Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA2) (THRIVE-AA2)

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

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.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 517
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Quebec, Canada, G1W4R4
    • Centre de Recherche Saint-Louis
  • Quebec, Canada, L4B1A5
    • Centre De Recherche Dermatologique Du Quebec Metropolitan
  • Alberta
    • Red Deer, Alberta, Canada, T4N6V7
      • Care Clinic Ltd.
  • British Columbia
    • Kelowna, British Columbia, Canada, V1Y 4N7
      • Medical Arts Health Research Group
  • Ontario
    • Etobicoke, Ontario, Canada, M8X1Y9
      • Kingsway Clinical Research
    • North Bay, Ontario, Canada, P1B 3Z7
      • North Bay Dermatology Centre
    • Richmond Hill, Ontario, Canada, L4B1A5
      • The Centre for Dermatology
• France
  • Alpes-maritimes
    • Nice, Alpes-maritimes, France, 06202 CEDEX 3
      • Centre Hospitalier Universitaire de Nice - Hôpital Archet 2
  • Finestère
    • Brest, Finestère, France, 29609 CEDEX
      • CHRU de Brest - Hôpital Morvan
• Germany
  • Berlin, Germany, 10117
    • Clinical Research Center for Hair and Skin Science, Dept of Dermatology, Charité Universitätsmedizin Berlin
  • Erlangen, Germany, 91054
    • Universitatsklinikum Erlangen Hautklinik
  • Baden-württemberg
    • Tübingen, Baden-württemberg, Germany, 72076
      • Universitaetsklinikum Tübingen
  • Bayern
    • Munich, Bayern, Germany, 80802
      • Klinikum rechts der Isar, Technische Universität München
  • Hessen
    • Frankfurt am main, Hessen, Germany, 60590
      • Universitätsklinikum Frankfurt Klinik Für Dermatologie, Venerologie Und Allergologie
  • Niedersachsen
    • Bad Bentheim, Niedersachsen, Germany, 48455
      • Fachklinik Bad Bentheim - Dermatologische Studienambulanz
  • Nordrhein-Westfalen
    • Bochum, Nordrhein-Westfalen, Germany, 44791
      • Dermatologische Studienambulanz
  • Nordrhein-westfalen
    • Münster, Nordrhein-westfalen, Germany, 48149
      • University Hospital Muenster
• Hungary
  • Baranya
    • Pécs, Baranya, Hungary, 7632
      • Pecsi Tudomanyegyetem Klinikai Kozpont, Bor-, Nemikortani es Onkodermatologiai Klinika
  • Csongrád-csanád
    • Szeged, Csongrád-csanád, Hungary, 6720
      • Szte Áok Szent-Györgyi Albert Klinikai Központ Bőrgyógyászati És Allergológiai Klinika
  • Heves
    • Gyöngyös, Heves, Hungary, 3200
      • Bugát Pál Kórház
  • Pest
    • Budapest, Pest, Hungary, 1085
      • Semmelweis Egyetem, Általános Orvostudományi Kar, Bőr-, Nemikórtani És Bőronkológiai Klinika
• Poland
  • Szczecin, Poland, 71-434
    • Twoja Przychodnia - Szczecinskie Centrum Medyczne
  • Dolnośląskie
    • Wrocław, Dolnośląskie, Poland, 50-685
      • WroMedica I. Bielicka, A. Strzalkowska s.c.
  • Lubuskie
    • Nowa Sól, Lubuskie, Poland, 67-100
      • Twoja Przychodnia - Centrum Medyczne Nowa Sol
  • Mazowieckie
    • Siedlce, Mazowieckie, Poland, 08-110
      • ETG Siedlce
    • Warszawa, Mazowieckie, Poland, 00-716
      • Ot.Co Clinic Klinika Osipowicz & Turkowski Sp. Z O.O.
    • Warszawa, Mazowieckie, Poland, 00-892
      • RCMed Oddzial Warszawa
    • Warszawa, Mazowieckie, Poland, 02-664
      • Carpe Diem Centrum Medycyny Estetycznej
    • Warszawa, Mazowieckie, Poland, 02-793
      • Etg Warszawa
  • Łódzkie
    • Skierniewice, Łódzkie, Poland, 96-100
      • Etg Skierniewice
• Spain
  • Barcelona, Spain, 08036
    • Hospital Clínic de Barcelona
  • Córdoba, Spain, 14004
    • Hospital Universitario Reina Sofia
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Madrid, Spain, 28041
    • Hospital Doce de Octubre
  • Madrid, Spain, 28034
    • Hospital Universitario Ramón Y Cajal - Edificio Consultas Externas
  • Madrid, Spain, 28081
    • Hospital Universitario Infanta Leonor
  • Cataluña
    • Barcelona, Cataluña, Spain, 08041
      • Hospital Santa Creu i Sant Pau
    • Barcelona, Cataluña, Spain, 08001
      • Hospital Del Mar
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85032
      • Alliance Dermatology & MOHS Center
  • Arkansas
    • Fort Smith, Arkansas, United States, 72916
      • Johnson Dermatology
  • California
    • Los Angeles, California, United States, 90045
      • Dermatology Research Associates
  • Florida
    • Miami, Florida, United States, 33124
      • University of Miami Hospital
    • Miami, Florida, United States, 33145
      • Floridian Research Institute Llc
  • Massachusetts
    • Brighton, Massachusetts, United States, 02135
      • MetroBoston Clinical Partners, LLC
  • Michigan
    • Chesterfield, Michigan, United States, 48087
      • Clinical Research Institute of Michigan, LLC
    • Fort Gratiot, Michigan, United States, 48059
      • Hamzavi Dermatology
  • Missouri
    • Chesterfield, Missouri, United States, 63005
      • Galen Research
  • Nevada
    • Las Vegas, Nevada, United States, 89148
      • Vivida Dermatology
  • New Jersey
    • Verona, New Jersey, United States, 07044
      • The Dermatology Group P.C.
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai
    • New York, New York, United States, 10032
      • Columbia University Medical Center - Department of Dermatology
    • Rochester, New York, United States, 14642
      • University of Rochester
  • Ohio
    • Mason, Ohio, United States, 45040
      • Dermatologists of Southwest Ohio
  • Oregon
    • Portland, Oregon, United States, 97223
      • Oregon Medical Research
  • Rhode Island
    • Warwick, Rhode Island, United States, 02886
      • Velocity Clinical Research - Providence
  • Tennessee
    • Knoxville, Tennessee, United States, 37922
      • The Skin Wellness Center
  • Texas
    • Frisco, Texas, United States, 75034
      • North Texas Center for Clinical Research
    • Houston, Texas, United States, 77077
      • Elixir Research Group
    • San Antonio, Texas, United States, 78213
      • Progressive Clinical Research
  • Utah
    • Salt Lake City, Utah, United States, 84101
      • Velocity Clinical Research/Gateway
    • Salt Lake City, Utah, United States, 84117
      • Velocity Clinical Research/Swinyer-Woseth Dermatology
    • South Jordan, Utah, United States, 84095
      • Jordan Valley Dermatology Center
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • The Rector and Visitors of the University of Virginia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CTP-543 8 mg BID; Participants received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.	Drug: CTP-543; Administered as tablets.
Placebo Comparator: Placebo; Participants received CTP-543 matched placebo tablets, orally, twice daily (BID) for up to 24 weeks.	Drug: CTP-543 matching placebo; Administered as tablets.
Experimental: CTP-543 12 mg BID; Participants received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.	Drug: CTP-543; Administered as tablets.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Achieving an Absolute Severity of Alopecia Tool (SALT) Score ≤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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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
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
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
Percentage of Responders on the Hair Satisfaction Participants 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
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
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, and 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
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
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 participants 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
Change in the Anxiety and Depression Scale Scores of the Hospital Anxiety and Depression Scale (HADS) From Baseline at Week 24	HADS is 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
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 from 0 (no scalp hair loss) to 100 (complete scalp hair loss).	Week 24

Collaborators and Investigators

• Sponsor: Concert Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2021
• Primary Completion (Actual): June 8, 2022
• Study Completion (Actual): June 29, 2022

Study Registration Dates

• First Submitted: March 10, 2021
• First Submitted That Met QC Criteria: March 10, 2021
• First Posted (Actual): March 15, 2021

Study Record Updates

• Last Update Posted (Actual): July 3, 2023
• Last Update Submitted That Met QC Criteria: June 30, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Alopecia; Hair loss; CTP-543; Hair disease
• Additional Relevant MeSH Terms: Skin Diseases; Pathological Conditions, Anatomical; Hypotrichosis; Hair Diseases; Alopecia; Alopecia Areata
• Other Study ID Numbers: CP543.3002; 2021-000387-30 (EudraCT Number)

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