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Not yet recruiting NCT07556120

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

Phase I Interventional Generalized Myasthenia Gravis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: HN2301 injection.
Who it may be relevant to
Registry conditions: Generalized Myasthenia Gravis. Basic parameters: 18 years — 80 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis

Overview

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.

Interventions

  • Drug HN2301 injection
    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.

Primary outcome measures

  • Incidence of treatment-emergent adverse events (TEAEs) [Time frame: Up to 3 months]
Secondary outcome measures (7)
  • 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]

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;
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • The First Affiliated Hospital of University of Science and Technology of China — Hefei

Identifiers

NCT: NCT07556120 · HN2301-02

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗