A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome

• Status: Completed
• Trial end date: 2020-01-07
• Target enrollment: 0
• Participant gender: All

Summary

This is a pilot study to determine the efficacy of Ruxolitinib in secondary hemophagocytic syndrome. The primary objective is to assess the efficacy of ruxolitinib 15 mg PO twice daily in patients with HPS. The primary endpoint is overall survival at two months.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Michigan Cancer Center; University of Michigan Rogel Cancer Center

Criteria

Inclusion Criteria:

• Patients, or their legally authorized representative, must voluntarily provide written IRB-approved informed consent.

• Males and females, 18 years of age or older at the time of enrollment.

• Patients must meet the diagnostic criteria for HPS (at least 5 of the following):

fever, splenomegaly, cytopenia involving ≥2 cell lines (Hemoglobin <9 g/dL; platelets <100,000/μL; absolute neutrophil count <1000/μL), hypertriglyceridemia or hypofibrinogenemia, tissue demonstration of hemophagocytosis, low or absent NK (Natural Killer) cell activity, serum ferritin ≥3000 ug/L, soluble IL-2 receptor (CD25) >2400 U/mL.

• In the investigator's opinion, the patient has the ability to participate fully in the study, and comply with all its requirements.

Exclusion Criteria:

• CNS (Central Nervous System) involvement

• Malabsorption

• Known secondary HPS (Hemophagocytic Syndrome) that is otherwise treatable (e.g. non-Hodgkin's lymphoma).

• Pregnant or lactating female: all females of child-bearing potential must have a negative serum pregnancy test within 7 days of treatment; lactating females must discontinue breast feeding.

• Estimated creatinine clearance <15mL/min

• Has received any prior systemic therapy, excluding corticosteroids, within 7 days (or 5 half-lives) of treatment.

• No active malignancy at the time of enrollment, except nonmelanoma skin cancers or carcinoma in situ. Patients with a prior history of malignancy are eligible if their malignancy has been definitely treated or is in remission and does not require ongoing adjuvant or cancer-directed therapies.

• Active hepatitis B or hepatitis C or known HIV infection

• Known (and biopsy-confirmed) liver cirrhosis; or, a reported history of liver cirrhosis with a Model for End-stage Liver Disease (MELD) score >20.