Acute Skin Graft Versus Host Disease (GVHD)Phase 1NCT07801248

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

A Phase 1 study testing Ruxolitinib 1.5% cream in people with Acute Skin Graft Versus Host Disease (GVHD), run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts

Official record

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Trial summary

Who is it for?: Ages 2 to 35 years
Conditions the study is for: Acute Skin Graft Versus Host Disease (GVHD)
What is being tested?: Ruxolitinib 1.5% cream
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 1 siteUnited States
Study dates: 1 Oct 2026to Oct 2029
Phase: Phase 1

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 to 35 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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.

Who cannot join

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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Monica Trapp, BSN, RN, TCTCN contact

513-803-8574

monica.trapp@cchmc.org

United States1 site

Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Monica Trapp, BSN, RN, TCTCN, 513-803-8574, monica.trapp@cchmc.org
Zahra Hudda, MD

Other trials in United States

About this study

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

Read the 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.

Conditions: Acute Skin Graft Versus Host Disease (GVHD)
Being tested: Ruxolitinib 1.5% cream

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Ruxolitinib 1.5% creamDrug
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 Cincinnati Children's Hospital Medical Center (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.

Study groups in NCT07801248
Study group	Role	What they receive
Ruxolitinib	Experimental	Ruxolitinib 1.5% cream will be applied topically twice a day to areas of erythroderma and pruritus. Receives: Drug: Ruxolitinib 1.5% cream

What is being measured

The primary outcome is the main question the study is designed to answer.

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).
Measured: 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).
Measured: From enrollment to Day 28 (± 3 days)

Secondary outcomes (3)

Number of patients needing systemic immune suppression due to lack of response from progressive symptoms or adverse events secondary to topical ruxolitinib
Measured: From enrollment to Day 28 (± 3 days)
Number of patients with a relapse of primary malignancy by 1 year post HSCT
Measured: 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.
Measured: Day 7 (± 3 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Oct 2026Oct 2029

First posted3 Sep 2026
Study start1 Oct 2026 (estimated)
Primary completionOct 2029 (estimated)
Study completionOct 2029 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Acute Skin Graft Versus Host Disease (GVHD)Studies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Single arm, open-label pilot study. No randomization
Masking: None (open label)
Enrolment: 10 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07801248
Sponsor's protocol ID: 2025-0873
Status last verified: Sep 2026
First submitted: 28 Aug 2026
Last updated: 8 Sep 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No