Generalized Myasthenia GravisPhase 1NCT07556120
Efficacy and Safety of HN2301 in Patients With Generalized Myasthenia Gravis (MG)
A Phase 1 study testing HN2301 injection in people with Generalized Myasthenia Gravis, run by Shenzhen MagicRNA Biotechnology Co., Ltd at 1 site in China.
Trial summary
This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- HN2301 injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 31 Dec 2026to 31 Dec 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 12Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of University of Science and Technology of China
Hefei, Anhui, 230036
Wen Xu, +86055162284920, Xuwen6779@ustc.edu.cn
About this study
Official title: An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis
This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.
Read the detailed description
This study consists of a screening period of up to 4 weeks, a treatment period, and a follow-up period of 1 year.
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- HN2301 injection
- Keywords
- MG, in vivo CART
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HN2301 injectionDrug
Also called in vivo CAR-T
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.
| Study group | Role | What they receive |
|---|---|---|
| HN2301 treatment group | Experimental | Participants will receive HN2301 Injection at the specified dose level and on the specified study days. Receives: Drug: HN2301 injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 3 months
Secondary outcomes (7)
in vivo CAR T cell production
Assessment of in vivo CAR-T cell production, defined by the proportion of CAR-expressing T cells in peripheral blood as measured by flow cytometry.
Measured: Up to14 days
Absolute B-cell count in peripheral blood
Assessment of peripheral blood absolute B-cell count (cells/μL)， including naive B cells and memory B cells, by flow cytometry.
Measured: Up to 12 months
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.
Measured: 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.
Measured: 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，including 8 items, each scored on a scale of 0 to 3, with 3 indicating the most severe degree.
Measured: Up to 12 months]
Changes from baseline in revised Myasthenia Gravis Quality of Life-15 score (MG-QOL15r)
To assess important aspects of the patient's experience related to MG, scores each of 15 items 0-2 (max score 30).The higher the score, the worse the patient's quality of life.
Measured: Up to 12 months
Changes in acetylcholine receptor (AchR) antibody levels after treatment
Differences in AchR antibody post-administration vs. baseline
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2026
- Study start31 Dec 2026 (estimated)
- Primary completion31 May 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen MagicRNA Biotechnology Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The First Affiliated Hospital of University of Science and Technology of China
- Responsible party
- Sponsor
- Investigators
- Wen Xu, Principal investigator, The First Affiliated Hospital of University of Science and Technology of ChinaYan Jiang, Principal investigator, The First Affiliated Hospital of University of Science and Technology of China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07556120
- Sponsor's protocol ID
- HN2301-N02
- Status last verified
- Aug 2026
- First submitted
- 16 Apr 2026
- Last updated
- 12 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT07556120
- Acronym / sponsor ID
- HN2301-N02
- Conditions
- Generalized Myasthenia Gravis
- Keywords
- MG, in vivo CART
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Shenzhen MagicRNA Biotechnology Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- The First Affiliated Hospital of University of Science and Technology of China
- Study start
- 31 Dec 2026 (estimated)
- Primary completion
- 31 May 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 29 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.
Detailed description
This study consists of a screening period of up to 4 weeks, a treatment period, and a follow-up period of 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HN2301 treatment group | Experimental | Drug: HN2301 injectionParticipants will receive HN2301 Injection at the specified dose level and on the specified study days. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HN2301 injectionAlso: in vivo CAR-TDosing will begin at a lower dose level and may be escalated to dose levels considered safe and potentially effective according to the study protocol. | Drug | HN2301 treatment group |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 3 months
Incidence, nature, and severity of treatment-emergent adverse events, assessed according to the study protocol and applicable toxicity grading criteria.
7 secondary outcomes
in vivo CAR T cell production
Time frame: Up to14 days
Absolute B-cell count in peripheral blood
Time frame: Up to 12 months
Changes from baseline in Myasthenia Gravis Activities of Daily Living(MG-ADL) score
Time frame: Up to 12 months]
Changes from baseline in Myasthenia Gravis Composite (MGC) score
Time frame: Up to 12 months
Changes from baseline in Quantitative Myasthenia Gravis (QMG) score
Time frame: Up to 12 months]
Changes from baseline in revised Myasthenia Gravis Quality of Life-15 score (MG-QOL15r)
Time frame: Up to 12 months
Changes in acetylcholine receptor (AchR) antibody levels after treatment
Time frame: Up to 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2026.
- Individual participant dataSharing planned: No
Related trials for Generalized Myasthenia Gravis
Active, not recruitingPhase 3
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Recruiting
Holistic Assessment and Remote Digital MONitoring of mYasthenia Gravis Via MyoSense 360
Myasthenia Gravis, Generalized
RecruitingPhase 3
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Myasthenia Gravis
RecruitingPhase 2/3
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Not yet recruitingEarly Phase 1
A Clinical Study to Evaluate the Safety and Efficacy of GT802 Injection in the Treatment of Refractory Generalized Myasthenia Gravis
Generalized Myasthenia Gravis (gMG)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Wen Xu principal investigator
The First Affiliated Hospital of University of Science and Technology of China
Yan Jiang principal investigator
The First Affiliated Hospital of University of Science and Technology of China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HN2301 injection in people with Generalized Myasthenia Gravis, run by Shenzhen MagicRNA Biotechnology Co., Ltd at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wen Xu (+86055162284920, Xuwen6779@ustc.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) (time frame: Up to 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2026.