New hope for rare blood disorder: drug targets overactive immune cells
NCT ID NCT03801434
First seen Jun 25, 2026 · Last updated Sep 02, 2026 · Updated 3 times
Summary
This phase 2 trial is testing the drug ruxolitinib in people with hypereosinophilic syndrome, a rare condition where the body produces too many eosinophils (a type of white blood cell), causing organ damage. The study aims to see if ruxolitinib can normalize blood cell counts and reduce symptoms. Ten participants are being recruited, and the main goal is to measure how many respond to treatment.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Ruxolitinib (a drug that blocks certain enzymes to reduce abnormal immune cell activity)
- What this could lead to
- If successful, this could provide a new treatment option for people with hypereosinophilic syndrome who do not respond to or cannot tolerate current therapies.
- What could go wrong
- This is a small, early-phase trial with only 10 participants, so results may not apply broadly. The drug may cause side effects or fail to improve outcomes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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8 people
The number who actually took part.
- Started
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Nov 2019
- Expected to finish
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Feb 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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). * 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). Exclusion Criteria: * 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. * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Fred Hutchinson cancer research center
Seattle, Washington, 98109, United States
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OHSU Knight Cancer Institute
Portland, Oregon, 97201, United States
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Stanford Cancer Institute Palo Alto
Palo Alto, California, 94304, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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