Trial Outcomes & Findings for Study of the Safety and Efficacy of Ruxolitinib Cream for Non-Sclerotic Chronic Cutaneous Graft-Versus-Host Disease (NCT NCT03954236)
NCT ID: NCT03954236
Last Updated: 2026-02-10
Results Overview
The difference in BSA of non-sclerotic cutaneous cGVHD between ruxolitinib treated side and vehicle treated sides of the face/body at study completion
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
28 days (+/-3 days)
Results posted on
2026-02-10
Participant Flow
Participant milestones
| Measure |
Group 1: Participants With Graft-versus-host Disease (GVHD)
Topical ruxolitinib BID to left side of face/body, And topical moisturizer BID to right side of face/body
|
Group 2: Participants With Graft-versus-host Disease (GVHD)
Topical ruxolitinib BID to right side of face/body And topical moisturizer BID to left side of face/body
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
12
|
|
Overall Study
COMPLETED
|
10
|
10
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
Group 1: Participants With Graft-versus-host Disease (GVHD)
Topical ruxolitinib BID to left side of face/body, And topical moisturizer BID to right side of face/body
|
Group 2: Participants With Graft-versus-host Disease (GVHD)
Topical ruxolitinib BID to right side of face/body And topical moisturizer BID to left side of face/body
|
|---|---|---|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
2
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Study of the Safety and Efficacy of Ruxolitinib Cream for Non-Sclerotic Chronic Cutaneous Graft-Versus-Host Disease
Baseline characteristics by cohort
| Measure |
Experimental: Participants With Graft-versus-host Disease (GVHD)
n=24 Participants
All Study Participants: topical ruxolitinib BID to one side of face/body
|
|---|---|
|
Age, Continuous
|
52 years
n=41 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=41 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=41 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=41 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=41 Participants
|
|
Region of Enrollment
United States
|
24 Participants
n=41 Participants
|
PRIMARY outcome
Timeframe: 28 days (+/-3 days)Population: 12 participants were assessed for each grouping of interventions
The difference in BSA of non-sclerotic cutaneous cGVHD between ruxolitinib treated side and vehicle treated sides of the face/body at study completion
Outcome measures
| Measure |
Experimental: Participants With Graft-versus-host Disease (GVHD)
n=24 Participants
All study participants: topical ruxolitinib BID to one side of face/body
|
|---|---|
|
Reduction in Body Surface Area (BSA) From Day 1 and Day 28
BSA on Day 1 for Topical Ruxolitinib BID to Right Side of Face/Body
|
14.5 % of body surface area
Standard Error 0.003
|
|
Reduction in Body Surface Area (BSA) From Day 1 and Day 28
BSA on Day 28 for Topical Ruxolitinib BID to Right Side of Face/Body
|
10.4 % of body surface area
Standard Error 0.003
|
|
Reduction in Body Surface Area (BSA) From Day 1 and Day 28
BSA on Day 1 for Topical Ruxolitinib BID to Left Side of Face/Body
|
14.4 % of body surface area
Standard Error 0.003
|
|
Reduction in Body Surface Area (BSA) From Day 1 and Day 28
BSA on Day 28 for Topical Ruxolitinib BID to Left Side of Face/Body
|
6.2 % of body surface area
Standard Error 0.003
|
SECONDARY outcome
Timeframe: 28 days (+/-3 days)Population: 12 participants were assessed for each grouping of interventions
The difference in the Physician's Global Assessment/PGA of non-sclerotic and superficially sclerotic cutaneous cGVHD between ruxolitinib treated side and vehicle treated sides of the face/body at study completion (day 28 visit). Grade of Rash as follows: Grade 0, Clinical Complete Response, Completely clear; NED (100% improvement); Grade 1-3, Partial Response, from very significant clearance to moderate improvement (\>90% to \>/= 50% improvement); Grade 4 and 5, Stable Disease, No change to some improvement (,50% to \>/=25%); Grade 6, Progressive Disease, Disease is worse than at baseline evaluation by \>/= 25%.
Outcome measures
| Measure |
Experimental: Participants With Graft-versus-host Disease (GVHD)
n=24 Participants
All study participants: topical ruxolitinib BID to one side of face/body
|
|---|---|
|
Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28
Topical Ruxolitinib BID to Left Side of Face/Body PGA on Day 1
|
5 units on a scale
Standard Error 0.0004
|
|
Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28
Topical Ruxolitinib BID to Left Side of Face/Body PGA on Day 28
|
1.9 units on a scale
Standard Error 0.0004
|
|
Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28
Topical Ruxolitinib BID to Right Side of Face/Body PGA on Day 1
|
5 units on a scale
Standard Error 0.0004
|
|
Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28
Topical Ruxolitinib BID to Right Side of Face/Body PGA on Day 28
|
3.7 units on a scale
Standard Error 0.0004
|
Adverse Events
Experimental: Participants With Graft-versus-host Disease (GVHD)
Serious events: 2 serious events
Other events: 7 other events
Deaths: 8 deaths
Experimental Left: Participants With Graft-versus-host Disease (GVHD)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Experimental Right: Participants With Graft-versus-host Disease (GVHD)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Experimental: Participants With Graft-versus-host Disease (GVHD)
n=24 participants at risk
All study participants: topical ruxolitinib BID to one side of face/body
|
Experimental Left: Participants With Graft-versus-host Disease (GVHD)
n=12 participants at risk
topical ruxolitinib BID to left side of face/body
|
Experimental Right: Participants With Graft-versus-host Disease (GVHD)
n=12 participants at risk
topical ruxolitinib BID to right side of face/body
|
|---|---|---|---|
|
Infections and infestations
Sinusitis
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm Benign, Malignant and Unspecified (incl cysts and polyps) - Other, specify - CNS Leukemia
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
Other adverse events
| Measure |
Experimental: Participants With Graft-versus-host Disease (GVHD)
n=24 participants at risk
All study participants: topical ruxolitinib BID to one side of face/body
|
Experimental Left: Participants With Graft-versus-host Disease (GVHD)
n=12 participants at risk
topical ruxolitinib BID to left side of face/body
|
Experimental Right: Participants With Graft-versus-host Disease (GVHD)
n=12 participants at risk
topical ruxolitinib BID to right side of face/body
|
|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Nummular eczema
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Skin and subcutaneous tissue disorders
Orofacial dermatitis
|
8.3%
2/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Eye disorders
Eyelash hypotrichosis
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Skin and subcutaneous tissue disorders
Seborrheic dermatitis
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
General disorders
Fever
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
General disorders
Flu like symptoms
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Infections and infestations
Lung infection
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Infections and infestations
Sinusitis
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Eye disorders
Eye irritation
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Skin and subcutaneous tissue disorders
Cracked/thick cuticles
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
|
Nervous system disorders
Headache
|
4.2%
1/24 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
0.00%
0/12 • 28 days (+/-3 days)
|
Additional Information
Dr. Alina Markova, MD
Memorial Sloan Kettering Cancer Center
Phone: 646-608-2380
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place