Back to resultsRuxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
Primary Purpose
Macrophage Activation Syndrome
Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Ruxolitinib
methylprednisolone
Sponsored by
About this trial
This is an interventional treatment trial for Macrophage Activation Syndrome
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ruxolitinib+methylprednisolone
Arm Description
Ruxolitinib and methylprednisolone administered as the first-line therapy
Outcomes
Primary Outcome Measures
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%.
Progression Free Survival
from date of inclusion to date of progression, relapse, or death from any cause
Adverse events
Adverse events including myelosuppression, infection, hemorrhage
Secondary Outcome Measures
Full Information
NCT ID
NCT05137496
First Posted
November 24, 2021
Last Updated
March 16, 2022
Sponsor
Beijing Friendship Hospital
1. Study Identification
Unique Protocol Identification Number
NCT05137496
Brief Title
Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
Official Title
A Single-center, Prospective, Non-comparative Clinical Trial of Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Not yet recruiting
Study Start Date
June 1, 2022 (Anticipated)
Primary Completion Date
June 1, 2023 (Anticipated)
Study Completion Date
June 1, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Beijing Friendship Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. 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 .
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Macrophage Activation Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Ruxolitinib+methylprednisolone
Arm Type
Experimental
Arm Description
Ruxolitinib and methylprednisolone administered as the first-line therapy
Intervention Type
Drug
Intervention Name(s)
Ruxolitinib
Intervention Description
0.3mg/kg/d, iv.gtt, for at least 2 weeks
Intervention Type
Drug
Intervention Name(s)
methylprednisolone
Intervention Description
2mg/kg, d1-d5, gradually reduced, for at least 2 weeks
Primary Outcome Measure Information:
Title
Response rate
Description
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%.
Time Frame
Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy
Title
Progression Free Survival
Description
from date of inclusion to date of progression, relapse, or death from any cause
Time Frame
2 years
Title
Adverse events
Description
Adverse events including myelosuppression, infection, hemorrhage
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yue Song
Phone
86-010-63139862
Email
xueqifeng1992@163.com
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
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