GTP Regimen in the Treatment of Refractory/Recurrent HLH
Study Details
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:
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Overall remission rate of GTP regimen in R/R HLH
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Adverse effect of GTP regimen Participants will be treated with GTP regimen
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Detailed Description
Refractory/recurrent hemophagocytic lymphohistiocytosis will be treated with emapalumab combined with teniposide and methylprednisolone. Observed the overall response rate and side effects.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: GTP regimen treated patients R/R HLH patients treated with GTP regimen |
Drug: GTP regimen
Refractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone)
|
Outcome Measures
Primary Outcome Measures
- ORR [1 year]
Overall remission rate
Eligibility Criteria
Criteria
Inclusion Criteria:
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Male and female patients ≥1 month of age with HLH
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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.
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The investigators assessed the presence of refractory/recurrent HLH disease. Patients must meet one of the following criteria assessed by the investigator:
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Previous conventional HLH treatment did not respond.
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The patients were not satisfied with the efficacy of conventional HLH treatment or their condition worsened.
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HLH reactivation occurs. Reactivation is defined as: worsening of two or more HLH clinical and laboratory criteria (after initial remission).
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According to the researchers, the expected survival was more than 2 weeks.
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The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative.
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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:
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There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmania infection.
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There is uncontrolled active gastrointestinal bleeding.
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The investigator believes that there are any serious comorbidities or any other conditions that make the patient unsuitable for treatment.
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A history of hypersensitivity or hypersensitivity to any component of the drug in the study protocol, such as polysorbate.
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Had received the BCG vaccine within 12 weeks prior to screening.
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Have received live or attenuated vaccine (except BCG) within 4 weeks prior to screening.
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Pregnancy patients.
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Within 4 weeks prior to enrollment in this study, another concurrent clinical intervention study was enrolled.
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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.
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.- BFH20230717001