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BCR-JAK2 Fusion Protein ExpressionPhase 2NCT03801434

Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders

A Phase 2 study testing Ruxolitinib in people with BCR-JAK2 Fusion Protein Expression and Blasts 20 Percent or Less of Peripheral Blood White Cells, run by Stanford University at 4 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

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

Who is it for?
Ages 18 years and older
What is being tested?
Ruxolitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
4 sitesUnited States
Study dates
15 Nov 2019to Feb 2029
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subject with idiopathic hypereosinophilic syndrome must meet the following:
  • 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).
  • Dependent, intolerant or refractory to corticosteroids OR has relapsed/refractory disease to other therapy besides corticosteroids.
  • Symptomatic from his/her disease OR has one or more signs of organ damage (assessed by the investigator as possibly-related to eosinophilia or biopsy-proven). This can include skin, lung, cardiac, central nervous system, liver, or gastrointestinal (GI) involvement, or evidence of symptomatic hepatic or splenic enlargement.
  • Subject with lymphocyte-variant hypereosinophilia must meet the following
  • Has 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).
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  • Dependent, intolerant or refractory to corticosteroids* OR has relapsed/refractory disease to other therapy besides corticosteroids.
  • Symptomatic from his/her disease OR has one or more signs of organ damage (assessed by the investigator as possibly-related to eosinophilia or biopsy-proven). This can include skin, lung, cardiac, central nervous system, liver, or GI involvement, or evidence of symptomatic hepatic or splenic enlargement
  • Has abnormal T-lymphocyte immuno-phenotype by flow cytometry.
  • Subject with chronic eosinophilic leukemia, not otherwise specified (CEL,NOS) must meet the following
  • Has at least 2 readings with an absolute eosinophil count >= 500/mm^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).
  • Newly-diagnosed OR receiving corticosteroids OR has relapsed/refractory disease to any therapy besides corticosteroids.
  • Has increased blasts in the blood or bone marrow (> 5% and < 20%), and/or a clonal cytogenetic or molecular abnormality
  • Subjects with JAK2 mutations are included within this group.
  • Subject with JAK2-rearranged eosinophilic neoplasm must meet the following
  • Has at least 2 readings with an absolute eosinophil count >= 500/mm^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).
  • Newly-diagnosed OR receiving corticosteroids OR has relapsed/refractory disease to any therapy besides corticosteroids.
  • This group includes subjects with PCM1-JAK2, BCR-JAK2, ETV6-JAK2 or other JAK2 rearrangements.
  • If receiving corticosteroids, must be a stable dose for >= 28 days prior to Day 1 (unstable dosing not eligible).
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 3.
  • Willing and able to review and execute informed consent (legally-authorized consent acceptable).

Who cannot join

  • Active life-threatening complication(s) from underlying eosinophilic disease (i.e., leukostasis; acute thromboembolic disease including central nervous system (CNS) involvement; severe pulmonary or cardiac dysfunction). Stabilization of acute, life-threatening eosinophil-related co-morbidities will allow enrollment of the patient.
  • World Health Organization (WHO)-defined myeloid neoplasm associated with eosinophilia other than CEL NOS and JAK2 rearranged neoplasms (e.g., myelodysplastic syndrome (MDS); myeloproliferative neoplasms (MPN); MDS/MPN overlap disorders; and systemic mastocytosis (SM).
  • Reactive hypereosinophilia due to connective tissue disease, sarcoidosis or eosinophilic granulomatosis with polyangiitis.
  • Organ-restricted ?tissue? eosinophilia with the absence of peripheral eosinophilia in the blood.
  • Invasive malignancy over the previous 2 years except treated early stage carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, and completely resected papillary thyroid and follicular thyroid cancers.
  • Myeloid or lymphoid neoplasm with eosinophilia and abnormalities of PDGFRA, PDGFRB or FGFR1.
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  • Anticipated to receive a hematopoietic stem cell transplant within the first 6 months of treatment on trial.
  • Major surgery within 4 weeks prior to entering the study.
  • Life expectancy of < 6 months.
  • Known diagnosis of human immunodeficiency virus (HIV).
  • Known diagnosis of chronic active hepatitis B or C (viral testing is not required). Subjects with a known history of hepatitis B and/or C are allowed on trial if at the time of enrollment, the virus is not active and undetected (testing required if there is a known history), and such patients are not actively receiving antiviral treatment specific for hepatitis B and/or C.
  • Clinically serious infections requiring ongoing antibiotic therapy.
  • Parasitic infection diagnosed within 24 weeks prior to enrollment.
  • Platelet count =< 25 x 10^9/L at baseline.
  • 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).
  • End-stage renal function (creatinine clearance [CrCl] < 15 mL/min or glomerular filtration rate [GFR] < 15 mL/min) regardless of whether hemodialysis is required.
  • Use of investigational or commercial therapies with the intent to treat the underlying eosinophilic disorder within 28 days of study start, including interferon; imatinib; alemtuzumab; cyclosporine; methotrexate; mepolizumab; benralizumab; or other antibody therapies.
  • Use of hydroxyurea within 7 days of study start.
  • Prior therapy with ruxolitinib or other JAK inhibitors.
  • Previous allergic reactions to JAK inhibitors or excipients.
  • Unwilling to commit to abstinence from heterosexual contact or agree to use and comply with highly effective contraception, 28 days prior to starting study drug, during the treatment period and for 12 weeks after discontinuation of study treatment.
  • Females of childbearing potential who have a positive pregnancy test (urine or serum) during screening period.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Stanford Cancer Institute Palo Alto

    Palo Alto, California, 94304

  • OHSU Knight Cancer Institute

    Portland, Oregon, 97201

  • University of Utah

    Salt Lake City, Utah, 84112

  • Fred Hutchinson cancer research center

    Seattle, Washington, 98109

Other trials in United States

About this study

Official title: Phase 2 Study of Ruxolitinib in Idiopathic Hypereosinophilic Syndrome and Primary Eosinophilic Disorders

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

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the overall hematologic response rate to ruxolitinib in patients with hypereosinophilic syndrome and primary eosinophilic disorders.

SECONDARY OBJECTIVES:

  1. To determine safety profile of ruxolitinib in patients with hypereosinophilic syndrome and primary eosinophilic disorders.

II. To determine the proportion of patients on corticosteroids who are able to become corticosteroid-independent and/or reduce the dose by >= 50%.

III. To evaluate the duration of response (DoR). IV. To evaluate the time-to-response (TTR). V. To evaluate progression-free survival (PFS) and overall survival.

Being tested
Ruxolitinib
Medical subject headings
Hypereosinophilic Syndrome, Eosinophilia, Hepatomegaly, Splenomegaly

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RuxolitinibDrug

    Also called INCB-18424, INCB18424, Oral JAK Inhibitor INCB18424

    Given PO

Study groups in NCT03801434
Study groupRoleWhat they receive
Treatment (ruxolitinib)ExperimentalPatients 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.
Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall response rate (ORR)

    ORR is the sum of complete response (CR) plus complete response with incomplete platelet recovery (CRp) plus partial response (PR). Complete response, with platelet incomplete platelet recovery (CRp) is defined as a response that meets CR criteria but platelet count remains below 100 x 10^9/L. This outcome will be reported as a number.

    • Complete response (CR) = normalization of white blood cell (WBC) count; absolute eosinophil count in blood; and % eosinophils in blood, without increased blasts or eosinophils in bone marrow, and with a non-palpable spleen and/ or normal spleen size by imaging.
    • Partial response (PR) is defined as ≥ 50% reduction (if above normal range) in all the following: total WBC count; absolute eosinophil count in blood; % eosinophils in blood; % eosinophils and myeloblasts in bone marrow; in addition to spleen size reduction of ≥ 50% by palpation and/ or ≥35% by imaging (if increased at baseline).

    Measured: 3 years

Secondary outcomes (6)

  1. Adverse events

    Adverse events will be reported including severity, seriousness, and relatedness of adverse events based on National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. The outcome will be reported as a number of all serious adverse events; with frequency ≥ 10%.

    Measured: 3 years

  2. Proportion of subjects who become corticosteroid-independent

    Descriptive analysis of the proportion of subjects who become corticosteroid-independent for 12 or more consecutive weeks will be reported. The outcomes will be reported as number

    Measured: 3 years

  3. Proportion of patients who reduce corticosteroid dose by >= 50% (including patients who become corticosteroid-independent)

    Descriptive analysis of the proportion of subjects who reduce corticosteroid dose by >= 50% (including patients who become corticosteroid-independent) for 12 or more consecutive weeks will be reported. The outcomes will be reported as number

    Measured: 3 years

  4. Duration of response (DoR)

    Median duration of response (DoR) defined as the time from first onset of confirmed response to the date of first documented and confirmed progression or death due to hypereosinophilic syndrome or a primary eosinophilic neoplasm. This outcome will be reported as the median with full range, for those subjects that achieve a clinical response. Response defined as per the Primary Outcome.

    Measured: 3 years

  5. Time to response (TTR)

    Median time to response (TTR) is defined as the time from start of treatment until the date of onset of a confirmed response. This outcome will be reported as the median with full range, for those subjects that achieve a clinical response.

    Measured: 3 years

  6. Median Progression-free survival (PFS)

    Median progression free survival (PFS) is defined as the time from start of treatment to the date of the first documented and confirmed progression or death or institution of new therapy. This outcome will be reported as the median with full range. Progression is defined as ≥ 25% in one of the following when compared to baseline: total WBC count; absolute eosinophil count in blood; or % eosinophils in blood; OR the presence of ≥ 20% blasts in the peripheral blood or bone marrow ("evolution to AML"), with any lab finding confirmed at 2 weeks; or ≥ 25% increase in spleen size.

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Jan 2019
  2. Study start15 Nov 2019
  3. Primary completionFeb 2029 (estimated)
  4. Study completionFeb 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
8 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03801434
Sponsor's protocol ID
IRB-47457
Other IDs
NCI-2018-03723, HEMMPD0035, IRB-47457
Status last verified
Aug 2026
First submitted
9 Jan 2019
Last updated
1 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib in people with BCR-JAK2 Fusion Protein Expression and Blasts 20 Percent or Less of Peripheral Blood White Cells, run by Stanford University at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 21 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall response rate (ORR) (time frame: 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03801434.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2026.

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