HN2302 for adults with refractory generalized myasthenia gravis

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

This open-label, single-arm trial will try HN2302 injections in adults with refractory generalized myasthenia gravis to see if the treatment is safe and reduces symptoms.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: The Affiliated Hospital of Xuzhou Medical University (other)
• Locations: 1 site (Xuzhou, Jiangsu)
• Trial ID: NCT07413835 on ClinicalTrials.gov

What this trial studies

This is an open-label, single-arm interventional protocol that gives HN2302 injections to adults with antibody-positive generalized refractory myasthenia gravis. After up to a 4-week screening period, eligible participants enter a treatment period followed by a one-year safety and outcome follow-up. Key eligibility requires AChR or MuSK antibody positivity, MGFA class IIa–IVb, and an MG-ADL score ≥6 with inadequate response to standard therapies. The trial focuses on safety, tolerability, and preliminary signals of clinical benefit using standard MG outcome measures.

Who should consider this trial

Good fit: Adults 18–80 years with generalized MG (MGFA IIa–IVb), positive AChR or MuSK antibodies, MG-ADL ≥6, and poor response to standard therapies are the intended participants.

Not a fit: Patients with only ocular MG, those whose disease is controlled by current therapies, or those with active untreated hepatitis B/C or significant organ dysfunction (per exclusion criteria) are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, HN2302 could provide a new treatment option that improves muscle strength and reduces relapses for patients with refractory MG.

How similar studies have performed: Other targeted biologic agents such as complement inhibitors and FcRn antagonists have shown benefit in refractory MG, but HN2302 itself appears to be a novel investigational agent with limited prior human data available.

Eligibility criteria

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.

Where this trial is running

Xuzhou, Jiangsu

• The Affiliated Hospital of Xuzhou Medical University — Xuzhou, Jiangsu, China (RECRUITING)

Study contacts

• Principal investigator: Guiyun Cui — The Affiliated Hospital of Xuzhou Medical University
• Study coordinator: Yong Zhang

Conditions: Refractory Myasthenia Gravis, MG, HN2302