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.
Trial summary
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). Read more about this study
- 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
- Are there results?
- Not yet: the study is under way
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.
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
About this study
Official title: 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).
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.
- 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 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.
- 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.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- 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)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Zahra Hudda, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
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
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Topical Ruxolitinib in Acute Skin GVHD: A Pilot Study
- ClinicalTrials.gov ID
- NCT07801248
- Acronym / sponsor ID
- 2025-0873
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 1 Oct 2026 (estimated)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Oct 2029 (estimated)
- First posted
- 3 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single arm, open-label pilot study. No randomization
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib | Experimental | Drug: Ruxolitinib 1.5% creamRuxolitinib 1.5% cream will be applied topically twice a day to areas of erythroderma and pruritus. |
Who may qualify
- Ages
- 2 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- Active uncontrolled skin infections.
- History of allergic reactions to topical ruxolitinib.
- Concomitant steroids or have received investigational therapies within the previous 30 days.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ruxolitinib 1.5% creamRuxolitinib 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. | Drug | Ruxolitinib |
Primary outcome
Number of patients with a clinical response at Day 7 (± 3 days)
Time frame: From enrollment to 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).
Number of patients with a clinical response at Day 28 (± 3 days)
Time frame: From enrollment to Day 28 (± 3 days)
Participants will be evaluated by monitoring the clinical response, tolerability and safety of topical ruxolitinib at Day 28 (± 3 days).
3 secondary outcomes
Number of patients needing systemic immune suppression due to lack of response from progressive symptoms or adverse events secondary to topical ruxolitinib
Time frame: From enrollment to Day 28 (± 3 days)
Number of patients with a relapse of primary malignancy by 1 year post HSCT
Time frame: 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.
Time frame: Day 7 (± 3 days)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Zahra Hudda, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Monica Trapp, BSN, RN, TCTCN (513-803-8574, monica.trapp@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with a clinical response at Day 7 (± 3 days) (time frame: From enrollment to Day 7 (± 3 days)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2026.