GTP Regimen in the Treatment of Refractory/Recurrent HLH

NCT06038422 | Unknown Phase 3

• 1 other identifier: BFH20230717001
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

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

Conditions

Refractory; Recurrence; Hemophagocytic Lymphohistiocytosis

Outcome Measures

Primary Outcomes (1)

• ORR

  Overall remission rate

  1 year

Study Arms (1)

GTP regimen treated patients

EXPERIMENTAL

R/R HLH patients treated with GTP regimen

Drug: GTP regimen

Interventions

GTP regimen DRUG

Refractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone)

GTP regimen treated patients

Eligibility Criteria

• Age: 1 Month+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Beijing Friendship Hospital: lead

MeSH Terms

Conditions

Recurrence; Lymphohistiocytosis, Hemophagocytic

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: director of department

Study Record Dates

• First Submitted: September 8, 2023
• First Posted: September 14, 2023
• Study Start: September 15, 2023
• Primary Completion: September 15, 2024
• Study Completion: September 15, 2025
• Last Updated: September 14, 2023

Record last verified: 2023-09