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

Sponsor
Beijing Friendship Hospital (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05137496
Collaborator
(none)
40
1
24

Study Details

Study Description

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 .

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
40 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Single-center, Prospective, Non-comparative Clinical Trial of Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
Anticipated Study Start Date :
Jun 1, 2022
Anticipated Primary Completion Date :
Jun 1, 2023
Anticipated Study Completion Date :
Jun 1, 2024

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

  1. 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%.

  2. Progression Free Survival [2 years]

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

  3. Adverse events [2 years]

    Adverse events including myelosuppression, infection, hemorrhage

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year to 75 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Beijing Friendship Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Zhao Wang, Clinical Professor, Beijing Friendship Hospital
ClinicalTrials.gov Identifier:
NCT05137496
Other Study ID Numbers:
  • Ruxolitinib-steroids-MAS
First Posted:
Nov 30, 2021
Last Update Posted:
Mar 17, 2022
Last Verified:
Mar 1, 2022
Individual Participant Data (IPD) Sharing Statement:
Undecided
Plan to Share IPD:
Undecided
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Mar 17, 2022