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NCT03954236

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

Active, enrolled Phase 2 Results posted Last updated 10 February 2026
What this trial tests

Phase 2 trial testing topical ruxolitinib 1.5% cream in Non-sclerotic Cutaneous Chronic Graft-versus-host Disease in 24 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline
14 May 2019
Primary endpoint
6 February 2027
6 February 2027

Quick facts

Lead sponsorMemorial Sloan Kettering Cancer Center
PhasePhase 2
StatusActive, enrolled
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment24
Start date14 May 2019
Primary completion6 February 2027
Estimated completion6 February 2027
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Memorial Sloan Kettering Cancer Center — full company profile →

Who can join

12 and older, any sex, with Non-sclerotic Cutaneous Chronic Graft-versus-host Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

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

BSA on Day 1 for Topical Ruxolitinib BID to Left Side of Face/Body
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)14.4± 0.003
BSA on Day 28 for Topical Ruxolitinib BID to Left Side of Face/Body
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)6.2± 0.003
BSA on Day 1 for Topical Ruxolitinib BID to Right Side of Face/Body
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)14.5± 0.003
BSA on Day 28 for Topical Ruxolitinib BID to Right Side of Face/Body
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)10.4± 0.003
Improvement in Physician's Global Assessment of Clinical Condition (PGA) at From Day 1 and Day 28 Secondary · 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 eval

Topical Ruxolitinib BID to Left Side of Face/Body PGA on Day 1
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)5± 0.0004
Topical Ruxolitinib BID to Left Side of Face/Body PGA on Day 28
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)1.9± 0.0004
Topical Ruxolitinib BID to Right Side of Face/Body PGA on Day 1
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)5± 0.0004
Topical Ruxolitinib BID to Right Side of Face/Body PGA on Day 28
GroupValue95% CI
Experimental: Participants With Graft-versus-host Disease (GVHD)3.7± 0.0004

Adverse events — posted to ClinicalTrials.gov

Time frame: 28 days (+/-3 days). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Experimental: Participants With Graft-versus-host Disease (GVHD)
Serious: 2/24 (8%)
Deaths: 8/24
Experimental Left: Participants With Graft-versus-host Disease (GVHD)
Serious: 0/12 (0%)
Deaths: 0/12
Experimental Right: Participants With Graft-versus-host Disease (GVHD)
Serious: 0/12 (0%)
Deaths: 0/12

Serious adverse events (2 terms)

ReactionSystemExperimental: Participants…Experimental Left: Partici…Experimental Right: Partic…
SinusitisInfections and infestations
Neoplasm Benign, Malignant and Unspecified (incl cysts and polyps) - Other, specify - CNS LeukemiaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Other adverse events (13 terms — click to expand)

ReactionSystemExperimental: Participants…Experimental Left: Partici…Experimental Right: Partic…
Orofacial dermatitisSkin and subcutaneous tissue disorders
SinusitisInfections and infestations
Eye irritationEye disorders
Cracked/thick cuticlesSkin and subcutaneous tissue disorders
HeadacheNervous system disorders
Nummular eczemaSkin and subcutaneous tissue disorders
Eyelash hypotrichosisEye disorders
Skin hyperpigmentationSkin and subcutaneous tissue disorders
Seborrheic dermatitisSkin and subcutaneous tissue disorders
FeverGeneral disorders
Flu like symptomsGeneral disorders
Lung infectionInfections and infestations
Rash acneiformSkin and subcutaneous tissue disorders

Most-reported serious reactions: Sinusitis, Neoplasm Benign, Malignant and Unspecified (incl cysts and polyps) - Other, specify - CNS Leukemia.

Data from ClinicalTrials.gov NCT03954236 adverse events section.

Sponsor's own description

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.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

  1. JAK-STAT signaling in inflammation and stress-related diseases: implications for therapeutic interventions.
    Sarapultsev A, Gusev E, Komelkova M, Utepova I, et al · · 2023 · cited 131× · PMID 37938494 · DOI 10.1186/s43556-023-00151-1
  2. Selectivity, efficacy and safety of JAKinibs: new evidence for a still evolving story.
    Bonelli M, Kerschbaumer A, Kastrati K, Ghoreschi K, et al · · 2024 · cited 95× · PMID 37923366 · DOI 10.1136/ard-2023-223850
  3. JAK-STAT signaling in human disease: From genetic syndromes to clinical inhibition.
    Luo Y, Alexander M, Gadina M, O'Shea JJ, et al · · 2021 · cited 92× · PMID 34625141 · DOI 10.1016/j.jaci.2021.08.004
  4. Protein kinases: drug targets for immunological disorders.
    Castelo-Soccio L, Kim H, Gadina M, Schwartzberg PL, et al · · 2023 · cited 67× · PMID 37188939 · DOI 10.1038/s41577-023-00877-7
  5. JAK inhibitors and black box warnings: what is the future for JAK inhibitors?
    Tokareva K, Reid P, Yang V, Liew D, et al · · 2023 · cited 20× · PMID 37596779 · DOI 10.1080/1744666x.2023.2249237
  6. Established and Emerging Treatments of Skin GvHD.
    Link-Rachner CS, Sockel K, Schuetz C. · · 2022 · cited 10× · PMID 35185931 · DOI 10.3389/fimmu.2022.838494
  7. Chronic graft-versus-host disease. Part II: Disease activity grading and therapeutic management.
    Baumrin E, Loren AW, Falk SJ, Mays JW, et al · · 2024 · cited 8× · PMID 36572064 · DOI 10.1016/j.jaad.2022.12.023

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