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

Phase 3

Completed

• Conditions: Alopecia Areata
• Interventions: Drug: CTP-543; Drug: CTP-543 matching placebo

• Registration Number: NCT04797650
• 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

Study Timeline

• Study First Submitted: 2021-03-10
• Study First Submitted QC: 2021-03-10
• Study First Posted: 2021-03-15
• Study Start: 2021-06-10
• Primary Completion: 2022-06-08
• Study Completion: 2022-06-29
• Status Verified: 2023-06
• Results First Submitted: 2023-06-05
• Results First Submitted QC: 2023-06-30
• Last Update Submitted: 2023-06-30
• Results First Posted: 2023-07-03
• Last Update Posted: 2023-07-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 517

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
CTP-543 8 mg BID	CTP-543	Participants received CTP-543 8 mg tablets, orally, BID for up to 24 weeks.
Placebo	CTP-543 matching placebo	Participants received CTP-543 matched placebo tablets, orally, twice daily (BID) for up to 24 weeks.
CTP-543 12 mg BID	CTP-543	Participants received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants Achieving an Absolute Severity of Alopecia Tool (SALT) Score ≤20 at Week 24	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).

Secondary Outcome Measures

Name	Time	Method
Change in the Clinician Global Impression of Severity (CGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24	Baseline, 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 Participants Achieving an Absolute SALT Score of ≤20 at Weeks 4, 8, 12, 16, and 20	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).
Percentage of Responders on the Hair Satisfaction Participants Reported Outcome (SPRO) Scale at Weeks 12, 16, 20, and 24	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".
Change in the Patient Global Impression of Severity (PGI-S) Scores From Baseline at Weeks 12, 16, 20, and 24	Baseline, 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.
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	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 24	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).
Percentage of Participants Achieving at Least a 75% and 90% Relative Reduction in SALT Score From Baseline at Weeks 12 and 24	Baseline, 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.
Change in the Brigham Eyebrow Tool for Alopecia (BETA) Scores From Baseline at Weeks 12 and 24	Baseline, 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.
Change in the Brigham Eyelash Tool for Alopecia (BELA) Scores From Baseline at Weeks 12 and 24	Baseline, 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 24	Baseline, 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 Anxiety and Depression Scale Scores of the Hospital Anxiety and Depression Scale (HADS) From Baseline at Week 24	Baseline and 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.
Percentage of Responders Assessed Using the Clinician Global Impression of Improvement (CGI-I) at Weeks 12, 16, 20, and 24	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).
Change in the Individual Items of the Hair Quality Patient Reported Outcome (QPRO) Scale From Baseline at Weeks 12, 16, 20, and 24	Baseline, 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.
Percentage of Participants Achieving a ≥2-point Change From Baseline in the SPRO Scale at Weeks 12, 16, 20, and 24	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.
Percentage of Participants Achieving an Absolute SALT Score of ≤10 at Week 24	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).

Trial Locations

Locations (63)

North Bay Dermatology Centre; 🇨🇦 North Bay, Ontario, Canada

Universitätsklinikum Erlangen Hautklinik; 🇩🇪 Erlangen, Germany

Hospital Universitario Reina Sofia; 🇪🇸 Córdoba, Spain

Columbia University Medical Center - Department of Dermatology; 🇺🇸 New York, New York, United States

Hospital Doce de Octubre; 🇪🇸 Madrid, Spain

Hospital Santa Creu I Sant Pau; 🇪🇸 Barcelona, Cataluña, Spain

Alliance Dermatology & Mohs Center; 🇺🇸 Phoenix, Arizona, United States

Dermatologische Studienambulanz; 🇩🇪 Bochum, Nordrhein-Westfalen, Germany

Fachklinik Bad Bentheim - Dermatologische Studienambulanz; 🇩🇪 Bad Bentheim, Niedersachsen, Germany

Icahn School of Medicine at Mount Sinai; 🇺🇸 New York, New York, United States

Chru de Brest - Hôpital Morvan; 🇫🇷 Brest, Finestère, France

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

The Rector and Visitors of the University of Virginia; 🇺🇸 Charlottesville, Virginia, United States

Pécsi Tudományegyetem Klinikai Központ, Bőr-, Nemikórtani És Onkodermatológiai Klinika; 🇭🇺 Pécs, Baranya, Hungary

Universitätsklinikum Frankfurt Klinik Für Dermatologie, Venerologie Und Allergologie; 🇩🇪 Frankfurt am main, Hessen, Germany

Hospital Universitario Infanta Leonor; 🇪🇸 Madrid, Spain

Hospital Del Mar; 🇪🇸 Barcelona, Cataluña, Spain

Dermatologists of Southwest Ohio; 🇺🇸 Mason, Ohio, United States

Oregon Medical Research; 🇺🇸 Portland, Oregon, United States

North Texas Center for Clinical Research; 🇺🇸 Frisco, Texas, United States

The Skin Wellness Center; 🇺🇸 Knoxville, Tennessee, United States

Velocity Clinical Research - Providence; 🇺🇸 Warwick, Rhode Island, United States

Centre Hospitalier Universitaire de Nice - Hôpital Archet 2; 🇫🇷 Nice, Alpes-maritimes, France

Klinikum Rechts Der Isar, Technische Universität München; 🇩🇪 Munich, Bayern, Germany

Hospital Universitario Ramón Y Cajal - Edificio Consultas Externas; 🇪🇸 Madrid, Spain

Progressive Clinical Research; 🇺🇸 San Antonio, Texas, United States

Velocity Clinical Research/Swinyer-Woseth Dermatology; 🇺🇸 Salt Lake City, Utah, United States

Dermatology Research Associates; 🇺🇸 Los Angeles, California, United States

University of Miami Hospital; 🇺🇸 Miami, Florida, United States

Floridian Research Institute LLC; 🇺🇸 Miami, Florida, United States

Galen Research; 🇺🇸 Chesterfield, Missouri, United States

Velocity Clinical Research/Gateway; 🇺🇸 Salt Lake City, Utah, United States

The Dermatology Group P.C.; 🇺🇸 Verona, New Jersey, United States

Elixir Research Group; 🇺🇸 Houston, Texas, United States

Szte Áok Szent-Györgyi Albert Klinikai Központ Bőrgyógyászati És Allergológiai Klinika; 🇭🇺 Szeged, Csongrád-csanád, Hungary

Universitaetsklinikum Tübingen; 🇩🇪 Tübingen, Baden-württemberg, Germany

MetroBoston Clinical Partners, LLC; 🇺🇸 Brighton, Massachusetts, United States

Vivida Dermatology; 🇺🇸 Las Vegas, Nevada, United States

Care Clinic Ltd.; 🇨🇦 Red Deer, Alberta, Canada

Clinical Research Institute of Michigan, LLC; 🇺🇸 Chesterfield, Michigan, United States

Johnson Dermatology; 🇺🇸 Fort Smith, Arkansas, United States

University of Rochester; 🇺🇸 Rochester, New York, United States

Hamzavi Dermatology; 🇺🇸 Fort Gratiot, Michigan, United States

Bugát Pál Kórház; 🇭🇺 Gyöngyös, Heves, Hungary

Wromedica I. Bielicka, A. Strzalkowska S.C.; 🇵🇱 Wrocław, Dolnośląskie, Poland

Twoja Przychodnia - Centrum Medyczne Nowa Sól; 🇵🇱 Nowa Sól, Lubuskie, Poland

Medical Arts Health Research Group; 🇨🇦 Kelowna, British Columbia, Canada

Ot.Co Clinic Klinika Osipowicz & Turkowski Sp. Z O.O.; 🇵🇱 Warszawa, Mazowieckie, Poland

Twoja Przychodnia - Szczecinskie Centrum Medyczne; 🇵🇱 Szczecin, Poland

Etg Siedlce; 🇵🇱 Siedlce, Mazowieckie, Poland

Etg Skierniewice; 🇵🇱 Skierniewice, Łódzkie, Poland

Semmelweis Egyetem, Általános Orvostudományi Kar, Bőr-, Nemikórtani És Bőronkológiai Klinika; 🇭🇺 Budapest, Pest, Hungary

Rcmed Oddzial Warszawa; 🇵🇱 Warszawa, Mazowieckie, Poland

Carpe Diem Centrum Medycyny Estetycznej; 🇵🇱 Warszawa, Mazowieckie, Poland

Etg Warszawa; 🇵🇱 Warszawa, Mazowieckie, Poland

Centre de Recherche Saint-Louis; 🇨🇦 Quebec, Canada

Kingsway Clinical Research; 🇨🇦 Etobicoke, Ontario, Canada

Centre de Recherche Dermatologique du Quebec Metropolitan; 🇨🇦 Quebec, Canada

The Centre for Dermatology; 🇨🇦 Richmond Hill, Ontario, Canada

University Hospital Muenster; 🇩🇪 Münster, Nordrhein-westfalen, Germany

Hospital Clinic de Barcelona; 🇪🇸 Barcelona, Spain

Jordan Valley Dermatology Center; 🇺🇸 South Jordan, Utah, United States

Clinical Research Center for Hair and Skin Science, Dept of Dermatology, Charité Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany