This is an open label, single arm study, to evaluate the safety , tolerability and preliminary efficacy of HN2302 for refractory myasthenia gravis.
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%;
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;
Single group
HN2302 Injection
DrugPatients will be administrated with specified dose on specified days at a lower dose level and escalated to safe and effective dose levels.
Incidence of Treatment-Emergent Adverse Events
Proportion and severity of adverse events (AEs)
Changes from baseline in Myasthenia Gravis Activities of Daily Living（MG-ADL） score
Proportion of patients ≥2 points. A total score can fall between 0 and 24, with a higher score representing a more significant degree of disease activity
Changes from baseline in Myasthenia Gravis Composite （MGC） score
Proportion of patients ≥3-point reduction.The total score is 50 points. The higher the score, the more severe the condition is indicated.
Changes from baseline in Quantitative Myasthenia Gravis (QMG) score
Proportion of patients ≥3-point reduction. To assess the muscle strength and endurance of the affected muscles in patients with myasthenia gravis, thereby reflecting the severity of the disease.
Changes from baseline in 15-item quality of life (MG-QOL15r) score
To assess important aspects of the patient's experience related to MG, scores each of 15 items 0-2 (max score 30).
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The Affiliated Hospital of Xuzhou Medical University
Lead sponsor
Shenzhen MagicRNA Biotechnology Co., Ltd
Collaborator
