Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorWilliam Shomali

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Ruxolitinib

    Drug

    Given PO

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: Treatment (ruxolitinib)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

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).

Time frame
3 years

Secondary outcomes

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%.

Time frame
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

Time frame
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

Time frame
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.

Time frame
3 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

William Shomali

Lead sponsor

Stanford University

Sponsor institution

Incyte Corporation

Collaborator