A Phase 3 Study to Evaluate the Efficacy and Safety of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata (THRIVE-AA2)

Sponsor

Concert Pharmaceuticals (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04797650

Collaborator

(none)

517

Enrollment

Locations

Arms

13.1

Anticipated Duration (Months)

8.2

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a multi-center Phase 3 study to evaluate 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. The study is placebo-controlled, meaning that some patients entering the study will not receive active study drug but will receive tablets with no active ingredients (a placebo). It is double-blinded, meaning that the Sponsor, the study doctors, the staff, and the patients will not know whether a patient is on active study drug (or the dose) or placebo.

Condition or Disease	Intervention/Treatment	Phase
Alopecia Areata	Drug: CTP-543, 8 mg BID Drug: CTP-543, 12 mg BID Drug: Placebo, BID	Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

517 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Actual Study Start Date :

May 27, 2021

Anticipated Primary Completion Date :

Jul 1, 2022

Anticipated Study Completion Date :

Jul 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: CTP-543, 8 mg BID Oral tablet for 24 weeks	Drug: CTP-543, 8 mg BID 8 mg twice-daily (BID)
Experimental: CTP-543, 12 mg BID Oral tablet for 24 weeks	Drug: CTP-543, 12 mg BID 12 mg twice-daily (BID)
Placebo Comparator: Placebo, BID Oral tablet for 24 weeks	Drug: Placebo, BID Placebo, twice-daily (BID)

Arm

Intervention/Treatment

Experimental: CTP-543, 8 mg BID

Oral tablet for 24 weeks

Drug: CTP-543, 8 mg BID

8 mg twice-daily (BID)

Experimental: CTP-543, 12 mg BID

Oral tablet for 24 weeks

Drug: CTP-543, 12 mg BID

12 mg twice-daily (BID)

Placebo Comparator: Placebo, BID

Oral tablet for 24 weeks

Drug: Placebo, BID

Placebo, twice-daily (BID)

Outcome Measures

Primary Outcome Measures

Percentage of patients achieving an absolute Severity of Alopecia Tool (SALT) score ≤20 [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).

Secondary Outcome Measures

Percentage of responders (defined as "satisfied" or "very satisfied") on the Hair Satisfaction Patient Reported Outcome (SPRO) [Week 24]
SPRO is a questionnaire answered by the patient designed to measure how satisfied alopecia areata patients are with their hair at the time of the assessment.
Percentage of patients achieving an absolute SALT score of ≤20 [Weeks 12, 16, and 20]
SALT is a quantitative assessment of scalp hair loss with scores ranging from 0 (no scalp hair loss) to 100 (complete scalp hair loss).
Relative change in SALT scores from Baseline [Weeks 4, 8, 12, 16, 20, and 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).
Percentage of responders (defined as "much improved" or "very much improved") using the CGI-I [Weeks 12, 16, 20, and 24]
The Clinician Global Impression of Improvement (CGI-I) is a questionnaire that asks the clinician to evaluate the improvement or worsening of the patient's alopecia areata as compared to the start of the study.
Percentage of responders (defined as "much improved" or "very much improved") using the PGI-I [Weeks 12, 16, 20, and 24]
The Patient Global Impression of Improvement (PGI-I) is a self-administered questionnaire that asks the patient to evaluate the improvement or worsening of their alopecia areata as compared to the start of the study.
Change from Baseline in the CGI-S [Weeks 12, 16, 20, and 24]
The Clinician Global Impression of Severity (CGI-S) is a questionnaire that asks the clinician to evaluate the severity of the patient's alopecia areata at the time of assessment.
Change from Baseline in the PGI-S [Weeks 12, 16, 20, and 24]
The Patient Global Impression of Severity (PGI-S) is a self-administered questionnaire that asks the patient to evaluate the severity of their alopecia areata at the time of assessment.
Percentage of patients achieving at least a 75% relative reduction in SALT score from Baseline [Weeks 12 and 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).
Percentage of patients achieving at least a 90% relative reduction in SALT score from Baseline [Weeks 12 and 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).
Change from Baseline on an eyebrow score [Weeks 12 and 24]
A clinician-rated scale that assesses the total eyebrow hair present. The score is derived by assessment of density and surface area of the patient's eyebrows.
Change from Baseline on an eyelash score [Weeks 12 and 24]
A clinician-rated scale that assesses the total eyelash hair present. The score is derived by assessment of density and distribution of the patient's eyelashes.
Percentage of responders (defined as "satisfied" or "very satisfied") on the Hair Satisfaction Patient Reported Outcome scale [Weeks 12, 16, and 20]
SPRO is a questionnaire answered by the patient designed to measure how satisfied alopecia areata patients are with their hair at the time of the assessment.
Change from Baseline on the individual items of the Hair Quality Patient Reported Outcome (QPRO) scale [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.
Change from Baseline in the depression scale of the Hospital Anxiety and Depression Scale (HADS) [Week 24]
HADS is a 14-item measure designed to assess anxiety and depression symptoms in medical patients. Items are rated on a 4-point severity scale.
Change from Baseline in the anxiety scale of the Hospital Anxiety and Depression Scale [Week 24]
HADS is a 14-item measure designed to assess anxiety and depression symptoms in medical patients. Items are rated on a 4-point severity scale.
Percentage of patients achieving an absolute SALT score of ≤10 [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).

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Alliance Dermatology & Mohs Center	Phoenix	Arizona	United States	85032
2	Johnson Dermatology	Fort Smith	Arkansas	United States	72916
3	Dermatology Research Associates	Los Angeles	California	United States	90045
4	University of Miami Hospital	Miami	Florida	United States	33124
5	Floridian Research Institute LLC	Miami	Florida	United States	33145
6	MetroBoston Clinical Partners, LLC	Brighton	Massachusetts	United States	02135
7	Clinical Research Institute of Michigan, LLC	Chesterfield	Michigan	United States	48087
8	Hamzavi Dermatology	Fort Gratiot	Michigan	United States	48059
9	Galen Research	Chesterfield	Missouri	United States	63005
10	Vivida Dermatology	Las Vegas	Nevada	United States	89148
11	The Dermatology Group P.C.	Verona	New Jersey	United States	07044
12	Icahn School of Medicine at Mount Sinai	New York	New York	United States	10029
13	Columbia University Medical Center - Department of Dermatology	New York	New York	United States	10032
14	University of Rochester	Rochester	New York	United States	14642
15	Dermatologists of Southwest Ohio	Mason	Ohio	United States	45040
16	Oregon Medical Research	Portland	Oregon	United States	97223
17	Velocity Clinical Research - Providence	Warwick	Rhode Island	United States	02886
18	The Skin Wellness Center	Knoxville	Tennessee	United States	37922
19	North Texas Center for Clinical Research	Frisco	Texas	United States	75034
20	Elixir Research Group	Houston	Texas	United States	77077
21	Progressive Clinical Research	San Antonio	Texas	United States	78213
22	Velocity Clinical Research/Gateway	Salt Lake City	Utah	United States	84101
23	Velocity Clinical Research/Swinyer-Woseth Dermatology	Salt Lake City	Utah	United States	84117
24	Jordan Valley Dermatology Center	South Jordan	Utah	United States	84095
25	The Rector and Visitors of the University of Virginia	Charlottesville	Virginia	United States	22903
26	Care Clinic Ltd.	Red Deer	Alberta	Canada	T4N6V7
27	Medical Arts Health Research Group	Kelowna	British Columbia	Canada	V1Y 4N7
28	Kingsway Clinical Research	Etobicoke	Ontario	Canada	M8X1Y9
29	North Bay Dermatology Centre	North Bay	Ontario	Canada	P1B 3Z7
30	The Centre for Dermatology	Richmond Hill	Ontario	Canada	L4B1A5
31	Centre de Recherche Saint-Louis	Quebec		Canada	G1W4R4
32	Centre de Recherche Dermatologique du Quebec Metropolitan	Quebec		Canada	L4B1A5
33	Centre Hospitalier Universitaire de Nice - Hôpital Archet 2	Nice	Alpes-maritimes	France	06202 CEDEX 3
34	Chru de Brest - Hôpital Morvan	Brest	Finestère	France	29609 CEDEX
35	Universitaetsklinikum Tübingen	Tübingen	Baden-württemberg	Germany	72076
36	Klinikum Rechts Der Isar, Technische Universität München	Munich	Bayern	Germany	80802
37	Universitätsklinikum Frankfurt Klinik Für Dermatologie, Venerologie Und Allergologie	Frankfurt am main	Hessen	Germany	60590
38	Fachklinik Bad Bentheim - Dermatologische Studienambulanz	Bad Bentheim	Niedersachsen	Germany	48455
39	Dermatologische Studienambulanz	Bochum	Nordrhein-Westfalen	Germany	44791
40	University Hospital Muenster	Münster	Nordrhein-westfalen	Germany	48149
41	Clinical Research Center for Hair and Skin Science, Dept of Dermatology, Charité Universitätsmedizin Berlin	Berlin		Germany	10117
42	Universitätsklinikum Erlangen Hautklinik	Erlangen		Germany	91054
43	Pécsi Tudományegyetem Klinikai Központ, Bőr-, Nemikórtani És Onkodermatológiai Klinika	Pécs	Baranya	Hungary	7632
44	Szte Áok Szent-Györgyi Albert Klinikai Központ Bőrgyógyászati És Allergológiai Klinika	Szeged	Csongrád-csanád	Hungary	6720
45	Bugát Pál Kórház	Gyöngyös	Heves	Hungary	3200
46	Semmelweis Egyetem, Általános Orvostudományi Kar, Bőr-, Nemikórtani És Bőronkológiai Klinika	Budapest	Pest	Hungary	1085
47	Wromedica I. Bielicka, A. Strzalkowska S.C.	Wrocław	Dolnośląskie	Poland	50-685
48	Twoja Przychodnia - Centrum Medyczne Nowa Sól	Nowa Sól	Lubuskie	Poland	67-100
49	Etg Siedlce	Siedlce	Mazowieckie	Poland	08-110
50	Ot.Co Clinic Klinika Osipowicz & Turkowski Sp. Z O.O.	Warszawa	Mazowieckie	Poland	00-716
51	Rcmed Oddzial Warszawa	Warszawa	Mazowieckie	Poland	00-892
52	Carpe Diem Centrum Medycyny Estetycznej	Warszawa	Mazowieckie	Poland	02-664
53	Etg Warszawa	Warszawa	Mazowieckie	Poland	02-793
54	Twoja Przychodnia - Szczecinskie Centrum Medyczne	Szczecin		Poland	71-434
55	Etg Skierniewice	Skierniewice	Łódzkie	Poland	96-100
56	Hospital Del Mar	Barcelona	Cataluña	Spain	08001
57	Hospital Santa Creu I Sant Pau	Barcelona	Cataluña	Spain	08041
58	Hospital Clinic de Barcelona	Barcelona		Spain	08036
59	Hospital Universitario Reina Sofia	Córdoba		Spain	14004
60	Hospital Universitario Ramón Y Cajal - Edificio Consultas Externas	Madrid		Spain	28034
61	Hospital Doce de Octubre	Madrid		Spain	28041
62	Hospital Universitario La Paz	Madrid		Spain	28046
63	Hospital Universitario Infanta Leonor	Madrid		Spain	28081