A Phase 2/3 interventional study of dose-modified E-Ru and Salvage treatment and follow-up in Hemophagocytic Lymphohistiocytosis (HLH), sponsored by The First Affiliated Hospital of Soochow University. Not yet recruiting at 1 site in China. Open to participants aged 1 Year to 70 Years. Per ClinicalTrials.gov, last updated 2025-04-30.
Sponsored by The First Affiliated Hospital of Soochow University · Phase 2/3, Interventional, and Treatment
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.
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:
99 studies on the registry are indexed under Lymphohistiocytosis, Hemophagocytic; 36 are open to participants now.
This study's planned enrollment of 30 is close to the median of 30 across 68 interventional studies indexed under Lymphohistiocytosis, Hemophagocytic.
Browse Lymphohistiocytosis, Hemophagocytic studies →The First Affiliated Hospital of Soochow University is the lead sponsor of 252 studies on the registry; 148 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
dose-modified Emapalumab+Ruxolitinib for the treatment of HLH patients.
Drug: dose-modified E-Ru · Other: Salvage treatment and follow-up
Emapalumab (1-2 mg/kg intravenously once weekly for 8 weeks) + Ruxolitinib (15-30 mg orally twice daily for 8 weeks)
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.
60-days OS
overall survival (OS) rate at 60 days (2-month OS)
Time frame: 60 days
Response rate
The ORR was defined as the percentage of patients with a complete response (CR), a partial response (PR), or an improvement in the measures of HLH.
Time frame: 7, 14, 28, and 56 days
Security
Adverse events (AEs) were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0 (CTCAE 5.0).
Time frame: 7, 14, 28, and 56 days
EFS
event-free survival, defined as the time from the first administration of E-Ru until no response on day 7, disease progression or death from any cause
Time frame: 6 months
Overall survival
OS (defined as the time from the first administration of E-Ru until death from any cause)
Time frame: 6 months
Trend of related indicators
Changes in biomarkers between baseline and post-treatment.
Time frame: 7, 14, 28, and 56 days
Plan to share: Undecided
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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Lymphohistiocytosis, Hemophagocytic→
The First Affiliated Hospital of Soochow University