Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome

Capital Medical University

Status and phase: Not yet enrolling; Phase 3

Conditions: Macrophage Activation Syndrome

Treatments: Drug: methylprednisolone; Drug: Ruxolitinib

Study type: Interventional

Funder types: Other

Identifiers

NCT05137496

Ruxolitinib-steroids-MAS

Details and patient eligibility

About

The present study was a single-center, prospective, non-comparative in which macrophage activation syndrome patients were selected as the main subjects to evaluate the effect and safety of Ruxolitinib and methylprednisolone regimens as the first-line therapy .

Enrollment: 40 estimated patients

Sex: All

Ages: 1 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS).
2. No HLH induction therapy was performed.
3. The expected survival time is more than 1 month.
4. Serum creatinine ≤ 1.5 times normal；Serum human immunodeficiency virus(HIV) antigen or antibody negative； Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；HBV copies less than 1E+03 copies/ml.
5. Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value
6. The left ventricular ejection fraction (LVEF) was normal.
7. No uncontrollable infection.
8. Contraception for both male or female.
9. Informed consent obtained.

Exclusion criteria

1. Pregnancy or lactating Women；
2. Allergic to ruxolitinib；
3. Active bleeding of the internal organs；
4. uncontrollable infection；
5. Serious mental illness;
6. Non-melanoma skin cancer history;
7. Patients unable to comply during the trial and/or follow-up phase;
8. Participate in other clinical research at the same time.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

Ruxolitinib+methylprednisolone

Experimental group

Description:

Ruxolitinib and methylprednisolone administered as the first-line therapy

Treatment:

Drug: methylprednisolone

Drug: Ruxolitinib