Study to Evaluate the Safety and Efficacy of CTP-543 in Adults With Moderate to Severe Alopecia Areata
Sponsor
Concert Pharmaceuticals (Industry)
Overall Status
Completed
CT.gov ID
NCT03137381
Collaborator
(none)
149
13
4
22.9
11.5
0.5
Study Details
Study Description
Brief Summary
This study will evaluate the safety and efficacy of CTP-543 on hair loss in adults with chronic, moderate to severe alopecia areata.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
This is a double-blind, randomized, placebo-controlled multi-center study consisting of 3 cohorts to assess the safety and efficacy of CTP-543. Each Cohort will be initiated sequentially in ascending dose order. Participants will be randomized to either an active dose of CTP-543 or placebo for a 24-week treatment period.
Study Design
Study Type:
Interventional
Actual Enrollment
:
149 participants
Allocation:
Randomized
Intervention Model:
Sequential Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata
Actual Study Start Date
:
Aug 9, 2017
Actual Primary Completion Date
:
Jul 8, 2019
Actual Study Completion Date
:
Jul 8, 2019
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Cohort 1: CTP-543 4 mg BID Participants will receive CTP-543 4 mg tablets, twice daily for up to 24 weeks. |
Drug: CTP-543
Administered as tablets.
|
| Experimental: Cohort 2: CTP-543 8 mg BID Participants will receive CTP-543 8 mg tablets, twice daily for up to 24 weeks. |
Drug: CTP-543
Administered as tablets.
|
| Experimental: Cohort 3: CTP-543 12 mg BID Participants will receive CTP-543 12 mg tablets, twice daily for up to 24 weeks. |
Drug: CTP-543
Administered as tablets.
|
| Placebo Comparator: Combined Placebo Participants will receive CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. |
Drug: CTP-543 matching placebo
Administered as tablets.
|
Outcome Measures
Primary Outcome Measures
- Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 [Week 24]
The SALT is a quantitative assessment of scalp hair loss. SALT scores range in severity from 0 (no hair loss) to a maximum of 100 (complete hair loss). Responders were defined as participants achieving at least a 50% relative reduction in SALT score from baseline at Week 24.
- Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) [From first dose of study drug up to safety follow up at Week 28]
An adverse event is any untoward medical occurrence that may appear or worsen in a participant during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the patient's health, including laboratory test values, regardless of etiology. Any worsening (ie, any clinically significant adverse change in the frequency or intensity of a pre-existing condition) should be considered an adverse event. TEAE is defined as any adverse event that occurs after administration of the first dose of study drug.
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Definitive diagnosis of 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.
-
Clinical lab results within the normal range
Exclusion Criteria:
-
Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or untreated actinic keratosis on the scalp.
-
Treatment with systemic immunosuppressive medications or biologics.
-
Vaccination with herpes zoster vaccine or any live virus within 6 weeks of screening or during the study
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California, Irvine | Irvine | California | United States | 92697 |
| 2 | Contour Dermatology & Cosmetic Surgery Center | Rancho Mirage | California | United States | 92270 |
| 3 | Stanford University School of Medicine | Redwood City | California | United States | 94064 |
| 4 | Kaiser Permanente Northern California | San Francisco | California | United States | 94118 |
| 5 | Yale School of Medicine | New Haven | Connecticut | United States | 06519 |
| 6 | Siperstein Dermatology Group | Boynton Beach | Florida | United States | 33472 |
| 7 | Northwestern University | Chicago | Illinois | United States | 60611 |
| 8 | Minnesota Clinical Study Center | Fridley | Minnesota | United States | 55432 |
| 9 | Icahn School of Medicine at Mt. Sinai | New York | New York | United States | 10029 |
| 10 | Wake Forest University Health Sciences | Winston-Salem | North Carolina | United States | 27104 |
| 11 | Cleveland Clinic | Cleveland | Ohio | United States | 44195 |
| 12 | Northwest Dermatology | Portland | Oregon | United States | 97210 |
| 13 | Suzanne Bruce & Associates, PA | Houston | Texas | United States | 77056 |
Sponsors and Collaborators
- Concert Pharmaceuticals
Investigators
- Study Director: Colleen E. Hamilton, Concert Pharmaceuticals, Inc.
Study Documents (Full-Text)
More Information
Publications
- Cassella J, Hamilton C, von Hehn J, Braman V. CTP-543, an oral JAK inhibitor, achieves primary endpoint in Phase 2 randomized, placebo-controlled, dose ranging trial in patients with moderate to severe alopecia areata. In: Proceedings from the 28th EADV Congress; 09-13 October 2019, Madrid, Spain. D3T01.1: Late breaking news.
- Cassella J, Hamilton C, von Hehn J, Braman V. JAK Inhibitor CTP-543 Achieves Primary Endpoint in Phase 2 Trial in Alopecia Areata. In: Proceedings from the 11th World Congress Hair Research; 24-27 April 2019; Sitges, Barcelona.
- Cassella J, Hamilton C, von Hehn J, Braman V. JAK Inhibitor CTP-543 Achieves Primary Endpoint in Phase 2 Trial in Alopecia Areata. In: Proceedings from the 2019 American Academy of Dermatology Meeting; 01-05 March 2019; Washington, DC. Abstract 11291.
Responsible Party:
Concert Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT03137381
Other Study ID Numbers:
- CP543.2001
First Posted:
May 2, 2017
Last Update Posted:
Jul 19, 2022
Last Verified:
Jun 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | Participants were enrolled at 13 study centers in the United States from 09 August 2017 to 08 July 2019. |
|---|---|
| Pre-assignment Detail | 235 participants were screened, out of which 149 participants who experienced an episode of hair loss due to alopecia areata were enrolled to receive CTP-543 or placebo. |
| Arm/Group Title | Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID |
|---|---|---|---|---|
| Arm/Group Description | Participants received CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | Participants received CTP-543 4 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 8 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 12 mg tablets, twice daily for up to 24 weeks. |
| Period Title: Overall Study | ||||
| STARTED | 44 | 30 | 38 | 37 |
| Safety Population | 44 | 29 | 38 | 36 |
| COMPLETED | 35 | 23 | 30 | 36 |
| NOT COMPLETED | 9 | 7 | 8 | 1 |
Baseline Characteristics
| Arm/Group Title | Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID | Total |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | Participants received CTP-543 4 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 8 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 12 mg tablets, twice daily for up to 24 weeks. | Total of all reporting groups |
| Overall Participants | 44 | 30 | 38 | 37 | 149 |
| Age (years) [Mean (Standard Deviation) ] | |||||
| Mean (Standard Deviation) [years] |
37.8
(13.50)
|
35.7
(11.01)
|
37.3
(14.18)
|
35.8
(12.37)
|
36.8
(12.85)
|
| Sex: Female, Male (Count of Participants) | |||||
| Female |
29
65.9%
|
22
73.3%
|
26
68.4%
|
28
75.7%
|
105
70.5%
|
| Male |
15
34.1%
|
8
26.7%
|
12
31.6%
|
9
24.3%
|
44
29.5%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |||||
| Hispanic or Latino |
5
11.4%
|
3
10%
|
4
10.5%
|
3
8.1%
|
15
10.1%
|
| Not Hispanic or Latino |
39
88.6%
|
26
86.7%
|
33
86.8%
|
34
91.9%
|
132
88.6%
|
| Unknown or Not Reported |
0
0%
|
1
3.3%
|
1
2.6%
|
0
0%
|
2
1.3%
|
| Race/Ethnicity, Customized (Count of Participants) | |||||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Asian |
2
4.5%
|
2
6.7%
|
2
5.3%
|
4
10.8%
|
10
6.7%
|
| Native Hawaiian or Other Pacific Islander |
1
2.3%
|
0
0%
|
0
0%
|
0
0%
|
1
0.7%
|
| Black or African American |
7
15.9%
|
2
6.7%
|
7
18.4%
|
3
8.1%
|
19
12.8%
|
| White |
33
75%
|
25
83.3%
|
26
68.4%
|
30
81.1%
|
114
76.5%
|
| Other |
1
2.3%
|
1
3.3%
|
3
7.9%
|
0
0%
|
5
3.4%
|
Outcome Measures
| Title | Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 |
|---|---|
| Description | The SALT is a quantitative assessment of scalp hair loss. SALT scores range in severity from 0 (no hair loss) to a maximum of 100 (complete hair loss). Responders were defined as participants achieving at least a 50% relative reduction in SALT score from baseline at Week 24. |
| Time Frame | Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Efficacy Population included all participants who received study drug and had at least 1 post-treatment SALT assessment during the treatment period. Participants were analyzed according to their randomized treatment group. |
| Arm/Group Title | Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID |
|---|---|---|---|---|
| Arm/Group Description | Participants received CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | Participants received CTP-543 4 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 8 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 12 mg tablets, twice daily for up to 24 weeks. |
| Measure Participants | 43 | 28 | 38 | 36 |
| Number [Percentage of participants] |
9.3
21.1%
|
21.4
71.3%
|
47.4
124.7%
|
58.3
157.6%
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Combined Placebo, Cohort 1: CTP-543 4 mg BID |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.177 |
| Comments | ||
| Method | Chi-squared | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Combined Placebo, Cohort 2: CTP-543 8 mg BID |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.001 |
| Comments | ||
| Method | Chi-squared | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Combined Placebo, Cohort 3: CTP-543 12 mg BID |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.001 |
| Comments | ||
| Method | Chi-squared | |
| Comments | ||
| Title | Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) |
|---|---|
| Description | An adverse event is any untoward medical occurrence that may appear or worsen in a participant during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the patient's health, including laboratory test values, regardless of etiology. Any worsening (ie, any clinically significant adverse change in the frequency or intensity of a pre-existing condition) should be considered an adverse event. TEAE is defined as any adverse event that occurs after administration of the first dose of study drug. |
| Time Frame | From first dose of study drug up to safety follow up at Week 28 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety Population included all participants who received study drug during the treatment period. Participants were analyzed according to the actual treatment received during the study. |
| Arm/Group Title | Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID |
|---|---|---|---|---|
| Arm/Group Description | Participants received CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | Participants received CTP-543 4 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 8 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 12 mg tablets, twice daily for up to 24 weeks. |
| Measure Participants | 44 | 29 | 38 | 36 |
| Count of Participants [Participants] |
31
70.5%
|
25
83.3%
|
31
81.6%
|
30
81.1%
|
Adverse Events
| Time Frame | From first dose of study drug up to safety follow up at Week 28 | |||||||
|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | All-cause mortality, Serious and other adverse events are reported for the Safety Population, included all participants who received study drug during the treatment period. Participants were analyzed according to the actual treatment received during the study. | |||||||
| Arm/Group Title | Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID | ||||
| Arm/Group Description | Participants received CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | Participants received CTP-543 4 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 8 mg tablets, twice daily for up to 24 weeks. | Participants received CTP-543 12 mg tablets, twice daily for up to 24 weeks. | ||||
All Cause Mortality |
||||||||
| Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/44 (0%) | 0/29 (0%) | 0/38 (0%) | 0/36 (0%) | ||||
Serious Adverse Events |
||||||||
| Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/44 (0%) | 0/29 (0%) | 0/38 (0%) | 1/36 (2.8%) | ||||
| Infections and infestations | ||||||||
| Cellulitis | 0/44 (0%) | 0/29 (0%) | 0/38 (0%) | 1/36 (2.8%) | ||||
Other (Not Including Serious) Adverse Events |
||||||||
| Combined Placebo | Cohort 1: CTP-543 4 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 3: CTP-543 12 mg BID | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 31/44 (70.5%) | 25/29 (86.2%) | 31/38 (81.6%) | 30/36 (83.3%) | ||||
| Blood and lymphatic system disorders | ||||||||
| Anemia | 0/44 (0%) | 0/29 (0%) | 3/38 (7.9%) | 3/36 (8.3%) | ||||
| Thrombocytosis | 0/44 (0%) | 0/29 (0%) | 2/38 (5.3%) | 0/36 (0%) | ||||
| Eye disorders | ||||||||
| Dry eye | 0/44 (0%) | 0/29 (0%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Gastrointestinal disorders | ||||||||
| Nausea | 4/44 (9.1%) | 4/29 (13.8%) | 4/38 (10.5%) | 1/36 (2.8%) | ||||
| Diarrhea | 3/44 (6.8%) | 3/29 (10.3%) | 1/38 (2.6%) | 0/36 (0%) | ||||
| Abdominal pain | 3/44 (6.8%) | 2/29 (6.9%) | 0/38 (0%) | 0/36 (0%) | ||||
| Vomiting | 0/44 (0%) | 2/29 (6.9%) | 0/38 (0%) | 1/36 (2.8%) | ||||
| General disorders | ||||||||
| Fatigue | 2/44 (4.5%) | 2/29 (6.9%) | 1/38 (2.6%) | 2/36 (5.6%) | ||||
| Infections and infestations | ||||||||
| Upper respiratory tract infection | 7/44 (15.9%) | 2/29 (6.9%) | 2/38 (5.3%) | 7/36 (19.4%) | ||||
| Nasopharyngitis | 1/44 (2.3%) | 3/29 (10.3%) | 3/38 (7.9%) | 9/36 (25%) | ||||
| Folliculitis | 0/44 (0%) | 3/29 (10.3%) | 2/38 (5.3%) | 1/36 (2.8%) | ||||
| Sinusitis | 3/44 (6.8%) | 0/29 (0%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Influenza | 0/44 (0%) | 1/29 (3.4%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Body tinea | 0/44 (0%) | 2/29 (6.9%) | 0/38 (0%) | 0/36 (0%) | ||||
| Gastroenteritis viral | 0/44 (0%) | 0/29 (0%) | 2/38 (5.3%) | 0/36 (0%) | ||||
| Viral upper respiratory tract infection | 0/44 (0%) | 2/29 (6.9%) | 0/38 (0%) | 0/36 (0%) | ||||
| Injury, poisoning and procedural complications | ||||||||
| Contusion | 0/44 (0%) | 0/29 (0%) | 3/38 (7.9%) | 0/36 (0%) | ||||
| Sunburn | 0/44 (0%) | 0/29 (0%) | 2/38 (5.3%) | 0/36 (0%) | ||||
| Investigations | ||||||||
| Blood creatine phosphokinase increased | 1/44 (2.3%) | 3/29 (10.3%) | 2/38 (5.3%) | 1/36 (2.8%) | ||||
| Red blood cell count decreased | 0/44 (0%) | 0/29 (0%) | 3/38 (7.9%) | 2/36 (5.6%) | ||||
| Blood triglycerides increased | 1/44 (2.3%) | 0/29 (0%) | 3/38 (7.9%) | 0/36 (0%) | ||||
| Low density lipoprotein increased | 0/44 (0%) | 0/29 (0%) | 4/38 (10.5%) | 0/36 (0%) | ||||
| Neutrophil count decreased | 1/44 (2.3%) | 0/29 (0%) | 3/38 (7.9%) | 0/36 (0%) | ||||
| Hemoglobin decreased | 0/44 (0%) | 1/29 (3.4%) | 2/38 (5.3%) | 0/36 (0%) | ||||
| Amylase increased | 0/44 (0%) | 0/29 (0%) | 2/38 (5.3%) | 0/36 (0%) | ||||
| Aspartate aminotransferase increased | 0/44 (0%) | 2/29 (6.9%) | 0/38 (0%) | 0/36 (0%) | ||||
| Hematocrit decreased | 0/44 (0%) | 0/29 (0%) | 2/38 (5.3%) | 0/36 (0%) | ||||
| Musculoskeletal and connective tissue disorders | ||||||||
| Arthralgia | 2/44 (4.5%) | 0/29 (0%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Pain in extremity | 0/44 (0%) | 0/29 (0%) | 0/38 (0%) | 3/36 (8.3%) | ||||
| Nervous system disorders | ||||||||
| Headache | 4/44 (9.1%) | 5/29 (17.2%) | 10/38 (26.3%) | 7/36 (19.4%) | ||||
| Migraine | 1/44 (2.3%) | 0/29 (0%) | 0/38 (0%) | 3/36 (8.3%) | ||||
| Dysgeusia | 0/44 (0%) | 1/29 (3.4%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Psychiatric disorders | ||||||||
| Depressed mood | 0/44 (0%) | 0/29 (0%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Respiratory, thoracic and mediastinal disorders | ||||||||
| Cough | 0/44 (0%) | 4/29 (13.8%) | 1/38 (2.6%) | 2/36 (5.6%) | ||||
| Oropharyngeal pain | 1/44 (2.3%) | 3/29 (10.3%) | 1/38 (2.6%) | 0/36 (0%) | ||||
| Sinus congestion | 0/44 (0%) | 1/29 (3.4%) | 0/38 (0%) | 2/36 (5.6%) | ||||
| Skin and subcutaneous tissue disorders | ||||||||
| Acne | 2/44 (4.5%) | 4/29 (13.8%) | 4/38 (10.5%) | 6/36 (16.7%) | ||||
| Dermatitis contact | 1/44 (2.3%) | 2/29 (6.9%) | 0/38 (0%) | 1/36 (2.8%) | ||||
| Rosacea | 0/44 (0%) | 0/29 (0%) | 2/38 (5.3%) | 0/36 (0%) | ||||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
If an Investigator wants to publish study data or results, the publication or presentation must be provided to Concert for review at least 60 days in advance. If Concert needs to file a patent application prior to publication, the publication can be delayed up to 90 days from Sponsor providing notice to the investigator of such need.
Results Point of Contact
| Name/Title | Colleen E. Hamilton |
|---|---|
| Organization | Concert Pharmaceuticals, Inc. |
| Phone | 781-860-0045 |
| AAclinicaltrial_inquiries@concertpharma.com |
Responsible Party:
Concert Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT03137381
Other Study ID Numbers:
- CP543.2001
First Posted:
May 2, 2017
Last Update Posted:
Jul 19, 2022
Last Verified:
Jun 1, 2022