Efficacy and Safety of HN2301 in Refractory Myasthenia Gravis(MG)

NCT06965309 | Terminated

• 1 other identifier: HN2301-002
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This is an investigator-initiated trial designed to evaluate the safety, and efficacy of HN2301 in refractory myasthenia gravis

Conditions

Refractory Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• Adverse events related to HN2301

  Incidence and severity of AEs associated with HN2301 as assessed by CTCAE v5.

  3 months

Secondary Outcomes (8)

• vivo CAR T cell production

  Day-28 to14 days

• B cell ratio and counts in peripheral blood

  Day-28 to12 months

• Change from baseline of MG-ADL score after HN2301 administration.

  Day-28 to12 months

• Change from baseline of MGC score after HN2301 administration.

  Day-28 to12 months

• Change from baseline of QMG score after HN2301 administration.

  Day-28 to12 months

Study Arms (1)

HN2301 treatment group

EXPERIMENTAL

Specified dose level

Drug: HN2301 injection

Interventions

HN2301 injection DRUG

Description: Three dose groups were set up, starting from the low dose group and climbing to explore the safe and effective dose.

Also known as: in vivo cart

HN2301 treatment group

Eligibility Criteria

• Age: 18 Years - 80 Years
• 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 (Myasthenia Gravis Activities of Daily Living) score ≥6, with ocular-related score \<50%;
• Poor response and/or lack of effective treatment under standard therapies, defined as no improvement or worsening of symptoms despite stable treatment with corticosteroids, immunosuppressants (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporin A, cyclophosphamide), or rituximab;
• Expected survival time greater than 12 weeks;
• Adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function；Bone marrow function: ANC ≥1.5×10⁹/L, ALC ≥0.8×10⁹/L, Hb ≥80g/L; transfusions or growth factors must not be used within 7 days prior to screening to meet these criteria.Coagulation function: INR or APTT ≤1.5×ULN.
• Cardiac function: left ventricular ejection fraction (LVEF) ≥40% assessed by echocardiogram (ECHO).
• Pulmonary function: CTCAE grade ≤1 dyspnea and room air SpO₂ ≥92% (measured by pulse oximetry).
• Liver function: ALT and AST ≤2.5×ULN; total bilirubin \<2.0 mg/dL (or \<3.0 mg/dL for subjects with Gilbert's syndrome).
• Renal function: calculated creatinine clearance (Cockcroft-Gault) ≥50 mL/min without hydration assistance.

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 upper 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

• Shenzhen MagicRNA Biotechnology Co., Ltd: lead
• Shanghai General Hospital (Songjiang Branch): collaborator

Study Sites (1)

Shanghai General Hospital (Songjiang Branch) of Shanghai Jiao Tong University School of Medicine

Shanghai, 201600, 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

• Ailian Du

  Shanghai Songshan Hospital

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: April 29, 2025
• First Posted: May 11, 2025
• Study Start: January 19, 2026
• Primary Completion: February 11, 2026
• Study Completion: February 11, 2026
• Last Updated: February 12, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)