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

• ClinicalTrials.gov Identifier: NCT03954236
• Recruitment Status: Recruiting
• First Posted: May 17, 2019
• Last Update Posted: March 4, 2022
• Sponsor: Memorial Sloan Kettering Cancer Center
• Collaborators: Hackensack Meridian Health; Incyte Corporation
• Information provided by (Responsible Party): Memorial Sloan Kettering Cancer Center

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Non-sclerotic Cutaneous Chronic Graft-versus-host Disease	Drug: topical ruxolitinib 1.5% cream; Other: Topical vehicle/moisturizer cream	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 24 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention 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 (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: May 14, 2019
• Estimated Primary Completion Date: May 2023
• Estimated Study Completion Date: May 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: topical ruxolitinib BID to left side of face/body; And topical moisturizer BID to right side of face/body.	Drug: topical ruxolitinib 1.5% cream; 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.; Other: Topical 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.
Experimental: topical ruxolitinib BID to right side of face/body; And topical moisturizer BID to left side of face/body.	Drug: topical ruxolitinib 1.5% cream; 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.; Other: Topical 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.

Outcome Measures

Primary Outcome Measures :

1. 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 [ Time Frame: 28 days (+/-3 days) ]

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Contacts

Contact: Alina Markova, MD; 646-608-2342; markovaa@mskcc.org

Contact: Doris Ponce, MD; 212-639-4838

Locations

United States, New York

Memorial Sloan Kettering Cancer Center; Recruiting

New York, New York, United States, 10065

Contact: Alina Markova, MD 646-608-2342

Contact: Doris Ponce, MD 212-639-4838

Sponsors and Collaborators

Memorial Sloan Kettering Cancer Center

Hackensack Meridian Health

Incyte Corporation

Investigators

• Principal Investigator: Alina Markova, MD; Memorial Sloan Kettering Cancer Center

More Information

Additional Information:

Memorial Sloan Kettering Cancer Center 

• Responsible Party: Memorial Sloan Kettering Cancer Center
• ClinicalTrials.gov Identifier: NCT03954236
• Other Study ID Numbers: 18-412
• First Posted: May 17, 2019
• Last Update Posted: March 4, 2022
• Last Verified: March 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
• Supporting Materials: Statistical Analysis Plan (SAP); Informed Consent Form (ICF)

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Memorial Sloan Kettering Cancer Center:

Ruxolitinib Cream; 18-412

Additional relevant MeSH terms:

Graft vs Host Disease; Immune System Diseases