Golidocitinib (GO) treatment for hemophagocytic lymphohistiocytosis

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

This trial will test whether the oral drug golidocitinib (GO) can safely treat people aged 14–70 with newly diagnosed or relapsed/refractory hemophagocytic lymphohistiocytosis who are not candidates for stem cell transplant.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 28 (estimated)
• Ages: 14 Years to 70 Years
• Sex: All
• Sponsor: Beijing Friendship Hospital (Academic / other)
• Drugs / interventions: Golidocitinib
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07339345 on ClinicalTrials.gov

What this trial studies

This is a prospective, single-arm Phase 2/3 trial of golidocitinib (GO) given alone to patients with primary or secondary HLH who meet molecular criteria or at least five of the HLH‑2004 diagnostic criteria. The protocol enrolls patients aged 14–70 who are currently unsuitable for allogeneic hematopoietic stem cell transplantation and who meet predefined organ‑function and infection‑screening limits. Efficacy will be measured by clinical and laboratory response rates and survival endpoints, while safety will be monitored by adverse events and cardiac and infectious screening. Detailed dosing and duration are not provided in the registry entry, and participants are treated at the single site in Beijing.

Who should consider this trial

Good fit: Ideal candidates are people 14–70 years old with newly diagnosed or relapsed/refractory HLH who meet molecular or HLH‑2004 diagnostic criteria and who are judged unsuitable for allo‑HSCT.

Not a fit: Patients who are eligible for curative allogeneic stem cell transplant, have uncontrolled serious infections, or have excluded viral/hepatic/cardiac findings per the protocol may not benefit from this trial.

Why it matters

Potential benefit: If successful, golidocitinib could provide an effective medical option to control HLH symptoms and improve survival for patients who cannot undergo transplant.

How similar studies have performed: Similar strategies using JAK‑pathway inhibitors (for example ruxolitinib) have shown encouraging results in small series and early-phase work, so this approach builds on prior, limited clinical experience.

Eligibility criteria

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.

Where this trial is running

Beijing, Beijing Municipality

• beijing Friendship Hospital, Capital Medical University — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: zhao Wang — Beijing Friendship Hospital
• Study coordinator: jingshi Wang, M.M.

Conditions Lymphohistiocytosis, Hemophagocytic; Hemophagocytic Lymphohistocytosis; Golidocitinib