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

Prospective Single-Arm Clinical Trial of Golidocitinib Monotherapy for Hemophagocytic Lymphohistiocytosis.

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

Study Overview

• Status: Recruiting
• Conditions: Lymphohistiocytosis, Hemophagocytic
• Intervention / Treatment: Drug: Golidocitinib

• Study Type: Interventional
• Enrollment (Estimated): 28
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: jingshi Wang, M.M.; Phone Number: 86-13520280731; Email: wangjingshi987@126.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100050
      • Recruiting
      • Beijing Friendship Hospital, Capital Medical University
      • Contact: jingshi Wang, M.M.; Phone Number: 86-13520280731; Email: wangjingshi987@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HLH patients; Initial diagnosis or presence of refractory/relapsed HLH disease or confirmed diagnosis of HLH.	Drug: Golidocitinib; Administer the GO regimen to HLH patients who meet the inclusion criteria, specifically Golicitinib 150 mg once daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	An adverse event is defined as an adverse medical condition (including worsening of a pre-existing condition) that occurs in a subject or clinical trial subject after receiving medication, regardless of whether it is causally related to the investigational drug. Serious adverse events are defined as AEs that meet one or more of the following criteria during any study period: death; immediate threat to life; and hospitalization or prolongation of hospitalization.	30 days after the last dose is administered.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survival	Patient's HLH-related symptoms and auxiliary examination results	1 year

Collaborators and Investigators

• Sponsor: Beijing Friendship Hospital
• Investigators: Principal Investigator: zhao Wang, Beijing Friendship Hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 27, 2025
• Primary Completion (Estimated): June 27, 2026
• Study Completion (Estimated): June 27, 2027

Study Registration Dates

• First Submitted: January 1, 2026
• First Submitted That Met QC Criteria: January 12, 2026
• First Posted (Actual): January 14, 2026

Study Record Updates

• Last Update Posted (Actual): January 14, 2026
• Last Update Submitted That Met QC Criteria: January 12, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Golidocitinib; Hemophagocytic Lymphohistocytosis
• Additional Relevant MeSH Terms: Lymphatic Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Hemic and Lymphatic Diseases; Lymphohistiocytosis, Hemophagocytic
• Other Study ID Numbers: Golidocitinib(GO),HLH

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No