Efficacy and Safety of HN2301 in Patients With Generalized Myasthenia Gravis (MG)

An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis

This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.

Study Overview

• Status: Not yet recruiting
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: HN2301 injection

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 Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Zexiu Xiao; Phone Number: +86075527109036; Email: xiaozexiu@magicrna.com
• Study Contact Backup: Name: Wen Xu; Phone Number: +86055162284920; Email: Xuwen6779@ustc.edu.cn

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230036
      • The First Affiliated Hospital of University of Science and Technology of China
      • Contact: Wen Xu; Phone Number: +86055162284920; Email: Xuwen6779@ustc.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HN2301 treatment group; Participants will receive HN2301 Injection at the specified dose level and on the specified study days.	Drug: HN2301 injection; Dosing will begin at a lower dose level and may be escalated to dose levels considered safe and potentially effective according to the study protocol.; Other Names: in vivo CAR-T

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Up to 3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in acetylcholine receptor (AchR) antibody levels after treatment	Differences in AchR antibody post-administration vs. baseline	Up to 12 months
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.	Up to14 days
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.	Up to 12 months
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.	Up to 12 months]
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.	Up to 12 months
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，including 8 items, each scored on a scale of 0 to 3, with 3 indicating the most severe degree.	Up to 12 months]
Changes from baseline in revised Myasthenia Gravis Quality of Life-15 score (MG-QOL15r)	To assess important aspects of the patient's experience related to MG, scores each of 15 items 0-2 (max score 30).The higher the score, the worse the patient's quality of life.	Up to 12 months

Collaborators and Investigators

• Sponsor: Shenzhen MagicRNA Biotechnology Co., Ltd
• Collaborators: The First Affiliated Hospital of University of Science and Technology of China
• Investigators: Principal Investigator: Wen Xu, The First Affiliated Hospital of University of Science and Technology of China; Principal Investigator: Yan Jiang, The First Affiliated Hospital of University of Science and Technology of China

Study record dates

Study Major Dates

• Study Start (Estimated): May 5, 2026
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: April 16, 2026
• First Submitted That Met QC Criteria: April 26, 2026
• First Posted (Actual): April 29, 2026

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 26, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: MG; in vivo CART
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis
• Other Study ID Numbers: HN2301-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No