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Ruxolitinib

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.

Table of contents

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]

Trial ID Phase Condition studied Status Enrollment
NCT06959225Phase 3Hidradenitis SuppurativaAuthorised550
NCT06958211Phase 3Hidradenitis SuppurativaAuthorised550
2024-518156-24-00Phase 3Atopic DermatitisAuthorised218
NCT06804811Phase 3VitiligoAuthorised250
2022-501621-20-00Phase 3Prurigo NodularisCompleted180
2022-502461-23-00Phase 3Prurigo NodularisCompleted207
2022-502827-23-00Phase 2Chronic Hand EczemaCompleted180
2023-503737-22-00Phase 2Nonsegmental vitiligo with genital involvementCompleted45
2023-504724-25-00Phase 3MyelofibrosisAuthorised183
2023-507754-33-00Phase 2Steroid-refractory chronic GvHDAuthorised40
2022-502168-19-00Phase 2Newly diagnosed chronic GvHDAuthorised120
NCT04116502Phase 3Polycythaemia VeraAuthorised586
NCT02577926Phase 2High-risk polycythemia vera or essential thrombocythemiaAuthorised223
NCT04562389Phase 3Treatment-naïve myelofibrosisAuthorised325
2024-516105-23-01Phase 2Haemophagocytic lymphohistiocytosis in childrenAuthorised20

FAQ

  • What kinds of conditions are being studied with Ruxolitinib? The trials include skin conditions such as hidradenitis suppurativa, atopic dermatitis, vitiligo, prurigo nodularis, and chronic hand eczema. Other studies look at blood and immune diseases such as myelofibrosis, polycythemia vera, graft-versus-host disease, hemophagocytic lymphohistiocytosis, and inclusion body myositis.
  • Who can take part in Ruxolitinib trials? It depends on the study. Some trials are for children, some for adolescents and adults, and others are for people with specific disease types or previous treatment history, such as treatment-naïve patients, steroid-refractory patients, or people who did not respond well to earlier therapies.
  • What phases are these Ruxolitinib trials in? The trials range from Phase 1 to Phase 4. Phase 1 studies focus more on safety and dose questions, while Phase 2 to Phase 4 studies look more at response, safety, and longer-term results.
  • What are the main outcomes being measured? Common outcomes include response rates, symptom improvement, spleen volume reduction, survival, and safety measures. Some studies measure skin scores, while others track blood counts, organ failure scores, or time until relapse after transplant.
  • Are these studies only about Ruxolitinib alone? No. Some trials test Ruxolitinib alone, while others study it in combination with other treatments or compare it with vehicle cream or best available therapy. This helps researchers see whether Ruxolitinib adds benefit in a specific setting.

Ongoing Clinical Trials on Ruxolitinib

  • Study of Pacritinib for Patients with Severe Thrombocytopenia in Myelofibrosis Conditions

    Not recruiting

    1 1 1 1
    Investigated drugs:
    Bulgaria Czechia France Hungary Italy Poland +2

Glossary

  • Adverse event (AE): Any unwanted medical problem that happens during a study, whether or not it is caused by the study treatment.
  • Best available therapy (BAT): The treatment thought to be the best standard option for a patient group at that time.
  • Complete response (CR): A very strong treatment result where signs of disease are no longer seen based on the study rules.
  • Chronic graft-versus-host disease (cGVHD): A long-lasting condition that can happen after stem cell transplant, when donor cells attack the patient’s body.
  • EASI75: A skin improvement measure showing at least 75% improvement in the Eczema Area and Severity Index.
  • Event-free survival (EFS): The length of time a patient stays free from a major study event such as relapse, death, or another defined failure event.
  • HiSCR75: A hidradenitis suppurativa response measure showing at least 75% improvement in abscess and nodule counts without worsening of draining tunnels.
  • MRI / CT scan: Imaging tests used to measure organs like the spleen and see how a disease is changing over time.
  • Myelofibrosis (MF): A bone marrow disease that can cause symptoms such as an enlarged spleen and fatigue.
  • Overall response rate (ORR): The percentage of patients who reach a defined level of improvement, such as complete or partial response.
  • Vehicle cream: A cream with the same base as the study cream but without the active study drug.
  • White Itch Numeric Rating Scale (WI-NRS): A patient-reported scale used to measure itch severity and improvement.

References