Study of the Safety and Efficacy of Ruxolitinib Cream for Non-Sclerotic Chronic Cutaneous Graft-Versus-Host Disease

Study Overview
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Brief Summary
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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.

Official Title
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A Pilot, Prospective, Randomized, Double-Blinded, Vehicle- and Comparator-Controlled Trial on Safety and Efficacy of a Topical Inhibitor of Janus Kinase 1/2 (Ruxolitinib INCB018424 Phosphate 1.5% Cream) for Non-Sclerotic Chronic Cutaneous Graft-Versus-Host Disease

Conditions
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Non-sclerotic Cutaneous Chronic Graft-versus-host Disease

Intervention / Treatment
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* Drug: topical ruxolitinib 1.5% cream
* Other: Topical vehicle/moisturizer cream


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: This is a prospective, randomized double-blinded, vehicle- and comparator controlled pilot study followed by an open label extension for all patients who wish to continue on study.
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: topical ruxolitinib BID to left side of face/body<br>And topical moisturizer BID to right side of face/body. | Drug: topical ruxolitinib 1.5% cream<br>* 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.<br>Other: Topical vehicle/moisturizer cream<br>* 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.<br>|
| Experimental: topical ruxolitinib BID to right side of face/body<br>And topical moisturizer BID to left side of face/body. | Drug: topical ruxolitinib 1.5% cream<br>* 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.<br>Other: Topical vehicle/moisturizer cream<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| the difference in body surface area (BSA) of non-sclerotic cutaneous cGVHD between ruxolitinib treated side and vehicle treated sides of the face/body at study completion |  | 28 days (+/-3 days) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Ruxolitinib Cream, 18-412