Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic LymphohistiocytosisPhase 3NCT07208058
A Phase III Clinical Study to Evaluate the Efficacy, Safety, and Tolerability of Plonmarlimab in Subjects With Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis (Also Known as Macrophage Activation Syndrome [MAS])
A Phase 3 study testing Plonmarlimab in people with Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis, run by TJ Biopharma Co., Ltd. at 18 sites in China.
Trial summary
A study designed to evaluate the efficacy, safety, immunogenicity, PK, and PD characteristics of Plonmarlimab in patients with relapsed/refractory rheumatic and immunologic disease-associated MAS, and to explore biomarkers related to the efficacy of Plonmarlimab. Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis
- 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
- 30 participants
- Where
- 18 sitesChina
- Study dates
- 21 Nov 2025to 30 Nov 2027
- Phase
- Phase 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 16 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age: 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 a rheumatic and immunological disease.
- Diagnosed with haemophagocytic lymphohistiocytosis (HLH) according to the HLH-2004 diagnostic criteria (excluding molecular diagnosis)
- No response to/or dependence on/intolerance to/or worsening during high-dose corticosteroid therapy. High-dose corticosteroid therapy: at least 1.5-2.0 mg/kg/d of prednisone or its equivalent dose for 3 consecutive days, including methylprednisolone pulse therapy (15-30 mg/kg/d, maximum dose 1 g/d, for 3-5 days).
Who cannot join
- Confirmed or suspected primary haemophagocytic lymphohistiocytosis (pHLH).
- HLH induced by infection (including but not limited to EBV-HLH) or treatment (including but not limited to drugs such as CAR-T cells, TCEs, ADCs).
- History of other active neoplasm malignant within 5 years prior to screening, with the exception of successfully treated cutaneous basal cell or squamous cell carcinoma, or localised neoplasms that have been adequately treated with curative intent, including but not limited to, uterine carcinoma in situ, breast cancer in situ, thyroid cancer, etc.; asymptomatic, localised prostate cancer confirmed to have no metastasis and not requiring treatment, etc. Prior to receiving the investigational drug, an assessment by an oncology specialist is required to clarify the current status of the neoplasm malignant and to rule out the possibility of HLH secondary to the neoplasm malignant.
- History of allergy to any component of the investigational drug.
- Lung disorder: including but not limited to asthma, chronic obstructive pulmonary disease, 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 the investigator's comprehensive assessment concludes that the subject has a pre-existing lung disease that significantly affects pulmonary function and is unsuitable for participation in this clinical study.
- Cardiovascular disorder: history of acute myocardial infarction or unstable angina pectoris, severe arrhythmia (multifocal frequent premature ventricular contractions, ventricular tachycardia, ventricular fibrillation), etc., within the last 6 months; New York Heart Association (NYHA) functional class III-IV.
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- Infection: Presence of an infection deemed uncontrollable by the investigator during the screening period [including but not limited to tuberculosis, active syphilis infection, viral infection (EBV, CMV, COVID-19, active hepatitis B, active hepatitis C, human immunodeficiency virus), other bacterial infections (including but not limited to atypical mycobacteria, Shigella, Salmonella, Campylobacter, etc.)].
- Abnormal renal function: creatinine (Cr) or urea/blood urea nitrogen (BUN) test value >1.5 times the upper limit of normal (ULN); or eGFR <60 mL/min during the screening period, calculated using the MDRD formula: eGFR [mL/(min × 1.73 m2)] = 186 × serum creatinine (mg/dL)-1.154 × age (years)-0.203 × (0.742 if female) × 1.233.
- Haematological diseases: Subjects with a past or current history of haematological diseases (including but not limited to, myelofibrosis, aplastic anaemia, leukaemia, lymphoma, etc.).
- Surgery or other conditions: Planned surgery or any other medical history, laboratory test abnormal, or other condition that, in the investigator's judgment, makes the subject unsuitable for participation in this study.
- Transplant: History of major organ transplant (e.g., heart, lung, kidney, liver) or haematopoietic stem cell/bone marrow transplant.
- Other diseases: Subject currently has a clinically significant and clinically unstable or inadequately controlled acute or chronic disease (e.g., acute pneumonia, pulmonary arterial hypertension, diabetic ketoacidosis, pancreatitis acute, etc.).
- Pregnant or lactating women.
- Participation in any clinical trial (including investigational vaccines) treatment or use of an invasive investigational medical device within 3 months prior to enrolment, or currently enrolled in an interventional study.
- Received live vaccine within 30 days prior to screening.
- Evidence of alcohol abuse within 3 months prior to screening or current abuse, identified through medical history inquiry.
- Other conditions that, in the investigator's opinion, make the subject unsuitable for participation in this clinical study. For example, any condition that may increase the risks associated with the subject's participation in the study, or may interfere with the evaluation of the investigational drug or confound the interpretation of the study results.
Trial locations and contacts
18 sites in 1 country. Contact the study team or a site near you to ask about taking part.
18 sites in 1 country
China18 sites, 16 recruiting
- Recruiting
Beijing Friendship Hospital, Capital Medical University
Beijing, Beijing Municipality
Zhao Wang, wangzhao@ccmu.edu.cn
- Recruiting
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality
Qian Wang
- Recruiting
Peking University People's Hospital
Beijing, Beijing Municipality
Yuan Jia
- Recruiting
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Guangzhou, Guangdong
Lie Dai
- Recruiting
Sir Run Run Shaw Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang
Yongmei Han
- Recruiting
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang
De Zhou
- Recruiting
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi
Fei Li
- Recruiting
The Second Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi
Cundong Mi
- Recruiting
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality
Li Sun
- Recruiting
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality
Yu Xue
- Recruiting
Renji Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality
Qiong Fu
- Withdrawn
The First Affiliated Hospital of China Medical University
Shenyang, Liaoning
- Recruiting
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei
Huifang Guo
- Withdrawn
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality
- Recruiting
The First Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang
Li Sun
- Recruiting
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei
Rong Du
- Recruiting
Henan Children's Hospital Zhengzhou Children's Hospital
Zhengzhou, Henan
Caifeng Li
- Recruiting
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan
Shengyun Liu
About this study
Official title: An Open-label, Single-arm, Multicenter, Phase III Clinical Study to Evaluate the Efficacy, Safety, and Tolerability of Plonmarlimab in Subjects With Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis (Also Known as Macrophage Activation Syndrome [MAS])
A study designed to evaluate the efficacy, safety, immunogenicity, PK, and PD characteristics of Plonmarlimab in patients with relapsed/refractory rheumatic and immunologic disease-associated MAS, and to explore biomarkers related to the efficacy of Plonmarlimab.
- Conditions
- Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis
- Being tested
- Plonmarlimab
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlonmarlimabDrug
Subjects will receive Plonmarlimab 10 mg/kg by intravenous injection once weekly from Week 1 to Week 3, and Plonmarlimab 6 mg/kg by intravenous injection once weekly from Week 4 to Week 8 (10 mg/kg QW×3 + 6 mg/kg QW×5).
| 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 posted6 Oct 2025
- Study start21 Nov 2025
- Primary completion20 Dec 2026 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic LymphohistiocytosisStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
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
- NCT07208058
- Sponsor's protocol ID
- THZ0103-MAS301
- Status last verified
- Oct 2026
- First submitted
- 27 Sep 2025
- Last updated
- 15 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Multicenter, Phase III Clinical Study to Evaluate the Efficacy, Safety, and Tolerability of Plonmarlimab in Subjects With Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis (Also Known as Macrophage Activation Syndrome [MAS])
- ClinicalTrials.gov ID
- NCT07208058
- Acronym / sponsor ID
- THZ0103-MAS301
- Conditions
- Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- TJ Biopharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 21 Nov 2025 (actual)
- Primary completion
- 20 Dec 2026 (estimated)
- Study completion
- 30 Nov 2027 (estimated)
- First posted
- 6 Oct 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.
A study designed to evaluate the efficacy, safety, immunogenicity, PK, and PD characteristics of Plonmarlimab in patients with relapsed/refractory rheumatic and immunologic disease-associated 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: 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 a rheumatic and immunological disease.
- Diagnosed with haemophagocytic lymphohistiocytosis (HLH) according to the HLH-2004 diagnostic criteria (excluding molecular diagnosis)
- No response to/or dependence on/intolerance to/or worsening during high-dose corticosteroid therapy. High-dose corticosteroid therapy: at least 1.5-2.0 mg/kg/d of prednisone or its equivalent dose for 3 consecutive days, including methylprednisolone pulse therapy (15-30 mg/kg/d, maximum dose 1 g/d, for 3-5 days).
Key exclusion criteria (17 listed)
- Confirmed or suspected primary haemophagocytic lymphohistiocytosis (pHLH).
- HLH induced by infection (including but not limited to EBV-HLH) or treatment (including but not limited to drugs such as CAR-T cells, TCEs, ADCs).
- History of other active neoplasm malignant within 5 years prior to screening, with the exception of successfully treated cutaneous basal cell or squamous cell carcinoma, or localised neoplasms that have been adequately treated with curative intent, including but not limited to, uterine carcinoma in situ, breast cancer in situ, thyroid cancer, etc.; asymptomatic, localised prostate cancer confirmed to have no metastasis and not requiring treatment, etc. Prior to receiving the investigational drug, an assessment by an oncology specialist is required to clarify the current status of the neoplasm malignant and to rule out the possibility of HLH secondary to the neoplasm malignant.
- History of allergy to any component of the investigational drug.
- Lung disorder: including but not limited to asthma, chronic obstructive pulmonary disease, 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 the investigator's comprehensive assessment concludes that the subject has a pre-existing lung disease that significantly affects pulmonary function and is unsuitable for participation in this clinical study.
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 will receive Plonmarlimab 10 mg/kg by intravenous injection once weekly from Week 1 to Week 3, and Plonmarlimab 6 mg/kg by intravenous injection once weekly from Week 4 to Week 8 (10 mg/kg QW×3 + 6 mg/kg QW×5). | Drug | Plonmarlimab |
Primary outcome
Overall response rate (ORR)
Time frame: at the end of Week 8
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 15 Oct 2026.
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.
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Plonmarlimab in people with Relapsed/Refractory Rheumatic and Immunologic Disease-associated Haemophagocytic Lymphohistiocytosis, run by TJ Biopharma Co., Ltd. at 18 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 17 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 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 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 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 Biwei Zeng (86 13540060494, biwei.zeng@tjbio.com), or a site near the patient. 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 15 Oct 2026; AzTrials last refreshed its copy on 31 Oct 2026.