Alopecia Areata
Conditions
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.
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.
Interventions
DRUGCTP-543
Administered as tablets.
Administered as tablets.
Sponsors
Concert Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
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 details
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.
Participants by arm
| Arm | Count |
|---|---|
| Combined Placebo Participants received CTP-543 matched placebo tablets, twice daily for up to 24 weeks in Cohorts 1, 2, and 3. | 44 |
| Cohort 1: CTP-543 4 mg BID Participants received CTP-543 4 mg tablets, twice daily for up to 24 weeks. | 30 |
| Cohort 2: CTP-543 8 mg BID Participants received CTP-543 8 mg tablets, twice daily for up to 24 weeks. | 38 |
| Cohort 3: CTP-543 12 mg BID Participants received CTP-543 12 mg tablets, twice daily for up to 24 weeks. | 37 |
| Total | 149 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Overall Study | Adverse Event | 3 | 0 | 2 | 0 |
| Overall Study | Lost to Follow-up | 2 | 1 | 1 | 0 |
| Overall Study | Pregnancy | 0 | 0 | 1 | 0 |
| Overall Study | Protocol deviation | 1 | 1 | 0 | 1 |
| Overall Study | Withdrawal by Investigator | 0 | 1 | 1 | 0 |
| Overall Study | Withdrawal by Subject | 3 | 4 | 3 | 0 |
Baseline characteristics
| Characteristic | Combined Placebo | Total | Cohort 3: CTP-543 12 mg BID | Cohort 2: CTP-543 8 mg BID | Cohort 1: CTP-543 4 mg BID |
|---|---|---|---|---|---|
| Age, Continuous | 37.8 years STANDARD_DEVIATION 13.5 | 36.8 years STANDARD_DEVIATION 12.85 | 35.8 years STANDARD_DEVIATION 12.37 | 37.3 years STANDARD_DEVIATION 14.18 | 35.7 years STANDARD_DEVIATION 11.01 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 5 Participants | 15 Participants | 3 Participants | 4 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 39 Participants | 132 Participants | 34 Participants | 33 Participants | 26 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 2 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 2 Participants | 10 Participants | 4 Participants | 2 Participants | 2 Participants |
| Race/Ethnicity, Customized Black or African American | 7 Participants | 19 Participants | 3 Participants | 7 Participants | 2 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 1 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Other | 1 Participants | 5 Participants | 0 Participants | 3 Participants | 1 Participants |
| Race/Ethnicity, Customized White | 33 Participants | 114 Participants | 30 Participants | 26 Participants | 25 Participants |
| Sex: Female, Male Female | 29 Participants | 105 Participants | 28 Participants | 26 Participants | 22 Participants |
| Sex: Female, Male Male | 15 Participants | 44 Participants | 9 Participants | 12 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 44 | 0 / 29 | 0 / 38 | 0 / 36 |
| other Total, other adverse events | 31 / 44 | 25 / 29 | 31 / 38 | 30 / 36 |
| serious Total, serious adverse events | 0 / 44 | 0 / 29 | 0 / 38 | 1 / 36 |
Outcome results
Primary
Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE)
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
Population: 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 | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Combined Placebo | Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) | 31 Participants |
| Cohort 1: CTP-543 4 mg BID | Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) | 25 Participants |
| Cohort 2: CTP-543 8 mg BID | Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) | 31 Participants |
| Cohort 3: CTP-543 12 mg BID | Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) | 30 Participants |
Primary
Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at 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.
Time frame: Week 24
Population: 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 | Measure | Value (NUMBER) |
|---|---|---|
| Combined Placebo | Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 | 9.3 Percentage of participants |
| Cohort 1: CTP-543 4 mg BID | Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 | 21.4 Percentage of participants |
| Cohort 2: CTP-543 8 mg BID | Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 | 47.4 Percentage of participants |
| Cohort 3: CTP-543 12 mg BID | Percentage of Participants Achieving at Least a 50% Relative Reduction in Severity of Alopecia Tool (SALT) Score From Baseline at Week 24 | 58.3 Percentage of participants |
p-value: 0.177Chi-squared
p-value: <0.001Chi-squared
p-value: <0.001Chi-squared