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Clinical Trial Results:
A Double-Blind, Vehicle-Controlled, Randomized Withdrawal and Treatment Extension Study to Assess the Long-Term Efficacy and Safety of Ruxolitinib Cream in Participants With Vitiligo

Summary
EudraCT number	2020-000987-53
Trial protocol	DE NL FR PL BG
Global end of trial date	14 Nov 2022

Results information
Results version number	v1(current)
This version publication date	28 May 2023
First version publication date	28 May 2023
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

INCB 18424-308

ISRCTN number

US NCT number

WHO universal trial number (UTN)

Sponsor organisation name

Incyte Corporation

Sponsor organisation address

1801 Augustine Cutoff Drive, Wilmington, United States, 19803

Public contact

Study Director, Incyte Corporation, 1 8554633463, medinfo@incyte.com

Scientific contact

Study Director, Incyte Corporation, 1 8554633463, medinfo@incyte.com

Is trial part of an agreed paediatric investigation plan (PIP)

Yes

EMA paediatric investigation plan number(s)

EMEA-002618-PIP02-20

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Final

Date of interim/final analysis

14 Nov 2022

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

14 Nov 2022

Was the trial ended prematurely?

Main objective of the trial

This study was conducted to evaluate the duration of clinical response of ruxolitinib cream in participants with vitiligo.

Protection of trial subjects

This study was to have been performed in accordance with ethical principles that have their origin in the Declaration of Helsinki and conducted in adherence to the study Protocol, applicable Good Clinical Practices, and applicable laws and country-specific regulations in which the study was being conducted.

Background therapy

Evidence for comparator

Actual start date of recruitment

25 Sep 2020

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Bulgaria: 30

Country: Number of subjects enrolled

Canada: 47

Country: Number of subjects enrolled

France: 24

Country: Number of subjects enrolled

Germany: 15

Country: Number of subjects enrolled

Netherlands: 8

Country: Number of subjects enrolled

Poland: 89

Country: Number of subjects enrolled

Spain: 7

Country: Number of subjects enrolled

United States: 238

Worldwide total number of subjects

458

EEA total number of subjects

173

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

368

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

This study was conducted at 87 study centers in North America and Europe.

Screening details

This randomized withdrawal and treatment-extension study (extension of treatment received in 2 parent studies: NCT04052425 or NCT04057573) was comprised of a 52-week extension treatment period and a 4-week safety follow-up period, starting 4 weeks (30 days) after the last application of study treatment or the last study visit.

Period 1 title

Overall Study (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Are arms mutually exclusive

Yes

Cohort A: Vehicle cream BID

Arm description

Participants who completed treatment and achieved ≥90% improvement from Baseline in the Facial Vitiligo Area Scoring Index score (≥F-VASI90) at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants were randomized to receive vehicle cream twice daily (BID) for 52 weeks. Participants who experienced relapse (<75% improvement from Baseline in the F-VASI score [<F-VASI75]) received open-label ruxolitinib 1.5% cream BID for the duration of the study.

Arm type

Vehicle

Investigational medicinal product name

Vehicle cream

Investigational medicinal product code

Other name

Pharmaceutical forms

Cream

Routes of administration

Cutaneous use, Topical use

Dosage and administration details

Matching cream

Cohort A: Ruxolitinib 1.5% cream BID

Arm description

Arm type

Experimental

Investigational medicinal product name

ruxolitinib

Investigational medicinal product code

INCB018424

Other name

Pharmaceutical forms

Cream

Routes of administration

Topical use

Dosage and administration details

1.5% weight/weight (W/W) BID

Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID

Arm description

Participants who completed treatment (vehicle cream BID for 24 weeks and ruxolitinib 1.5% BID for 28 weeks) and did not achieve ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants applied ruxolitinib 1.5% cream BID for 52 weeks.

Arm type

Experimental

Investigational medicinal product name

ruxolitinib

Investigational medicinal product code

INCB018424

Other name

Pharmaceutical forms

Cream

Routes of administration

Topical use

Dosage and administration details

1.5% weight/weight (W/W) BID

Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID

Arm description

Participants who completed treatment (ruxolitinib 1.5% BID for 52 weeks) and did not achieve ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants applied ruxolitinib 1.5% cream BID for 52 weeks.

Arm type

Experimental

Investigational medicinal product name

ruxolitinib

Investigational medicinal product code

INCB018424

Other name

Pharmaceutical forms

Cream

Routes of administration

Topical use

Dosage and administration details

1.5% weight/weight (W/W) BID

Number of subjects in period 1	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Started	58	58	118	224
Completed	41	50	92	173
Not completed	17	8	26	51
Missed Safety Follow-up Visit	-	-	1	-
Consent withdrawn by subject	14	5	18	33
Physician decision	-	-	1	1
Discontinued due to COVID-19 Pandemic	-	-	1	-
Adverse event, non-fatal	-	-	-	1
Pregnancy	-	-	2	1
Not Compliant with Protocol-specific Visit Window	-	-	-	1
Personal Reasons	-	-	-	1
Lost to follow-up	3	3	3	10
Site Closed Due to Noncompliance	-	-	-	1
Lack of efficacy	-	-	-	1
Protocol deviation	-	-	-	1

Baseline characteristics

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Reporting group title

Cohort A: Vehicle cream BID

Reporting group description

Reporting group title

Cohort A: Ruxolitinib 1.5% cream BID

Reporting group description

Reporting group title

Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID

Reporting group description

Reporting group title

Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID

Reporting group description

Reporting group values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID	Total
Number of subjects	58	58	118	224	458
Age categorical
Units: Subjects
Adolescents (12-17 years)	4	8	10	36	58
Adults (18-64 years)	53	46	99	170	368
From 65-84 years	1	4	9	18	32
Age Continuous
Units: years
arithmetic mean (standard deviation)	39.3 ± 12.49	42.9 ± 15.95	39.7 ± 14.62	39.3 ± 16.45	-
Sex: Female, Male
Units: participants
Female	31	33	61	129	254
Male	27	25	57	95	204
Race/Ethnicity, Customized
Units: Subjects
Asian	4	3	3	8	18
Black or African American	5	4	3	11	23
Native Hawaiian or Other Pacific Islander	1	0	0	1	2
Not Reported	2	1	3	13	19
White	42	48	107	180	377
Captured as Latino in Database	1	0	0	0	1
Persian	1	0	0	0	1
Indo-Caribbean	1	0	0	0	1
Jordanian	1	0	0	0	1
Brazilian	0	1	0	0	1
Guyana	0	1	0	0	1
American Indian or Alaska Native	0	0	0	1	1
Cape Verdean	0	0	1	1	2
Dominican Republic	0	0	1	0	1
Captured as Hispanic or Latino in Database	0	0	0	1	1
Iranian	0	0	0	1	1
Indian	0	0	0	1	1
North African	0	0	0	1	1
Middle Eastern	0	0	0	1	1
Arab//North-African	0	0	0	1	1
White/Black/Asian	0	0	0	1	1
Mexican	0	0	0	2	2
Race/Ethnicity, Customized
Units: Subjects
Hispanic or Latino	11	13	16	53	93
Not Hispanic or Latino	45	43	99	157	344
Not Reported	2	1	3	11	17
Unknown	0	0	0	1	1
Captured as Other in Database	0	1	0	2	3

Subject analysis set title

Cohort A: Vehicle cream BID to ruxolitinib 1.5% cream BID

Subject analysis set type

Full analysis

Subject analysis set description

Participants who completed treatment and achieved ≥90% improvement from Baseline in the Facial Vitiligo Area Scoring Index score (≥F-VASI90) at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants were randomized to receive vehicle cream twice daily (BID) for 52 weeks. These participants experienced relapse (<F-VASI75) and received open-label ruxolitinib 1.5% cream BID for the duration of the study.

Subject analysis set title

Cohort B: Ruxolitinib 1.5% cream BID

Subject analysis set type

Full analysis

Subject analysis set description

Participants who completed treatment (vehicle cream BID for 24 weeks and ruxolitinib 1.5% BID for 28 weeks or ruxolitinib 1.5% BID for 52 weeks) and did not achieve ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants applied ruxolitinib 1.5% cream BID for 52 weeks and had at least 1 post-Baseline pharmacokinetic (PK) assessment.

Subject analysis set title

Cohorts A and B: Ruxolitinib 1.5% cream BID

Subject analysis set type

Full analysis

Subject analysis set description

Participants who completed treatment and achieved ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). Participants who completed treatment (vehicle cream BID for 24 weeks and ruxolitinib 1.5% BID for 28 weeks or ruxolitinib 1.5% BID for 52 weeks) and did not achieve ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants (58 from Cohort A; 289 from Cohort B) applied ruxolitinib 1.5% cream BID for 52 weeks And had at least 1 post-Baseline PK assessment.

Subject analysis sets values	Cohort A: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream BID	Cohorts A and B: Ruxolitinib 1.5% cream BID
Number of subjects	23	293	347
Age categorical
Units: Subjects
Adolescents (12-17 years)
Adults (18-64 years)
From 65-84 years
Age Continuous
Units: years
arithmetic mean (standard deviation)	3 ±	±	±
Sex: Female, Male
Units: participants
Female
Male
Race/Ethnicity, Customized
Units: Subjects
Asian
Black or African American
Native Hawaiian or Other Pacific Islander
Not Reported
White
Captured as Latino in Database
Persian
Indo-Caribbean
Jordanian
Brazilian
Guyana
American Indian or Alaska Native
Cape Verdean
Dominican Republic
Captured as Hispanic or Latino in Database
Iranian
Indian
North African
Middle Eastern
Arab//North-African
White/Black/Asian
Mexican
Race/Ethnicity, Customized
Units: Subjects
Hispanic or Latino
Not Hispanic or Latino
Not Reported
Unknown
Captured as Other in Database

End points

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Reporting group title

Cohort A: Vehicle cream BID

Reporting group description

Reporting group title

Cohort A: Ruxolitinib 1.5% cream BID

Reporting group description

Reporting group title

Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID

Reporting group description

Reporting group title

Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID

Reporting group description

Subject analysis set title

Cohort A: Vehicle cream BID to ruxolitinib 1.5% cream BID

Subject analysis set type

Full analysis

Subject analysis set description

Participants who completed treatment and achieved ≥90% improvement from Baseline in the Facial Vitiligo Area Scoring Index score (≥F-VASI90) at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants were randomized to receive vehicle cream twice daily (BID) for 52 weeks. These participants experienced relapse (<F-VASI75) and received open-label ruxolitinib 1.5% cream BID for the duration of the study.

Subject analysis set title

Cohort B: Ruxolitinib 1.5% cream BID

Subject analysis set type

Full analysis

Subject analysis set description

Subject analysis set title

Cohorts A and B: Ruxolitinib 1.5% cream BID

Subject analysis set type

Full analysis

Subject analysis set description

Primary: Time to relapse (defined as <F-VASI75)

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End point title

Time to relapse (defined as <F-VASI75)

End point description

Relapse was defined as a loss of 75% improvement from Baseline in the Face Vitiligo Area Scoring Index score (F-VASI75) response, assessed as percentage improvement in the F-VASI score at Baseline (Day 1 of the parent study) to <75%. -9999, 9999=not estimable because there were too few events of loss of F-VASI75 response.

End point type

Primary

End point timeframe

up to Week 52 of Extension Study

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	56	55	0 ^[1]	0 ^[2]
Units: days
median (confidence interval 95%)	9999 (238.0 to 9999)	9999 (-9999 to 9999)	( to )	( to )

Notes
[1] - Only participants in Cohort A were analyzed.
[2] - Only participants in Cohort A were analyzed.

Time to relapse treatment/vehicle

Statistical analysis description

Cox regression model stratified by stratification factor (treatment assignment in the parent studies) was conducted to compare the difference in hazard rate between treatment and vehicle.

Comparison groups

Cohort A: Vehicle cream BID v Cohort A: Ruxolitinib 1.5% cream BID

Number of subjects included in analysis

111

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0414

Method

Logrank

Parameter type

Hazard ratio (HR)

Point estimate

0.422

Confidence interval

level

95%

sides

2-sided

lower limit

0.18

upper limit

0.99

Secondary: Time to loss of adequate response

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End point title

Time to loss of adequate response

End point description

Loss of adequate response was defined as a loss of 90% improvement from Baseline in the F-VASI score (F-VASI90) response, assessed as percentage improvement in the F-VASI score at Baseline (Day 1 of the parent study) to <90%. -9999, 9999=not estimable because there were too few events of loss of F-VASI75 response.

End point type

Secondary

End point timeframe

up to Week 52 of Extension Study

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	56	55	0 ^[3]	0 ^[4]
Units: days
median (confidence interval 95%)	195.0 (113.0 to 372.0)	9999 (-9999 to 9999)	( to )	( to )

Notes
[3] - Only participants in Cohort A were analyzed.
[4] - Only participants in Cohort A were analyzed.

Time to loss of response treatment/vehicle

Statistical analysis description

Cox regression model stratified by stratification factor (treatment assignment in the parent studies) was conducted to compare the difference in hazard rate between treatment and vehicle.

Comparison groups

Cohort A: Vehicle cream BID v Cohort A: Ruxolitinib 1.5% cream BID

Number of subjects included in analysis

111

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0003

Method

Logrank

Parameter type

Hazard ratio (HR)

Point estimate

0.316

Confidence interval

level

95%

sides

2-sided

lower limit

0.165

upper limit

0.606

Secondary: Percentage of participants achieving a ≥50% improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) score during the Extension Treatment Period

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End point title

Percentage of participants achieving a ≥50% improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) score during the Extension Treatment Period

End point description

An F-VASI50 responder achieved at least 50% 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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[5]	57 ^[6]	118 ^[7]	222 ^[8]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	98.2 (90.6 to 100.0)	98.2 (90.6 to 100.0)	45.8 (36.6 to 55.2)	65.6 (58.9 to 71.9)
Week 56, n=56, 55, 108, 216	98.2 (90.4 to 100.0)	96.4 (87.5 to 99.6)	50.0 (40.2 to 59.8)	69.4 (62.8 to 75.5)
Week 60, n=48, 52, 107, 211	97.9 (88.9 to 99.9)	98.1 (89.7 to 100.0)	54.2 (44.3 to 63.9)	69.2 (62.5 to 75.4)
Week 64, n=43, 45, 108, 207	97.7 (87.7 to 99.9)	100.0 (92.1 to 100.0)	58.3 (48.5 to 67.7)	72.0 (65.3 to 78.0)
Week 68, n=41, 45, 107, 210	95.1 (83.5 to 99.4)	100.0 (92.1 to 100.0)	63.6 (53.7 to 72.6)	77.1 (70.9 to 82.6)
Week 80, n=30, 45, 99, 194	96.7 (82.8 to 99.9)	100.0 (92.1 to 100.0)	65.7 (55.4 to 74.9)	78.9 (72.4 to 84.4)
Week 92, n=23, 43, 94, 179	100.0 (85.2 to 100.0)	97.7 (87.7 to 99.9)	66.0 (55.5 to 75.4)	84.4 (78.2 to 89.3)
Week 104, n=23, 38, 93, 177	95.7 (78.1 to 99.9)	100.0 (90.7 to 100.0)	69.9 (59.5 to 79.0)	86.4 (80.5 to 91.1)

Notes
[5] - Only participants with available data were analyzed.
[6] - Only participants with available data were analyzed.
[7] - Only participants with available data were analyzed.
[8] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percentage of participants achieving a ≥75% improvement from Baseline in the F-VASI (F-VASI75) score during the Extension Treatment Period

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End point title

Percentage of participants achieving a ≥75% improvement from Baseline in the F-VASI (F-VASI75) score during the Extension Treatment Period

End point description

An F-VASI75 responder achieved at least 75% 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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[9]	57 ^[10]	118 ^[11]	222 ^[12]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	98.2 (90.6 to 100.0)	98.2 (90.6 to 100.0)	16.1 (10.0 to 24.0)	30.8 (24.8 to 37.3)
Week 56, n=56, 55, 108, 216	96.4 (87.7 to 99.6)	96.4 (87.5 to 99.6)	23.1 (15.6 to 32.2)	34.7 (28.4 to 41.5)
Week 60, n=48, 52, 107, 211	95.8 (85.7 to 99.5)	90.4 (79.0 to 96.8)	29.0 (20.6 to 38.5)	40.8 (34.1 to 47.7)
Week 64, n=43, 45, 108, 207	95.3 (84.2 to 99.4)	97.8 (88.2 to 99.9)	30.6 (22.1 to 40.2)	43.5 (36.6 to 50.5)
Week 68, n=41, 45, 107, 210	87.8 (73.8 to 95.9)	100.0 (92.1 to 100.0)	34.6 (25.6 to 44.4)	48.6 (41.6 to 55.5)
Week 80, n=30, 45, 99, 194	90.0 (73.5 to 97.9)	100.0 (92.1 to 100.0)	43.4 (33.5 to 53.8)	54.6 (47.4 to 61.8)
Week 92, n=23, 43, 94, 179	100.0 (85.2 to 100.0)	97.7 (87.7 to 99.9)	47.9 (37.5 to 58.4)	60.3 (52.8 to 67.6)
Week 104, n=23, 38, 93, 177	95.7 (78.1 to 99.9)	97.4 (86.2 to 99.9)	47.3 (36.9 to 57.9)	66.1 (58.6 to 73.0)

Notes
[9] - Only participants with available data were analyzed.
[10] - Only participants with available data were analyzed.
[11] - Only participants with available data were analyzed.
[12] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percentage of participants achieving a ≥90% improvement from Baseline in the F-VASI (F-VASI90) score during the Extension Treatment Period

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End point title

Percentage of participants achieving a ≥90% improvement from Baseline in the F-VASI (F-VASI90) score during the Extension Treatment Period

End point description

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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[13]	57 ^[14]	118 ^[15]	222 ^[16]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	98.2 (90.6 to 100.0)	96.5 (87.9 to 99.6)	0.0 (0.0 to 3.1)	2.3 (0.7 to 5.2)
Week 56, n=56, 55, 108, 216	89.3 (78.1 to 96.0)	94.5 (84.9 to 98.9)	3.7 (1.0 to 9.2)	8.3 (5.0 to 12.9)
Week 60, n=48, 52, 107, 211	83.3 (69.8 to 92.5)	86.5 (74.2 to 94.4)	8.4 (3.9 to 15.4)	15.2 (10.6 to 20.7)
Week 64, n=43, 45, 108, 207	72.1 (56.3 to 84.7)	93.3 (81.7 to 98.6)	11.1 (5.9 to 18.6)	15.5 (10.8 to 21.1)
Week 68, n=41, 45, 107, 210	68.3 (51.9 to 81.9)	95.6 (84.9 to 99.5)	12.1 (6.6 to 19.9)	22.4 (16.9 to 28.6)
Week 80, n=30, 45, 99, 194	80.0 (61.4 to 92.3)	97.8 (88.2 to 99.9)	22.2 (14.5 to 31.7)	30.4 (24.0 to 37.4)
Week 92, n=23, 43, 94, 179	78.3 (56.3 to 92.5)	93.0 (80.9 to 98.5)	24.5 (16.2 to 34.4)	32.4 (25.6 to 39.8)
Week 104, n=23, 38, 93, 177	69.6 (47.1 to 86.8)	92.1 (78.6 to 98.3)	28.0 (19.1 to 38.2)	33.9 (27.0 to 41.4)

Notes
[13] - Only participants with available data were analyzed.
[14] - Only participants with available data were analyzed.
[15] - Only participants with available data were analyzed.
[16] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Mean F-VASI scores during the Extension Treatment Period

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End point title

Mean F-VASI scores during the Extension Treatment Period

End point description

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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[17]	57 ^[18]	118 ^[19]	222 ^[20]
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline, n=57, 57, 188, 222	0.86 ± 0.492	0.99 ± 0.644	0.88 ± 0.543	0.91 ± 0.550
Week 52, n=57, 57, 118, 221	0.05 ± 0.110	0.07 ± 0.181	0.51 ± 0.459	0.39 ± 0.374
Week 56, n=56, 55, 108, 216	0.05 ± 0.108	0.07 ± 0.186	0.50 ± 0.475	0.38 ± 0.372
Week 60, n=48, 52, 107, 211	0.06 ± 0.145	0.09 ± 0.186	0.45 ± 0.450	0.35 ± 0.375
Week 64, n=43, 45, 108, 207	0.08 ± 0.131	0.08 ± 0.230	0.43 ± 0.456	0.33 ± 0.350
Week 68, n=43, 45, 108, 207	0.11 ± 0.199	0.04 ± 0.081	0.40 ± 0.430	0.31 ± 0.352
Week 80, n=30, 45, 99, 194	0.05 ± 0.082	0.04 ± 0.108	0.36 ± 0.426	0.27 ± 0.345
Week 92, n=23, 43, 94, 179	0.04 ± 0.055	0.04 ± 0.076	0.38 ± 0.471	0.23 ± 0.281
Week 104, n=23, 38, 93, 177	0.06 ± 0.109	0.04 ± 0.083	0.37 ± 0.493	0.21 ± 0.280

Notes
[17] - Only participants with available data were analyzed.
[18] - Only participants with available data were analyzed.
[19] - Only participants with available data were analyzed.
[20] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Change from Baseline in F-VASI scores during the Extension Treatment Period

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End point title

Change from Baseline in F-VASI scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[21]	57 ^[22]	118 ^[23]	222 ^[24]
Units: scores on a scale
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-0.81 ± 0.464	-0.92 ± 0.591	-0.37 ± 0.346	-0.51 ± 0.448
Week 56, n=56, 55, 108, 216	-0.82 ± 0.492	-0.90 ± 0.594	-0.39 ± 0.370	-0.54 ± 0.457
Week 60, n=48, 52, 107, 211	-0.84 ± 0.447	-0.87 ± 0.570	-0.43 ± 0.402	-0.55 ± 0.457
Week 64, n=43, 45, 108, 207	-0.80 ± 0.442	-0.87 ± 0.527	-0.44 ± 0.423	-0.58 ± 0.452
Week 68, n=41, 45, 107, 210	-0.73 ± 0.316	-0.86 ± 0.518	-0.46 ± 0.437	-0.61 ± 0.476
Week 80, n=30, 45, 99, 194	-0.75 ± 0.360	-0.86 ± 0.508	-0.49 ± 0.471	-0.64 ± 0.478
Week 92, n=23, 43, 94, 179	-0.76 ± 0.341	-0.87 ± 0.548	-0.48 ± 0.531	-0.69 ± 0.488
Week 104, n=23, 38, 93, 177	-0.72 ± 0.380	-0.92 ± 0.561	-0.50 ± 0.557	-0.68 ± 0.514

Notes
[21] - Only participants with available data were analyzed.
[22] - Only participants with available data were analyzed.
[23] - Only participants with available data were analyzed.
[24] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percent change from Baseline in F-VASI scores during the Extension Treatment Period

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End point title

Percent change from Baseline in F-VASI scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline (BL); up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[25]	57 ^[26]	118 ^[27]	222 ^[28]
Units: percentage change
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-94.45 ± 14.069	-94.50 ± 8.990	-44.32 ± 27.775	-54.72 ± 31.006
Week 56, n=56, 55, 108, 216	-93.79 ± 14.981	-93.21 ± 12.573	-46.19 ± 30.728	-57.38 ± 31.256
Week 60, n=48, 52, 107,211	-93.85 ± 9.087	-92.04 ± 13.198	-50.55 ± 30.537	-60.17 ± 30.147
Week 64, n=43, 45, 108, 207	-89.64 ± 23.562	-94.87 ± 8.079	-52.32 ± 32.826	-62.94 ± 27.427
Week 68, n=41, 45, 107, 210	-87.98 ± 19.644	-96.25 ± 4.633	-55.27 ± 32.433	-65.55 ± 30.388
Week 80, n=30, 45, 99, 194	-93.24 ± 13.062	-96.65 ± 4.683	-58.60 ± 34.839	-69.38 ± 29.091
Week 92, n=23, 43, 94, 179	-95.77 ± 5.586	-94.95 ± 10.366	-55.44 ± 49.855	-73.77 ± 25.490
Week 104, n=23, 38, 93, 177	-90.53 ± 17.763	-95.86 ± 7.213	-56.53 ± 57.087	-73.84 ± 31.780

Notes
[25] - Only participants with available data were analyzed.
[26] - Only participants with available data were analyzed.
[27] - Only participants with available data were analyzed.
[28] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percentage of participants achieving a ≥50% improvement from Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) score during the Extension Treatment Period

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End point title

Percentage of participants achieving a ≥50% improvement from Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) score during the Extension Treatment Period

End point description

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 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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[29]	57 ^[30]	118 ^[31]	222 ^[32]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	80.7 (68.1 to 90.0)	71.9 (58.5 to 83.0)	16.9 (10.7 to 25.0)	42.5 (35.9 to 49.3)
Week 56, n=56, 55, 108, 216	76.8 (63.6 to 87.0)	72.7 (59.0 to 83.9)	16.7 (10.2 to 25.1)	45.8 (39.1 to 52.7)
Week 60, n=48, 52, 107, 211	83.3 (69.8 to 92.5)	75.0 (61.1 to 86.0)	19.6 (12.6 to 28.4)	49.8 (42.8 to 56.7)
Week 64, n=43, 45, 108, 207	74.4 (58.8 to 86.5)	82.2 (67.9 to 92.0)	21.3 (14.0 to 30.2)	50.7 (43.7 to 57.7)
Week 68, n=41, 45, 107, 210	70.7 (54.5 to 83.9)	84.4 (70.5 to 93.5)	29.9 (21.4 to 39.5)	54.3 (47.3 to 61.2)
Week 80, n=30, 45, 99, 194	80.0 (61.4 to 92.3)	86.7 (73.2 to 94.9)	39.4 (29.7 to 49.7)	57.7 (50.4 to 64.8)
Week 92, n=23, 43, 94, 179	73.9 (51.6 to 89.8)	86.0 (72.1 to 94.7)	48.9 (38.5 to 59.5)	61.5 (53.9 to 68.6)
Week 104, n=23, 38, 93, 177	60.9 (38.5 to 80.3)	89.5 (75.2 to 97.1)	54.8 (44.2 to 65.2)	63.8 (56.3 to 70.9)

Notes
[29] - Only participants with available data were analyzed.
[30] - Only participants with available data were analyzed.
[31] - Only participants with available data were analyzed.
[32] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percentage of participants achieving a ≥75% improvement from Baseline in the T-VASI (T-VASI75) score during the Extension Treatment Period

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End point title

Percentage of participants achieving a ≥75% improvement from Baseline in the T-VASI (T-VASI75) score during the Extension Treatment Period

End point description

A T-VASI75 responder achieved at least 75% 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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[33]	57 ^[34]	118 ^[35]	222 ^[36]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	38.6 (26.0 to 52.4)	42.1 (29.1 to 55.9)	3.4 (0.9 to 8.5)	12.2 (8.2 to 17.3)
Week 56, n=56, 55, 108, 216	42.9 (29.7 to 56.8)	43.6 (30.3 to 57.7)	4.6 (1.5 to 10.5)	13.4 (9.2 to 18.7)
Week 60, n=48, 52, 107, 211	45.8 (31.4 to 60.8)	38.5 (25.3 to 53.0)	4.7 (1.5 to 10.6)	14.2 (9.8 to 19.7)
Week 64, n=43, 45, 108, 207	39.5 (25.0 to 55.6)	48.9 (33.7 to 64.2)	3.7 (1.0 to 9.2)	16.9 (12.1 to 22.7)
Week 68, n=41, 45, 107, 210	39.0 (24.2 to 55.5)	42.2 (27.7 to 57.8)	6.5 (2.7 to 13.0)	22.4 (16.9 to 28.6)
Week 80, n=30, 45, 99, 194	53.3 (34.3 to 71.7)	48.9 (33.7 to 64.2)	10.1 (5.0 to 17.8)	23.7 (17.9 to 30.3)
Week 92, n=23, 43, 94, 179	43.5 (23.2 to 65.5)	53.5 (37.7 to 68.8)	12.8 (6.8 to 21.2)	29.1 (22.5 to 36.3)
Week 104, n=23, 38, 93, 177	39.1 (19.7 to 61.5)	55.3 (38.3 to 71.4)	18.3 (11.0 to 27.6)	30.5 (23.8 to 37.9)

Notes
[33] - Only participants with available data were analyzed.
[34] - Only participants with available data were analyzed.
[35] - Only participants with available data were analyzed.
[36] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percentage of participants achieving a ≥90% improvement from Baseline in the T-VASI (T-VASI90) score during the Extension Treatment Period

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End point title

Percentage of participants achieving a ≥90% improvement from Baseline in the T-VASI (T-VASI90) score during the Extension Treatment Period

End point description

A T-VASI90 responder achieved at least 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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[37]	57 ^[38]	118 ^[39]	222 ^[40]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	12.3 (5.1 to 23.7)	12.3 (5.1 to 23.7)	0.0 (0.0 to 3.1)	2.3 (0.7 to 5.2)
Week 56, n=56, 55, 108, 216	12.5 (5.2 to 24.1)	14.5 (6.5 to 26.7)	0.0 (0.0 to 3.4)	3.2 (1.3 to 6.6)
Week 60, n=48, 52, 107, 211	16.7 (7.5 to 30.2)	17.3 (8.2 to 30.3)	0.0 (0.0 to 3.4)	3.8 (1.7 to 7.3)
Week 64, n=43, 45, 108, 207	20.9 (10.0 to 36.0)	20.0 (9.6 to 34.6)	0.0 (0.0 to 3.4)	2.9 (1.1 to 6.2)
Week 68, n=41, 45, 107, 210	22.0 (10.6 to 37.6)	20.0 (9.6 to 34.6)	0.9 (0.0 to 5.1)	4.3 (2.0 to 8.0)
Week 80, n=30, 45, 99, 194	20.0 (7.7 to 38.6)	20.0 (9.6 to 34.6)	1.0 (0.0 to 5.5)	6.7 (3.6 to 11.2)
Week 92, n=23, 43, 94, 179	21.7 (7.5 to 43.7)	20.9 (10.0 to 36.0)	2.1 (0.3 to 7.5)	8.9 (5.2 to 14.1)
Week 104, n=23, 38, 93, 177	21.7 (7.5 to 43.7)	23.7 (11.4 to 40.2)	3.2 (0.7 to 9.1)	9.6 (5.7 to 14.9)

Notes
[37] - Only participants with available data were analyzed.
[38] - Only participants with available data were analyzed.
[39] - Only participants with available data were analyzed.
[40] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Mean T-VASI scores during the Extension Treatment Period

Top of page

End point title

Mean T-VASI scores during the Extension Treatment Period

End point description

T-VASI was 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).

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[41]	57 ^[42]	118 ^[43]	222 ^[44]
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline, n=57, 57, 118, 222	6.06 ± 2.056	6.27 ± 2.030	6.69 ± 2.150	6.74 ± 2.006
Week 52, n=57, 57, 118, 221	2.38 ± 2.206	2.36 ± 1.805	5.25 ± 3.248	3.90 ± 2.132
Week 56, n=56, 55, 108, 216	2.39 ± 2.253	2.31 ± 1.843	5.06 ± 3.151	3.80 ± 2.213
Week 60, n=48, 52, 107, 211	2.21 ± 2.176	2.30 ± 1.841	4.86 ± 3.150	3.58 ± 2.122
Week 64, n=43, 45, 108, 207	2.33 ± 2.210	1.94 ± 1.717	4.85 ± 3.317	3.57 ± 2.051
Week 68, n=41, 45, 107, 210	2.45 ± 2.231	1.76 ± 1.378	4.51 ± 3.060	3.43 ± 2.160
Week 80, n=30, 45, 99, 194	2.05 ± 2.233	1.73 ± 1.331	4.18 ± 3.036	3.18 ± 2.017
Week 92, n=23, 43, 94, 179	2.17 ± 1.913	1.65 ± 1.244	4.12 ± 3.477	3.04 ± 2.128
Week 104, n=23, 38, 93, 177	2.86 ± 3.269	1.54 ± 1.227	4.00 ± 4.208	2.92 ± 2.061

Notes
[41] - Only participants with available data were analyzed.
[42] - Only participants with available data were analyzed.
[43] - Only participants with available data were analyzed.
[44] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Change from Baseline in T-VASI scores during the Extension Treatment Period

Top of page

End point title

Change from Baseline in T-VASI scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[45]	57 ^[46]	118 ^[47]	222 ^[48]
Units: scores on a scale
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-3.68 ± 1.564	-3.91 ± 1.526	-1.45 ± 2.271	-2.83 ± 1.938
Week 56, n=56, 55, 108, 216	-3.71 ± 1.615	-3.91 ± 1.503	-1.69 ± 2.217	-2.97 ± 2.086
Week 60, n=48, 52, 107, 211	-3.79 ± 1.692	-3.95 ± 1.585	-1.83 ± 2.161	-3.16 ± 2.027
Week 64, n=43, 45, 108, 207	-3.60 ± 2.033	-4.20 ± 1.569	-1.84 ± 2.375	-3.21 ± 2.060
Week 68, n=41, 45, 107, 210	-3.44 ± 1.789	-4.24 ± 1.534	-2.21 ± 2.273	-3.35 ± 2.143
Week 80, n=30, 45, 99, 194	-3.79 ± 1.663	-4.26 ± 1.519	-2.49 ± 2.175	-3.55 ± 2.109
Week 92, n=23, 43, 94, 179	-3.85 ± 1.545	-4.33 ± 1.673	-2.61 ± 2.624	-3.73 ± 2.246
Week 104, n=23, 38, 93, 177	-3.05 ± 2.156	-4.48 ± 1.702	-2.71 ± 3.479	-3.84 ± 2.151

Notes
[45] - Only participants with available data were analyzed.
[46] - Only participants with available data were analyzed.
[47] - Only participants with available data were analyzed.
[48] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percent change from Baseline in T-VASI scores during the Extension Treatment Period

Top of page

End point title

Percent change from Baseline in T-VASI scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[49]	57 ^[50]	118 ^[51]	222 ^[52]
Units: percentage change
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-65.01 ± 23.969	-64.95 ± 21.547	-24.75 ± 2.271	-42.14 ± 25.783
Week 56, n=56, 55, 108, 216	-65.16 ± 24.916	-65.69 ± 22.042	-28.05 ± 30.487	-43.91 ± 27.408
Week 60, n=48, 52, 107, 211	-66.83 ± 26.478	-65.58 ± 22.910	-31.01 ± 28.944	-47.00 ± 26.089
Week 64, n=43, 45, 108, 207	-62.82 ± 33.328	-70.44 ± 20.212	-30.85 ± 32.898	-46.96 ± 26.395
Week 68, n=41, 45, 107, 210	-61.40 ± 29.204	-71.89 ± 17.942	-35.53 ± 32.135	-49.41 ± 27.629
Week 80, n=30, 45, 99, 194	-68.99 ± 26.870	-72.21 ± 17.852	-40.97 ± 30.033	-52.48 ± 26.991
Week 92, n=23, 43, 94, 179	-66.95 ± 24.885	-72.55 ± 19.152	-43.34 ± 34.425	-55.00 ± 28.995
Week 104, n=23, 38, 93, 177	-58.38 ± 35.533	-74.66 ± 17.727	-45.52 ± 41.968	-56.96 ± 27.409

Notes
[49] - Only participants with available data were analyzed.
[50] - Only participants with available data were analyzed.
[51] - Only participants with available data were analyzed.
[52] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Mean Facial Body Surface Area (F-BSA) scores during the Extension Treatment Period

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End point title

Mean Facial Body Surface Area (F-BSA) scores during the Extension Treatment Period

End point description

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.

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[53]	57 ^[54]	118 ^[55]	222 ^[56]
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline, n=57, 57, 118, 222	0.92 ± 0.498	1.10 ± 0.745	1.01 ± 0.632	1.02 ± 0.636
Week 52, n=57, 57, 118, 221	0.18 ± 0.240	0.27 ± 0.401	0.81 ± 0.641	0.70 ± 0.568
Week 56, n=56, 55, 108, 216	0.18 ± 0.198	0.27 ± 0.416	0.77 ± 0.638	0.67 ± 0.572
Week 60, n=48, 51, 107, 211	0.20 ± 0.225	0.25 ± 0.309	0.73 ± 0.616	0.63 ± 0.557
Week 64, n=43, 45, 108, 207	0.20 ± 0.206	0.20 ± 0.271	0.71 ± 0.607	0.61 ± 0.566
Week 68, n=41, 45, 107, 210	0.22 ± 0.258	0.16 ± 0.183	0.67 ± 0.581	0.57 ± 0.548
Week 80, n=30, 45, 99, 194	0.12 ± 0.138	0.15 ± 0.200	0.62 ± 0.565	0.55 ± 0.564
Week 92, n=23, 43, 94, 179	0.10 ± 0.130	0.15 ± 0.180	0.64 ± 0.595	0.49 ± 0.527
Week 104, n=23, 38, 93, 177	0.13 ± 0.154	0.15 ± 0.181	0.62 ± 0.602	0.47 ± 0.509

Notes
[53] - Only participants with available data were analyzed.
[54] - Only participants with available data were analyzed.
[55] - Only participants with available data were analyzed.
[56] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Change from Baseline in F-BSA scores during the Extension Treatment Period

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End point title

Change from Baseline in F-BSA scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[57]	57 ^[58]	118 ^[59]	222 ^[60]
Units: scores on a scale
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-0.74 ± 0.402	-0.83 ± 0.638	-0.20 ± 0.313	-0.31 ± 0.435
Week 56, n=56, 55, 108, 216	-0.76 ± 0.458	-0.82 ± 0.672	-0.25 ± 0.345	-0.35 ± 0.433
Week 60, n=48, 51, 107, 211	-0.77 ± 0.441	-0.80 ± 0.601	-0.28 ± 0.364	-0.38 ± 0.454
Week 64, n=43, 45, 108, 207	-0.74 ± 0.446	-0.83 ± 0.625	-0.29 ± 0.417	-0.41 ± 0.457
Week 68, n=41, 45, 107, 210	-0.69 ± 0.345	-0.86 ± 0.657	-0.31 ± 0.425	-0.45 ± 0.489
Week 80, n=30, 45, 99, 194	-0.74 ± 0.381	-0.87 ± 0.637	-0.37 ± 0.494	-0.48 ± 0.521
Week 92, n=23, 43, 94, 179	-0.76 ± 0.374	-0.87 ± 0.678	-0.36 ± 0.558	-0.54 ± 0.532
Week 104, n=23, 38, 93, 177	-0.70 ± 0.400	-0.93 ± 0.716	-0.39 ± 0.604	-0.54 ± 0.548

Notes
[57] - Only participants with available data were analyzed.
[58] - Only participants with available data were analyzed.
[59] - Only participants with available data were analyzed.
[60] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percent change from Baseline in F-BSA scores during the Extension Treatment Period

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End point title

Percent change from Baseline in F-BSA scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[61]	57 ^[62]	118 ^[63]	222 ^[64]
Units: percentage change
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-80.75 ± 18.726	-76.75 ± 23.470	-22.15 ± 28.268	-29.06 ± 36.354
Week 56, n=56, 55, 108, 216	-80.23 ± 21.828	-74.42 ± 35.048	-26.20 ± 30.604	-33.66 ± 33.944
Week 60, n=48, 51, 107, 211	-79.20 ± 19.463	-77.67 ± 20.946	-30.04 ± 30.751	-36.86 ± 33.289
Week 64, n=43, 45, 108, 207	-75.92 ± 32.175	-80.06 ± 22.142	-30.67 ± 33.473	-39.58 ± 31.992
Week 68, n=41, 45, 107, 210	-77.11 ± 23.565	-83.14 ± 21.999	-32.60 ± 34.575	-43.61 ± 32.549
Week 80, n=30, 45, 99, 194	-84.55 ± 18.730	-84.67 ± 22.014	-36.79 ± 36.826	-46.38 ± 34.407
Week 92, n=23, 43, 94, 179	-87.81 ± 15.738	-83.24 ± 23.746	-34.05 ± 50.762	-51.96 ± 31.510
Week 104, n=23, 38, 93, 177	-82.87 ± 23.245	-84.09 ± 22.920	-35.82 ± 52.646	-52.85 ± 32.983

Notes
[61] - Only participants with available data were analyzed.
[62] - Only participants with available data were analyzed.
[63] - Only participants with available data were analyzed.
[64] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Mean Total Body Surface Area (T-BSA) scores during the Extension Treatment Period

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End point title

Mean Total Body Surface Area (T-BSA) scores during the Extension Treatment Period

End point description

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.

End point type

Secondary

End point timeframe

up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[65]	57 ^[66]	118 ^[67]	222 ^[68]
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline, n=57, 57, 118, 222	6.79 ± 2.157	6.85 ± 1.924	7.42 ± 2.064	7.49 ± 2.006
Week 52, n=57, 57, 118, 221	4.09 ± 2.838	4.20 ± 2.640	6.95 ± 3.403	5.96 ± 2.352
Week 56, n=56, 55, 108, 216	4.11 ± 2.880	4.13 ± 2.788	6.73 ± 3.281	5.81 ± 2.558
Week 60, n=48, 51, 107, 211	3.77 ± 2.756	4.09 ± 2.833	6.61 ± 3.262	5.64 ± 2.479
Week 64, n=43, 45, 108, 207	3.98 ± 3.087	3.49 ± 2.452	6.67 ± 3.448	5.64 ± 2.483
Week 68, n=41, 45, 107, 210	3.92 ± 2.813	3.27 ± 2.310	6.46 ± 3.239	5.51 ± 2.682
Week 80, n=30, 45, 99, 194	3.72 ± 3.151	3.18 ± 2.232	6.09 ± 3.313	5.27 ± 2.581
Week 92, n=23, 43, 94, 179	3.94 ± 2.834	3.04 ± 2.194	5.96 ± 3.646	5.15 ± 2.741
Week 104, n=23, 38, 93, 177	4.56 ± 4.156	2.93 ± 2.301	5.91 ± 4.475	5.07 ± 2.699

Notes
[65] - Only participants with available data were analyzed.
[66] - Only participants with available data were analyzed.
[67] - Only participants with available data were analyzed.
[68] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Change from Baseline in T-BSA scores during the Extension Treatment Period

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End point title

Change from Baseline in T-BSA scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[69]	57 ^[70]	118 ^[71]	222 ^[72]
Units: scores on a scale
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-2.70 ± 1.830	-2.64 ± 1.906	-0.47 ± 2.325	-1.52 ± 1.711
Week 56, n=56, 55, 108, 216	-2.74 ± 1.918	-2.70 ± 1.983	-0.73 ± 2.238	-1.71 ± 2.059
Week 60, n=48, 51, 107, 211	-2.95 ± 1.910	-2.75 ± 2.195	-0.80 ± 2.128	-1.86 ± 1.978
Week 64, n=43, 45, 108, 207	-2.73 ± 2.528	-3.22 ± 1.883	-0.73 ± 2.432	-1.90 ± 2.052
Week 68, n=41, 45, 107, 210	-2.73 ± 1.958	-3.34 ± 1.922	-0.98 ± 2.262	-2.03 ± 2.180
Week 80, n=30, 45, 99, 194	-3.05 ± 2.083	-3.42 ± 1.809	-1.31 ± 2.341	-2.21 ± 2.169
Week 92, n=23, 43, 94, 179	-3.03 ± 1.664	-3.58 ± 1.938	-1.49 ± 2.753	-2.36 ± 2.380
Week 104, n=23, 38, 93, 177	-2.18 ± 2.723	-3.72 ± 2.075	-1.52 ± 3.672	-2.42 ± 2.307

Notes
[69] - Only participants with available data were analyzed.
[70] - Only participants with available data were analyzed.
[71] - Only participants with available data were analyzed.
[72] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percent change from Baseline in T-BSA scores during the Extension Treatment Period

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End point title

Percent change from Baseline in T-BSA scores during the Extension Treatment Period

End point description

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[73]	57 ^[74]	118 ^[75]	222 ^[76]
Units: percentage change
arithmetic mean (standard deviation)
Week 52, n=57, 57, 118, 221	-43.81 ± 27.916	-41.50 ± 28.747	-8.54 ± 28.806	-20.57 ± 23.042
Week 56, n=56, 55, 108, 216	-44.22 ± 29.590	-43.12 ± 29.489	-11.96 ± 28.156	-22.98 ± 26.032
Week 60, n=48, 51, 107, 211	-47.61 ± 29.237	-43.09 ± 31.608	-13.40 ± 26.410	-25.12 ± 25.564
Week 64, n=43, 45, 108, 207	-43.29 ± 40.045	-50.59 ± 27.166	-12.00 ± 30.202	-25.15 ± 26.860
Week 68, n=41, 45, 107, 210	-44.53 ± 30.086	-52.51 ± 26.921	-15.01 ± 29.093	-27.59 ± 28.476
Week 80, n=30, 45, 99, 194	-50.51 ± 33.794	-53.98 ± 25.682	-20.22 ± 30.154	-29.92 ± 27.996
Week 92, n=23, 43, 94, 179	-48.53 ± 29.457	-55.56 ± 26.401	-22.84 ± 33.708	-31.98 ± 30.805
Week 104, n=23, 38, 93, 177	-40.84 ± 40.076	-57.40 ± 27.898	-24.26 ± 42.193	-32.96 ± 29.907

Notes
[73] - Only participants with available data were analyzed.
[74] - Only participants with available data were analyzed.
[75] - Only participants with available data were analyzed.
[76] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Percentage of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 or 5 during the Extension Treatment Period

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End point title

Percentage of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 or 5 during the Extension Treatment Period

End point description

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.

End point type

Secondary

End point timeframe

Baseline; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	57 ^[77]	57 ^[78]	118 ^[79]	222 ^[80]
Units: percentage of participants
number (confidence interval 95%)
Week 52, n=57, 57, 118, 221	49.1 (35.6 to 62.7)	42.1 (29.1 to 55.9)	11.9 (6.6 to 19.1)	35.3 (29.0 to 42.0)
Week 56, n=56, 55, 108, 216	48.2 (34.7 to 62.0)	34.5 (22.2 to 48.6)	20.4 (13.2 to 29.2)	30.1 (24.1 to 36.7)
Week 60, n=49, 52, 107, 210	46.9 (32.5 to 61.7)	28.8 (17.1 to 43.1)	19.6 (12.6 to 28.4)	27.6 (21.7 to 34.2)
Week 64, n=43, 45, 108, 206	53.5 (37.7 to 68.8)	37.8 (23.8 to 53.5)	23.1 (15.6 to 32.2)	28.2 (22.1 to 34.8)
Week 68, n=41, 45, 107, 210	46.3 (30.7 to 62.6)	46.7 (31.7 to 62.1)	20.6 (13.4 to 29.5)	33.8 (27.4 to 40.6)
Week 80, n=31, 45, 99, 195	38.7 (21.8 to 57.8)	42.2 (27.7 to 57.8)	28.3 (19.7 to 38.2)	32.8 (26.3 to 39.9)
Week 92, n=23, 43, 95, 180	39.1 (19.7 to 61.5)	37.2 (23.0 to 53.3)	28.4 (19.6 to 38.6)	41.1 (33.8 to 48.7)
Week 104, n=23, 38, 93, 178	56.5 (34.5 to 76.8)	50.0 (33.4 to 66.6)	30.1 (21.0 to 40.5)	43.3 (35.9 to 50.9)

Notes
[77] - Only participants with available data were analyzed.
[78] - Only participants with available data were analyzed.
[79] - Only participants with available data were analyzed.
[80] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Change from Week 52 in Dermatology Life Quality Index (DLQI) total score during the Extension Treatment Period

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End point title

Change from Week 52 in Dermatology Life Quality Index (DLQI) total score during the Extension Treatment Period

End point description

The DLQI is a simple, 10-question validated questionnaire to measure how much the skin problem has affected the participant over the previous 7 days. Participants age ≥16 years answered the questionnaire with: (1) very much; (2) a lot; (3) a little; or (4) not at all. The questionnaire was analyzed under 6 headings: Symptoms and feelings (Questions 1 and 2); Daily activities (Questions 3 and 4); Leisure (Questions 5 and 6); Work and school (Question 7); Personal relations (Questions 8 and 9); and Treatment (Question 10). The total score range from 10 to 40; higher scores indicate higher quality of life. Change from Week 52 was calculated as the post-Week 52 value minus the Week 52 value.

End point type

Secondary

End point timeframe

Week 52; up to up to Week 104 of Extension Study (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	54 ^[81]	51 ^[82]	114 ^[83]	200 ^[84]
Units: scores on a scale
arithmetic mean (standard deviation)
Week 52, n=54, 51, 114, 200	2.87 ± 3.059	4.10 ± 4.784	3.69 ± 3.428	3.53 ± 4.150
Change from Week 52 at Week 56, n=53, 49, 104, 194	0.11 ± 2.054	-0.47 ± 1.757	-0.05 ± 2.138	-0.22 ± 2.483
Change from Week 52 at Week 60, n=46, 46, 103, 188	0.37 ± 2.388	0.13 ± 2.227	0.28 ± 2.491	0.24 ± 2.929
Change from Week 52 at Week 64, n=40, 40, 104, 184	0.35 ± 2.578	0.18 ± 2.630	-0.06 ± 2.712	-0.14 ± 2.350
Change from Week 52 at Week 68, n=38, 40, 103, 188	-0.08 ± 2.508	-0.15 ± 2.202	-0.17 ± 2.501	0.08 ± 2.454
Change from Week 52 at Week 80, n=29, 40, 95, 175	0.03 ± 3.006	-0.23 ± 2.315	-0.77 ± 2.871	-0.13 ± 2.809
Change from Week 52 at Week 92, n=22, 39, 91, 160	0.27 ± 2.354	-0.23 ± 1.898	-0.89 ± 2.383	-0.22 ± 2.941
Change from Week 52 at Week 104, n=21, 35, 89, 158	0.57 ± 2.135	-0.40 ± 1.538	-0.48 ± 2.277	-0.06 ± 2.813

Notes
[81] - Only participants with available data were analyzed.
[82] - Only participants with available data were analyzed.
[83] - Only participants with available data were analyzed.
[84] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Change from Week 52 in Children's Dermatology Life Quality Index (CDLQI) total score during the Extension Treatment Period

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End point title

Change from Week 52 in Children's Dermatology Life Quality Index (CDLQI) total score during the Extension Treatment Period

End point description

The CDLQI is the youth/children’s version of the DLQI. The DLQI is a simple, 10-question validated questionnaire to measure how much the skin problem has affected the participant over the previous 7 days. Participants age <16 years answered the questionnaire with: (1) very much; (2) a lot; (3) a little; or (4) not at all. The questionnaire was analyzed under 6 headings: Symptoms and feelings (Questions 1 and 2); Leisure (Questions 4, 5, and 6); School or holidays (Question 7); Personal relationships (Questions 3 and 8); Sleep (Question 9); and Treatment (Question 10). The total score ranges from 10 to 40; higher scores indicate higher quality of life. Change from Week 52 was calculated as the post-Week 52 value minus the Week 52 value. 9999=the mean and standard deviation cannot be calculated for a single participant.

End point type

Secondary

End point timeframe

Week 52; up to Week 104 of Treatment Extension (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID
Number of subjects analysed	3 ^[85]	6 ^[86]	4 ^[87]	22 ^[88]
Units: scores on a scale
arithmetic mean (standard deviation)
Week 52, n=3, 6, 4, 22	1.67 ± 2.887	1.00 ± 0.632	1.25 ± 1.893	2.32 ± 3.872
Change from Week 52 at Week 56, n=3, 6, 4, 22	-1.00 ± 2.646	-0.17 ± 0.753	0.00 ± 0.816	0.36 ± 1.989
Change from Week 52 at Week 60, n=3, 6, 4, 22	0.00 ± 0.000	-0.50 ± 0.548	-0.50 ± 0.577	-0.41 ± 2.108
Change from Week 52 at Week 64, n=3, 5, 4, 22	-1.33 ± 2.309	0.00 ± 0.707	-1.00 ± 1.414	-0.36 ± 1.399
Change from Week 52 at Week 68, n=3, 5, 4, 22	-0.67 ± 1.155	0.40 ± 1.140	0.50 ± 0.577	-0.23 ± 1.541
Change from Week 52 at Week 80, n=2, 5, 4, 20	-2.00 ± 2.828	-0.40 ± 0.548	-0.75 ± 1.708	0.15 ± 1.814
Change from Week 52 at Week 92, n=1, 4, 4, 20	9999 ± 9999	0.25 ± 1.258	-0.25 ± 0.957	0.70 ± 3.614
Change from Week 52 at Week 104, n=2, 3, 4, 20	-0.50 ± 0.707	-0.67 ± 1.155	-1.25 ± 1.893	-0.20 ± 4.086

Notes
[85] - Only participants with available data were analyzed.
[86] - Only participants with available data were analyzed.
[87] - Only participants with available data were analyzed.
[88] - Only participants with available data were analyzed.

No statistical analyses for this end point

Secondary: Number of participants with any treatment-emergent adverse event (TEAE)

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End point title

Number of participants with any treatment-emergent adverse event (TEAE)

End point description

A TEAE was defined as any adverse event (AE) reported for the first time or the worsening of a pre-existing event after the first application of study drug in this study. 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 therefore 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.

End point type

Secondary

End point timeframe

up to approximately Week 108 (Week 52 was the first visit of this Treatment Extension study.)

End point values	Cohort A: Vehicle cream BID	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Vehicle cream BID to ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream to ruxolitinib 1.5% cream BID	Cohort A: Vehicle cream BID to ruxolitinib 1.5% cream BID
Number of subjects analysed	58	58	118	224	23
Units: participants	21	32	59	114	3

No statistical analyses for this end point

Secondary: Trough plasma concentrations of ruxolitinib at Week 80 and Week 104

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End point title

Trough plasma concentrations of ruxolitinib at Week 80 and Week 104 ^[89]

End point description

The steady-state plasma concentration was assessed. Pharmacokinetic blood samples could have been collected at any time prior to study drug application at the site at the Week 80 visit and at any time at the Week 104 (End of Trial) visit.

End point type

Secondary

End point timeframe

Weeks 80 (predose); Week 104 (any time post-dose) (Week 52 was the first visit of this Treatment Extension study.)

Notes
[89] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistical analysis was not conducted for this endpoint.

End point values	Cohort A: Ruxolitinib 1.5% cream BID	Cohort B: Ruxolitinib 1.5% cream BID	Cohorts A and B: Ruxolitinib 1.5% cream BID
Number of subjects analysed	54 ^[90]	293 ^[91]	347 ^[92]
Units: nanomolar
arithmetic mean (standard deviation)
12 to <18 years, n=8, 40, 48	7.28 ± 11.7	5.56 ± 10.5	5.85 ± 10.6
18 to <65 years, n=43, 229, 272	15.1 ± 19.5	12.7 ± 17.2	13.1 ± 17.6
≥65 years, n=3, 24, 27	13.0 ± 9.44	25.7 ± 20.9	24.3 ± 20.2
Overall, n=54, 293, 347	13.8 ± 18.1	12.8 ± 17.4	12.9 ± 17.5

Notes
[90] - Only participants with available data were analyzed.
[91] - Only participants with available data were analyzed.
[92] - Only participants with available data were analyzed.

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

up to approximately Week 108 (Week 52 was the first visit of this Treatment Extension study.)

Adverse event reporting additional description

Treatment-emergent adverse events, defined as adverse events reported for the first time or the worsening of pre-existing events after the first application of study drug, have been reported.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

23.1

Reporting group title

Vehicle cream BID

Reporting group description

Reporting group title

Total

Reporting group description

Total

Reporting group title

Ruxolitinib 1.5% cream BID

Reporting group description

Participants who completed treatment and achieved ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants were randomized to receive ruxolitinib 1.5% cream BID for 52 weeks, or were randomized to receive vehicle cream BID but experienced relapse (<F-VASI75) and received open-label ruxolitinib 1.5% cream BID for the duration of the study. Participants who completed treatment (vehicle cream BID for 24 weeks and ruxolitinib 1.5% BID for 28 weeks) and did not achieve ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants applied ruxolitinib 1.5% cream BID for 52 weeks. Participants who completed treatment (ruxolitinib 1.5% BID for 52 weeks) and did not achieve ≥F-VASI90 at Week 52 in either of 2 parent studies (NCT04052425 or NCT04057573). In this study, these participants applied ruxolitinib 1.5% cream BID for 52 weeks.

Serious adverse events	Vehicle cream BID	Total	Ruxolitinib 1.5% cream BID
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 58 (0.00%)	12 / 458 (2.62%)	12 / 423 (2.84%)
number of deaths (all causes)	0	0	0
number of deaths resulting from adverse events	0	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Injury, poisoning and procedural complications
Spinal fracture
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Hip fracture
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Cardiac disorders
Angina pectoris
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Ear and labyrinth disorders
Otosclerosis
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Reproductive system and breast disorders
Pelvic prolapse
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Rectocele
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Uterine prolapse
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Cystocele
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Hepatobiliary disorders
Cholelithiasis
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Psychiatric disorders
Bipolar I disorder
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Mental status changes
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Spinal osteoarthritis
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 2	0 / 2
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Intervertebral disc disorder
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Infections and infestations
COVID-19 pneumonia
subjects affected / exposed	0 / 58 (0.00%)	1 / 458 (0.22%)	1 / 423 (0.24%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Vehicle cream BID	Total	Ruxolitinib 1.5% cream BID
Total subjects affected by non serious adverse events
subjects affected / exposed	6 / 58 (10.34%)	61 / 458 (13.32%)	55 / 423 (13.00%)
Infections and infestations
COVID-19
subjects affected / exposed	6 / 58 (10.34%)	61 / 458 (13.32%)	55 / 423 (13.00%)
occurrences all number	6	63	57

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Were there any global substantial amendments to the protocol? Yes

28 Sep 2020

The primary purpose of this amendment was to accommodate the European Medicines Agency Pediatric Committee recommendation regarding the Dermatology Life Quality Index/Children's Dermatology Life Quality Index endpoint and to align the pharmacokinetic endpoints with those of parent studies INCB 18424-306 and INCB 18424-307.

10 Nov 2020

The main purpose of this amendment was to add language to the Protocol to inform sites of alternative strategies to guarantee continuity of the clinical trial conduct and oversight in response to the coronavirus disease 2019 (COVID-19) pandemic.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

Data from participants at a single site (N = 2) were removed from all efficacy analyses performed on the ITT-Ext Population and FAS Cohort A owing to noncompliance with the Protocol in the parent study resulting in serious concerns with data quality.

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Clinical trials

Clinical Trial Results: A Double-Blind, Vehicle-Controlled, Randomized Withdrawal and Treatment Extension Study to Assess the Long-Term Efficacy and Safety of Ruxolitinib Cream in Participants With Vitiligo

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Time to relapse (defined as <F-VASI75)

Primary: Time to relapse (defined as <F-VASI75)

Secondary: Time to loss of adequate response

Secondary: Time to loss of adequate response

Secondary: Percentage of participants achieving a ≥50% improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥50% improvement from Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥75% improvement from Baseline in the F-VASI (F-VASI75) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥75% improvement from Baseline in the F-VASI (F-VASI75) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥90% improvement from Baseline in the F-VASI (F-VASI90) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥90% improvement from Baseline in the F-VASI (F-VASI90) score during the Extension Treatment Period

Secondary: Mean F-VASI scores during the Extension Treatment Period

Secondary: Mean F-VASI scores during the Extension Treatment Period

Secondary: Change from Baseline in F-VASI scores during the Extension Treatment Period

Secondary: Change from Baseline in F-VASI scores during the Extension Treatment Period

Secondary: Percent change from Baseline in F-VASI scores during the Extension Treatment Period

Secondary: Percent change from Baseline in F-VASI scores during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥50% improvement from Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥50% improvement from Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥75% improvement from Baseline in the T-VASI (T-VASI75) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥75% improvement from Baseline in the T-VASI (T-VASI75) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥90% improvement from Baseline in the T-VASI (T-VASI90) score during the Extension Treatment Period

Secondary: Percentage of participants achieving a ≥90% improvement from Baseline in the T-VASI (T-VASI90) score during the Extension Treatment Period

Secondary: Mean T-VASI scores during the Extension Treatment Period

Secondary: Mean T-VASI scores during the Extension Treatment Period

Secondary: Change from Baseline in T-VASI scores during the Extension Treatment Period

Secondary: Change from Baseline in T-VASI scores during the Extension Treatment Period

Secondary: Percent change from Baseline in T-VASI scores during the Extension Treatment Period

Secondary: Percent change from Baseline in T-VASI scores during the Extension Treatment Period

Secondary: Mean Facial Body Surface Area (F-BSA) scores during the Extension Treatment Period

Secondary: Mean Facial Body Surface Area (F-BSA) scores during the Extension Treatment Period

Secondary: Change from Baseline in F-BSA scores during the Extension Treatment Period

Secondary: Change from Baseline in F-BSA scores during the Extension Treatment Period

Secondary: Percent change from Baseline in F-BSA scores during the Extension Treatment Period

Secondary: Percent change from Baseline in F-BSA scores during the Extension Treatment Period

Secondary: Mean Total Body Surface Area (T-BSA) scores during the Extension Treatment Period

Secondary: Mean Total Body Surface Area (T-BSA) scores during the Extension Treatment Period

Secondary: Change from Baseline in T-BSA scores during the Extension Treatment Period

Secondary: Change from Baseline in T-BSA scores during the Extension Treatment Period

Secondary: Percent change from Baseline in T-BSA scores during the Extension Treatment Period

Secondary: Percent change from Baseline in T-BSA scores during the Extension Treatment Period

Secondary: Percentage of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 or 5 during the Extension Treatment Period

Secondary: Percentage of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 or 5 during the Extension Treatment Period

Secondary: Change from Week 52 in Dermatology Life Quality Index (DLQI) total score during the Extension Treatment Period

Secondary: Change from Week 52 in Dermatology Life Quality Index (DLQI) total score during the Extension Treatment Period

Secondary: Change from Week 52 in Children's Dermatology Life Quality Index (CDLQI) total score during the Extension Treatment Period

Secondary: Change from Week 52 in Children's Dermatology Life Quality Index (CDLQI) total score during the Extension Treatment Period

Secondary: Number of participants with any treatment-emergent adverse event (TEAE)

Secondary: Number of participants with any treatment-emergent adverse event (TEAE)

Secondary: Trough plasma concentrations of ruxolitinib at Week 80 and Week 104

Secondary: Trough plasma concentrations of ruxolitinib at Week 80 and Week 104

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Double-Blind, Vehicle-Controlled, Randomized Withdrawal and Treatment Extension Study to Assess the Long-Term Efficacy and Safety of Ruxolitinib Cream in Participants With Vitiligo