GTP Regimen in the Treatment of Refractory/Recurrent HLH

Last updated: September 8, 2023
Sponsor: Beijing Friendship Hospital
Overall Status: Active - Not Recruiting

Phase

3

Condition

Histiocytoma

White Cell Disorders

Red Blood Cell Disorders

Treatment

GTP regimen

Clinical Study ID

NCT06038422
BFH20230717001
  • Ages > 1
  • All Genders

Study 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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Male and female patients ≥1 month of age with HLH
  2. Definitive diagnosis of HLH: meets the molecular diagnosis of pHLH or has a familyhistory of HLH, or meets at least 5 of the 8 diagnostic criteria of HLH-2004.
  3. The investigators assessed the presence of refractory/recurrent HLH disease. Patientsmust 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 treatmentor their condition worsened.
  • HLH reactivation occurs. Reactivation is defined as: worsening of two or more HLHclinical and laboratory criteria (after initial remission).
  1. According to the researchers, the expected survival was more than 2 weeks.
  2. The informed consent is signed by the patient or his legal guardian (patients under 18years), or by the patient's legally authorized representative.
  3. Women who are fertile are willing to use a highly effective contraceptive method fromthe start of the study until 6 months after the end of the last course of treatment.

Exclusion

Exclusion Criteria:

  1. There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmaniainfection.
  2. There is uncontrolled active gastrointestinal bleeding.
  3. The investigator believes that there are any serious comorbidities or any otherconditions that make the patient unsuitable for treatment.
  4. A history of hypersensitivity or hypersensitivity to any component of the drug in thestudy protocol, such as polysorbate.
  5. Had received the BCG vaccine within 12 weeks prior to screening.
  6. Have received live or attenuated vaccine (except BCG) within 4 weeks prior toscreening.
  7. Pregnancy patients.
  8. Within 4 weeks prior to enrollment in this study, another concurrent clinicalintervention study was enrolled.
  9. There is any condition or circumstance that the investigator believes may cause thepatient to be unable to complete the study or to comply with study procedures orrequirements.

Study Design

Total Participants: 15
Treatment Group(s): 1
Primary Treatment: GTP regimen
Phase: 3
Study Start date:
September 15, 2023
Estimated Completion Date:
September 15, 2025

Study Description

Refractory/recurrent hemophagocytic lymphohistiocytosis will be treated with emapalumab combined with teniposide and methylprednisolone. Observed the overall response rate and side effects.