Trial Outcomes & Findings for Topical Ruxolitinib Evaluation in Vitiligo Study 2 (TRuE-V2) (NCT NCT04057573)

Last Updated: 2025-08-22

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

344 participants

Primary outcome timeframe

Baseline; Week 24

Results posted on

2025-08-22

Participant Flow

This study was conducted at 49 study centers in North America and Europe.

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).

Participant milestones

Participant milestones
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
24-Week Double-Blind Period STARTED	229	115	0	0
24-Week Double-Blind Period ITT Population	229	115	0	0
24-Week Double-Blind Period ITT Population, Excluding Non-compliant Site	222	109	0	0
24-Week Double-Blind Period COMPLETED	199	98	0	0
24-Week Double-Blind Period NOT COMPLETED	30	17	0	0
28-Week Treatment Extension Period STARTED	0	0	199	98
28-Week Treatment Extension Period COMPLETED	0	0	178	79
28-Week Treatment Extension Period NOT COMPLETED	0	0	21	19

Reasons for withdrawal

Reasons for withdrawal
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
24-Week Double-Blind Period Adverse Event	2	0	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 Physician Decision	1	0	0	0
24-Week Double-Blind Period Pregnancy	1	0	0	0
24-Week Double-Blind Period Withdrawal by Subject	10	8	0	0
24-Week Double-Blind Period Discontinued Treatment due to COVID-19 Pandemic	3	1	0	0
24-Week Double-Blind Period Non-compliance with Attending Study Visits	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 Not Treated	1	0	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 Withdrawal by Subject	0	0	9	9
28-Week Treatment Extension Period Unable to Perform Safety Follow-up Visit	0	0	1	0

Baseline Characteristics

Baseline data are reported for members of the Intent-to-Treat (ITT) Population (all randomized participants), minus those participants enrolled at the noncompliant site. Treatment groups for this population were defined according to the treatment assignment at the time of randomization).

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=228 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=115 Participants Participants applied matching vehicle cream BID for 24 weeks.	Total n=343 Participants Total of all reporting groups
Age, Continuous	38.4 years STANDARD_DEVIATION 15.22 • n=228 Participants	39.8 years STANDARD_DEVIATION 12.12 • n=115 Participants	38.9 years STANDARD_DEVIATION 14.25 • n=343 Participants
Sex: Female, Male Female	112 Participants n=228 Participants	60 Participants n=115 Participants	172 Participants n=343 Participants
Sex: Female, Male Male	116 Participants n=228 Participants	55 Participants n=115 Participants	171 Participants n=343 Participants
Race/Ethnicity, Customized White	182 Participants n=228 Participants	93 Participants n=115 Participants	275 Participants n=343 Participants
Race/Ethnicity, Customized Black/African American	12 Participants n=228 Participants	5 Participants n=115 Participants	17 Participants n=343 Participants
Race/Ethnicity, Customized Asian	12 Participants n=228 Participants	7 Participants n=115 Participants	19 Participants n=343 Participants
Race/Ethnicity, Customized American Indian/Alaska Native	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Native Hawaiian/Pacific Islander	2 Participants n=228 Participants	0 Participants n=115 Participants	2 Participants n=343 Participants
Race/Ethnicity, Customized Not Reported	2 Participants n=228 Participants	3 Participants n=115 Participants	5 Participants n=343 Participants
Race/Ethnicity, Customized North African	2 Participants n=228 Participants	0 Participants n=115 Participants	2 Participants n=343 Participants
Race/Ethnicity, Customized White/Caucasian and Asian	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Guyana	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Cape Verdean	1 Participants n=228 Participants	1 Participants n=115 Participants	2 Participants n=343 Participants
Race/Ethnicity, Customized Persian	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Middle Eastern	3 Participants n=228 Participants	0 Participants n=115 Participants	3 Participants n=343 Participants
Race/Ethnicity, Customized Arab/North-African	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized White/Black/Asian	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Mexican	2 Participants n=228 Participants	1 Participants n=115 Participants	3 Participants n=343 Participants
Race/Ethnicity, Customized Jordanian	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Arabic	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Argentinian	1 Participants n=228 Participants	0 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Brazilian	0 Participants n=228 Participants	2 Participants n=115 Participants	2 Participants n=343 Participants
Race/Ethnicity, Customized Dominican Republic	0 Participants n=228 Participants	1 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Indo-Caribbean	0 Participants n=228 Participants	1 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Multi-National	0 Participants n=228 Participants	1 Participants n=115 Participants	1 Participants n=343 Participants
Race/Ethnicity, Customized Hispanic or Latino	50 Participants n=228 Participants	32 Participants n=115 Participants	82 Participants n=343 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	174 Participants n=228 Participants	80 Participants n=115 Participants	254 Participants n=343 Participants
Race/Ethnicity, Customized Captured as "Other"	2 Participants n=228 Participants	0 Participants n=115 Participants	2 Participants n=343 Participants
Face Vitiligo Area Scoring Index (F-VASI)	0.900 scores on a scale STANDARD_DEVIATION 0.5248 • n=228 Participants	0.834 scores on a scale STANDARD_DEVIATION 0.5233 • n=115 Participants	0.878 scores on a scale STANDARD_DEVIATION 0.5245 • n=343 Participants
Facial Body Surface Area (F-BSA) Involvement	0.98 percentage of facial surface area STANDARD_DEVIATION 0.570 • n=228 Participants	0.92 percentage of facial surface area STANDARD_DEVIATION 0.569 • n=115 Participants	0.96 percentage of facial surface area STANDARD_DEVIATION 0.569 • n=343 Participants
Total Body Vitiligo Area Scoring Index (T-VASI)	6.844 scores on a scale STANDARD_DEVIATION 2.0574 • n=228 Participants	7.022 scores on a scale STANDARD_DEVIATION 2.1986 • n=115 Participants	6.904 scores on a scale STANDARD_DEVIATION 2.1043 • n=343 Participants
Total Body Surface Area (T-BSA) Involvement	7.44 percentage of total body surface area STANDARD_DEVIATION 2.011 • n=228 Participants	7.68 percentage of total body surface area STANDARD_DEVIATION 2.040 • n=115 Participants	7.52 percentage of total body surface area STANDARD_DEVIATION 2.021 • n=343 Participants
Age Continuous, without Noncompliant Site	38.4 years STANDARD_DEVIATION 15.44 • n=222 Participants • Baseline data are reported for members of the Intent-to-Treat (ITT) Population (all randomized participants), minus those participants enrolled at the noncompliant site. Treatment groups for this population were defined according to the treatment assignment at the time of randomization).	39.4 years STANDARD_DEVIATION 12.29 • n=109 Participants • Baseline data are reported for members of the Intent-to-Treat (ITT) Population (all randomized participants), minus those participants enrolled at the noncompliant site. Treatment groups for this population were defined according to the treatment assignment at the time of randomization).	38.7 years STANDARD_DEVIATION 14.46 • n=331 Participants • Baseline data are reported for members of the Intent-to-Treat (ITT) Population (all randomized participants), minus those participants enrolled at the noncompliant site. Treatment groups for this population were defined according to the treatment assignment at the time of randomization).
Gender, without Noncompliant Site Female	110 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	58 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	168 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Gender, without Noncompliant Site Male	112 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	51 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	163 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site White	178 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	88 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	266 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Black/African American	11 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	5 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	16 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Asian	11 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	6 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	17 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site American Indian/Alaska Native	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Native Hawaiian/Pacific Islander	2 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	2 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Not Reported	3 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	3 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	6 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site North African	2 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	2 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site White/Caucasian and Asian	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Guyana	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Cape Verdean	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	2 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Persian	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Middle Eastern	3 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	3 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Arab/North-African	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site White/Black/Asian	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Mexican	2 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	3 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Jordanian	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Arabic	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Argentinian	1 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Brazilian	0 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	2 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	2 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Dominican Republic	0 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Indo-Caribbean	0 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Race, without Noncompliant Site Multi-National	0 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	1 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Ethnicity, without Noncompliant Site Hispanic or Latino	49 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	30 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	79 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Ethnicity, without Noncompliant Site Not Hispanic or Latino	169 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	76 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	245 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Ethnicity, without Noncompliant Site Not Reported	2 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	3 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	5 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
Ethnicity, without Noncompliant Site Captured as "Other"	2 Participants n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0 Participants n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	2 Participants n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
F-VASI, without Noncompliant Site	0.898 scores on a scale STANDARD_DEVIATION 0.5256 • n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0.834 scores on a scale STANDARD_DEVIATION 0.5342 • n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0.877 scores on a scale STANDARD_DEVIATION 0.5285 • n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
F-BSA Involvement, without Noncompliant Site	0.98 percentage of facial surface area STANDARD_DEVIATION 0.571 • n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0.92 percentage of facial surface area STANDARD_DEVIATION 0.582 • n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	0.96 percentage of facial surface area STANDARD_DEVIATION 0.575 • n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
T-VASI, without Noncompliant Site	6.790 scores on a scale STANDARD_DEVIATION 2.0435 • n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	6.979 scores on a scale STANDARD_DEVIATION 2.1953 • n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	6.852 scores on a scale STANDARD_DEVIATION 2.0933 • n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.
T-BSA Involvement, without Noncompliant Site	7.38 percentage of total body surface area STANDARD_DEVIATION 2.025 • n=222 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	7.66 percentage of total body surface area STANDARD_DEVIATION 2.040 • n=109 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.	7.47 percentage of total body surface area STANDARD_DEVIATION 2.031 • n=331 Participants • Baseline data are reported for members of the ITT Population, minus those participants enrolled at the noncompliant site.

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	11.4 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	20.9 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	1.3 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	6.8 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24	20.5 percentage of participants	4.9 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24	-26.4 percentage change Standard Error 2.57	-7.0 percentage change Standard Error 3.82	—	—

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=228 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=115 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period	114 Participants	39 Participants	—	—

SECONDARY outcome

Timeframe: 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

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=199 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=98 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period	82 Participants	38 Participants	—	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	32.0 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=81 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	71.6 percentage of participants	—	—
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	49.4 percentage of participants	—	—
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	29.6 percentage of participants	—	—
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	16.0 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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).

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=199 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=98 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in F-VASI at Week 24	-44.39 percentage change Standard Error 2.85	-15.80 percentage change Standard Error 4.04	—	—

SECONDARY outcome

Timeframe: Baseline; Week 52

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=81 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in F-VASI at Week 52	-63.75 percentage change Standard Deviation 34.103	-43.50 percentage change Standard Deviation 47.509	—	—

SECONDARY outcome

Timeframe: Baseline; Week 52

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=81 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in F-BSA at Week 52	-41.81 percentage change Standard Deviation 35.694	-23.45 percentage change Standard Deviation 46.360	—	—

SECONDARY outcome

Timeframe: 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).

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=199 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=98 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in T-VASI at Week 24	-28.85 percentage change Standard Error 1.96	-8.99 percentage change Standard Error 2.78	—	—

SECONDARY outcome

Timeframe: Baseline; Week 52

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=81 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in T-VASI at Week 52	-46.80 percentage change Standard Deviation 29.903	-30.11 percentage change Standard Deviation 30.315	—	—

SECONDARY outcome

Timeframe: 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).

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=199 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=98 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in T-BSA at Week 24	-14.23 percentage change Standard Error 1.55	-2.28 percentage change Standard Error 2.19	—	—

SECONDARY outcome

Timeframe: Baseline; Week 52

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=81 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Percentage Change From Baseline in T-BSA at Week 52	-26.04 percentage change Standard Deviation 28.481	-13.54 percentage change Standard Deviation 27.144	—	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=222 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=109 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	21.2 percentage of participants	—	—
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	1.8 percentage of participants	—	—
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	0.0 percentage of participants	—	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=81 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	53.1 percentage of participants	—	—
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	22.2 percentage of participants	—	—
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	8.6 percentage of participants	—	—
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	1.2 percentage of participants	—	—

SECONDARY outcome

Timeframe: Baseline; Week 24 and Week 52

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=199 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=98 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID n=177 Participants Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID n=81 Participants Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	11.2 percentage of participants	—	—
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	58.2 percentage of participants	—	—
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	25.5 percentage of participants	—	—
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	4.1 percentage of participants	—	—
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	1.0 percentage of participants	—	—
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	11.1 percentage of participants
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	25.9 percentage of participants
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	49.4 percentage of participants
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	13.6 percentage of participants
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	0.0 percentage of participants

SECONDARY outcome

Timeframe: 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).

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=182 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=94 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24	-1.19 scores on a scale Standard Error 0.30	-0.59 scores on a scale Standard Error 0.41	—	—

SECONDARY outcome

Timeframe: Baseline; Week 52

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=205 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=105 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Change From Baseline in DLQI at Week 52 Baseline	4.37 scores on a scale Standard Deviation 4.485	5.38 scores on a scale Standard Deviation 4.876	—	—
Change From Baseline in DLQI at Week 52 Week 52	-0.84 scores on a scale Standard Deviation 3.976	-1.15 scores on a scale Standard Deviation 4.264	—	—

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=17 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID n=3 Participants Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID n=16 Participants Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID n=3 Participants Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
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	6.33 scores on a scale Standard Deviation 10.116	—	—
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	-2.33 scores on a scale Standard Deviation 8.505	—	—
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	-1.00 scores on a scale Standard Deviation 4.583

SECONDARY outcome

Timeframe: 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

Trough plasma concentration was defined as the measurement of the plasma concentration of ruxolitinib before drug application.

Outcome measures

Outcome measures
Measure	Double-Blind Period: Ruxolitinib Cream 1.5% BID n=208 Participants Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.	Double-Blind Period: Vehicle Cream BID Participants applied matching vehicle cream BID for 24 weeks.	Treatment-Extension Period: Ruxolitinib Cream 1.5% BID n=184 Participants Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.	Treatment-Extension Period: Vehicle Cream to Ruxolitinib Cream 1.5% BID n=83 Participants Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 Week 4	61.0 nanomoles Standard Deviation 68.6	—	—	—
Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 Week 24	54.5 nanomoles Standard Deviation 79.1	—	—	—
Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40 Week 40	—	—	57.0 nanomoles Standard Deviation 73.3	48.2 nanomoles Standard Deviation 57.0

Adverse Events

Vehicle Cream BID

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Ruxolitinib Cream 1.5% BID

Serious events: 6 serious events

Other events: 56 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Vehicle Cream BID n=115 participants at risk Participants applied matching vehicle cream twice a day (BID) for 24 weeks in the Double-Blind Period.	Ruxolitinib Cream 1.5% BID n=326 participants at risk Participants applied ruxolitinib cream during the Double-Blind Treatment Period and the Treatment-Extension Period. Participants applied ruxolitinib 1.5% cream BID for 24 weeks. Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5% BID for 28 weeks in the Treatment-Extension Period.
Infections and infestations Appendiceal abscess	0.00% 0/115 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	0.31% 1/326 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Cardiac disorders Coronary artery stenosis	0.00% 0/115 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	0.31% 1/326 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Injury, poisoning and procedural complications Joint dislocation	0.00% 0/115 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	0.31% 1/326 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Papillary thyroid cancer	0.00% 0/115 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	0.31% 1/326 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Musculoskeletal and connective tissue disorders Rhabdomyolysis	0.00% 0/115 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	0.31% 1/326 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Renal and urinary disorders Ureterolithiasis	0.00% 0/115 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	0.31% 1/326 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.

Other adverse events

Other adverse events
Measure	Vehicle Cream BID n=115 participants at risk Participants applied matching vehicle cream twice a day (BID) for 24 weeks in the Double-Blind Period.	Ruxolitinib Cream 1.5% BID n=326 participants at risk Participants applied ruxolitinib cream during the Double-Blind Treatment Period and the Treatment-Extension Period. Participants applied ruxolitinib 1.5% cream BID for 24 weeks. Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5% BID for 28 weeks in the Treatment-Extension Period.
General disorders Application site acne	2.6% 3/115 • Number of events 3 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	6.1% 20/326 • Number of events 24 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Infections and infestations COVID-19	1.7% 2/115 • Number of events 2 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	7.4% 24/326 • Number of events 25 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.
Infections and infestations Nasopharyngitis	0.87% 1/115 • Number of events 1 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.	5.2% 17/326 • Number of events 17 • from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 52 + 30 days) Treatment-emergent adverse events (TEAEs): AEs reported for the first time or the worsening of a pre-existing event after the first application of study drug. For the Double-Blind Period, TEAEs are reported for members of the Safety Population: all participants who applied ruxolitinib cream or vehicle cream at least once. For the Treatment-Extension (TE) Period, TEAEs are reported for the TE Evaluable Population: all participants who applied ruxolitinib cream at least once in the TE Period.

Additional Information

Study Director

Incyte Corporation

Phone: 1-855-463-3463

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
Publication restrictions are in place

Restriction type: OTHER