Trial Outcomes & Findings for Topical Ruxolitinib for the Treatment of Vitiligo (NCT NCT02809976)
NCT ID: NCT02809976
Last Updated: 2020-08-31
Results Overview
The VASI for each body region (hands, upper extremities, trunk, lower extremities, feet) is determined by the product of the area involved in hand prints and the extent of depigmentation within each hand print unit measured patch (0-100). Area involved is measured by hand prints (1 hand print = 1%) with a possible range of 0-100. Degree of depigmentation is measured as: 1.00 (100%) = complete depigmentation, no pigment present, 0.90(90%)=specks of pigment present, 0.75(75%)=depigmented area exceeds the pigmented area, 0.50(50%)=pigmented and depigmented areas are equal, 0.25(25%)=pigmented area exceeds depigmented area, 0.10(10%)=only specks of depigmentation present, and 0.0(0%)=no depigmentation present.
COMPLETED
PHASE2
11 participants
Baseline to Week 20
2020-08-31
Participant Flow
Participant milestones
| Measure |
Ruxolitinib 1.5% Phosphate Cream
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Ruxolitinib 1.5% Phosphate Cream
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Topical Ruxolitinib for the Treatment of Vitiligo
Baseline characteristics by cohort
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
52 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
11 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 20The VASI for each body region (hands, upper extremities, trunk, lower extremities, feet) is determined by the product of the area involved in hand prints and the extent of depigmentation within each hand print unit measured patch (0-100). Area involved is measured by hand prints (1 hand print = 1%) with a possible range of 0-100. Degree of depigmentation is measured as: 1.00 (100%) = complete depigmentation, no pigment present, 0.90(90%)=specks of pigment present, 0.75(75%)=depigmented area exceeds the pigmented area, 0.50(50%)=pigmented and depigmented areas are equal, 0.25(25%)=pigmented area exceeds depigmented area, 0.10(10%)=only specks of depigmentation present, and 0.0(0%)=no depigmentation present.
Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Percent Change in Vitiligo Area Severity Index (VASI) Score From Baseline to Week 20
|
23 percentage of change in VASI
Interval 4.0 to 43.0
|
SECONDARY outcome
Timeframe: Baseline and Week 20Area involved is measured by hand prints (1 hand print = 1%) with a possible range of 0-100.
Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Percent Change in Body Surface Area (BSA) of Repigmentation
|
11.2 percentage of change
Standard Deviation 26.4
|
SECONDARY outcome
Timeframe: Baseline and Week 20Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Number of Subjects Who Achieve a Physician Global Vitiligo Assessment (PGVA) of Clear or Almost Clear
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline and Week 20Vitiligo European Task Force assessment consisted of three components: Extent of disease reflecting the body surface area (0-100%), disease staging (0-20), and disease progression (-5 +5). Staging is based on cutaneous and hair pigmentation assessing the largest patch in each body area (head/neck, trunk, arms, legs, and hands/feet); Stage 0=normal pigmentation, Stage 1=incomplete pigmentation, Stage 2=complete depigmentation, Stage 3= partial hair whitening (\<30%), Stage 4= complete hair whitening. Disease progression is based on assessing the largest patch in each body area; Score 0= similar limits, Score 1= progressive vitiligo (ongoing subclinical depigmentation), Score -1= regressive vitiligo (ongoing subclinical repigmentation). Where a higher number indicates more severe disease spread and a negative number indicates improving disease. These three subsets are evaluated and reported independently and not mutually related to each other.
Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Percent Change in Vitiligo European Task Force (VETF) Assessment - Body Surface Area
|
11.2 percent change in body surface area
Standard Deviation 26.4
|
SECONDARY outcome
Timeframe: Baseline and Week 20DLQI is 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. The percent change was calculated from the mean DLQI at baseline and week 20
Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Mean Dermatology Life Quality Index (DLQI) Scores
Week 20
|
3 score on a scale
Standard Deviation 3.46
|
|
Mean Dermatology Life Quality Index (DLQI) Scores
Baseline
|
2.9 score on a scale
Standard Deviation 2.67
|
SECONDARY outcome
Timeframe: Baseline and Week 20Vitiligo European Task Force assessment consisted of three components: Extent of disease reflecting the body surface area (0-100%), disease staging (0-20), and disease progression (-5 +5). Staging is based on cutaneous and hair pigmentation assessing the largest patch in each body area (head/neck, trunk, arms, legs, and hands/feet); Stage 0=normal pigmentation, Stage 1=incomplete pigmentation, Stage 2=complete depigmentation, Stage 3= partial hair whitening (\<30%), Stage 4= complete hair whitening. Disease progression is based on assessing the largest patch in each body area; Score 0= similar limits, Score 1= progressive vitiligo (ongoing subclinical depigmentation), Score -1= regressive vitiligo (ongoing subclinical repigmentation). Where a higher number indicates more severe disease spread and a negative number indicates improving disease. These three subsets are evaluated and reported independently and not mutually related to each other.
Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Percent Change in Vitiligo European Task Force (VETF) Assessment - Disease Staging
|
4.5 percent change in disease staging score
Standard Deviation 2.4
|
SECONDARY outcome
Timeframe: Baseline and Week 20Vitiligo European Task Force assessment consisted of three components: Extent of disease reflecting the body surface area (0-100%), disease staging (0-20), and disease progression (-5 +5). Staging is based on cutaneous and hair pigmentation assessing the largest patch in each body area (head/neck, trunk, arms, legs, and hands/feet); Stage 0=normal pigmentation, Stage 1=incomplete pigmentation, Stage 2=complete depigmentation, Stage 3= partial hair whitening (\<30%), Stage 4= complete hair whitening. Disease progression is based on assessing the largest patch in each body area; Score 0= similar limits, Score 1= progressive vitiligo (ongoing subclinical depigmentation), Score -1= regressive vitiligo (ongoing subclinical repigmentation). Where a higher number indicates more severe disease spread and a negative number indicates improving disease. These three subsets are evaluated and reported independently and not mutually related to each other.
Outcome measures
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 Participants
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Percent Change in Vitiligo European Task Force (VETF) Assessment - Disease Progression
|
-0.5 percent of change
Standard Deviation 1.1
|
Adverse Events
Ruxolitinib 1.5% Phosphate Cream
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ruxolitinib 1.5% Phosphate Cream
n=11 participants at risk
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
Ruxolitinib 1.5% Phosphate Cream: twice daily topical application of Ruxolitinib 1.5% Phosphate Cream beginning at baseline and ending at week 20
|
|---|---|
|
Skin and subcutaneous tissue disorders
Rim of hyperpigmentation surrounding vitiligo lesion
|
81.8%
9/11
|
|
Skin and subcutaneous tissue disorders
Intermittent erythema over affected lesions
|
72.7%
8/11
|
|
Skin and subcutaneous tissue disorders
Transient acne
|
18.2%
2/11
|
|
Respiratory, thoracic and mediastinal disorders
URI symptoms
|
36.4%
4/11
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
9.1%
1/11
|
|
Musculoskeletal and connective tissue disorders
Muscle aches
|
9.1%
1/11
|
|
Metabolism and nutrition disorders
hypercholesterolemia
|
9.1%
1/11
|
|
Social circumstances
Traumatic blister
|
9.1%
1/11
|
|
Social circumstances
Bee sting
|
9.1%
1/11
|
|
General disorders
Dental pain
|
9.1%
1/11
|
|
Gastrointestinal disorders
Gastroenteritis
|
9.1%
1/11
|
Additional Information
David Romarin, MD Prinicpal Investigator
Tufts Medical Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place