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Ruxolitinib 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
Beijing Friendship Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Macrophage Activation Syndrome

Eligibility Criteria

1 Year - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

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

    First Posted
    November 24, 2021
    Last Updated
    March 16, 2022
    Sponsor
    Beijing Friendship Hospital
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    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

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

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