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

Alopecia Areata Clinical Trial

— THRIVE-AA1  

Official title:

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata (THRIVE-AA1)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04518995
• Other study ID #: CP543.3001
• Secondary ID: 2020-002704-40
• Status: Completed
• Phase: Phase 3
• Start date: November 23, 2020
• Est. completion date: April 19, 2022

Study information

• Verified date: April 2023
• Source: Concert Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 706
• Est. completion date: April 19, 2022
• Est. primary completion date: April 19, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Alopecia
• Alopecia Areata

Intervention

Drug:
• CTP-543 matching placebo
Administered as tablets.
• CTP-543
Administered as tablets.
• CTP-543
Administered as tablets.

Locations

Country	Name	City	State
Canada	SimcoDerm Medical and Surgical Dermatology Center	Barrie	Ontario
Canada	Dermatology Research Institute	Calgary	Alberta
Canada	Kirk Barber Research	Calgary	Alberta
Canada	Guelph Dermatology Research	Guelph	Ontario
Canada	Dr Wei Jing Loo Medicine Professional Corporation	London	Ontario
Canada	Lynderm Research Inc.	Markham	Ontario
Canada	Innovaderm Research Inc. - Clinic	Montreal	Quebec
Canada	Dr. S. K. Siddha Medicine Professional Corporation	Newmarket	Ontario
Canada	The Centre for Clinical Trials	Oakville	Ontario
Canada	JRB Research Inc.	Ottawa	Ontario
Canada	Enverus Medical Research	Surrey	British Columbia
Canada	Research Toronto	Toronto	Ontario
Canada	K. Papp Clinical Research Inc.	Waterloo	Ontario
Canada	Wiseman Dermatology Research Inc.	Winnipeg	Manitoba
France	Hôpital Saint-André - Chu de Bordeaux	Bordeaux	Nouvelle-aquitaine
France	Hôpital Saint-Louis - Gh de La Rochelle	La Rochelle	Nouvelle-aquitaine
France	Hôpital La Timone-Dermatologie	Marseille
France	Hôpital Hotel Dieu - Chu de Nantes	Nantes	PAYS DE LA Loire
France	Hôpital Saint Louis, Centre de Santé Sabouraud	Paris
Poland	Vita Longa Sp.Zo.O	Katowice
Poland	SGD S.C.	Krakow
Poland	My Clinic	Warsaw
Poland	Royalderm Agnieszka Nawrocka	Warsaw
Spain	Hospital Clínico Y Provincial de Barcelona	Barcelona
Spain	H. U. Ramón y Cajal	Madrid
United States	Kern Research, Inc.	Bakersfield	California
United States	Bellaire Dermatology Associates	Bellaire	Texas
United States	Bexley Dermatology Research	Bexley	Ohio
United States	Total Skin and Beauty Dermatology Center	Birmingham	Alabama
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital - Clinic	Boston	Massachusetts
United States	Colorado Center for Dermatology and Skin Surgery	Centennial	Colorado
United States	Clinical Research Center of the Carolinas	Charleston	South Carolina
United States	Darst Dermatology	Charlotte	North Carolina
United States	Dermatology Specialists of Charlotte	Charlotte	North Carolina
United States	Denova Research	Chicago	Illinois
United States	Northwestern Memorial Hospital, Department of Dermatology	Chicago	Illinois
United States	Clarkston Skin Research	Clarkston	Michigan
United States	Dermatology Treatment and Research Center	Dallas	Texas
United States	Colorado Medical Research Center	Denver	Colorado
United States	California Dermatology & Clinical Research Institute	Encinitas	California
United States	Palmetto Clinical Trial Services	Fountain Inn	South Carolina
United States	Minnesota Clinical Study Center	Fridley	Minnesota
United States	Skin Laser and Surgery Specialists of NJ	Hackensack	New Jersey
United States	Austin Institute for Clinical Research, Inc.	Houston	Texas
United States	Dawes Fretzin Clinical Research Group, LLC	Indianapolis	Indiana
United States	UC Irvine Dermatology Clinical Research Center	Irvine	California
United States	Clinical Investigation Specialists, Inc.	Kenosha	Wisconsin
United States	DS Research	Louisville	Kentucky
United States	University of Minnesota Department of Dermatology	Minneapolis	Minnesota
United States	DS Research	New Albany	Indiana
United States	Yale University, Church Street Research Unit	New Haven	Connecticut
United States	Sadick Research Group	New York	New York
United States	Quest Dermatology Research	Northridge	California
United States	Kaiser Permanente	Oakland	California
United States	Qualmedica Research LLC	Owensboro	Kentucky
United States	Austin Institute for Clinical Research, Inc.	Pflugerville	Texas
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	The Indiana Clinical Trials Center, PC	Plainfield	Indiana
United States	Dermatology Associates Of Plymouth Meeting	Plymouth Meeting	Pennsylvania
United States	Northwest Dermatology Institute	Portland	Oregon
United States	Rhode Island Hospital	Providence	Rhode Island
United States	West End Dermatology Associates	Richmond	Virginia
United States	Kaiser Permanente South Sacramento	Sacramento	California
United States	Dermatology Clinical Research Center of San Antonio	San Antonio	Texas
United States	Kaiser Permanente	San Francisco	California
United States	Lenus Research & Medical Group, LLC	Sweetwater	Florida
United States	Clinical Research Trials of Florida, Inc.	Tampa	Florida
United States	Forward Clinical Trials Inc.	Tampa	Florida
United States	Vital Prospects Clinical Research Institute, P.C.	Tulsa	Oklahoma
United States	PEAK Research	Upper Saint Clair	Pennsylvania
United States	Wake Forest University Health Sciences, Department of Dermatology	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Concert Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada,  France,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants Achieving an Absolute Severity of Alopecia Tool (SALT) Score of =20 at Week 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).	Week 24
Secondary	Percentage of Responders on the Hair Satisfaction Patient Reported Outcome (SPRO) Scale at 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. Responders were defined as participants with responses of "satisfied" or "very satisfied".	Weeks 12, 16, 20, and 24
Secondary	Percentage of Participants Achieving an Absolute SALT Score of =20 at Weeks 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).	Weeks 4, 8, 12, 16, and 20
Secondary	Relative Change in SALT Scores From Baseline at 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).	Baseline, Weeks 4, 8, 12, 16, 20, and 24
Secondary	Percentage of Responders Assessed Using the Clinician Global Impression of Improvement (CGI-I) at Weeks 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).	Weeks 12, 16, 20, and 24
Secondary	Percentage of Responders Assessed Using the Patient Global Impression of Improvement (PGI-I) at Weeks 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).	Weeks 12, 16, 20, and 24
Secondary	Change in the Clinician Global Impression of Severity (CGI-S) Scores From Baseline at 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.	Baseline, Weeks 12, 16, 20, and 24
Secondary	Change in the Patient Global Impression of Severity (PGI-S) Scores From Baseline at 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.	Baseline, Weeks 12, 16, 20, and 24
Secondary	Percentage of Participants Achieving at Least a 75% and 90% Relative Reduction in SALT Score From Baseline at 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.	Baseline, Weeks 12, and 24
Secondary	Change in the Brigham Eyebrow Tool for Alopecia (BETA) Scores From Baseline at 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.	Baseline, Weeks 12, and 24
Secondary	Change in the Brigham Eyelash Tool for Alopecia (BELA) Scores From Baseline at 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.	Baseline, Weeks 12, and 24
Secondary	Change in the SPRO Scale From Baseline at 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.	Baseline, Weeks 12, 16, 20, and 24
Secondary	Percentage of Participants Achieving a =2-point Change From Baseline in the SPRO Scale at 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.	Weeks 12, 16, 20, and 24
Secondary	Change in the Individual Items of the Hair Quality Patient Reported Outcome (QPRO) Scale From Baseline at 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.	Baseline, Weeks 12, 16, 20, and 24
Secondary	Change in the Anxiety and Depression Scale Scores of the Hospital Anxiety and Depression Scale (HADS) From Baseline at 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.	Baseline and Week 24
Secondary	Percentage of Participants Achieving an Absolute SALT Score of =10 at Week 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).	Week 24