Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD)
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ClinicalTrials.gov Identifier: NCT03395340 |
Recruitment Status :
Terminated
(Forced to close study due to poor recruitment (company withdrew drug support).)
First Posted : January 10, 2018
Results First Posted : July 12, 2022
Last Update Posted : July 12, 2022
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Background:
About half the people who have a hematopoietic stem cell transplant using donor cells get Chronic Graft Versus Host Disease (cGVHD). This is chronic graft versus host disease. Immune cells from the donor may see the body tissues in the person as foreign and attack, causing damage. The skin is the most commonly affected organ. Most cGVHD therapies have serious side effects. The cream ruxolitinib inhibits proteins that may play a role in cGVHD.
Objective:
To test the safety and effectiveness of topical ruxolitinib 1.5 percent cream in people with cGVHD of the skin.
Eligibility:
People ages 12 and older with epidermal skin cGVHD
Design:
Participants will be screened with:
Medical history
Physical exam
Blood and urine tests
Skin sample taken (biopsy) to confirm the diagnosis.
At the baseline visit, participants will have:
Skin disease measured with rulers, photographs, and tracing the outline of skin lesions
To complete questionnaires about their symptoms
Blood and urine tests
Some participants will also have a skin biopsy, or total body photographs while they wear only underwear.
Participants will get the ruxolitinib cream and a placebo cream to apply to 2 separate areas of disease. They will do this twice a day for 6 weeks, if they do not have serious side effects. Neither the study team nor the participant will know which area will get ruxolitinib cream and the placebo cream.
Participants will write down:
- When they apply the creams
- Any side effects
- Any medications they take
Most participants will have 4 visits during the 6 weeks they use the creams. Some will have 3 visits and a phone call to see how they are doing. All participants will get a call 4-6 weeks after they stop. Visits include physical exams, blood tests, skin disease measurements, questionnaires, and photos.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Graft Versus Host Disease JNS Kinase Topical Administration | Drug: Ruxolitinib 1.5% cream Drug: vehicle cream | Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 1 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | Phase II Study of Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD) |
Actual Study Start Date : | November 19, 2018 |
Actual Primary Completion Date : | January 31, 2019 |
Actual Study Completion Date : | January 31, 2019 |

Arm | Intervention/treatment |
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Experimental: Ruxolitinib cream
Investigational cream to 1 location; vehicle cream to 2nd location
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Drug: Ruxolitinib 1.5% cream
Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.
Other Name: Opzelura |
Placebo Comparator: Vehicle cream
Investigational cream to 1 location; vehicle cream to 2nd location
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Drug: vehicle cream
Matching vehicle cream applied as a thin film |
- Percent Change in the Surface Area Measurement of the Target Lesions for Ruxolitinib Treated Versus Placebo Treated Lesions [ Time Frame: Baseline to week 6 ]The surface area will be measured by tracing the lesion on transparency paper and measuring the area from the transparency. Differences in remaining active surface area at 6 weeks from baseline between the treated and placebo sites were compared to calculate efficacy. A decrease in active surface area would signify improvement in skin disease.
- Number of Participants With Grade 3 and Grade 4 Adverse Events [ Time Frame: date on study, up to approximately 2 months and 12 days. ]Adverse events were measured by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade 3 is severe. Grade 4 is life-threatening.
- Change in Overall Severity Visual Analog Scale (VAS) [ Time Frame: Baseline and week 6 ]The Overall Severity VAS is a psychometric response questionnaire to measure subjective characteristics or attitudes that cannot be directly measured on a scale of 0-10; a participants assessment of degree of disease activity on the skin. 0=none and 10 = worst imaginable.
- Change in Pain Visual Analog Scale (VAS) [ Time Frame: Baseline and week 6 ]The Pain VAS is a psychometric response questionnaire to measure subjective characteristics or attitudes that cannot be directly measured on a scale of 0 (no pain) -10 (worst imaginable pain). The participant draws a line on the scale representing how they feel between 0 (none) and 10 (worst). That line is measured and assigned a value. Scores from Baseline and week 6 will be reported. A change is considered a higher number (worsening of disease) or a lower number (improvement of disease) from baseline.
- Change in Pruritus Visual Analog Scale (VAS) [ Time Frame: Baseline and week 6 ]The pruritus VAS is a psychometric response questionnaire to measure subjective characteristics or attitudes that cannot be directly measured on a scale of 0 (no itching) -10 (worst imaginable itching). Scores from Baseline and week 6 will be reported. A change is considered a higher number (worsening of disease) or a lower number (improvement of disease) from baseline.
- Area Under the Serum Concentration Versus Time Curve (AUC) of Ruxolitinib [ Time Frame: A pre-dose trough sample will be collected prior to application of the treatment on the day of the follow up visit. After application in clinic, blood samples will be collected at 1 hour, 2 hour and 4 hours. ]The AUC is a measure of the serum concentration of the drug over time. It is used to characterize drug absorption.
- Total Clearance (CL) of Ruxolitinib [ Time Frame: A pre-dose trough sample will be collected prior to application of the treatment on the day of the follow up visit. After application in clinic, blood samples will be collected at 1 hour, 2 hour and 4 hours. ]The CL is a quantitative measure of the rate at which a drug substance is removed from the body.
- Half-Life of Ruxolitinib [ Time Frame: A pre-dose trough sample will be collected prior to application of the treatment on the day of the follow up visit. After application in clinic, blood samples will be collected at 1 hour, 2 hour and 4 hours. ]Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half.
- Number of Participants With Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) [ Time Frame: Date treatment consent signed to date off study, approximately 2 months and 12 days. ]Here is the number of participants with non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Years to 100 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
- INCLUSION CRITERIA:
- Patients must have histologically confirmed epidermal Chronic Graft Versus Host Disease (cGVHD) including lichen planus-like, papulosquamous, and erythematous cGVHD with clinical involvement at 2 separate body regions (e.g. right forearm and left forearm).
- Patients must have measurable disease, defined as at least 2 areas of cutaneous, nonulcerated, epidermal cGVHD involvement. Each site must involve at least 0.5% body surface area (1 palm equivalent) and cannot be a site of current or previous nonmelanoma skin cancer (NMSC).
- Stable immunosuppressant or immunomodulatory systemic cGVHD treatment, including phototherapy and extracorporeal photopheresis, for 4 weeks prior to enrollment.
- Age greater than or equal to 12 years. There is no available safety or adverse events data available for children younger than 12 years of age.
- Karnofsky or Lansky greater than or equal to 60
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Patients must have normal organ and marrow function as defined below:
- absolute neutrophil count greater than or equal to 1,000/mcL
- platelets greater than or equal to 50,000/mcL
- hemoglobin > 9 g/dL
- total bilirubin <1.5X institutional upper limit of normal except if known history of Gilbert's disease
- Aspartate aminotransferase (AST) serum glutamic-oxaloacetic transaminase (SGOT)/alanine aminotransferase (ALT) serum glutamic-pyruvic transaminase (SGPT) less than or equal to 5X institutional upper limit of normal
- creatinine clearance greater than or equal to 50 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal.
- Willingness to comply with twice daily application of 2 different creams to 2 separate, prespecified sites.
- The effects of ruxolitinib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
- Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
EXCLUSION CRITERIA:
- Patients concurrently receiving a Janus kinase (JAK) inhibitor (topical or systemic).
- Patients receiving any other investigational agents.
- Patients concurrently taking oral fluconazole.
- Patients concurrently taking strong Cytochrome P450 3A4 (CYP3A4) inhibitors.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to ruxolitinib or other agents used in study.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection including Epstein-Barr virus (EBV), Cytomegalovirus (CMV), human immunodeficiency virus (HIV), hepatitis B virus (HBV), and hepatitis C virus (HCV), symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study because ruxolitinib is an agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ruxolitinib, breastfeeding should be discontinued if the mother is treated with ruxolitinib. These potential risks may also apply to other agents used in this study.
- Recurrent or progressive malignancy requiring anticancer treatment.
- Other cancer (except that for which hematopoietic cell transplantation (HCT) was performed) within 2 years of study entry, except nonmelanoma skin cancer or carcinoma in situ of the breast, uterus, or cervix.
- History of cutaneous malignancy at target lesion site.
- Any participant who, in the investigator's opinion, would be unable to comply with study requirements or for whom participation may pose a greater medical risk.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03395340
United States, Maryland | |
National Institutes of Health Clinical Center | |
Bethesda, Maryland, United States, 20892 |
Principal Investigator: | Edward W Cowen, M.D. | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
Documents provided by Edward Cowen, M.D., National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS):
Publications:
Responsible Party: | Edward Cowen, M.D., Principal Investigator, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
ClinicalTrials.gov Identifier: | NCT03395340 |
Other Study ID Numbers: |
180035 18-AR-0035 |
First Posted: | January 10, 2018 Key Record Dates |
Results First Posted: | July 12, 2022 |
Last Update Posted: | July 12, 2022 |
Last Verified: | June 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
cGVHD Epidermal JAK Inhibitor Skin JAK-STAT |
Graft vs Host Disease Immune System Diseases |