Lymphohistiocytosis, HemophagocyticPhase 2/3NCT07339345
Prospective Single-Arm Clinical Trial of GO Regimen for HLH.
A Phase 2/3 study testing Golidocitinib in people with Lymphohistiocytosis, Hemophagocytic, run by Beijing Friendship Hospital at 1 site in China.
Trial summary
This study aims to investigate the efficacy and safety of Golidocitinib(GO) monotherapy in the treatment of hemophagocytic lymphohistiocytosis. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Lymphohistiocytosis, Hemophagocytic
- What is being tested?
- Golidocitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteChina
- Study dates
- 27 Jun 2025to 27 Jun 2027
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
beijing Friendship Hospital, Capital Medical University
Beijing, Beijing Municipality, 100050
jingshi Wang, M.M., 86-13520280731, wangjingshi987@126.com
About this study
Official title: 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.
- Conditions
- Lymphohistiocytosis, Hemophagocytic
- Being tested
- Golidocitinib
- Keywords
- Hemophagocytic Lymphohistocytosis, Golidocitinib
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GolidocitinibDrug
Administer the GO regimen to HLH patients who meet the inclusion criteria, specifically Golicitinib 150 mg once daily.
| Study group | Role | What they receive |
|---|---|---|
| HLH patients | Experimental | Initial diagnosis or presence of refractory/relapsed HLH disease or confirmed diagnosis of HLH. Receives: Drug: Golidocitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
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.
Measured: 30 days after the last dose is administered.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2026
- Study start27 Jun 2025
- Primary completion27 Jun 2026 (estimated)
- Study completion27 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphohistiocytosis, HemophagocyticStudies of the same condition, open and under way.
- More trials run by Beijing Friendship HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Friendship Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhao Wang, Beijing Friendship Hospital
- Investigators
- zhao Wang, Principal investigator, Beijing Friendship Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07339345
- Sponsor's protocol ID
- Golidocitinib(GO),HLH
- Status last verified
- Jan 2026
- First submitted
- 1 Jan 2026
- Last updated
- 14 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Single-Arm Clinical Trial of Golidocitinib Monotherapy for Hemophagocytic Lymphohistiocytosis.
- ClinicalTrials.gov ID
- NCT07339345
- Acronym / sponsor ID
- Golidocitinib(GO),HLH
- Conditions
- Lymphohistiocytosis, Hemophagocytic
- Keywords
- Hemophagocytic Lymphohistocytosis, Golidocitinib
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Study start
- 27 Jun 2025 (actual)
- Primary completion
- 27 Jun 2026 (estimated)
- Study completion
- 27 Jun 2027 (estimated)
- First posted
- 14 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to investigate the efficacy and safety of Golidocitinib(GO) monotherapy in the treatment of hemophagocytic lymphohistiocytosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HLH patients | Experimental | Drug: GolidocitinibInitial diagnosis or presence of refractory/relapsed HLH disease or confirmed diagnosis of HLH. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GolidocitinibAdminister the GO regimen to HLH patients who meet the inclusion criteria, specifically Golicitinib 150 mg once daily. | Drug | HLH patients |
Primary outcome
Evaluation of treatment response
Time frame: Weeks 2, 4, 6, and 8 after the start of treatment
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.
1 secondary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 30 days after the last dose is administered.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
zhao Wang principal investigator
Beijing Friendship Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Golidocitinib in people with Lymphohistiocytosis, Hemophagocytic, run by Beijing Friendship Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact jingshi Wang, M.M. (86-13520280731, wangjingshi987@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of treatment response (time frame: Weeks 2, 4, 6, and 8 after the start of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2026.