In short
Clinical trials are investigating Ruxolitinib in many different patient groups, including people with skin, blood, immune, and cancer-related conditions. These studies look at safety, effectiveness, and response measures in children and adults across several trial phases.
Key points
- Clinical trials of Ruxolitinib are being done in both skin diseases and serious blood or immune conditions. In skin studies, researchers are testing Ruxolitinib cream in hidradenitis suppurativa, atopic dermatitis, vitiligo, prurigo nodularis, and chronic hand eczema. In blood and immune studies, trials are looking at myelofibrosis, polycythemia vera, graft-versus-host disease, hemophagocytic lymphohistiocytosis, and some cancers. The studies include Phase 1, Phase 2, Phase 3, and Phase 4 designs, so they range from early safety work to larger efficacy and long-term safety studies. Many trials compare Ruxolitinib with placebo, vehicle cream, or other active treatments, and they measure outcomes such as response rates, symptom improvement, spleen size, survival, and safety.
Trial overview
These studies investigate Ruxolitinib in many different diseases, with a strong focus on skin disorders and blood-related conditions. The trial list includes both completed and authorised studies, and the phases range from Phase 1 to Phase 4.
Some trials test Ruxolitinib alone, while others study it with another treatment or against a placebo or vehicle cream. The main goal across the trials is to see whether the treatment is safe and whether it helps patients improve in a measurable way.
Skin condition studies
Several trials focus on skin diseases and use Ruxolitinib cream. In hidradenitis suppurativa, two Phase 3 trials called TRuE-HS1 and TRuE-HS2 are studying whether Ruxolitinib cream can improve disease signs by Week 16, using the HiSCR75 response measure.
In atopic dermatitis, a Phase 3b study in children and adolescents aged 6 to under 18 years is testing Ruxolitinib cream against vehicle cream, with the main outcome being EASI75 at Week 8. A similar Phase 3b study in adults with moderate atopic dermatitis also compares Ruxolitinib cream with vehicle cream and measures EASI75 and IGA-TS at Week 8.
Ruxolitinib cream is also being studied in children aged 6 to under 12 years with non-segmental vitiligo, where the main outcome is F-VASI75 at Week 24. Another Phase 2 study looks at genital vitiligo and measures a genital visibility score at Week 48, asking whether the area becomes less noticeable.
Two Phase 3 studies in prurigo nodularis tested whether Ruxolitinib cream could reduce itching, using the WI-NRS4 response at Week 12. A Phase 2 study in chronic hand eczema also compared Ruxolitinib cream with vehicle cream and measured the IGA-CHE-TS score at Week 16.
Blood and immune system studies
Ruxolitinib is also being studied in myelofibrosis, a bone marrow disease that can cause an enlarged spleen and symptoms such as tiredness. These studies examine Ruxolitinib alone or in combination with other drugs such as selinexor, navtemadlin, axatilimab, navitoclax, KER-050, or roginolisib, depending on the trial.
Common myelofibrosis outcomes include spleen volume reduction, symptom score improvement, and safety measures. For example, several trials measure whether the spleen shrinks by at least 35% after 24 weeks, which is called SVR35. Other studies measure total symptom score, using questionnaires such as MFSAF v4.0a.
Trials in polycythemia vera and essential thrombocythemia compare Ruxolitinib with other standard treatments or best available therapy. One Phase 3 study in high-risk polycythemia vera measures event-free survival, while a Phase 2 study in high-risk polycythemia vera or essential thrombocythemia measures complete clinicohematologic response at month 6.
Ruxolitinib is also being tested in graft-versus-host disease, including newly diagnosed chronic graft-versus-host disease, steroid-refractory chronic graft-versus-host disease, and steroid-refractory acute graft-versus-host disease. These studies measure overall response, usually defined as complete or partial response without needing new systemic therapy.
Other immune-related studies include haemophagocytic lymphohistiocytosis in children and acquired hemophagocytic syndrome in adults in intensive care units. These trials focus on survival, organ failure scores, or survival until stem cell transplant.
Children and young people
Some trials are built specifically for younger patients. These include studies in children with hidradenitis suppurativa, atopic dermatitis, vitiligo, haemophagocytic lymphohistiocytosis, acute lymphoblastic leukemia, and relapsed or refractory leukemia or lymphoma with IL-7R/JAK-STAT pathway changes.
In these studies, the trial teams use child-appropriate outcomes such as skin scores, survival until transplant, minimal residual disease, or best overall response rate. Minimal residual disease means a very small number of cancer cells that may still be present after treatment and can only be found with special tests.
Some pediatric studies also include patients up to age 25 years, especially in transplant and leukemia-related research. This shows that Ruxolitinib research is not limited to adults and is being explored across a wide age range.
Study design and comparators
The trial designs include interventional studies, which means the researchers assign a treatment and then watch what happens. Several studies are randomized and double-blind, meaning patients are assigned by chance and neither the patient nor the study team knows which treatment is being given during the blinded part.
Many skin trials compare Ruxolitinib cream with vehicle cream, which is the same cream base without the active study drug. In blood and immune studies, Ruxolitinib is often compared with corticosteroids, best available therapy, or other active treatments, depending on the condition being studied.
Some studies are combination trials, where Ruxolitinib is used together with another medicine to see whether the pair works better than one treatment alone. Other studies are rollover or extension trials that allow patients to keep receiving treatment while researchers continue to collect safety data over time.
Outcomes measured in the trials
The main outcomes differ by disease, but they all aim to measure a clear treatment result. In skin studies, the outcomes often include improvement in rash, itch, visible lesions, or disease severity scores such as EASI, IGA, WI-NRS, HiSCR75, F-VASI75, and IGA-CHE-TS.
In blood cancer and marrow disease studies, the outcomes often include spleen volume reduction, symptom score changes, complete response, overall response rate, or event-free survival. In transplant and immune studies, outcomes may include survival until HSCT, organ failure scores such as SOFA, relapse rate, graft failure, or time to relapse after transplant.
Safety is also a major endpoint in many trials. Safety outcomes may include adverse events, serious adverse events, vital signs, blood tests, ECG results, and physical examination findings.
Who may join these studies
Eligibility depends on the disease being studied and the exact trial rules. Some trials enroll people who have not yet received treatment, while others look for patients who did not respond to earlier therapy, cannot use standard treatment, or have steroid-refractory disease.
Age limits also vary, with some trials for children only, some for adults only, and some for both children and young adults. Because the studies are focused on specific diseases and treatment histories, not every patient with the same diagnosis can join every trial.