Topical Ruxolitinib in Acute Skin GVHD (Graft Versus Host Disease)

Efficacy of Topical Ruxolitinib in Acute Skin GVHD: A Pilot Study

The purpose of this study is to see if ruxolitinib, a Janus Kinase (JAK) 1/2 inhibitor, is safe for people who have acute skin graft versus host disease (GVHD) following allogenic hematopoietic stem cell transplantation (HSCT).

Study Overview

• Status: Not yet recruiting
• Conditions: Acute Skin Graft Versus Host Disease (GVHD)
• Intervention / Treatment: Drug: Ruxolitinib 1.5% cream

Detailed Description

The purpose of this study is to see if ruxolitinib, a Janus Kinase (JAK) 1/2 inhibitor, is safe for people who have acute skin graft versus host disease (GVHD) following allogenic hematopoietic stem cell transplantation (HSCT). This is a single arm, open-label pilot study. There is no randomization. This study will enroll approximately 10 patients with underlying malignancies with acute skin GVHD. Participants will be treated with topical ruxolitinib for 28 days or until the end of treatment if earlier resolution of GVHD is achieved.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Monica Trapp, BSN, RN, TCTCN; Phone Number: 513-803-8574; Email: monica.trapp@cchmc.org

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
      • Contact: Monica Trapp, BSN, RN, TCTCN; Phone Number: 513-803-8574; Email: monica.trapp@cchmc.org
      • Principal Investigator: Zahra Hudda, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age ≥2 through age 35 years
• Acute skin GVHD (any grade).
• Underlying diagnosis of malignancy.
• No concurrent systemic immune suppression to treat acute GVHD. This excludes agents like calcineurin inhibitors, mycophenolate mofetil, and abatacept which prevent acute GVHD and are not used for treatment as per clinical practice may continue.
• Use of prior topical steroids or topical tacrolimus will be allowed but these will need to be discontinued 24 hours prior to study enrollment.

Exclusion Criteria:

• Active uncontrolled skin infections.
• History of allergic reactions to topical ruxolitinib.
• Concomitant steroids or have received investigational therapies within the previous 30 days.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Ruxolitinib; Ruxolitinib 1.5% cream will be applied topically twice a day to areas of erythroderma and pruritus.

Drug: Ruxolitinib 1.5% cream; Ruxolitinib is a Janus Kinase (JAK) 1/2 inhibitor. JAKs are intracellular tyrosine kinases that play an important role in the development and function of immune cells that are involved in the pathogenesis of acute GVHD. For this study, ruxolitinib cream will be manufactured by Incyte and stored and distributed by the investigational pharmacy at CCHMC using standard operating procedures. Ruxolitinib 1.5% cream will be applied as a thin film topically twice a day to areas of erythroderma and pruritus. Participants and guardians will receive an informational instruction sheet describing the amount, frequency, and technique of applying the cream. Patients may continue to use standard of care GVHD prophylaxis and moisturizers.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with a clinical response at Day 7 (± 3 days)	Participants will be evaluated by monitoring the clinical response, tolerability and safety of topical ruxolitinib at Day 7 (± 3 days). Patients who respond by Day 7 (± 3 days) will continue treatment until resolution of symptoms or till Day 28 (± 3 days).	From enrollment to Day 7 (± 3 days)
Number of patients with a clinical response at Day 28 (± 3 days)	Participants will be evaluated by monitoring the clinical response, tolerability and safety of topical ruxolitinib at Day 28 (± 3 days).	From enrollment to Day 28 (± 3 days)

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of patients needing systemic immune suppression due to lack of response from progressive symptoms or adverse events secondary to topical ruxolitinib	From enrollment to Day 28 (± 3 days)
Number of patients with a relapse of primary malignancy by 1 year post HSCT	From enrollment to 1 year post HSCT
Blood ruxolitinib concentration levels at documented body surface area (BSA) applications and their association with adverse effects and relapse rates.	Day 7 (± 3 days)

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Zahra Hudda, MD, Children's Hospital Medical Center, Cincinnati

Study record dates

Study Major Dates

• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2029

Study Registration Dates

• First Submitted: August 28, 2026
• First Submitted That Met QC Criteria: August 28, 2026
• First Posted (Actual): September 3, 2026

Study Record Updates

• Last Update Posted (Actual): September 3, 2026
• Last Update Submitted That Met QC Criteria: August 28, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: ruxolitinib
• Other Study ID Numbers: 2025-0873

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No