

HADS is a questionnaire designed to assess anxiety and depression symptoms which is completed by participants. The questionnaire is comprised of two separate scales with a total of 14 items: A 7-item scale related to anxiety and 7-item scale related to depression. Each item within both scales is scored using a 4-point scale, ranging from 0 to 3 and the total scores in each scale can range from 0 to 21. Separate scores were created for anxiety and depression. A score between 0-7 is considered normal, 8-10 is mild, 11-14 is moderate, and \>14 is severe anxiety or depression. Higher scores indicate greater severity. A negative change from baseline indicates less severity.

Trial Locations

Locations (69)

Dermatology Treatment and Research Center

🇺🇸

Dallas, Texas, United States

Research Toronto

🇨🇦

Toronto, Ontario, Canada

West End Dermatology Associates

🇺🇸

Richmond, Virginia, United States

UC Irvine Dermatology Clinical Research Center

🇺🇸

Irvine, California, United States

Darst Dermatology

🇺🇸

Charlotte, North Carolina, United States

H. U. Ramón y Cajal

🇪🇸

Madrid, Spain

Quest Dermatology Research

🇺🇸

Northridge, California, United States

Kern Research, Inc.

🇺🇸

Bakersfield, California, United States

California Dermatology & Clinical Research Institute

🇺🇸

Encinitas, California, United States

Colorado Center for Dermatology and Skin Surgery

🇺🇸

Centennial, Colorado, United States

Lenus Research & Medical Group, LLC

🇺🇸

Sweetwater, Florida, United States

The Indiana Clinical Trials Center, PC

🇺🇸

Plainfield, Indiana, United States

Qualmedica Research LLC

🇺🇸

Owensboro, Kentucky, United States

Clarkston Skin Research

🇺🇸

Clarkston, Michigan, United States

Minnesota Clinical Study Center

🇺🇸

Fridley, Minnesota, United States

Skin Laser and Surgery Specialists of NJ

🇺🇸

Hackensack, New Jersey, United States

Sadick Research Group

🇺🇸

New York, New York, United States

Dermatology Specialists of Charlotte

🇺🇸

Charlotte, North Carolina, United States

Vital Prospects Clinical Research Institute, P.C.

🇺🇸

Tulsa, Oklahoma, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Bexley Dermatology Research

🇺🇸

Bexley, Ohio, United States

Dermatology Associates Of Plymouth Meeting

🇺🇸

Plymouth Meeting, Pennsylvania, United States

PEAK Research

🇺🇸

Upper Saint Clair, Pennsylvania, United States

Palmetto Clinical Trial Services

🇺🇸

Fountain Inn, South Carolina, United States

Clinical Investigation Specialists, Inc.

🇺🇸

Kenosha, Wisconsin, United States

Austin Institute for Clinical Research, Inc.

🇺🇸

Pflugerville, Texas, United States

Bellaire Dermatology Associates

🇺🇸

Bellaire, Texas, United States

Dermatology Research Institute

🇨🇦

Calgary, Alberta, Canada

My Clinic

🇵🇱

Warsaw, Poland

Kirk Barber Research

🇨🇦

Calgary, Alberta, Canada

Enverus Medical Research

🇨🇦

Surrey, British Columbia, Canada

Wiseman Dermatology Research Inc.

🇨🇦

Winnipeg, Manitoba, Canada

Guelph Dermatology Research

🇨🇦

Guelph, Ontario, Canada

SimcoDerm Medical and Surgical Dermatology Center

🇨🇦

Barrie, Ontario, Canada

Dr Wei Jing Loo Medicine Professional Corporation

🇨🇦

London, Ontario, Canada

Lynderm Research Inc.

🇨🇦

Markham, Ontario, Canada

Hôpital Hotel Dieu - Chu de Nantes

🇫🇷

Nantes, PAYS DE LA Loire, France

Innovaderm Research Inc. - Clinic

🇨🇦

Montreal, Quebec, Canada

The Centre for Clinical Trials

🇨🇦

Oakville, Ontario, Canada

K. Papp Clinical Research Inc.

🇨🇦

Waterloo, Ontario, Canada

Dr. S. K. Siddha Medicine Professional Corporation

🇨🇦

Newmarket, Ontario, Canada

JRB Research Inc.

🇨🇦

Ottawa, Ontario, Canada

Hôpital Saint-Louis - Gh de La Rochelle

🇫🇷

La Rochelle, Nouvelle-aquitaine, France

Vita Longa Sp.Zo.O

🇵🇱

Katowice, Poland

Hôpital La Timone-Dermatologie

🇫🇷

Marseille, France

Hôpital Saint Louis, Centre de Santé Sabouraud

🇫🇷

Paris, France

SGD S.C.

🇵🇱

Krakow, Poland

Royalderm Agnieszka Nawrocka

🇵🇱

Warsaw, Poland

Hospital Clínico Y Provincial de Barcelona

🇪🇸

Barcelona, Spain

Hôpital Saint-André - Chu de Bordeaux

🇫🇷

Bordeaux, Nouvelle-aquitaine, France

Kaiser Permanente

🇺🇸

San Francisco, California, United States

Kaiser Permanente South Sacramento

🇺🇸

Sacramento, California, United States

Total Skin and Beauty Dermatology Center

🇺🇸

Birmingham, Alabama, United States

Northwestern Memorial Hospital, Department of Dermatology

🇺🇸

Chicago, Illinois, United States

Colorado Medical Research Center

🇺🇸

Denver, Colorado, United States

Yale University, Church Street Research Unit

🇺🇸

New Haven, Connecticut, United States

Clinical Research Trials of Florida, Inc.

🇺🇸

Tampa, Florida, United States

Forward Clinical Trials Inc.

🇺🇸

Tampa, Florida, United States

Denova Research

🇺🇸

Chicago, Illinois, United States

Dawes Fretzin Clinical Research Group, LLC

🇺🇸

Indianapolis, Indiana, United States

DS Research

🇺🇸

Louisville, Kentucky, United States

Massachusetts General Hospital - Clinic

🇺🇸

Boston, Massachusetts, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

University of Minnesota Department of Dermatology

🇺🇸

Minneapolis, Minnesota, United States

Wake Forest University Health Sciences, Department of Dermatology

🇺🇸

Winston-Salem, North Carolina, United States

Northwest Dermatology Institute

🇺🇸

Portland, Oregon, United States

Clinical Research Center of the Carolinas

🇺🇸

Charleston, South Carolina, United States

Dermatology Clinical Research Center of San Antonio

🇺🇸

San Antonio, Texas, United States

Rhode Island Hospital

🇺🇸

Providence, Rhode Island, United States

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