The Safety and Efficacy of Universal CAR-T Cells Targeting BCMA in the Treatment of Refractory AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disease
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 18
- 主要终点
- DLT
研究概览
简要总结
This is an open label, Multi-center，dose-escalation study in up to 18 participants with refractory NMOSD. This study aims to evaluate the safety and efficacy of universal CAR-T Cells targeting BCMA in the Treatment of refractory NMOSD.
详细描述
This is a multi-center, single-arm, open-label clinical study, and the sample size is set to 12-18 subjects. Based on the "3 + 3" dose escalation design principle, subjects will be divided into 3 groups from low dose to high dose in sequence (Group A; Group B; Group C).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18-65 years ; both genders eligible.
- •Meets the criteria for Refractory NMOSD.
- •Anticipated survival of ≥ 12 weeks as judged by the researcher.
- •Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.
- •Provides written informed consent.
排除标准
- •History of solid organ transplantation.
- •Malignant tumor within the last two years.
- •Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.
- •Primary immunodeficiency (congenital or acquired).
- •Severe cardiac disease.
- •History of psychiatric disorders or history of psychotropic drug abuse, with no history of withdrawal.
- •Allergic constitution or a history of severe allergies.
- •Pregnant or breastfeeding women.
研究组 & 干预措施
BCMA CART Group
1.0-4.0×10^6 CAR- Tcells/kg
干预措施: Universal BCMA-CD19 CART (Drug)
结局指标
主要结局
DLT
时间窗: Within28 Days After BRL-302 Infusion
The number and severity of dose-limiting toxicity (DLT) events
AEs
时间窗: Up to 12 Months After BRL-302 Infusion
The total number, incidence, and severity of AEs
次要结局
- Annualized relapse rate (ARR)(1,3,6,12 month after BRL-302 infusion)