A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetic Characteristics, and Immu… (NCT07034209) | Clinical Trial Compass
CompletedPhase 2
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))
China18 participantsStarted 2023-04-07
Plain-language 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 Plonmarlimab.
Who can participate
Age range
16 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. 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.
. 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).