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

NCT03954236 | Active Not Recruiting Phase 2 Results FDA Drug

• 1 other identifier: 18-412
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Non-sclerotic Cutaneous Chronic Graft-versus-host Disease

Keywords

Ruxolitinib Cream; 18-412

Outcome Measures

Primary Outcomes (1)

• Reduction in Body Surface Area (BSA) From Day 1 and Day 28

  The difference in 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)

Secondary Outcomes (1)

• Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28

  28 days (+/-3 days)

Study Arms (1)

Participants with graft-versus-host disease (GVHD)

EXPERIMENTAL

Participants will have non-sclerotic chronic cutaneous graft-versus-host disease (GVHD) or you have a condition called superficially sclerotic chronic cutaneous graft-versus-host disease (GVHD) after having received an allogeneic hematopoietic stem cell transplant.

Drug: topical ruxolitinib 1.5% cream; Other: Topical vehicle/moisturizer cream

Interventions

topical ruxolitinib 1.5% cream DRUG

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.

Participants with graft-versus-host disease (GVHD)

Topical vehicle/moisturizer cream OTHER

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.

Participants with graft-versus-host disease (GVHD)

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• Hackensack Meridian Health: collaborator
• Incyte Corporation: collaborator

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Interventions

ruxolitinib

Results Point of Contact

• Title: Dr. Alina Markova, MD
• Organization: Memorial Sloan Kettering Cancer Center

markovaa@mskcc.org

646-608-2380

Study Officials

• Alina Markova, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2019
• First Posted: May 17, 2019
• Study Start: May 14, 2019
• Primary Completion (Estimated): February 6, 2027
• Study Completion (Estimated): February 6, 2027
• Last Updated: July 10, 2026
• Results First Posted: February 10, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: SAP, ICF

Locations

US (1)