Efficacy and Safety of HN2301 in Refractory Myasthenia Gravis(MG)
- Conditions
- Refractory Myasthenia Gravis
- Interventions
- Registration Number
- NCT06965309
- Lead Sponsor
- Shenzhen MagicRNA Biotechnology Co., Ltd
- Brief Summary
This is an investigator-initiated trial designed to evaluate the safety, and efficacy of HN2301 in refractory myasthenia gravis
- 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.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 9
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description HN2301 treatment group HN2301 injection -
- Primary Outcome Measures
Name Time Method Adverse events related to HN2301 3 months Incidence and severity of AEs associated with HN2301 as assessed by CTCAE v5.
- Secondary Outcome Measures
Name Time Method vivo CAR T cell production Day-28 to14 days Assessment of CAR T production (CAR expression ratio in T cells) in the peripheral blood of MG patients by flow cytometry (FACS)
B cell ratio and counts in peripheral blood Day-28 to12 months 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
Change from baseline of MG-ADL score after HN2301 administration. Day-28 to12 months 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.Change from baseline of MGC score after HN2301 administration. Day-28 to12 months 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.Change from baseline of QMG score after HN2301 administration. Day-28 to12 months 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.Change from baseline of MG-QOL15r score after HN2301 administration. Day-28 to12 months 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.
Change of patients after HN2301 administration. Day-28 to12 months 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 reductionTime to achieve a ≥2 point reduction in MG-ADL score following HN2301 administration. 14 days-12 months The time (months) to achieve a ≥2 point reduction in MG-ADL score following HN2301 administration of the patient.
Trial Locations
- Locations (1)
Shanghai General Hospital (Songjiang Branch) of Shanghai Jiao Tong University School of Medicine
🇨🇳Shanhai, China