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GTP Regimen in the Treatment of Refractory/Recurrent HLH

Primary Purpose

Refractory, Recurrence, Hemophagocytic Lymphohistiocytosis

Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
GTP regimen
Sponsored by
Beijing Friendship Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Refractory

Eligibility Criteria

1 Month - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Male and female patients ≥1 month of age with HLH Definitive diagnosis of HLH: meets the molecular diagnosis of pHLH or has a family history of HLH, or meets at least 5 of the 8 diagnostic criteria of HLH-2004. The investigators assessed the presence of refractory/recurrent HLH disease. Patients must meet one of the following criteria assessed by the investigator: Previous conventional HLH treatment did not respond. The patients were not satisfied with the efficacy of conventional HLH treatment or their condition worsened. HLH reactivation occurs. Reactivation is defined as: worsening of two or more HLH clinical and laboratory criteria (after initial remission). According to the researchers, the expected survival was more than 2 weeks. The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative. Women who are fertile are willing to use a highly effective contraceptive method from the start of the study until 6 months after the end of the last course of treatment. Exclusion Criteria: There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmania infection. There is uncontrolled active gastrointestinal bleeding. The investigator believes that there are any serious comorbidities or any other conditions that make the patient unsuitable for treatment. A history of hypersensitivity or hypersensitivity to any component of the drug in the study protocol, such as polysorbate. Had received the BCG vaccine within 12 weeks prior to screening. Have received live or attenuated vaccine (except BCG) within 4 weeks prior to screening. Pregnancy patients. Within 4 weeks prior to enrollment in this study, another concurrent clinical intervention study was enrolled. There is any condition or circumstance that the investigator believes may cause the patient to be unable to complete the study or to comply with study procedures or requirements.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    GTP regimen treated patients

    Arm Description

    R/R HLH patients treated with GTP regimen

    Outcomes

    Primary Outcome Measures

    ORR
    Overall remission rate

    Secondary Outcome Measures

    Full Information

    First Posted
    September 8, 2023
    Last Updated
    September 8, 2023
    Sponsor
    Beijing Friendship Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06038422
    Brief Title
    GTP Regimen in the Treatment of Refractory/Recurrent HLH
    Official Title
    Clinical Study on Efficacy and Safety of GTP Regimen in the Treatment of Refractory/Recurrent Hemophagocytic Lymphohistiocytosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    September 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    September 15, 2023 (Anticipated)
    Primary Completion Date
    September 15, 2024 (Anticipated)
    Study Completion Date
    September 15, 2025 (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 goal of this clinical trial is to learn about efficacy and safety of GTP regimen in refractory/recurrent hemophagocytic lymphohistiocytosis. The main questions it aims to answer are: Overall remission rate of GTP regimen in R/R HLH Adverse effect of GTP regimen Participants will be treated with GTP regimen
    Detailed Description
    Refractory/recurrent hemophagocytic lymphohistiocytosis will be treated with emapalumab combined with teniposide and methylprednisolone. Observed the overall response rate and side effects.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Refractory, Recurrence, Hemophagocytic Lymphohistiocytosis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    15 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    GTP regimen treated patients
    Arm Type
    Experimental
    Arm Description
    R/R HLH patients treated with GTP regimen
    Intervention Type
    Drug
    Intervention Name(s)
    GTP regimen
    Intervention Description
    Refractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone)
    Primary Outcome Measure Information:
    Title
    ORR
    Description
    Overall remission rate
    Time Frame
    1 year

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    1 Month
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Male and female patients ≥1 month of age with HLH Definitive diagnosis of HLH: meets the molecular diagnosis of pHLH or has a family history of HLH, or meets at least 5 of the 8 diagnostic criteria of HLH-2004. The investigators assessed the presence of refractory/recurrent HLH disease. Patients must meet one of the following criteria assessed by the investigator: Previous conventional HLH treatment did not respond. The patients were not satisfied with the efficacy of conventional HLH treatment or their condition worsened. HLH reactivation occurs. Reactivation is defined as: worsening of two or more HLH clinical and laboratory criteria (after initial remission). According to the researchers, the expected survival was more than 2 weeks. The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative. Women who are fertile are willing to use a highly effective contraceptive method from the start of the study until 6 months after the end of the last course of treatment. Exclusion Criteria: There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmania infection. There is uncontrolled active gastrointestinal bleeding. The investigator believes that there are any serious comorbidities or any other conditions that make the patient unsuitable for treatment. A history of hypersensitivity or hypersensitivity to any component of the drug in the study protocol, such as polysorbate. Had received the BCG vaccine within 12 weeks prior to screening. Have received live or attenuated vaccine (except BCG) within 4 weeks prior to screening. Pregnancy patients. Within 4 weeks prior to enrollment in this study, another concurrent clinical intervention study was enrolled. There is any condition or circumstance that the investigator believes may cause the patient to be unable to complete the study or to comply with study procedures or requirements.

    12. IPD Sharing Statement

    Learn more about this trial

    GTP Regimen in the Treatment of Refractory/Recurrent HLH

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