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

Phase 3

• Conditions: Macrophage Activation Syndrome
• Interventions: Drug: Ruxolitinib; Drug: methylprednisolone

• Registration Number: NCT05137496
• Lead Sponsor: Beijing Friendship Hospital

Brief Summary

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 .

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-24
• Study First Submitted QC: 2021-11-29
• Study First Posted: 2021-11-30
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-16
• Last Update Posted: 2022-03-17
• Study Start: 2022-06-01
• Primary Completion: 2023-06-01
• Study Completion: 2024-06-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ruxolitinib+methylprednisolone	Ruxolitinib	Ruxolitinib and methylprednisolone administered as the first-line therapy
Ruxolitinib+methylprednisolone	methylprednisolone	Ruxolitinib and methylprednisolone administered as the first-line therapy

Primary Outcome Measures

Name	Time	Method
Response rate	Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy	A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT).; A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of\<0.5 ×109/L, a response was defined as an increase by at least 100% to\>0.5× 109/L; for patients with a neutrophil count of 0.5 to 2.0 × 109/L, an increase by at least 100% to \>2.0 × 109/L was considered a response; and for patients with ALT \>400 U/L, response was defined as an ALT decrease of at least 50%.
Progression Free Survival	2 years	from date of inclusion to date of progression, relapse, or death from any cause
Adverse events	2 years	Adverse events including myelosuppression, infection, hemorrhage