Alopecia Areata
Conditions
Keywords
Hair loss, alopecia areata, alopecia totalis, alopecia universalis, JAK inhibitors, topical hair growth
Brief summary
A phase 2 study to find out if the drug ruxolitinib Phosphate Cream is safe and has beneficial effects in people who have alopecia areata (partial or complete hair loss) when applied to the skin.
Interventions
DRUGPlacebo Cream
Sponsors
Incyte Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
Yes
Inclusion criteria
* Diagnosis of AA * Duration and extent of current episode of AA * Evidence of active hair loss
Exclusion criteria
* Evidence of diffuse, spontaneous terminal hair regrowth * Receipt of treatment known to potentially affect the course of AA within last month
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Up to Week 24 | Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss. |
| Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented) | Week 24 | Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Weeks 4, 8, 12, 18, and 24. | Number of participants achieving 90% or greater improvement in their SALT score (SALT90) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes. |
| Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3 | Baseline to Week 24 | Number of subjects achieving a Physician's Global Assessment (PGA) score of 3 or above at week 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). |
| Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. | Up to Week 24 | Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes. |
| Part B: Mean Change From Baseline in SALT Score | Weeks 4, 8, 12, 18, and 24. | Severity of Alopecia Tool Score (SALT) calculation is based on a scoring system. The scalp is divided into the following 4 areas: 1) Vertex: 40% (0.4) of scalp surface area, 2) Right profile of scalp: 18% (0.18) of scalp surface area, 3) Left profile of scalp: 18% (0.18) of scalp surface area, and 4) Posterior aspect of scalp: 24% (0.24) of scalp surface area. The percentage of hair loss in any of these areas is the percentage hair loss multiplied by percent surface area of the scalp in that area. The SALT score is the sum of percentage of hair loss in all the above-mentioned areas, so a lower number indicates a better outcome. The reported SALT score range is from a minimum of 0 (no hair loss) to a maximum of 100 (100% hair loss). |
| Part A and B : Number of Treatment-emergent Adverse Events | Up to 100 weeks | Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment |
| Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Weeks 4, 8, 12, and 18. | Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes |
| Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Week 24 | Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes. |
Countries
United States
Participant flow
Recruitment details
This study was conducted at 17 study centers in the United States.
Pre-assignment details
Twelve participants were enrolled at 4 sites and were evaluable in the Part A treatment period; In Part B, 78 additional participants were enrolled at 17 sites.
Participants by arm
| Arm | Count |
|---|---|
| Part A: Ruxolitinib Cream Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks | 12 |
| Part B : Placebo Cream Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks | 39 |
| Part B : Ruxolitinib Cream Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks. | 39 |
| Total | 90 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 | 0 |
| Overall Study | Lost to Follow-up | 0 | 4 | 4 |
| Overall Study | Non compliance to study treatment | 0 | 1 | 0 |
| Overall Study | Other | 0 | 1 | 0 |
| Overall Study | Study Terminated by Sponsor | 2 | 13 | 12 |
| Overall Study | Withdrawal by Subject | 3 | 19 | 21 |
Baseline characteristics
| Characteristic | Part A: Ruxolitinib Cream | Part B : Placebo Cream | Part B : Ruxolitinib Cream | Total |
|---|---|---|---|---|
| Age, Continuous | 47.6 Years STANDARD_DEVIATION 13.17 | 44.3 Years STANDARD_DEVIATION 15.92 | 42.4 Years STANDARD_DEVIATION 14.69 | 43.4 Years STANDARD_DEVIATION 15.25 |
| Race/Ethnicity, Customized Asian | 1 Participants | 1 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants | 5 Participants | 4 Participants | 9 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 1 Participants | 3 Participants | 5 Participants | 9 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 11 Participants | 36 Participants | 33 Participants | 80 Participants |
| Race/Ethnicity, Customized Not Reported | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Other | 0 Participants | 0 Participants | 5 Participants | 5 Participants |
| Race/Ethnicity, Customized Unknown | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized White or Caucasian | 11 Participants | 33 Participants | 29 Participants | 73 Participants |
| Sex: Female, Male Female | 9 Participants | 24 Participants | 27 Participants | 60 Participants |
| Sex: Female, Male Male | 3 Participants | 15 Participants | 12 Participants | 30 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 38 | 0 / 83 |
| other Total, other adverse events | 14 / 38 | 31 / 83 |
| serious Total, serious adverse events | 0 / 38 | 4 / 83 |
Outcome results
Primary
Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented).
Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.
Time frame: Up to Week 24
Population: Part A evaluable population in treatment period includes all subjects exposed to at least 1 dose of study drug in that period.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: Ruxolitinib Cream | Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Week 4 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Week 8 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Week 12 | 3 Participants |
| Part A: Ruxolitinib Cream | Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Week 18 | 4 Participants |
| Part A: Ruxolitinib Cream | Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | Week 24 | 6 Participants |
Primary
Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented)
Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.
Time frame: Week 24
Population: Intent to Treat population in the Part B treatment period i.e all participants who are randomized
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented) | 5 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented) | 5 Participants |
Secondary
Part A and B : Number of Treatment-emergent Adverse Events
Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment
Time frame: Up to 100 weeks
Population: All enrolled subjects who received at least 1 dose of INCB018424 or Placebo.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: Ruxolitinib Cream | Part A and B : Number of Treatment-emergent Adverse Events | Treatment Period | 9 Participants |
| Part A: Ruxolitinib Cream | Part A and B : Number of Treatment-emergent Adverse Events | Extension Period | 7 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Number of Treatment-emergent Adverse Events | Treatment Period | 28 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Number of Treatment-emergent Adverse Events | Extension Period | 23 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Number of Treatment-emergent Adverse Events | Treatment Period | 23 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Number of Treatment-emergent Adverse Events | Extension Period | 22 Participants |
Secondary
Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair.
Number of participants achieving 90% or greater improvement in their SALT score (SALT90) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.
Time frame: Weeks 4, 8, 12, 18, and 24.
Population: Part A evaluable population in treatment period includes all subjects exposed to at least 1 dose of study drug in that period.~Intent to Treat population in the Part B treatment period i.e all participants who are randomized.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 18 | 1 Participants |
| Part A: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 12 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week4 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 8 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 24 | 2 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 12 | 0 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week4 | 0 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 8 | 0 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 18 | 0 Participants |
| Part B: Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 24 | 0 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 24 | 2 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 18 | 2 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week4 | 0 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 12 | 1 Participants |
| Part B : Ruxolitinib Cream | Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. | Week 8 | 0 Participants |
Secondary
Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair.
Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.
Time frame: Up to Week 24
Population: The subgroup analysis was performed on subjects exposed to at least 1 dose of study drug in that period with a baseline SALT score of 50-100%.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: Ruxolitinib Cream | Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. | Week 4 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. | Week 8 | 0 Participants |
| Part A: Ruxolitinib Cream | Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. | Week 12 | 2 Participants |
| Part A: Ruxolitinib Cream | Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. | Week 18 | 3 Participants |
| Part A: Ruxolitinib Cream | Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. | Week 24 | 4 Participants |
Secondary
Part B: Mean Change From Baseline in SALT Score
Severity of Alopecia Tool Score (SALT) calculation is based on a scoring system. The scalp is divided into the following 4 areas: 1) Vertex: 40% (0.4) of scalp surface area, 2) Right profile of scalp: 18% (0.18) of scalp surface area, 3) Left profile of scalp: 18% (0.18) of scalp surface area, and 4) Posterior aspect of scalp: 24% (0.24) of scalp surface area. The percentage of hair loss in any of these areas is the percentage hair loss multiplied by percent surface area of the scalp in that area. The SALT score is the sum of percentage of hair loss in all the above-mentioned areas, so a lower number indicates a better outcome. The reported SALT score range is from a minimum of 0 (no hair loss) to a maximum of 100 (100% hair loss).
Time frame: Weeks 4, 8, 12, 18, and 24.
Population: Intent to Treat population in the Part B treatment period i.e all participants who are randomized.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Part A: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 8 | 2.13 Units on a Scale | Standard Error 2.233 |
| Part A: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 18 | -0.01 Units on a Scale | Standard Error 3.55 |
| Part A: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 12 | 1.23 Units on a Scale | Standard Error 2.956 |
| Part A: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 24 | -1.68 Units on a Scale | Standard Error 3.886 |
| Part A: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 4 | 1.02 Units on a Scale | Standard Error 1.577 |
| Part B: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 24 | -3.92 Units on a Scale | Standard Error 3.794 |
| Part B: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 4 | 0.13 Units on a Scale | Standard Error 1.534 |
| Part B: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 8 | -1.11 Units on a Scale | Standard Error 2.16 |
| Part B: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 12 | -2.55 Units on a Scale | Standard Error 2.873 |
| Part B: Ruxolitinib Cream | Part B: Mean Change From Baseline in SALT Score | Change from Baseline to Week 18 | -4.16 Units on a Scale | Standard Error 3.461 |
Secondary
Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented).
Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes
Time frame: Weeks 4, 8, 12, and 18.
Population: Intent to Treat population in the Part B treatment period i.e all participants who are randomized.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 12 | 1 Participants |
| Part A: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 4 | 0 Participants |
| Part A: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 18 | 4 Participants |
| Part A: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 8 | 0 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 18 | 5 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 4 | 0 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 12 | 3 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). | Week 8 | 2 Participants |
Secondary
Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented).
Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.
Time frame: Week 24
Population: The subgroup analysis was performed on subjects exposed to at least 1 dose of study drug in that period with a baseline SALT score of 50-100%.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A: Ruxolitinib Cream | Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | 2 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). | 4 Participants |
Secondary
Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3
Number of subjects achieving a Physician's Global Assessment (PGA) score of 3 or above at week 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).
Time frame: Baseline to Week 24
Population: Intent to Treat population in the Part B treatment period i.e all participants who are randomized. Participants dropped out of study by week 24 were excluded from analysis.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A: Ruxolitinib Cream | Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3 | 5 Participants |
| Part B: Ruxolitinib Cream | Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3 | 2 Participants |