Non-segmental Vitiligo
Conditions
Keywords
Vitiligo, non-segmental, JAK inhibitor
Brief summary
The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in adolescent and adult participants with non-segmental vitiligo for whom total body involved vitiligo area (facial and nonfacial) does not exceed 10% body surface area (BSA).
Interventions
DRUGRuxolitinib cream
Ruxolitinib cream is a topical formulation applied as a thin film to affected areas.
DRUGVehicle
Vehicle cream is a topical formulation applied as a thin film to affected areas.
Sponsors
Incyte Corporation
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Intervention model description
Participants will receive ruxolitinib cream or vehicle for 24 weeks, after which they will be offered the opportunity to continue in the treatment extension period. Participants initially randomized to vehicle will be crossed over to active drug, and participants treated with ruxolitinib cream will receive an additional 28 weeks of treatment with ruxolitinib cream.
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Clinical diagnosis of non-segmental vitiligo with depigmented area including ≥ 0.5% BSA on the face, ≥ 0.5 F-VASI, ≥ 3% BSA on nonfacial areas, ≥ 3 T-VASI, and total body vitiligo area (facial and nonfacial) not exceeding 10% BSA. * Agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit. Over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted. * Must be willing to take appropriate contraceptive measures to avoid pregnancy or fathering a child for the duration of study participation. Key
Exclusion criteria
* No pigmented hair within any of the vitiligo areas on the face. * Other forms of vitiligo (eg, segmental) or other differential diagnosis of vitiligo or other skin depigmentation disorders (eg, piebaldism, pityriasis alba, leprosy, postinflammatory hypopigmentation, progressive macule hypomelanosis, nevus anemicus, chemical leukoderma, and tinea versicolor). * Have used depigmentation treatments (eg, monobenzone) for past treatment of vitiligo or other pigmented areas. * Use of protocol-defined treatments within the indicated washout period before baseline.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24 | Baseline; Week 24 | An F-VASI75 responder achieved at least 75% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving a ≥ 90% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24 | Baseline; Week 24 | An F-VASI90 responder achieved at least 90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). |
| Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24 | Baseline; Week 24 | A T-VASI50 responder achieved at least 50% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). |
| Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24 | Baseline; Week 24 | The VNS is a patient-reported measure of vitiligo treatment success that is rated on a 5-point scale. The Baseline facial photograph was shown to the participants for reference, and a mirror was provided for the participants to assess the vitiligo on their face. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable. |
| Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24 | Baseline; Week 24 | F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area Face was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area Face did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-Baseline (BL) value minus BL value\]/BL value) X 100. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period | from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 24) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE reported for the first time or the worsening of a pre-existing event after the first application of study drug. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period | from the completion of the Week 24 assessments until at least 30 days after the last application of study drug (up to Week 52 + 30 days) | An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE reported for the first time or the worsening of a pre-existing event after the first application of study drug. |
| Percentage of Participants Achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24 | Baseline; Week 24 | An F-VASI25 responder achieved at least 25% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). |
| Percentage Change From Baseline in T-BSA at Week 52 | Baseline; Week 52 | T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
| Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | Baseline; Week 52 | An F-VASI25/50/75/90 responder achieved at least 25/50/75/90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). |
| Percentage Change From Baseline in F-VASI at Week 24 | Baseline; Week 24 | F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
| Percentage Change From Baseline in F-VASI at Week 52 | Baseline; Week 52 | F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
| Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24 | Baseline; Week 24 | An F-VASI50 responder achieved at least 50% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). |
| Percentage Change From Baseline in T-VASI at Week 24 | Baseline; Week 24 | T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
| Percentage Change From Baseline in T-VASI at Week 52 | Baseline; Week 52 | T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
| Percentage Change From Baseline in T-BSA at Week 24 | Baseline; Week 24 | T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
| Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | Baseline; Week 24 | A T-VASI25/75/90 responder achieved at least 25/75/90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). |
| Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | Baseline; Week 52 | A T-VASI25/50/75/90 responder achieved ≥25/50/75/90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). |
| Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Baseline; Week 24 and Week 52 | The VNS is a patient-reported measure of vitiligo treatment success that is rated on a 5-point scale. The Baseline facial photograph was shown to the participants for reference, and a mirror was provided for the participants to assess the vitiligo on their face. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable. |
| Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24 | Baseline; Week 24 | The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. Each question is scored as: very much = 3; a lot = 2; a little = 1; not at all = 0; not relevant = 0. For Question 7, Prevented work or studying = 3. The DLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. |
| Change From Baseline in DLQI at Week 52 | Baseline; Week 52 | The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. Each question is scored as: very much = 3; a lot = 2; a little = 1; not at all = 0; not relevant = 0. For Question 7, Prevented work or studying = 3. The DLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. |
| Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Baseline; Week 24 and Week 52 | The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. The CDLQI is the youth/children's version of the DLQI and was completed by adolescents aged ≥ 12 years to \< 16 years. Each question is scored as: very much = 3; quite a lot = 2; only a little = 1; not at all = 0; question unanswered = 0. For Question 7: Prevented school = 3. The CDLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. |
| Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 | pre-dose at Weeks 4, 24, and 40 | Trough plasma concentration was defined as the measurement of the plasma concentration of ruxolitinib before drug application. |
| Percentage Change From Baseline in F-BSA at Week 52 | Baseline; Week 52 | F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area Face was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area Face did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100. |
Countries
Bulgaria, Canada, France, Germany, Italy, Netherlands, Poland, Spain, United States
Participant flow
Recruitment details
This study was conducted at 49 study centers in North America and Europe.
Pre-assignment details
A total of 344 participants were randomized into the study. Of the 344 randomized participants (Intent-to-Treat Population), 343 applied study drug at least once (Safety Population); 297 participants applied ruxolitinib cream at least once during the Treatment-Extension (TE) Period (TE Evaluable Population).
Participants by arm
| Arm | Count |
|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks. | 228 |
| Double-Blind Period: Vehicle Cream BID Participants applied matching vehicle cream BID for 24 weeks. | 115 |
| Total | 343 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| 24-Week Double-Blind Period | Adverse Event | 2 | 0 | 0 | 0 |
| 24-Week Double-Blind Period | Discontinued Treatment due to COVID-19 Pandemic | 3 | 1 | 0 | 0 |
| 24-Week Double-Blind Period | Lack of Efficacy | 0 | 1 | 0 | 0 |
| 24-Week Double-Blind Period | Lost to Follow-up | 11 | 6 | 0 | 0 |
| 24-Week Double-Blind Period | Non-compliance with Attending Study Visits | 1 | 0 | 0 | 0 |
| 24-Week Double-Blind Period | Not Treated | 1 | 0 | 0 | 0 |
| 24-Week Double-Blind Period | Physician Decision | 1 | 0 | 0 | 0 |
| 24-Week Double-Blind Period | Pregnancy | 1 | 0 | 0 | 0 |
| 24-Week Double-Blind Period | Unable to Attend Study Visits Due to Travel | 0 | 1 | 0 | 0 |
| 24-Week Double-Blind Period | Withdrawal by Subject | 10 | 8 | 0 | 0 |
| 28-Week Treatment Extension Period | Adverse Event | 0 | 0 | 1 | 0 |
| 28-Week Treatment Extension Period | Lack of Efficacy | 0 | 0 | 1 | 0 |
| 28-Week Treatment Extension Period | Lost to Follow-up | 0 | 0 | 9 | 10 |
| 28-Week Treatment Extension Period | Unable to Perform Safety Follow-up Visit | 0 | 0 | 1 | 0 |
| 28-Week Treatment Extension Period | Withdrawal by Subject | 0 | 0 | 9 | 9 |
Baseline characteristics
| Characteristic | Total | Double-Blind Period: Ruxolitinib Cream 1.5% BID | Double-Blind Period: Vehicle Cream BID |
|---|---|---|---|
| Age, Continuous | 38.9 years STANDARD_DEVIATION 14.25 | 38.4 years STANDARD_DEVIATION 15.22 | 39.8 years STANDARD_DEVIATION 12.12 |
| Age Continuous, without Noncompliant Site | 38.7 years STANDARD_DEVIATION 14.46 | 38.4 years STANDARD_DEVIATION 15.44 | 39.4 years STANDARD_DEVIATION 12.29 |
| Ethnicity, without Noncompliant Site Captured as Other | 2 Participants | 2 Participants | 0 Participants |
| Ethnicity, without Noncompliant Site Hispanic or Latino | 79 Participants | 49 Participants | 30 Participants |
| Ethnicity, without Noncompliant Site Not Hispanic or Latino | 245 Participants | 169 Participants | 76 Participants |
| Ethnicity, without Noncompliant Site Not Reported | 5 Participants | 2 Participants | 3 Participants |
| Face Vitiligo Area Scoring Index (F-VASI) | 0.878 scores on a scale STANDARD_DEVIATION 0.5245 | 0.900 scores on a scale STANDARD_DEVIATION 0.5248 | 0.834 scores on a scale STANDARD_DEVIATION 0.5233 |
| Facial Body Surface Area (F-BSA) Involvement | 0.96 percentage of facial surface area STANDARD_DEVIATION 0.569 | 0.98 percentage of facial surface area STANDARD_DEVIATION 0.57 | 0.92 percentage of facial surface area STANDARD_DEVIATION 0.569 |
| F-BSA Involvement, without Noncompliant Site | 0.96 percentage of facial surface area STANDARD_DEVIATION 0.575 | 0.98 percentage of facial surface area STANDARD_DEVIATION 0.571 | 0.92 percentage of facial surface area STANDARD_DEVIATION 0.582 |
| F-VASI, without Noncompliant Site | 0.877 scores on a scale STANDARD_DEVIATION 0.5285 | 0.898 scores on a scale STANDARD_DEVIATION 0.5256 | 0.834 scores on a scale STANDARD_DEVIATION 0.5342 |
| Gender, without Noncompliant Site Female | 168 Participants | 110 Participants | 58 Participants |
| Gender, without Noncompliant Site Male | 163 Participants | 112 Participants | 51 Participants |
| Race/Ethnicity, Customized American Indian/Alaska Native | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Arabic | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Arab/North-African | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Argentinian | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 19 Participants | 12 Participants | 7 Participants |
| Race/Ethnicity, Customized Black/African American | 17 Participants | 12 Participants | 5 Participants |
| Race/Ethnicity, Customized Brazilian | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Cape Verdean | 2 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Captured as Other | 2 Participants | 2 Participants | 0 Participants |
| Race/Ethnicity, Customized Dominican Republic | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Guyana | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 82 Participants | 50 Participants | 32 Participants |
| Race/Ethnicity, Customized Indo-Caribbean | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Jordanian | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Mexican | 3 Participants | 2 Participants | 1 Participants |
| Race/Ethnicity, Customized Middle Eastern | 3 Participants | 3 Participants | 0 Participants |
| Race/Ethnicity, Customized Multi-National | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Native Hawaiian/Pacific Islander | 2 Participants | 2 Participants | 0 Participants |
| Race/Ethnicity, Customized North African | 2 Participants | 2 Participants | 0 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 254 Participants | 174 Participants | 80 Participants |
| Race/Ethnicity, Customized Not Reported | 5 Participants | 2 Participants | 3 Participants |
| Race/Ethnicity, Customized Persian | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized White | 275 Participants | 182 Participants | 93 Participants |
| Race/Ethnicity, Customized White/Black/Asian | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized White/Caucasian and Asian | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site American Indian/Alaska Native | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site Arabic | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site Arab/North-African | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site Argentinian | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site Asian | 17 Participants | 11 Participants | 6 Participants |
| Race, without Noncompliant Site Black/African American | 16 Participants | 11 Participants | 5 Participants |
| Race, without Noncompliant Site Brazilian | 2 Participants | 0 Participants | 2 Participants |
| Race, without Noncompliant Site Cape Verdean | 2 Participants | 1 Participants | 1 Participants |
| Race, without Noncompliant Site Dominican Republic | 1 Participants | 0 Participants | 1 Participants |
| Race, without Noncompliant Site Guyana | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site Indo-Caribbean | 1 Participants | 0 Participants | 1 Participants |
| Race, without Noncompliant Site Jordanian | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site Mexican | 3 Participants | 2 Participants | 1 Participants |
| Race, without Noncompliant Site Middle Eastern | 3 Participants | 3 Participants | 0 Participants |
| Race, without Noncompliant Site Multi-National | 1 Participants | 0 Participants | 1 Participants |
| Race, without Noncompliant Site Native Hawaiian/Pacific Islander | 2 Participants | 2 Participants | 0 Participants |
| Race, without Noncompliant Site North African | 2 Participants | 2 Participants | 0 Participants |
| Race, without Noncompliant Site Not Reported | 6 Participants | 3 Participants | 3 Participants |
| Race, without Noncompliant Site Persian | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site White | 266 Participants | 178 Participants | 88 Participants |
| Race, without Noncompliant Site White/Black/Asian | 1 Participants | 1 Participants | 0 Participants |
| Race, without Noncompliant Site White/Caucasian and Asian | 1 Participants | 1 Participants | 0 Participants |
| Sex: Female, Male Female | 172 Participants | 112 Participants | 60 Participants |
| Sex: Female, Male Male | 171 Participants | 116 Participants | 55 Participants |
| T-BSA Involvement, without Noncompliant Site | 7.47 percentage of total body surface area STANDARD_DEVIATION 2.031 | 7.38 percentage of total body surface area STANDARD_DEVIATION 2.025 | 7.66 percentage of total body surface area STANDARD_DEVIATION 2.04 |
| Total Body Surface Area (T-BSA) Involvement | 7.52 percentage of total body surface area STANDARD_DEVIATION 2.021 | 7.44 percentage of total body surface area STANDARD_DEVIATION 2.011 | 7.68 percentage of total body surface area STANDARD_DEVIATION 2.04 |
| Total Body Vitiligo Area Scoring Index (T-VASI) | 6.904 scores on a scale STANDARD_DEVIATION 2.1043 | 6.844 scores on a scale STANDARD_DEVIATION 2.0574 | 7.022 scores on a scale STANDARD_DEVIATION 2.1986 |
| T-VASI, without Noncompliant Site | 6.852 scores on a scale STANDARD_DEVIATION 2.0933 | 6.790 scores on a scale STANDARD_DEVIATION 2.0435 | 6.979 scores on a scale STANDARD_DEVIATION 2.1953 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 115 | 0 / 326 |
| other Total, other adverse events | 6 / 115 | 56 / 326 |
| serious Total, serious adverse events | 0 / 115 | 6 / 326 |
Outcome results
Primary
Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24
An F-VASI75 responder achieved at least 75% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Time frame: Baseline; Week 24
Population: Intent-to-Treat Population: all randomized participants. Treatment groups for this population were defined according to the treatment assignment at the time of randomization. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing F-VASI scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24 | 30.9 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24 | 11.4 percentage of participants |
p-value: 0.000495% CI: [1.737, 6.835]exact logistic regression
Secondary
Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods)
The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. The CDLQI is the youth/children's version of the DLQI and was completed by adolescents aged ≥ 12 years to \< 16 years. Each question is scored as: very much = 3; quite a lot = 2; only a little = 1; not at all = 0; question unanswered = 0. For Question 7: Prevented school = 3. The CDLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Time frame: Baseline; Week 24 and Week 52
Population: ITT Population: participants aged \< 16 years. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Baseline | 1.29 scores on a scale | Standard Deviation 2.201 |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24 | 0.00 scores on a scale | Standard Deviation 1.837 |
| Double-Blind Period: Vehicle Cream BID | Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Baseline | 6.33 scores on a scale | Standard Deviation 10.116 |
| Double-Blind Period: Vehicle Cream BID | Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24 | -2.33 scores on a scale | Standard Deviation 8.505 |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52 | 1.19 scores on a scale | Standard Deviation 4.665 |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52 | -1.00 scores on a scale | Standard Deviation 4.583 |
Secondary
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24
The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. Each question is scored as: very much = 3; a lot = 2; a little = 1; not at all = 0; not relevant = 0. For Question 7, Prevented work or studying = 3. The DLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Time frame: Baseline; Week 24
Population: ITT Population: participants aged ≥ 16 years. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed. MMRM model: (Response Variable = Treatment + Stratification Factors \[Skin Type Fitzpatrick scale Type I and II vs Type III, IV, V, and VI, Region North America/Europe\] + Visit + Treatment\*Visit).
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24 | -1.19 scores on a scale | Standard Error 0.3 |
| Double-Blind Period: Vehicle Cream BID | Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24 | -0.59 scores on a scale | Standard Error 0.41 |
p-value: 0.23595% CI: [-1.6, 0.39]mixed-effect model; repeated measurement
Secondary
Change From Baseline in DLQI at Week 52
The DLQI is a 10-question validated questionnaire for use in participants aged 16 years and over to measure how much the skin problem has affected the participant over the previous 7 days. Each question is scored as: very much = 3; a lot = 2; a little = 1; not at all = 0; not relevant = 0. For Question 7, Prevented work or studying = 3. The DLQI was calculated by summing the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Time frame: Baseline; Week 52
Population: ITT Population: participants aged ≥ 16 years. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Change From Baseline in DLQI at Week 52 | Baseline | 4.37 scores on a scale | Standard Deviation 4.485 |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Change From Baseline in DLQI at Week 52 | Week 52 | -0.84 scores on a scale | Standard Deviation 3.976 |
| Double-Blind Period: Vehicle Cream BID | Change From Baseline in DLQI at Week 52 | Baseline | 5.38 scores on a scale | Standard Deviation 4.876 |
| Double-Blind Period: Vehicle Cream BID | Change From Baseline in DLQI at Week 52 | Week 52 | -1.15 scores on a scale | Standard Deviation 4.264 |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE reported for the first time or the worsening of a pre-existing event after the first application of study drug.
Time frame: from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 24)
Population: Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. Treatment groups for this population were determined according to the actual treatment the participant applied on Day 1.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period | 114 Participants |
| Double-Blind Period: Vehicle Cream BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period | 39 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE could have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE reported for the first time or the worsening of a pre-existing event after the first application of study drug.
Time frame: from the completion of the Week 24 assessments until at least 30 days after the last application of study drug (up to Week 52 + 30 days)
Population: Treatment-Extension (TE) Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period | 82 Participants |
| Double-Blind Period: Vehicle Cream BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period | 38 Participants |
Secondary
Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24
F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area Face was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area Face did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-Baseline (BL) value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing F-BSA scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24 | -26.4 percentage change | Standard Error 2.57 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24 | -7.0 percentage change | Standard Error 3.82 |
p-value: <0.000195% CI: [-28.46, -10.45]ANCOVA
Secondary
Percentage Change From Baseline in F-BSA at Week 52
F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area Face was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area Face did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in F-BSA at Week 52 | -41.81 percentage change | Standard Deviation 35.694 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in F-BSA at Week 52 | -23.45 percentage change | Standard Deviation 46.36 |
Secondary
Percentage Change From Baseline in F-VASI at Week 24
F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed. MMRM model: (Response Variable = Treatment + Stratification Factors \[Skin Type Fitzpatrick Scale Type I and II versus Type III, IV, V, and VI, Region North America/Europe\] + Visit + Treatment\*Visit).
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in F-VASI at Week 24 | -44.39 percentage change | Standard Error 2.85 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in F-VASI at Week 24 | -15.80 percentage change | Standard Error 4.04 |
p-value: <0.000195% CI: [-38.33, -18.86]mixed-effect model; repeated measurement
Secondary
Percentage Change From Baseline in F-VASI at Week 52
F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in F-VASI at Week 52 | -63.75 percentage change | Standard Deviation 34.103 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in F-VASI at Week 52 | -43.50 percentage change | Standard Deviation 47.509 |
Secondary
Percentage Change From Baseline in T-BSA at Week 24
T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed. MMRM model: (Response Variable = Treatment + Stratification Factors \[Skin Type Fitzpatrick scale Type I and II vs Type III, IV, V, and VI, Region North America/Europe\] + Visit + Treatment\*Visit).
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in T-BSA at Week 24 | -14.23 percentage change | Standard Error 1.55 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in T-BSA at Week 24 | -2.28 percentage change | Standard Error 2.19 |
p-value: <0.000195% CI: [-17.23, -6.67]mixed-effect model; repeated measurement
Secondary
Percentage Change From Baseline in T-BSA at Week 52
T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in T-BSA at Week 52 | -26.04 percentage change | Standard Deviation 28.481 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in T-BSA at Week 52 | -13.54 percentage change | Standard Deviation 27.144 |
Secondary
Percentage Change From Baseline in T-VASI at Week 24
T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed. MMRM model: (Response Variable = Treatment + Stratification Factors \[Skin Type Fitzpatrick scale Type I and II vs Type III, IV, V, and VI, Region North America/Europe\] + Visit + Treatment\*Visit).
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in T-VASI at Week 24 | -28.85 percentage change | Standard Error 1.96 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in T-VASI at Week 24 | -8.99 percentage change | Standard Error 2.78 |
p-value: <0.000195% CI: [-26.55, -13.16]mixed-effect model; repeated measurement
Secondary
Percentage Change From Baseline in T-VASI at Week 52
T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.
Time frame: Baseline; Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage Change From Baseline in T-VASI at Week 52 | -46.80 percentage change | Standard Deviation 29.903 |
| Double-Blind Period: Vehicle Cream BID | Percentage Change From Baseline in T-VASI at Week 52 | -30.11 percentage change | Standard Deviation 30.315 |
Secondary
Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52
An F-VASI25/50/75/90 responder achieved at least 25/50/75/90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Time frame: Baseline; Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI25 | 82.5 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI50 | 74.0 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI75 | 48.0 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI90 | 27.7 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI90 | 16.0 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI25 | 71.6 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI75 | 29.6 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52 | F-VASI50 | 49.4 percentage of participants |
Secondary
Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52
A T-VASI25/50/75/90 responder achieved ≥25/50/75/90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).
Time frame: Baseline; Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI25 | 76.8 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI50 | 49.2 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI75 | 20.9 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI90 | 6.8 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI90 | 1.2 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI25 | 53.1 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI75 | 8.6 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52 | T-VASI50 | 22.2 percentage of participants |
Secondary
Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24
A T-VASI25/75/90 responder achieved at least 25/75/90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing T-VASI scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | T-VASI25 | 50.2 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | T-VASI75 | 8.0 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | T-VASI90 | 1.0 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | T-VASI25 | 21.2 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | T-VASI75 | 1.8 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24 | T-VASI90 | 0.0 percentage of participants |
Comparison: T-VASI25p-value: <0.000195% CI: [2.121, 6.63]exact logistic regression
Comparison: T-VASI75p-value: 0.043695% CI: [1.045, 20.192]exact logistic regression
Comparison: T-VASI90
Secondary
Percentage of Participants Achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24
An F-VASI25 responder achieved at least 25% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing F-VASI scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24 | 63.9 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24 | 32.0 percentage of participants |
p-value: <0.000195% CI: [2.267, 6.246]exact logistic regression
Secondary
Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24
An F-VASI50 responder achieved at least 50% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing F-VASI scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24 | 51.4 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24 | 20.9 percentage of participants |
p-value: <0.000195% CI: [2.296, 6.949]exact logistic regression
Secondary
Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24
A T-VASI50 responder achieved at least 50% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing T-VASI scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24 | 23.9 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24 | 6.8 percentage of participants |
p-value: 0.000695% CI: [1.865, 9.853]exact logistic regression
Secondary
Percentage of Participants Achieving a ≥ 90% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24
An F-VASI90 responder achieved at least 90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing F-VASI scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a ≥ 90% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24 | 16.3 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a ≥ 90% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24 | 1.3 percentage of participants |
p-value: 0.006595% CI: [2.15, 108.739]exact logistic regression
Secondary
Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24
The VNS is a patient-reported measure of vitiligo treatment success that is rated on a 5-point scale. The Baseline facial photograph was shown to the participants for reference, and a mirror was provided for the participants to assess the vitiligo on their face. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable.
Time frame: Baseline; Week 24
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Missing VNS scores were imputed by Multiple Imputation with Fully Conditional Specification. The multiple imputation method used treatment and observed stratification factors as predicators.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24 | 20.5 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24 | 4.9 percentage of participants |
p-value: 0.001395% CI: [1.851, 12.755]exact logistic regression
Secondary
Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods)
The VNS is a patient-reported measure of vitiligo treatment success that is rated on a 5-point scale. The Baseline facial photograph was shown to the participants for reference, and a mirror was provided for the participants to assess the vitiligo on their face. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable.
Time frame: Baseline; Week 24 and Week 52
Population: ITT Population. Data from participants at a single site were excluded from this analysis owing to serious noncompliance with the protocol. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, as noticeable | 30.7 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, a lot less noticeable | 20.6 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, slightly less noticeable | 40.2 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, no longer noticeable | 0.0 percentage of participants |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, more noticeable | 8.5 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, no longer noticeable | 1.0 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, more noticeable | 11.2 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, as noticeable | 58.2 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, slightly less noticeable | 25.5 percentage of participants |
| Double-Blind Period: Vehicle Cream BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 24, a lot less noticeable | 4.1 percentage of participants |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, more noticeable | 4.5 percentage of participants |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, as noticeable | 16.4 percentage of participants |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, slightly less noticeable | 46.3 percentage of participants |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, a lot less noticeable | 32.2 percentage of participants |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, no longer noticeable | 0.6 percentage of participants |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, slightly less noticeable | 49.4 percentage of participants |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, more noticeable | 11.1 percentage of participants |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, no longer noticeable | 0.0 percentage of participants |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, as noticeable | 25.9 percentage of participants |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods) | Week 52, a lot less noticeable | 13.6 percentage of participants |
Secondary
Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40
Trough plasma concentration was defined as the measurement of the plasma concentration of ruxolitinib before drug application.
Time frame: pre-dose at Weeks 4, 24, and 40
Population: Pharmacokinetic/Pharmacodynamic (PK/PD) Evaluable Population: all participants who applied ruxolitinib cream at least once and provided at least 1 postdose blood sample that complied with the Protocol
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 | Week 4 | 61.0 nanomoles | Standard Deviation 68.6 |
| Double-Blind Period: Ruxolitinib Cream 1.5% BID | Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 | Week 24 | 54.5 nanomoles | Standard Deviation 79.1 |
| Treatment-Extension Period: Ruxolitinib Cream 1.5% BID | Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 | Week 40 | 57.0 nanomoles | Standard Deviation 73.3 |
| Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID | Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 | Week 40 | 48.2 nanomoles | Standard Deviation 57 |