NCT06965309终止不适用
Dose-escalation Study to Assess the Safety, Tolerability, and Preliminary Efficacy of HN2301 in Patients With Refractory Myasthenia Gravis (MG)
Shenzhen MagicRNA Biotechnology Co., Ltd1 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2026年1月19日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Adverse events related to HN2301
研究概览
简要总结
This is an investigator-initiated trial designed to evaluate the safety, and efficacy of HN2301 in refractory myasthenia gravis
详细描述
This study is a prospective exploratory clinical trial in subjects with refractory myasthenia gravis. The objective is to evaluate the safety, initial efficacy of HN2301injection in refractory myasthenia gravis.
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 (Myasthenia Gravis Activities of Daily Living) score ≥6, with ocular-related score <50%;
- •Poor response and/or lack of effective treatment under standard therapies, defined as no improvement or worsening of symptoms despite stable treatment with corticosteroids, immunosuppressants (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporin A, cyclophosphamide), or rituximab;
- •Expected survival time greater than 12 weeks;
- •Adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function；Bone marrow function: ANC ≥1.5×10⁹/L, ALC ≥0.8×10⁹/L, Hb ≥80g/L; transfusions or growth factors must not be used within 7 days prior to screening to meet these criteria.Coagulation function: INR or APTT ≤1.5×ULN.
- •Cardiac function: left ventricular ejection fraction (LVEF) ≥40% assessed by echocardiogram (ECHO).
- •Pulmonary function: CTCAE grade ≤1 dyspnea and room air SpO₂ ≥92% (measured by pulse oximetry).
- •Liver function: ALT and AST ≤2.5×ULN; total bilirubin <2.0 mg/dL (or <3.0 mg/dL for subjects with Gilbert's syndrome).
- •Renal function: calculated creatinine clearance (Cockcroft-Gault) ≥50 mL/min without hydration assistance.
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 upper 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
HN2301 treatment group
Experimental
Specified dose level
干预措施: HN2301 injection (Drug)
结局指标
主要结局
Adverse events related to HN2301
时间窗: 3 months
Incidence and severity of AEs associated with HN2301 as assessed by CTCAE v5.
次要结局
- vivo CAR T cell production(Day-28 to14 days)
- B cell ratio and counts in peripheral blood(Day-28 to12 months)
- Change from baseline of MG-ADL score after HN2301 administration.(Day-28 to12 months)
- Change from baseline of MGC score after HN2301 administration.(Day-28 to12 months)
- Change from baseline of QMG score after HN2301 administration.(Day-28 to12 months)
- Change from baseline of MG-QOL15r score after HN2301 administration.(Day-28 to12 months)
- Change of patients after HN2301 administration.(Day-28 to12 months)
- Time to achieve a ≥2 point reduction in MG-ADL score following HN2301 administration.(14 days-12 months)
研究者
研究点 (1)
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