jRCT2053240204招募中不适用
An open-label, multi-center, phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis (CYTB323O12101)
Novartis Pharma. K.K.0 个研究点目标入组 2 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 2
- 主要终点
- Occurrence of Adverse Events (AEs)
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 18age old over 至 65age old under（—）
- 性别
- All
入选标准
- •Confirmed gMG diagnosis supported by the following:
- •Documented report of positive serology testing for either AChR antibodies or MuSK antibodies at screening AND at least one of the following:
- •History of abnormal neuromuscular transmission test demonstrated by repetitive nerve stimulation or single-fiber electromyography
- •History of positive acetylcholinesterase inhibitor test
- •Improvement in MG signs on an oral acetylcholinesterase inhibitor as assessed by the treating physician
- •MGFA Class III-IVa (gMG) at screening
- •Treatment-resistant gMG as defined by: MG-ADL score >= 6 at screening despite adequate treatment trials with at least two different non-steroidal immunosuppressive drugs given at adequate doses and duration of therapy.
- •If on chronic corticosteroids, the ability and willingness to taper to a maximum dose of 10 mg prednisolone daily or equivalent at least one week before leukapheresis
- •If treated with cholinesterase inhibitors, patients must be on a stable dose for at least two weeks prior to screening
排除标准
- •Exclusively ocular myasthenia gravis (MGFA I), mild symptoms (MGFA II), or severe bulbar disease or MG crisis, MGFA Class IVb or V at screening
- •History of bone marrow/hematopoietic stem cell or solid organ transplantation.
- •Clinically significant active, opportunistic, chronic or recurrent infection (including positive for hepatitis B or hepatitis C) confirmed by clinical evidence, imaging, or positive laboratory tests one month prior to leukapheresis
- •Other uncontrolled disease states, such as asthma, or inflammatory bowel disease, where flares are commonly treated with oral or parenteral corticosteroids, at screening
- •Participants with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug induced immune deficiency), or tested positive for HIV antibody, at screening
- •Prior treatment with anti-CD19 therapy, adoptive T cell therapy or any prior gene therapy product (e.g. CAR-T cell therapy).
结局指标
主要结局
Occurrence of Adverse Events (AEs)
Occurrence of Adverse Events (AEs) (including CRS and ICANs)
Severity of Adverse Events (AEs)
Severity of Adverse Events (AEs) (including CRS and ICANs)
Frequency of Adverse Events (AEs)
Frequency of Adverse Events (AEs) (including CRS and ICANs)
Change from baseline in safety parameters
Change from baseline in safety parameters including, but not limited to: Vital signs, laboratory parameters, ECG, and neurological status
次要结局
未报告次要终点
研究者
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