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

NCT07556120 | Not Yet Recruiting Phase 1

• 1 other identifier: HN2301-02
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Generalized Myasthenia Gravis

Keywords

MG; in vivo CART

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• 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]

Study Arms (1)

HN2301 treatment group

EXPERIMENTAL

Participants will receive HN2301 Injection at the specified dose level and on the specified study days.

Drug: HN2301 injection

Interventions

HN2301 injection DRUG

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.

Also known as: in vivo CAR-T

HN2301 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;
• 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.

Sponsors & Collaborators

• Shenzhen MagicRNA Biotechnology Co., Ltd: lead
• The First Affiliated Hospital of University of Science and Technology of China: collaborator

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China

Hefei, Anhui, 230036, China

Location

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

• 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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wen Xu

CONTACT

+86055162284920; Xuwen6779@ustc.edu.cn

Zexiu Xiao

CONTACT

+86075527109036; xiaozexiu@magicrna.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2026
• First Posted: April 29, 2026
• Study Start: May 5, 2026
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)