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

NCT07339345 | Recruiting Phase 2 DMC

• 1 other identifier: Golidocitinib(GO),HLH
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Lymphohistiocytosis, Hemophagocytic

Keywords

Hemophagocytic Lymphohistocytosis; Golidocitinib

Outcome Measures

Primary Outcomes (1)

• Evaluation of treatment response

  A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, triglyceride, blood cell count, hemophagocytosis, and level of consciousness (if CNS HLH is present). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; without blood transfusion: Neutrophils \<500/ml must increase by 100% and be \>500/ml; neutrophils 500-2000/ml must increase by 100% and return to normal.; and for patients with ALT \>400 U/L, response was defined as an ALT decrease of at least 50%. No response is defined as not meeting the criteria for partial response.

  Weeks 2, 4, 6, and 8 after the start of treatment

Secondary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  30 days after the last dose is administered.

Other Outcomes (1)

• Survival

  1 year

Study Arms (1)

HLH patients

EXPERIMENTAL

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

Drug: Golidocitinib

Interventions

Golidocitinib DRUG

Administer the GO regimen to HLH patients who meet the inclusion criteria, specifically Golicitinib 150 mg once daily.

HLH patients

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Researchers evaluate patients with newly diagnosed or refractory/relapsed HLH disease.
• Definite diagnosis of HLH: molecular diagnosis consistent with pHLH, or at least 5 of the 8 criteria in the HLH-2004 diagnostic criteria.
• Patients who are currently unsuitable or unable to undergo allo-HSCT.
• Researchers estimate that the expected survival period exceeds one month.
• Patients must be at least 14 years of age and no older than 70 years of age, with no gender restrictions.
• Before the study began, TB was ≤10 times the upper limit of normal; Cr was ≤1.5 times the normal value.
• Serum HIV antigen or antibody negative。
• HCV antibody negative, or HCV antibody positive but HCV RNA negative.
• 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.
• Echocardiography showed LVEF ≥ 50%.
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Beijing Friendship Hospital: lead

Study Sites (1)

beijing Friendship Hospital, Capital Medical University

Beijing, Beijing Municipality, 100050, China

RECRUITING

MeSH Terms

Conditions

Lymphohistiocytosis, Hemophagocytic

Condition Hierarchy (Ancestors)

Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Study Officials

• zhao Wang

  Beijing Friendship Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

jingshi Wang, M.M.

CONTACT

86-13520280731; wangjingshi987@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Doctor

Study Record Dates

• First Submitted: January 1, 2026
• First Posted: January 14, 2026
• Study Start: June 27, 2025
• Primary Completion (Estimated): June 27, 2026
• Study Completion (Estimated): June 27, 2027
• Last Updated: January 14, 2026

Record last verified: 2026-01

Locations

CN (1)