NCT06951971

Dose-modified Emapalumab and Ruxolitinib (E-Ru) Regimens for Hemophagocytic Lymphohistiocytosis

A Multiple Center, Prospective, Single Arm, Phase III Clinical Study to Evaluate the Efficacy and Safety of Dose-modified Emapalumab and Ruxolitinib in the Treatment of Hemophagocytic Lymphohistiocytosis

• Status: Not Yet Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: The First Affiliated Hospital of Soochow University · Academic / Other
• Sex: All
• Age: 1 Year – 70 Years
• Healthy volunteers: Not accepted

Summary

This study is trying to evaluate the efficacy and safety of dose-modified Emapalumab and Ruxolitinib (E-Ru) regimens for the treatment of active hemophagocytic lymphohistiocytosis.

Detailed description

This clinical trial is designed to evaluate a new treatment strategy for patients with active Hemophagocytic Lymphohistiocytosis (HLH)-a rare but severe immune disorder characterized by excessive inflammation and immune system activation. HLH can be life-threatening if not treated effectively. The study is prospective and multicenter, meaning it will be conducted at multiple hospitals and medical institutions, and patients will be followed over time to assess treatment outcomes. We aim to test the combination of two medications: 1. Emapalumab, a monoclonal antibody that blocks interferon-gamma, a key driver of the overactive immune response seen in HLH. In this trial, it will be used at a lower-than-standard dose to reduce potential side effects. 2. Ruxolitinib, a JAK1/2 inhibitor that can reduce inflammation by interfering with immune signaling pathways. It will be administered at a higher-than-standard dose to enhance its therapeutic effects.

Conditions

• Hemophagocytic Lymphohistiocytosis (HLH)

Interventions

Type	Name	Description
DRUG	dose-modified E-Ru	Emapalumab (1-2 mg/kg intravenously once weekly for 8 weeks) + Ruxolitinib (15-30 mg orally twice daily for 8 weeks)
OTHER	Salvage treatment and follow-up	1\) for patients who did not respond to the E-Ru regimen after 7 days or who relapsed at any point during treatment, a rescue regimen such as HLH-94 or doxorubicin-etoposide-methylprednisolone (DEP) was administered; 2) for patients diagnosed with lymphoma after enrollment, chemotherapy was initiated; 3) for patients who completed 8 weeks of treatment without recurrence and had no detected HLH-related gene mutations, follow-up was initiated; and 4) for patients who met the criteria for allo-HSCT, allo-HSCT was performed.

Timeline

• Start date: 2025-06-01
• Primary completion: 2027-06-01
• Completion: 2028-06-01
• First posted: 2025-04-30
• Last updated: 2025-04-30

Locations

1 site across 1 country: China