Hemophagocytic LymphohistiocytosesPhase 2NCT07034209
A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetic Characteristics, and Immunogenicity of Plonmarlimab in Subjects With Rheumatic and Immunological Disease-associated Haemophagocytic Lymphohistiocytosis (HLH) (Also Known as Macrophage Activation Syndrome (MAS))
A Phase 2 study testing Plonmarlimab in people with Hemophagocytic Lymphohistiocytoses, run by TJ Biopharma Co., Ltd. at 6 sites in China.
Trial summary
This study adopts an open-label, single-arm, multicenter design to evaluate the efficacy, safety, tolerability, immunogenicity, and PK characteristics of Plonmarlimab administration in patients with rheumatic and immunological disease-associated HLH (MAS), and to explore biomarkers related to the efficacy of… Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Hemophagocytic Lymphohistiocytoses
- What is being tested?
- Plonmarlimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 6 sitesChina
- Study dates
- 7 Apr 2023to 18 Apr 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 16 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age: This study will include subjects aged 16 to 80 years (inclusive), of any gender.
- The subject is willing to participate in this study and voluntarily signs the informed consent form. For minor subjects aged 16 years (inclusive) to less than 18 years, written informed consent must be signed by both the subject and the subject's legal guardian
- Diagnosed with haemophagocytic lymphohistiocytosis (HLH) according to the HLH-2004 diagnostic criteria (see Appendix 1 for details. HLH-2004 diagnostic criteria)，
- Diagnosed with a rheumatic and immunological disease,including:Systemic juvenile idiopathic arthritis (sJIA);Adult Onset Still's Disease (AOSD);Systemic lupus erythematosus (SLE)
- The subject (including the subject's partner) has no plans for pregnancy from the screening period until 28 days after the last dose and is willing to use contraceptive measures (oral oestrogens, oestrogens, vaginal rings, etc., cannot be used; see Appendix 5. Contraceptive Measures, Definition of Women of Childbearing Potential, and Contraception Requirements for acceptable contraceptive measures).
Who cannot join
- Known pathogenic gene mutation or abnormal perforin expression and CD107a degranulation assay indicating primary haemophagocytic lymphohistiocytosis, or a family history of primary haemophagocytic lymphohistiocytosis.
- Subjects who:
- Are receiving tumour necrosis factor (TNF) antagonists (anti-TNF), interleukin-1 (IL-1) antagonists [anti-IL-1, e.g., canakinumab, anakinra], Janus kinase inhibitors (JAKi), or interleukin-6 (IL-6) antagonists [anti-IL-6, e.g., tocilizumab] at the time of initiating Plonmarlimab treatment;
- Received Etoposide (VP-16) for MAS treatment within 7 days before the first dose;
- Increased the dose or type of non-biologic agents (e.g., immunosuppressants, immunomodulators, antimalarials) for the treatment of rheumatic and immunological diseases within 3 days before the first dose. Unless the investigator determines it is expected to be ineffective and it is discontinued before the first dose. Specific drugs for immunosuppressants, immunomodulators.
- History of allergy to any component of the investigational drug.
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- Lung disorder: Including but not limited to asthma, chronic obstructive pulmonary disease (COPD), interstitial lung disease, alveolar proteinosis, pulmonary granulomatosis, etc., AND abnormal pulmonary function tests: forced vital capacity (FVC) <80% of predicted value, or FEV1/FVC <70%, etc.; or cases where the investigator's comprehensive assessment indicates that the subject has a pre-existing lung disorder significantly affecting pulmonary function and is unsuitable for participation in this clinical study.
- Cardiovascular disorder: History of acute myocardial infarction or unstable angina pectoris, severe arrhythmia (e.g., multifocal frequent premature ventricular contractions, ventricular tachycardia, ventricular fibrillation) within the last 6 months; New York Heart Association (NYHA) functional classification III-IV (see Appendix 6. NYHA Functional Classification).
- History of neoplasm malignant within the past 5 years (whether treated or not), with the exception of successfully treated cutaneous basal cell or squamous cell carcinoma.
- Other diseases: Subjects currently have clinically significant and clinically unstable or uncontrolled acute or chronic disease (e.g., acute pneumonia, pulmonary arterial hypertension, diabetic ketoacidosis, pancreatitis acute, etc.), or planned medical/surgery procedures; or place the subject at undue risk, or affect the subject's ability to voluntarily participate in the study.
- Infection: Subjects with investigator-assessed uncontrolled infection during the screening period.
- Subjects with Mycobacterium tuberculosis infection, including those with latent infection positive by "T-SPOT" or "QuantiFERON" test.
- Positive for any of the following: hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab), treponema pallidum antibody test. If hepatitis B core antibody (HBcAb) test is positive, an additional hepatitis B DNA (HBV-DNA) test will be performed; subjects with HBV-DNA above the lower limit of detection will be excluded.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
China6 sites
Beijing Friendship Hospital, Capital Medical University
Beijing, Beijing Municipality
Beijing Peking Union Medical College Hospital
Beijing
Beijing Peking University People's Hospital
Beijing
The First Affiliated Hospital of Nanjing Medical University
Nanjing
Renji Hospital of Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality
Ruijin Hospital Of Shanghai Jiao tong University School of Medicine
Shanghai
About this study
Official title: An Open-label, Single-arm, Multicenter Clinical Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetic Characteristics, and Immunogenicity of Plonmarlimab in Subjects With Rheumatic and Immunological Disease-associated Haemophagocytic Lymphohistiocytosis (HLH) (Also Known as Macrophage Activation Syndrome (MAS))
This study adopts an open-label, single-arm, multicenter design to evaluate the efficacy, safety, tolerability, immunogenicity, and PK characteristics of Plonmarlimab administration in patients with rheumatic and immunological disease-associated HLH (MAS), and to explore biomarkers related to the efficacy of Plonmarlimab.
- Conditions
- Hemophagocytic Lymphohistiocytoses
- Being tested
- Plonmarlimab
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlonmarlimabDrug
Subjects receive Plonmarlimab 6 mg/kg or 10 mg/kg, administered intravenously, once weekly for 8 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Plonmarlimab | Experimental | Not described Receives: Drug: Plonmarlimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate,ORR
Measured: At the end of Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2025
- Study start7 Apr 2023
- Primary completion18 Apr 2025
- Study completion18 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophagocytic LymphohistiocytosesStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- TJ Biopharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07034209
- Sponsor's protocol ID
- THZ0103-201
- Status last verified
- Jun 2025
- First submitted
- 13 Jun 2025
- Last updated
- 24 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Multicenter Clinical Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetic Characteristics, and Immunogenicity of Plonmarlimab in Subjects With Rheumatic and Immunological Disease-associated Haemophagocytic Lymphohistiocytosis (HLH) (Also Known as Macrophage Activation Syndrome (MAS))
- ClinicalTrials.gov ID
- NCT07034209
- Acronym / sponsor ID
- THZ0103-201
- Conditions
- Hemophagocytic Lymphohistiocytoses
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- TJ Biopharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 7 Apr 2023 (actual)
- Primary completion
- 18 Apr 2025 (actual)
- Study completion
- 18 Apr 2025 (actual)
- First posted
- 24 Jun 2025 (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 adopts an open-label, single-arm, multicenter design to evaluate the efficacy, safety, tolerability, immunogenicity, and PK characteristics of Plonmarlimab administration in patients with rheumatic and immunological disease-associated HLH (MAS), and to explore biomarkers related to the efficacy of Plonmarlimab.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Plonmarlimab | Experimental | Drug: Plonmarlimab |
Who may qualify
- Ages
- 16 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Age: This study will include subjects aged 16 to 80 years (inclusive), of any gender.
- The subject is willing to participate in this study and voluntarily signs the informed consent form. For minor subjects aged 16 years (inclusive) to less than 18 years, written informed consent must be signed by both the subject and the subject's legal guardian
- Diagnosed with haemophagocytic lymphohistiocytosis (HLH) according to the HLH-2004 diagnostic criteria (see Appendix 1 for details. HLH-2004 diagnostic criteria)，
- Diagnosed with a rheumatic and immunological disease,including:Systemic juvenile idiopathic arthritis (sJIA);Adult Onset Still's Disease (AOSD);Systemic lupus erythematosus (SLE)
- The subject (including the subject's partner) has no plans for pregnancy from the screening period until 28 days after the last dose and is willing to use contraceptive measures (oral oestrogens, oestrogens, vaginal rings, etc., cannot be used; see Appendix 5. Contraceptive Measures, Definition of Women of Childbearing Potential, and Contraception Requirements for acceptable contraceptive measures).
Key exclusion criteria (13 listed)
- Known pathogenic gene mutation or abnormal perforin expression and CD107a degranulation assay indicating primary haemophagocytic lymphohistiocytosis, or a family history of primary haemophagocytic lymphohistiocytosis.
- Subjects who:
- History of allergy to any component of the investigational drug.
- Lung disorder: Including but not limited to asthma, chronic obstructive pulmonary disease (COPD), interstitial lung disease, alveolar proteinosis, pulmonary granulomatosis, etc., AND abnormal pulmonary function tests: forced vital capacity (FVC) <80% of predicted value, or FEV1/FVC <70%, etc.; or cases where the investigator's comprehensive assessment indicates that the subject has a pre-existing lung disorder significantly affecting pulmonary function and is unsuitable for participation in this clinical study.
- Cardiovascular disorder: History of acute myocardial infarction or unstable angina pectoris, severe arrhythmia (e.g., multifocal frequent premature ventricular contractions, ventricular tachycardia, ventricular fibrillation) within the last 6 months; New York Heart Association (NYHA) functional classification III-IV (see Appendix 6. NYHA Functional Classification).
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 |
|---|---|---|
| PlonmarlimabSubjects receive Plonmarlimab 6 mg/kg or 10 mg/kg, administered intravenously, once weekly for 8 weeks. | Drug | Plonmarlimab |
Primary outcome
Overall Response Rate,ORR
Time frame: At the end of Week 8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2025.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Plonmarlimab in people with Hemophagocytic Lymphohistiocytoses, run by TJ Biopharma Co., Ltd. at 6 sites 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 5 inclusion and 13 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: finished, as of the sponsor's last update on 24 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate,ORR (time frame: At the end of Week 8). 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 24 Jun 2025; AzTrials last refreshed its copy on 6 Nov 2026.