NCT07556120Not yet recruitingPhase 1
An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis
Shenzhen MagicRNA Biotechnology Co., Ltd1 site in 1 country6 target enrollmentStarted: December 31, 2026Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Not yet recruiting
- Sponsor
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Incidence of treatment-emergent adverse events (TEAEs)
Study Overview
Brief Summary
This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.
Detailed Description
This study consists of a screening period of up to 4 weeks, a treatment period, and a follow-up period of 1 year.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 80 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •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;
- •Patients combined with other malignant tumors;
- •Any condition that, in the investigator's opinion, may increase the patient's risk or interfere with study assessments.
Arms & Interventions
HN2301 treatment group
Experimental
Participants will receive HN2301 Injection at the specified dose level and on the specified study days.
Intervention: HN2301 injection (Drug)
Outcomes
Primary Outcomes
Incidence of treatment-emergent adverse events (TEAEs)
Time Frame: Up to 3 months
Incidence, nature, and severity of treatment-emergent adverse events, assessed according to the study protocol and applicable toxicity grading criteria.
Secondary Outcomes
- in vivo CAR T cell production(Up to14 days)
- Absolute B-cell count in peripheral blood(Up to 12 months)
- 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 revised Myasthenia Gravis Quality of Life-15 score (MG-QOL15r)(Up to 12 months)
- Changes in acetylcholine receptor (AchR) antibody levels after treatment(Up to 12 months)
Investigators
Study Sites (1)
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