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

Macrophage Activation Syndrome Clinical Trial

Official title:

A Single-center, Prospective, Non-comparative Clinical Trial of Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05137496
• Other study ID #: Ruxolitinib-steroids-MAS
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: June 1, 2022
• Est. completion date: June 1, 2024

Study information

• Verified date: March 2022
• Source: Beijing Friendship Hospital
• Contact: Yue Song
• Phone: 86-010-63139862
• Email: xueqifeng1992[@]163.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 .

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 40
• Est. completion date: June 1, 2024
• Est. primary completion date: June 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Macrophage Activation Syndrome
• Syndrome

Intervention

Drug:
• Ruxolitinib
0.3mg/kg/d, iv.gtt, for at least 2 weeks
• methylprednisolone
2mg/kg, d1-d5, gradually reduced, for at least 2 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Beijing Friendship Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Response rate	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%.	Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy
Primary	Progression Free Survival	from date of inclusion to date of progression, relapse, or death from any cause	2 years
Primary	Adverse events	Adverse events including myelosuppression, infection, hemorrhage	2 years