GTP Regimen in the Treatment of Refractory/Recurrent HLH

Recurrence Clinical Trial

Official title:

Clinical Study on Efficacy and Safety of GTP Regimen in the Treatment of Refractory/Recurrent Hemophagocytic Lymphohistiocytosis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06038422
• Other study ID #: BFH20230717001
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: September 15, 2023
• Est. completion date: September 15, 2025

Study information

• Verified date: September 2023
• Source: Beijing Friendship Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Description:

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 15
• Est. completion date: September 15, 2025
• Est. primary completion date: September 15, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month and older

Eligibility

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 family history 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. 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).

4. According to the researchers, the expected survival was more than 2 weeks.

5. The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative.

6. 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:

1. There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmania infection.

2. There is uncontrolled active gastrointestinal bleeding.

3. The investigator believes that there are any serious comorbidities or any other conditions that make the patient unsuitable for treatment.

4. A history of hypersensitivity or hypersensitivity to any component of the drug in the study 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 to screening.

7. Pregnancy patients.

8. Within 4 weeks prior to enrollment in this study, another concurrent clinical intervention study was enrolled.

9. 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.

Related Conditions & MeSH terms

• Hemophagocytic Lymphohistiocytosis
• Lymphohistiocytosis, Hemophagocytic
• Recurrence
• Refractory

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Beijing Friendship Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ORR	Overall remission rate	1 year