Clinical trial NCT06951971: Dose-modified Emapalumab and Ruxolitinib (E-Ru) Regimens for Hemophagocytic Lymphohistiocytosis
Official title: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Hemophagocytic Lymphohistiocytosis (HLH)
Lead sponsor: The First Affiliated Hospital of Soochow University
Sponsor class: OTHER
First posted: 2025-04-30
Enrollment: 30
Sex: ALL
Minimum age (years): 1
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: Inclusion Criteria:

1. Fulfillment of at least five of the eight HLH-2004 criteria for HLH;
2. Age 1-70 years old;
3. No prior chemotherapy for HLH;
4. Confirmed not pregnant and willing to use effective contraceptive measures during the study period, with the last dose of medication administered at least 12 months prior;
5. Signed informed consent prior to study participation.

Exclusion Criteria:

1. According to the New York Heart Association (NYHA) score, patients with heart disease of grade II or above (including grade II);
2. HIV-infected patients;
3. Patients with severe renal dysfunction (glomerular filtration rate \<15 mL/min);
4. Patients with severe liver cirrhosis (MELD score\>20);
5. Uncontrollable infections (including lung infections, intestinal infections, etc.);
6. Have serious mental illness;
7. Have a history of active tumor;
8. Participate in other clinical investigators at the same time;
9. People with central nervous system involvement;
10. Exclusion criteria related to concomitant medication.

Primary outcomes:
measure: 60-days OS
timeFrame: 60 days
Secondary outcomes:
measure: Response rate
timeFrame: 7, 14, 28, and 56 days
measure: Security
timeFrame: 7, 14, 28, and 56 days
measure: EFS
timeFrame: 6 months
measure: Overall survival
timeFrame: 6 months
measure: Trend of related indicators
timeFrame: 7, 14, 28, and 56 days
Other outcomes:
Locations:
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
state: Jiangsu
Study officials:
affiliation: The First Affiliated Hospital of Soochow University
name: Xuefeng He, Dr.
role: PRINCIPAL_INVESTIGATOR
References: