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

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

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 .

Study Overview

• Status: Not yet recruiting
• Conditions: Macrophage Activation Syndrome
• Intervention / Treatment: Drug: Ruxolitinib; Drug: methylprednisolone

• Study Type: Interventional
• Enrollment (Anticipated): 40
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Yue Song; Phone Number: 86-010-63139862; Email: xueqifeng1992@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib+methylprednisolone; Ruxolitinib and methylprednisolone administered as the first-line therapy	Drug: Ruxolitinib; 0.3mg/kg/d, iv.gtt, for at least 2 weeks; Drug: methylprednisolone; 2mg/kg, d1-d5, gradually reduced, for at least 2 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Beijing Friendship Hospital

Study record dates

Study Major Dates

• Study Start (Anticipated): June 1, 2022
• Primary Completion (Anticipated): June 1, 2023
• Study Completion (Anticipated): June 1, 2024

Study Registration Dates

• First Submitted: November 24, 2021
• First Submitted That Met QC Criteria: November 29, 2021
• First Posted (Actual): November 30, 2021

Study Record Updates

• Last Update Posted (Actual): March 17, 2022
• Last Update Submitted That Met QC Criteria: March 16, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Disease; Syndrome; Macrophage Activation Syndrome; Physiological Effects of Drugs; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Antineoplastic Agents; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Neuroprotective Agents; Protective Agents; Prednisolone; Methylprednisolone Acetate; Methylprednisolone; Methylprednisolone Hemisuccinate; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate
• Other Study ID Numbers: Ruxolitinib-steroids-MAS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No