Non-sclerotic Cutaneous Chronic Graft-versus-host Disease
Conditions
Keywords
Ruxolitinib Cream, 18-412
Brief summary
The purpose of this study is to compare the safety and effects of ruxolitinib 1.5% cream with those of standard moisturizers in people with non-sclerotic chronic cutaneous GVHD.
Interventions
Patients will be prescribed twice daily use of topical ruxolitinib 1.5% cream (randomized half of face/body) to a maximum of 20% BSA on each side for 28 ± 3 days. Topical ruxolitinib will be provided as topical cream. At the end of the 28 ± 3 days study visit, patients will remain blinded to treatment arms. After trial completion (day 28 ± 3 days), all patients will be offered repeated treatment cycle with topical ruxolitinib or standard of care therapy.
OTHERTopical vehicle/moisturizer cream
Patients will be prescribed twice daily use of vehicle/moisturizer (for contralateral side of face/body) to a maximum of 20% BSA on each side for 28 ± 3 days.At the end of the 28 ± 3 days study visit, patients will remain blinded to treatment arms. After trial completion (day 28 ± 3 days), all patients will be offered repeated treatment cycle with topical ruxolitinib or standard of care therapy.
Sponsors
Memorial Sloan Kettering Cancer Center
Hackensack Meridian Health
Incyte Corporation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients (≥ 12 years) * History of allogeneic hematopoietic stem cell transplantation * BSA of at least 2% of clinically or histologically confirmed non-sclerotic cutaneous chronic graft-versus-host disease (diagnosed and BSA calculated in accordance with the National Institutes of Health Chronic Graft-versus-Host Disease Consensus for Clinical Trials: I. The 2014 Diagnosis and Staging Working Group Report) * Patients age ≥ 18 years must provide written informed consent; or patients age ≥12 years and \<18 years must provide assent and have at least one guardian provide written informed consent to participate in the study. * Able to self-administer topical interventions or provide for another person to apply the topical interventions (while wearing nitrile gloves) * If on systemic therapy for GVHD, systemic therapy must be stable for past 4 weeks; however, any planned systemic corticosteroid taper during the study will be permitted.Changes in systemic therapy during the study period will be allowed for the management of non-skin GVHD. * Any concurrent topical therapies including topical corticosteroids, topical calcienurin inhibitors, moisturizers, phototherapy (narrowband UVB or UVA1); or excimer laser therapy must be discontinued on Study Day 0.
Exclusion criteria
* Known history of allergy to any ingredient of the study medication * Patients with deep sclerotic cutaneous graft-versus-host disease including deep sclerotic subtypes of chronic cutaneous GVHD * Use of concurrent topical therapy including topical corticosteroids, topical calcienurin inhibitors, moisturizers, phototherapy (narrowband UVB or UVA1); or excimer laser therapy after Study Day 0 up to and including Study Day 28. * Changes in systemic therapy during study period for the purpose of treating skin GVHD. * Special populations: * vulnerable populations e.g. decisionally impaired (cognitive, psychiatric), terminally ill, prisoners * patients who, in the opinion of the investigator have a condition that precludes their ability to provide an informed consent * Concurrent participation in another topical trial of a drug(s) or medical device, or the subject is in an exclusion period after a previous trial of drug(s) or medical device * Pregnancy or lactation * Patients with inadequate liver function (ALT above 4 × upper limit of normal \[ULN\] for the patient's age or direct bilirubin 4 × ULN for the patient's age and the laboratory abnormalities are considered to be due to underlying liver dysfunction) unless attributed to GVHD (if direct bilirubin is not in the medical record, it is acceptable to use total bilirubin x4 ULN). * Active uncontrolled infection requiring systemic therapy. Subjects with a controlled infection receiving definitive therapy for 48 hours prior to enrollment are eligible.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Reduction in Body Surface Area (BSA) From Day 1 and Day 28 | 28 days (+/-3 days) | The difference in BSA of non-sclerotic cutaneous cGVHD between ruxolitinib treated side and vehicle treated sides of the face/body at study completion |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28 | 28 days (+/-3 days) | 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%. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORAlina Markova, MD
Memorial Sloan Kettering Cancer Center
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 52 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 5 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 19 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 14 Participants |
| Region of Enrollment United States | 24 Participants |
| Sex: Female, Male Female | 13 Participants |
| Sex: Female, Male Male | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 8 / 24 | 0 / 12 | 0 / 12 |
| other Total, other adverse events | 7 / 24 | 0 / 12 | 0 / 12 |
| serious Total, serious adverse events | 2 / 24 | 0 / 12 | 0 / 12 |
Outcome results
None listed