This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized 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
HN2301 injection
DrugDosing will begin at a lower dose level and may be escalated to dose levels considered safe and potentially effective according to the study protocol.
Incidence of treatment-emergent adverse events (TEAEs)
Incidence, nature, and severity of treatment-emergent adverse events, assessed according to the study protocol and applicable toxicity grading criteria.
in vivo CAR T cell production
Assessment of in vivo CAR-T cell production, defined by the proportion of CAR-expressing T cells in peripheral blood as measured by flow cytometry.
Absolute B-cell count in peripheral blood
Assessment of peripheral blood absolute B-cell count (cells/μL)， including naive B cells and memory B cells, by flow cytometry.
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.
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Shenzhen MagicRNA Biotechnology Co., Ltd
Lead sponsor
The First Affiliated Hospital of University of Science and Technology of China
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
