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New treatment option for Immune Effector Associated Hemophagocytic Lymphohistiocytosis-like...
Official title Ruxolitinib for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (RISE)
ClinicalTrials.gov ID: NCT07424222
What this study is testing
What is Ruxolitinib?
Ruxolitinib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for immune effector associated hemophagocytic lymphohistiocytosis-like syndrome (iec-hs).
Also referred to as JAKAFI.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- Time commitment: about 2 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 99
You may be able to join if
- 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)...
- 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)
You likely can't join if
- Life expectancy greater than 6 months
- Patients with known active malignancy or known progressive underlying disease
- Women who are pregnant or breastfeeding. Women must also refrain from breastfeeding during the course of study and for 60 days after the last dose of...
- Presence of chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment. Participants with acute...
- Any prior investigational agent used to treat IEC-HS
- Patient on treatment with rifampin, St Johns wart or other JAK inhibitors
See the full eligibility 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...
- 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 infiltrates
- pulmonary edema with radiologic findings and moderate dyspnea on exertion
- New onset renal insufficiency defined by:
- an absolute increase in serum creatinine of ≥0.3 mg/dL within 48 hours.
- A baseline increase in serum creatinine of ≥1.5 times within the prior 7 days.
- Oliguria defined as urine volume \<0.5 mL/kg/h for at least 6 hours.
- Hypertriglyceridemia (fasting level, \>265 mg/dL‖)
- Patients who have received a CAR T product, commercially available or investigational, will be allowed to enroll
- Eastern Cooperative Oncology Group (ECOG) Performance Score43 of 0-2
- Patients may be treatment naïve for the IECH-HS diagnosis or may have received prior or ongoing treatment. Corticosteroids can be continued along with RUX initiation as noted above.
- Patient is able to take oral medication or is willing to have an NG tube placed if unable to take oral medication. Patients in whom NG tube was placed due to acute decompensation (resulting in an ECOG of \>2), will be...
- Willing and able to sign an informed consent form
- Life expectancy greater than 6 months
- Patients with known active malignancy or known progressive underlying disease
- Women who are pregnant or breastfeeding. Women must also refrain from breastfeeding during the course of study and for 60 days after the last dose of study treatment.
- Presence of chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment. Participants with acute infection requiring antibiotic, antifungal, or antiviral treatment use...
- Any prior investigational agent used to treat IEC-HS
- Patient on treatment with rifampin, St Johns wart or other JAK inhibitors
- Patient is unable to tolerate medicine administration either orally or via NG tube.
- Known history of allergic reaction to ruxolitinib
- History of clinically significant or uncontrolled cardiac disease, including recent (within the last 12 months) unstable angina or acute myocardial infarction, or New York Heart Association Class III or IV congestive...
- Any major surgery within 28 days before the first dose of study treatment.
- Active HBV (or at risk of reactivation), defined as follows: positive HBsAg result (laboratory test required at screening), and/or positive total anti-HBc result (laboratory test required at screening), and/or...
- Active HCV, defined as follows: positive anti-HCV result (laboratory test required at screening) and quantitative HCV RNA test result greater than the lower limits of detection of the assay (laboratory test only...
- Known history of HIV (1/2 antibodies).
- Any condition or circumstance that would, in the investigator's judgment, interfere with full participation in the study (eg, unable, unlikely, or unwilling to comply with the dose schedule and study evaluations, active...
The study team makes the final eligibility decision.
Where it's taking place
- Baltimore, Maryland, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 99 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Baltimore, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.