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

NCT ID: NCT06951971

Last Updated: 2025-04-30

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-01
• Study Completion Date: 2028-06-01

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.

Conditions

Hemophagocytic Lymphohistiocytosis (HLH)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

E-Ru for HLH

dose-modified Emapalumab+Ruxolitinib for the treatment of HLH patients.

Group Type: EXPERIMENTAL

dose-modified E-Ru

Intervention Type: DRUG

Emapalumab (1-2 mg/kg intravenously once weekly for 8 weeks) + Ruxolitinib (15-30 mg orally twice daily for 8 weeks)

Salvage treatment and follow-up

Intervention Type: OTHER

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.

Interventions

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)

Intervention Type: DRUG

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.

Intervention Type: OTHER

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;

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The First Affiliated Hospital of Soochow University

OTHER

Sponsor Role: lead

Responsible Party

Xuefeng He

The First Affiliated Hospital of Soochow University

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Xuefeng He, Dr.

Role: PRINCIPAL_INVESTIGATOR

The First Affiliated Hospital of Soochow University

Locations

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China

Countries

China

Central Contacts

Yue Song, Dr

Role: CONTACT

Phone: +86 18810253070

Email: xueqifeng1992@163.com

Facility Contacts

Yue Song, Dr.

Role: primary

Other Identifiers

E-Ru

Identifier Type: -

Identifier Source: org_study_id