Ruxolitinib for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (RISE)
16 participantsStarted 2026-07-15
Plain-language summary
This is a pilot study to gather information about safety and efficacy of using ruxolitinib (RUX) to treat Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (IEC-HS) occurring after CAR-T therapy. In addition, correlative studies will be done to 1) estimate the optimal duration of RUX therapy, 2) to identify immunological biomarkers associated with response (3) To evaluate the dynamics of CAR T expansion following RUX treatment.
Oral RUX will be administered twice daily, with dosing determined by the participant's baseline platelet count. Treatment will continue for up to 8 weeks unless significant adverse events occur or the treating physician concludes that the therapy is no longer providing clinical benefit.
The study expects to accrue 16 evaluable patients diagnosed with IEC-HS over 2 years.
Who can participate
Age range
18 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Patients of age 18 or older
* Diagnosis of for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (IEC-HS) per ASTCT consensus criteria
* Patients must have an elevated ferritin (\>2 × ULN) at time of infusion and/or have a ferritin count that is rapidly rising (per clinical assessment) and at least 2 of the following manifestations as described in the ASTCT consensus criteria:
* Onset with resolving/resolved CRS or worsening inflammatory response after initial improvement with CRS-directed therapy
* Hepatic transaminase elevation§ (\>5 × ULN (if baseline was normal) or \>5 × baseline if baseline was abnormal)
* Hypofibrinogenemia (\<150 mg/dL or \<LLN)
* Hemophagocytosis in bone marrow or other tissue
* Grade 1 cytopenias (new onset, worsening, or refractory) defined as:
* Anemia (Hgb) \<LLN\* - 10.0 g/dL(gr1)
* Neutropenia (ANC) 1,000 - 1,499/µL (Gr1)
* Thrombocytopenia \<LLN - 75,000/µL (Gr1)
* Lactate dehydrogenase elevations (\>ULN)
* Other coagulation abnormalities (e.g. elevated PT/PTT)
* Direct hyperbilirubinemia
* New-onset splenomegaly that is palpable ≥5 cm below costal margin or \>450cc on imaging
* Fever of 100.4 or greater (new or persistent)
* Neurologic changes greater than baseline that are consistent with a grade 1 Immune Effector Cell Encephalopathy (ICE) Score or greater
* Pulmonary manifestations
* Gr 2 Hypoxia with decreased oxygen saturation with exercise (e.g. pulse ox \<88%)
* pulmonary …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants with Clinical Response
Timeframe: 8 weeks
2
Number of Adverse events
Timeframe: 8 weeks
Trial details
NCT IDNCT07424222
SponsorSidney Kimmel Comprehensive Cancer Center at Johns Hopkins