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

NCT07413835 | Recruiting

• 1 other identifier: HN2302-N04
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Refractory Myasthenia Gravis

Keywords

MG; HN2302

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events

  Proportion and severity of adverse events (AEs)

  Up to 3 months

Secondary Outcomes (9)

• 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 15-item quality of life (MG-QOL15r) score

  Up to 12 months

• Percentage of patients with symptom changes after treatment

  Up to 12 months

Study Arms (1)

HN2302 treatment group

EXPERIMENTAL

Started at a lower dose level

Drug: HN2302 Injection

Interventions

HN2302 Injection DRUG

Patients will be administrated with specified dose on specified days at a lower dose level and escalated to safe and effective dose levels.

Also known as: in vivo CAR-T

HN2302 treatment group

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• The Affiliated Hospital of Xuzhou Medical University: lead
• Shenzhen MagicRNA Biotechnology Co., Ltd: collaborator

Study Sites (1)

The Affiliated Hospital of Xuzhou Medical University

Xuzhou, Jiangsu, 221000, China

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Guiyun Cui

  The Affiliated Hospital of Xuzhou Medical University

  PRINCIPAL INVESTIGATOR

• Yong Zhang

  The Affiliated Hospital of Xuzhou Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yong Zhang

CONTACT

86-0516-85802193; zy20037416@163.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2026
• First Posted: February 17, 2026
• Study Start: March 17, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: March 18, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)