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

NCT05137496 | Unknown Phase 3

• 1 other identifier: Ruxolitinib-steroids-MAS
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

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 .

Conditions

Macrophage Activation Syndrome

Outcome Measures

Primary Outcomes (3)

• 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

• Progression Free Survival

  from date of inclusion to date of progression, relapse, or death from any cause

  2 years

• Adverse events

  Adverse events including myelosuppression, infection, hemorrhage

  2 years

Study Arms (1)

Ruxolitinib+methylprednisolone

EXPERIMENTAL

Ruxolitinib and methylprednisolone administered as the first-line therapy

Drug: Ruxolitinib; Drug: methylprednisolone

Interventions

Ruxolitinib DRUG

0.3mg/kg/d, iv.gtt, for at least 2 weeks

Ruxolitinib+methylprednisolone

methylprednisolone DRUG

2mg/kg, d1-d5, gradually reduced, for at least 2 weeks

Ruxolitinib+methylprednisolone

Eligibility Criteria

• Age: 1 Year - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS).
• No HLH induction therapy was performed.
• The expected survival time is more than 1 month.
• 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.
• Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value
• The left ventricular ejection fraction (LVEF) was normal.
• No uncontrollable infection.
• Contraception for both male or female.
• Informed consent obtained.

You may not qualify if:

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

Sponsors & Collaborators

• Beijing Friendship Hospital: lead

MeSH Terms

Conditions

Macrophage Activation Syndrome

Interventions

ruxolitinib; Methylprednisolone

Condition Hierarchy (Ancestors)

Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Central Study Contacts

Yue Song

CONTACT

86-010-63139862; xueqifeng1992@163.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: November 24, 2021
• First Posted: November 30, 2021
• Study Start: June 1, 2022
• Primary Completion: June 1, 2023
• Study Completion: June 1, 2024
• Last Updated: March 17, 2022

Record last verified: 2022-03