10 Participants Needed

Ruxolitinib for Eosinophilia

JA
DD
TN
Overseen ByTiffany Nguyen
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

This phase II trial studies how well ruxolitinib works in treating patients with hypereosinophilic syndrome or primary eosinophilic disorders.

Will I have to stop taking my current medications?

The trial requires that you stop using certain medications, such as hydroxyurea within 7 days of starting the study and other specific therapies for eosinophilic disorders within 28 days. If you are on corticosteroids, you must be on a stable dose for at least 28 days before starting the trial.

What data supports the effectiveness of the drug Ruxolitinib for treating eosinophilia?

Ruxolitinib has shown effectiveness in treating conditions with high eosinophil counts, like lymphocytic-variant hypereosinophilic syndrome, where it led to complete clinical remission and normalized eosinophil counts in patients. Additionally, it is effective in reducing symptoms and improving quality of life in myelofibrosis, a different condition, suggesting its potential in managing eosinophilia.12345

Is ruxolitinib generally safe for humans?

Ruxolitinib has been used for over a decade to treat myelofibrosis, and while it is generally well tolerated, some patients may experience side effects like anemia (low red blood cell count) and thrombocytopenia (low platelet count), which are usually manageable. Rarely, it can cause skin reactions or increase the risk of infections, so monitoring by a healthcare provider is important.36789

How is the drug Ruxolitinib unique for treating eosinophilia?

Ruxolitinib is unique because it is a Janus kinase (JAK) inhibitor, which means it works by blocking specific enzymes involved in the immune response, potentially offering a novel approach for conditions like eosinophilia where standard treatments may not exist.2471011

Research Team

WE

William Shomali, MD

Principal Investigator

Stanford Cancer Institute Palo Alto

Eligibility Criteria

This trial is for patients with hypereosinophilic syndrome or primary eosinophilic disorders, including those newly-diagnosed, on corticosteroids, or with relapsed/refractory disease. Participants must have symptoms of organ damage or enlargement and an absolute eosinophil count >= 1,500/mm^3 (or >= 500/mm^3 in certain conditions). They should be able to consent and have a performance status <= 3. Exclusions include life-threatening complications from the disease, recent serious infections, low platelet counts, prior JAK inhibitor therapy like ruxolitinib.

Inclusion Criteria

- Has as at least 2 readings with an absolute eosinophil count >= 1,500/mm^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).
- Willing and able to review and execute informed consent (legally-authorized consent acceptable).
My cancer has specific genetic changes involving the JAK2 gene.
See 16 more

Exclusion Criteria

I am currently on antibiotics for a serious infection.
- Alanine aminotransferase (ALT)/serum glutamate pyruvate transaminase (SGPT) > 4 x upper limit of normal (ULN) or direct bilirubin > 4 x ULN (if considered to be unrelated to the underlying eosinophilic disorder).
I have not taken hydroxyurea in the last 7 days.
See 19 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive ruxolitinib PO BID on days 1-28. Treatment repeats for up to 6 cycles (28 days each) in the absence of disease progression or unacceptable toxicity.

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

Treatment Details

Interventions

  • Ruxolitinib
Trial OverviewThe trial is testing Ruxolitinib's effectiveness for treating hypereosinophilic syndrome or primary eosinophilic disorders. It's a phase II study which means they're looking at how well it works and its safety in people who fit the criteria.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Treatment (ruxolitinib)Experimental Treatment1 Intervention
Patients receive ruxolitinib PO BID on days 1-28. Treatment repeats for up to 6 cycles (28 days each) in the absence of disease progression or unacceptable toxicity.

Ruxolitinib is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Jakafi for:
  • Intermediate or high-risk myelofibrosis
  • Polycythemia vera
  • Steroid-refractory acute graft-versus-host disease
  • Chronic graft-versus-host disease
  • Vitiligo
🇪🇺
Approved in European Union as Jakavi for:
  • Intermediate or high-risk myelofibrosis
  • Polycythemia vera
  • Steroid-refractory acute graft-versus-host disease
  • Chronic graft-versus-host disease
  • Non-segmental vitiligo

Find a Clinic Near You

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials
2,527
Recruited
17,430,000+

Jason Robert Gotlib

Lead Sponsor

Trials
5
Recruited
50+

William Shomali

Lead Sponsor

Trials
1
Recruited
10+

Incyte Corporation

Industry Sponsor

Trials
408
Recruited
66,800+
Steven Stein profile image

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot profile image

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Findings from Research

In a study of five patients with CD3- CD4+ lymphocytic-variant hypereosinophilic syndrome (L-HES), treatment with JAK inhibitors (ruxolitinib and tofacitinib) resulted in complete clinical remission within three months for all patients, allowing for prednisone withdrawal in four of them.
No adverse events were reported during 3-13 months of follow-up, suggesting that JAK inhibitors may be a safe and effective alternative for patients with L-HES who are unresponsive to conventional therapies.
JAK inhibition for CD3- CD4+ lymphocytic-variant hypereosinophilic syndrome.Faguer, S., Groh, M., Vergez, F., et al.[2023]
Ruxolitinib is strongly recommended for patients with myelofibrosis to improve severe splenomegaly and systemic symptoms, particularly in those with specific risk scores and symptoms like severe itching or unexplained weight loss.
However, there is weak evidence supporting the use of ruxolitinib for improving survival, and these recommendations do not apply to patients eligible for allogeneic stem cell transplant.
Which patients with myelofibrosis should receive ruxolitinib therapy? ELN-SIE evidence-based recommendations.Marchetti, M., Barosi, G., Cervantes, F., et al.[2021]
In a 3-year follow-up of the COMFORT-II Trial involving 219 patients with myelofibrosis, ruxolitinib demonstrated sustained reductions in spleen size and improved quality of life, with 45% of patients remaining on treatment after 3 years.
Ruxolitinib was well tolerated, with manageable side effects like anemia and thrombocytopenia, and it was associated with longer overall survival compared to the best available therapy, indicating its efficacy and safety as a treatment option.
Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis.Cervantes, F., Vannucchi, AM., Kiladjian, JJ., et al.[2022]

References

JAK inhibition for CD3- CD4+ lymphocytic-variant hypereosinophilic syndrome. [2023]
Which patients with myelofibrosis should receive ruxolitinib therapy? ELN-SIE evidence-based recommendations. [2021]
Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. [2022]
Mycobacterial Infections With Ruxolitinib: A Retrospective Pharmacovigilance Review. [2021]
Fedratinib: a pharmacotherapeutic option for JAK-inhibitor naïve and exposed patients with myelofibrosis. [2022]
Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety. [2023]
Erythematous skin lesions with necrotic centers on lower extremities due to the use of ruxolitinib for primary myelofibrosis. [2021]
Case-report: EBV driven lymphoproliferative disorder associated with Ruxolitinib. [2022]
Safety and efficacy of jaktinib (a novel JAK inhibitor) in patients with myelofibrosis who are intolerant to ruxolitinib: A single-arm, open-label, phase 2, multicenter study. [2023]
10.United Statespubmed.ncbi.nlm.nih.gov
Off-label Studies on the Use of Ruxolitinib in Dermatology. [2021]
Pneumocystis jiroveci pneumonitis complicating ruxolitinib therapy. [2022]