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Refractory Myasthenia GravisNCT06965309

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

A study testing HN2301 injection in people with Refractory Myasthenia Gravis, run by Shenzhen MagicRNA Biotechnology Co., Ltd at 1 site in China.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Refractory 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
9 participants
Where
1 siteChina
Study dates
19 Jan 2026to 11 Feb 2026
Phase
Interventional
Are there results?
None posted in the registry

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:
  • 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;
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  • 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.

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 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;
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  • 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.

Trial locations and contacts

1 site in 1 country.

China1 site

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

    Shanghai, 201600

Other trials in China

About this study

Official title: Dose-escalation Study to Assess the Safety, Tolerability, and Preliminary Efficacy of HN2301 in Patients With Refractory Myasthenia Gravis (MG)

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

Read the detailed description

This study is a prospective exploratory clinical trial in subjects with refractory myasthenia gravis. The objective is to evaluate the safety, initial efficacy of HN2301injection in refractory myasthenia gravis.

Being tested
HN2301 injection
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 cart

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

Study groups in NCT06965309
Study groupRoleWhat they receive
HN2301 treatment groupExperimentalSpecified dose level
Receives: Drug: HN2301 injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse events related to HN2301

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

    Measured: 3 months

Secondary outcomes (8)

  1. vivo CAR T cell production

    Assessment of CAR T production (CAR expression ratio in T cells) in the peripheral blood of MG patients by flow cytometry (FACS)

    Measured: Day-28 to14 days

  2. B cell ratio and counts in peripheral blood

    Assessment of B cell ratio and counts (B cell counts per μl peripheral blood) and B cell subsets(naive B cell, memory B cell) by flow cytometry (FACS) in peripheral blood

    Measured: Day-28 to12 months

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

    1. Assessment of MG-ADL score from baseline administration at various timepoints up to month 12 follow-up visit. A total score can fall between 0 and 24 with a higher score representing a more significant degree of disease activity.
    2. Proportion of patients achieving a ≥2-point reduction in MG-ADL score following HN2301 administration.

    Measured: Day-28 to12 months

  4. Change from baseline of MGC score after HN2301 administration.

    1. Assessment of MGC score from baseline administration at various timepoints up to month 12 follow-up visit. A total score can fall between 0 and 50 with a higher score representing a more significant degree of disease activity.
    2. Proportion of patients achieving a ≥3-point reduction in MGC score following HN2301 administration.

    Measured: Day-28 to12 months

  5. Change from baseline of QMG score after HN2301 administration.

    1. Assessment of QMG from baseline administration at various timepoints up to month 12 follow-up visit. A total score can fall between 0 and 32, with a higher score representing a more significant degree of disease activity.
    2. Proportion of patients achieving a ≥3-point reduction in QMG score following HN2301 administration.

    Measured: Day-28 to12 months

  6. Change from baseline of MG-QOL15r score after HN2301 administration.

    Assessment of MG-QOL15r from baseline administration at various timepoints up to month 12 follow-up visit. A total score can fall between 0 and 100, with a lower score representing a more significant degree of disease activity.

    Measured: Day-28 to12 months

  7. Change of patients after HN2301 administration.

    1. Proportion of patients without symptom worsening
    2. Proportion of patients without symptom relapse
    3. Proportion of patients achieving minimal clinical manifestations
    4. Proportion of patients undergoing corticosteroid dose reduction

    Measured: Day-28 to12 months

  8. Time to achieve a ≥2 point reduction in MG-ADL score following HN2301 administration.

    The time (months) to achieve a ≥2 point reduction in MG-ADL score following HN2301 administration of the patient.

    Measured: 14 days-12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 May 2025
  2. Study start19 Jan 2026
  3. Primary completion11 Feb 2026
  4. Study completion11 Feb 2026

Why it stopped: Change of registrant

Resources

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 N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06965309
Sponsor's protocol ID
HN2301-002
Status last verified
Feb 2026
First submitted
29 Apr 2025
Last updated
12 Feb 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A study testing HN2301 injection in people with Refractory 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 12 inclusion and 11 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: stopped early, as of the sponsor's last update on 12 Feb 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.

Are results available for this study?

The study was stopped early (reason given: Change of registrant). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse events related to HN2301 (time frame: 3 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06965309.

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 Feb 2026.

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