Trial Outcomes & Findings for Pilot Study of the Safety and Efficacy of Apremilast in Subjects With Moderate to Severe Alopecia Areata (NCT NCT02684123)
NCT ID: NCT02684123
Last Updated: 2023-11-07
Results Overview
Number of patients achieving 50% or greater improvement in their Severity of Alopecia Tool (SALT) score (SALT50) at Week 24 SALT50 is defined as the percent of patients achieving \>=50% reduction in SALT score compared to baseline. Scalp is divided into 4 areas namely, Vertex - 40% (0.4) of scalp surface area; right profile of scalp - 18% (0.18) of scalp surface area; left profile of scalp - 18% (0.18) of scalp surface area; Posterior aspect of scalp - 24% (0.24) of scalp surface area. Percentage of hair loss in any of these areas is percentage hair loss multiplied by percent surface area of the scalp in that area. SALT score is the sum of percentage of hair loss in all above mentioned areas.
COMPLETED
NA
30 participants
Baseline and Week 24
2023-11-07
Participant Flow
Participant milestones
| Measure |
Apremilast
30mg twice daily
|
Placebo
pills twice daily
|
|---|---|---|
|
Phase 1
STARTED
|
20
|
10
|
|
Phase 1
COMPLETED
|
12
|
9
|
|
Phase 1
NOT COMPLETED
|
8
|
1
|
|
Eligibility Determination for Phase 2
STARTED
|
12
|
9
|
|
Eligibility Determination for Phase 2
COMPLETED
|
1
|
9
|
|
Eligibility Determination for Phase 2
NOT COMPLETED
|
11
|
0
|
|
Phase 2
STARTED
|
1
|
9
|
|
Phase 2
COMPLETED
|
0
|
1
|
|
Phase 2
NOT COMPLETED
|
1
|
8
|
Reasons for withdrawal
| Measure |
Apremilast
30mg twice daily
|
Placebo
pills twice daily
|
|---|---|---|
|
Phase 1
Lack of Efficacy
|
5
|
0
|
|
Phase 1
Adverse Event
|
3
|
0
|
|
Phase 1
Lost to Follow-up
|
0
|
1
|
|
Phase 2
Lack of Efficacy
|
1
|
8
|
Baseline Characteristics
Pilot Study of the Safety and Efficacy of Apremilast in Subjects With Moderate to Severe Alopecia Areata
Baseline characteristics by cohort
| Measure |
Apremilast
n=20 Participants
30mg twice daily
|
Placebo
n=10 Participants
pills twice daily
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
37.1 years
STANDARD_DEVIATION 14.4 • n=5 Participants
|
44.15 years
STANDARD_DEVIATION 16.9 • n=7 Participants
|
41.8 years
STANDARD_DEVIATION 16.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Duration of AA
|
4.3 years
STANDARD_DEVIATION 2.7 • n=5 Participants
|
6.6 years
STANDARD_DEVIATION 2.8 • n=7 Participants
|
5.1 years
STANDARD_DEVIATION 2.9 • n=5 Participants
|
|
SALT score
|
88.0 units on a scale
STANDARD_DEVIATION 19.8 • n=5 Participants
|
87.7 units on a scale
STANDARD_DEVIATION 16.1 • n=7 Participants
|
87.8 units on a scale
STANDARD_DEVIATION 17.1 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 24Number of patients achieving 50% or greater improvement in their Severity of Alopecia Tool (SALT) score (SALT50) at Week 24 SALT50 is defined as the percent of patients achieving \>=50% reduction in SALT score compared to baseline. Scalp is divided into 4 areas namely, Vertex - 40% (0.4) of scalp surface area; right profile of scalp - 18% (0.18) of scalp surface area; left profile of scalp - 18% (0.18) of scalp surface area; Posterior aspect of scalp - 24% (0.24) of scalp surface area. Percentage of hair loss in any of these areas is percentage hair loss multiplied by percent surface area of the scalp in that area. SALT score is the sum of percentage of hair loss in all above mentioned areas.
Outcome measures
| Measure |
Apremilast
n=1 Participants
30mg twice daily
|
Placebo
n=9 Participants
pills twice daily
|
|---|---|---|
|
Number of Patients With SALT50
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline and week 4, 8, 12, 16, 20, 24Population: Data for participants who returned for particular visits.
Mean percent change in SALT score at 4, 8, 12, 16, 20, and 24 weeks as compared to baseline
Outcome measures
| Measure |
Apremilast
n=20 Participants
30mg twice daily
|
Placebo
n=10 Participants
pills twice daily
|
|---|---|---|
|
Mean Change in SALT Score
Week 4
|
-0.13 Mean percent change
Standard Error 0.56
|
-0.11 Mean percent change
Standard Error 0.11
|
|
Mean Change in SALT Score
Week 8
|
-2.39 Mean percent change
Standard Error 2.58
|
-2.86 Mean percent change
Standard Error 1.47
|
|
Mean Change in SALT Score
Week 12
|
-0.99 Mean percent change
Standard Error 1.52
|
-4.07 Mean percent change
Standard Error 2.21
|
|
Mean Change in SALT Score
Week 16
|
-8.15 Mean percent change
Standard Error 7.37
|
-10.55 Mean percent change
Standard Error 6.68
|
|
Mean Change in SALT Score
Week 20
|
1.17 Mean percent change
Standard Error 2.00
|
-9.92 Mean percent change
Standard Error 6.48
|
|
Mean Change in SALT Score
Week 24
|
-1.45 Mean percent change
Standard Error 5.39
|
-9.01 Mean percent change
Standard Error 6.37
|
SECONDARY outcome
Timeframe: Week 24 and Week 48Population: Only those who completed treatments as respective week analyzed.
Number of subjects achieving an alopecia areata Physician's Global Assessment (aaPGA) score of 3 or above at Weeks 24 (0, no regrowth; 1, \<25% of regrowth; 2, 25%-49% of regrowth; 3, 50%-74% of regrowth; 4, 75%-99% of re- growth; 5, 100% of regrowth).
Outcome measures
| Measure |
Apremilast
n=1 Participants
30mg twice daily
|
Placebo
n=9 Participants
pills twice daily
|
|---|---|---|
|
Phase 2: Number of Patients Achieving aaPGA Score of 3 or Above
week 24
|
1 Participants
|
1 Participants
|
|
Phase 2: Number of Patients Achieving aaPGA Score of 3 or Above
week 48
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Week 24 and Weeks 48Population: Data for participants who returned for particular visits
Change from Baseline in the Alopecia Areata Symptom Impact Scale (AASIS) at Weeks 24 and Week 48 The AASIS is a 13-items disease-specific patient-reported outcomes measure that asks participants about symptoms related to alopecia areata and how these symptoms interfere with daily functioning. Total scale ranges from 0-130, with higher score indicating more symptoms.
Outcome measures
| Measure |
Apremilast
n=19 Participants
30mg twice daily
|
Placebo
n=10 Participants
pills twice daily
|
|---|---|---|
|
Phase 2: Change in AASIS
Baseline
|
56.79 score on a scale
Standard Deviation 30.44
|
51.4 score on a scale
Standard Deviation 25.68
|
|
Phase 2: Change in AASIS
Week 24
|
48.0 score on a scale
Standard Deviation 29.58
|
53.57 score on a scale
Standard Deviation 23.59
|
|
Phase 2: Change in AASIS
week 48
|
—
|
75.67 score on a scale
Standard Deviation 3.79
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Weeks 48Population: Data for participants who returned for particular visits
Change from baseline in the Alopecia Areata Quality of Life questionnaire (AA-QoL) at Weeks 24 and Weeks 48. Total scale from 0 to 100, with higher score indicating better quality of life.
Outcome measures
| Measure |
Apremilast
n=20 Participants
30mg twice daily
|
Placebo
n=10 Participants
pills twice daily
|
|---|---|---|
|
Phase 2: AA-QoL
Baseline
|
30.75 score on a scale
Standard Deviation 11.88
|
34.0 score on a scale
Standard Deviation 20.93
|
|
Phase 2: AA-QoL
Week 24
|
29.55 score on a scale
Standard Deviation 11.52
|
27.14 score on a scale
Standard Deviation 11.55
|
|
Phase 2: AA-QoL
week 48
|
—
|
35.33 score on a scale
Standard Deviation 2.08
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Weeks 48Population: Data for participants who returned for particular visits
Semiquantitative score using SALT subclasses (0, no hair loss; 1, \<25% hair loss; 2, 25%-49% hair loss; 3, 50%-74% hair loss; 4, 75%-99% hair loss; 5, 100% hair loss) at week 24 and week 48 compared to baseline
Outcome measures
| Measure |
Apremilast
n=20 Participants
30mg twice daily
|
Placebo
n=10 Participants
pills twice daily
|
|---|---|---|
|
Phase 2: Semiquantitative Score
Baseline
|
4.35 score on a scale
Standard Deviation 0.75
|
4.3 score on a scale
Standard Deviation 0.82
|
|
Phase 2: Semiquantitative Score
Week 24
|
4.42 score on a scale
Standard Deviation 1.16
|
4.1 score on a scale
Standard Deviation 1.27
|
|
Phase 2: Semiquantitative Score
week 48
|
—
|
3.67 score on a scale
Standard Deviation 2.31
|
Adverse Events
Apremilast
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Apremilast
n=20 participants at risk
30mg twice daily
|
Placebo
n=10 participants at risk
pills twice daily
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
20.0%
4/20 • 48 weeks
|
30.0%
3/10 • 48 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
15.0%
3/20 • 48 weeks
|
10.0%
1/10 • 48 weeks
|
|
Infections and infestations
Upper respiratory tract infections
|
5.0%
1/20 • 48 weeks
|
10.0%
1/10 • 48 weeks
|
|
General disorders
Fatigue
|
0.00%
0/20 • 48 weeks
|
10.0%
1/10 • 48 weeks
|
|
Skin and subcutaneous tissue disorders
Tinea pedis
|
5.0%
1/20 • 48 weeks
|
0.00%
0/10 • 48 weeks
|
|
Musculoskeletal and connective tissue disorders
Diffuse arthralgias
|
5.0%
1/20 • 48 weeks
|
0.00%
0/10 • 48 weeks
|
|
General disorders
Headache
|
5.0%
1/20 • 48 weeks
|
0.00%
0/10 • 48 weeks
|
|
Nervous system disorders
Migraine
|
5.0%
1/20 • 48 weeks
|
0.00%
0/10 • 48 weeks
|
|
Cardiac disorders
Hypertension
|
5.0%
1/20 • 48 weeks
|
0.00%
0/10 • 48 weeks
|
|
Infections and infestations
Acute sinusitis
|
0.00%
0/20 • 48 weeks
|
10.0%
1/10 • 48 weeks
|
Additional Information
Dr. Emma Guttman-Yassky
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place