Table of contents

• Trial overview
• Skin condition studies
• Blood and immune system studies
• Children and young people
• Study design and comparators
• Outcomes measured in the trials
• Who may join these studies

Trial overview

These studies investigate Ruxolitinib in many different diseases, with a strong focus on skin disorders and blood-related conditions.^[1] The trial list includes both completed and authorised studies, and the phases range from Phase 1 to Phase 4.^[1]

Some trials test Ruxolitinib alone, while others study it with another treatment or against a placebo or vehicle cream.^[1] The main goal across the trials is to see whether the treatment is safe and whether it helps patients improve in a measurable way.^[1]

Skin condition studies

Several trials focus on skin diseases and use Ruxolitinib cream.^[1] 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.^[1]

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.^[1] 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.^[1]

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.^[1] Another Phase 2 study looks at genital vitiligo and measures a genital visibility score at Week 48, asking whether the area becomes less noticeable.^[1]

Two Phase 3 studies in prurigo nodularis tested whether Ruxolitinib cream could reduce itching, using the WI-NRS4 response at Week 12.^[1] 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.^[1]

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.^[1] 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.^[1]

Common myelofibrosis outcomes include spleen volume reduction, symptom score improvement, and safety measures.^[1] For example, several trials measure whether the spleen shrinks by at least 35% after 24 weeks, which is called SVR35.^[1] Other studies measure total symptom score, using questionnaires such as MFSAF v4.0a.^[1]

Trials in polycythemia vera and essential thrombocythemia compare Ruxolitinib with other standard treatments or best available therapy.^[1] 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.^[1]

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.^[1] These studies measure overall response, usually defined as complete or partial response without needing new systemic therapy.^[1]

Other immune-related studies include haemophagocytic lymphohistiocytosis in children and acquired hemophagocytic syndrome in adults in intensive care units.^[1] These trials focus on survival, organ failure scores, or survival until stem cell transplant.^[1]

Children and young people

Some trials are built specifically for younger patients.^[1] 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.^[1]

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.^[1] 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.^[1]

Some pediatric studies also include patients up to age 25 years, especially in transplant and leukemia-related research.^[1] This shows that Ruxolitinib research is not limited to adults and is being explored across a wide age range.^[1]

Study design and comparators

The trial designs include interventional studies, which means the researchers assign a treatment and then watch what happens.^[1] 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.^[1]

Many skin trials compare Ruxolitinib cream with vehicle cream, which is the same cream base without the active study drug.^[1] In blood and immune studies, Ruxolitinib is often compared with corticosteroids, best available therapy, or other active treatments, depending on the condition being studied.^[1]

Some studies are combination trials, where Ruxolitinib is used together with another medicine to see whether the pair works better than one treatment alone.^[1] Other studies are rollover or extension trials that allow patients to keep receiving treatment while researchers continue to collect safety data over time.^[1]

Outcomes measured in the trials

The main outcomes differ by disease, but they all aim to measure a clear treatment result.^[1] 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.^[1]

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.^[1] 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.^[1]

Safety is also a major endpoint in many trials.^[1] Safety outcomes may include adverse events, serious adverse events, vital signs, blood tests, ECG results, and physical examination findings.^[1]

Who may join these studies

Eligibility depends on the disease being studied and the exact trial rules.^[1] 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.^[1]

Age limits also vary, with some trials for children only, some for adults only, and some for both children and young adults.^[1] Because the studies are focused on specific diseases and treatment histories, not every patient with the same diagnosis can join every trial.^[1]