Efficacy and Safety of HN2302 in Refractory Myasthenia Gravis(MG)

A Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HN2302 in Patients With Refractory Myasthenia Gravis

This is an open label, single arm study, to evaluate the safety , tolerability and preliminary efficacy of HN2302 for refractory myasthenia gravis.

Study Overview

• Status: Recruiting
• Conditions: Refractory Myasthenia Gravis
• Intervention / Treatment: Drug: HN2302 Injection

Detailed Description

The study will consist of an up to 4-week Screening Period, Treatment Period and one year Follow-up Period.

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Yong Zhang; Phone Number: 86-0516-85802193; Email: zy20037416@163.com

Study Locations

• China
  • Jiangsu
    • Xuzhou, Jiangsu, China, 221000
      • Recruiting
      • The Affiliated Hospital of Xuzhou Medical University
      • Contact: Yong Zhang; Phone Number: 86-0516-85802193; Email: zy20037416@163.com

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;
• 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: HN2302 treatment group; Started at a lower dose level	Drug: HN2302 Injection; Patients will be administrated with specified dose on specified days at a lower dose level and escalated to safe and effective dose levels.; 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	Proportion and severity of adverse events (AEs)	Up to 3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Up to 12 months
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).	Up to 12 months
Percentage of patients with symptom changes after treatment	Proportion of patients without symptom worsening or relapse	Up to 12 months
in vivo CAR T cell production	Assessment of CAR T production (CAR expression ratio in T cells) in the peripheral blood of MG patients by flow cytometry (FACS)	Day-28 to14 days
B cell ratio and counts in peripheral blood	Assessment of B cell ratio and counts (B cell counts per μl peripheral blood) and B cell subsets(naive B cell, memory B cell) by flow cytometry (FACS) in peripheral blood	Up to 12 months
Dynamic changes in cytokine levels after treatment	Differences in cytokine post-administration vs. baseline	Up to 12 months
Changes in acetylcholine receptor (AchR) antibody levels after treatment	Differences in AchR antibody post-administration vs. baseline	Up to 12 months

Collaborators and Investigators

• Sponsor: The Affiliated Hospital of Xuzhou Medical University
• Collaborators: Shenzhen MagicRNA Biotechnology Co., Ltd
• Investigators: Principal Investigator: Guiyun Cui, The Affiliated Hospital of Xuzhou Medical University; Principal Investigator: Yong Zhang, The Affiliated Hospital of Xuzhou Medical University

Study record dates

Study Major Dates

• Study Start (Actual): March 17, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: February 3, 2026
• First Submitted That Met QC Criteria: February 13, 2026
• First Posted (Actual): February 17, 2026

Study Record Updates

• Last Update Posted (Actual): March 18, 2026
• Last Update Submitted That Met QC Criteria: March 16, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: MG; HN2302
• 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: HN2302-N04

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