NCT07413835招募中不适用
A Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HN2302 in Patients With Refractory Myasthenia Gravis
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Incidence of Treatment-Emergent Adverse Events
研究概览
简要总结
This is an open label, single arm study, to evaluate the safety , tolerability and preliminary efficacy of HN2302 for refractory myasthenia gravis.
详细描述
The study will consist of an up to 4-week Screening Period, Treatment Period and one year Follow-up Period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: 18-80 years, no gender restriction;
- •Confirmed diagnosis of generalized myasthenia gravis (MG) with positive AchR or MuSK antibodies, meeting at least one of the following conditions：(1) Repetitive nerve stimulation suggesting neuromuscular transmission defect; (2) Positive response to neostigmine test; (3) Clinically judged improvement of --MG symptoms after oral cholinesterase inhibitor therapy;
- •Clinical classification of MG according to MGFA types IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa, IVb);
- •Baseline MG-ADL score ≥6, ocular-related score <50%;
- •Poor response and/or lack of efficacy under standard therapies;
- •Minimum life expectancy > 12 weeks;
- •Adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function.
排除标准
- •Subjects positive for hepatitis B surface antigen (HBsAg) and/or hepatitis B core antibody (HBcAb) with detectable or quantifiable HBV DNA, positive for hepatitis C antibody (HCV Ab) with detectable or quantifiable HCV RNA, positive for HIV antibody, positive CMV DNA, or CMV DNA above the lower limit of detection; positive for syphilis antigen or antibody;
- •Presence of other uncontrolled active infections;
- •History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation;
- •Pregnant or breastfeeding women;
- •Receipt of any mRNA-LNP products or other LNP-based drugs within the past two years;
- •History of any of the following cardiovascular conditions within 6 months prior to screening: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant cardiac disease;
- •History of ≥Grade 2 bleeding events within 30 days prior to screening, or requiring long-term continuous anticoagulation therapy (e.g., warfarin, low molecular weight heparin, Xa factor inhibitors);
- •History of live vaccination within 30 days prior to screening;
- •Severe central nervous system diseases or pathological changes, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, seizures/convulsions, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disorders, organic brain syndromes, or psychosis;
- •History of asthma or severe allergies;
- •Any condition that, in the investigator's opinion, may increase the patient's risk or interfere with study assessments.
研究组 & 干预措施
HN2302 treatment group
Experimental
Started at a lower dose level
干预措施: HN2302 Injection (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events
时间窗: Up to 3 months
Proportion and severity of adverse events (AEs)
次要结局
- Changes from baseline in Myasthenia Gravis Activities of Daily Living（MG-ADL） score(Up to 12 months)
- Changes from baseline in Myasthenia Gravis Composite （MGC） score(Up to 12 months)
- Changes from baseline in Quantitative Myasthenia Gravis (QMG) score(Up to 12 months)
- Changes from baseline in 15-item quality of life (MG-QOL15r) score(Up to 12 months)
- Percentage of patients with symptom changes after treatment(Up to 12 months)
- in vivo CAR T cell production(Day-28 to14 days)
- B cell ratio and counts in peripheral blood(Up to 12 months)
- Dynamic changes in cytokine levels after treatment(Up to 12 months)
- Changes in acetylcholine receptor (AchR) antibody levels after treatment(Up to 12 months)
研究者
研究点 (1)
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