Ruxolitinib for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (RISE)

Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Study ID
NCT07424222
Phase
PHASE1
Status
Not Yet Recruiting

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Conditions

  • Immune Effector Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (IEC-HS)

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Ruxolitinib — DRUG
    In this study, Ruxolitinib will be supplied as 5 mg tablets which will be administered orally twice daily (BID) as an open-label, investigational product. Ruxolitinib dosing based on platelet numbers: * 5 mg twice a day if platelets are under 30,000/µL, * 10 mg twice a day if platelets are more than or equal to 30,000/µL but less than 50,000/µL, or * 15 mg twice a day if platelets are more than or equal to 50,000/µL Patients who respond may continue treatment for at least 8 weeks. Therapy will be discontinued for significant toxicity or evidence of IEC-HS progression. After 8 weeks, the dose may be tapered as clinically appropriate, with continued therapy permitted for up to 6 additional months if clinical benefit persists.

Study Details

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.

Key Dates

Start date
Jun 1, 2026
Status verified
May 2026
Primary completion
Jun 1, 2028
Completion
Jan 1, 2029

Study Design

Enrollment
16 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Ruxolitinib (RUX) therapy
    Patients will receive ruxolitinib at a dose of 5 mg twice a day if platelets are under 30,000/µL, ruxolitinib 10 mg twice a day if platelets are ≥30,000/µL but under 50,000/µL, or ruxolitinib 15 mg twice a day if platelets are ≥50,000/µL at the start of treatment. If a favorable response to ruxolitinib is noted (as defined in the protocol), patients will continue on treatment for at least 8 weeks if it is tolerated without significant side effects.

Primary Outcome Measure

Number of Participants with Clinical Response [ Time Frame: 8 weeks ]

Central Contacts