This is an open label, single arm study, to evaluate the safety , tolerability and preliminary efficacy of HN2302 for refractory myasthenia gravis.
The study will consist of an up to 4-week Screening Period, Treatment Period and one year Follow-up Period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Patients will be administrated with specified dose on specified days at a lower dose level and escalated to safe and effective dose levels.
The Affiliated Hospital of Xuzhou Medical University
Xuzhou, Jiangsu, China
RECRUITINGIncidence of Treatment-Emergent Adverse Events
Proportion and severity of adverse events (AEs)
Time frame: Up to 3 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
Time frame: 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.
Time frame: 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.
Time frame: Up to 12 months
Changes from baseline in 15-item quality of life (MG-QOL15r) score
To assess important aspects of the patient's experience related to MG, scores each of 15 items 0-2 (max score 30).
Time frame: Up to 12 months
Percentage of patients with symptom changes after treatment
Proportion of patients without symptom worsening or relapse
Time frame: Up to 12 months
in 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)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: Day-28 to14 days
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
Time frame: Up to 12 months
Dynamic changes in cytokine levels after treatment
Differences in cytokine post-administration vs. baseline
Time frame: Up to 12 months
Changes in acetylcholine receptor (AchR) antibody levels after treatment
Differences in AchR antibody post-administration vs. baseline
Time frame: Up to 12 months