Prospective Single-Arm Clinical Trial of GO Regimen for HLH.

Capital Medical University

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Lymphohistiocytosis, Hemophagocytic

Treatments: Drug: Golidocitinib

Study type: Interventional

Funder types: Other

Identifiers

NCT07339345

Golidocitinib(GO),HLH

Details and patient eligibility

About

This study aims to investigate the efficacy and safety of Golidocitinib(GO) monotherapy in the treatment of hemophagocytic lymphohistiocytosis.

Enrollment: 28 estimated patients

Sex: All

Ages: 14 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Researchers evaluate patients with newly diagnosed or refractory/relapsed HLH disease.
2. Definite diagnosis of HLH: molecular diagnosis consistent with pHLH, or at least 5 of the 8 criteria in the HLH-2004 diagnostic criteria.
3. Patients who are currently unsuitable or unable to undergo allo-HSCT.
4. Researchers estimate that the expected survival period exceeds one month.
5. Patients must be at least 14 years of age and no older than 70 years of age, with no gender restrictions.
6. Before the study began, TB was ≤10 times the upper limit of normal; Cr was ≤1.5 times the normal value.
7. Serum HIV antigen or antibody negative。
8. HCV antibody negative, or HCV antibody positive but HCV RNA negative.
9. .HBsAg and HBcAb are both negative. If either of the above is positive, peripheral blood hepatitis B virus DNA titer testing is required, with a titer of less than 1×10³ copies/ml.
10. Echocardiography showed LVEF ≥ 50%.
11. Women of childbearing age must be confirmed as not pregnant by a pregnancy test and must be willing to use effective contraception during the study period and for at least 12 months after the last dose; all male participants must use contraception during the study period and for at least 3 months after the last dose.

Exclusion criteria

1. Heart function above grade II(including II) (NYHA).
2. Pregnancy or lactating Women and reproductive-age patients who refused to use appropriate contraceptive measures during this trial.
3. Individuals who are allergic to GO or have a severe allergic constitution.
4. Active bleeding of the internal organs.
5. uncontrollable infection.
6. Severe mental illness.
7. History of non-melanoma skin cancer.
8. Unable to comply during the trial and/or follow-up phase. Participate in other clinical research at the same time.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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28 participants in 1 patient group

HLH patients

Experimental group

Description:

Initial diagnosis or presence of refractory/relapsed HLH disease or confirmed diagnosis of HLH.

Treatment:

Drug: Golidocitinib