A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapsed / Refractory Neurological Autoimmune Diseases
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- The number and severity of dose-limiting toxicity (DLT) events
研究概览
简要总结
This is an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.
详细描述
This is an investigator-initiated trial to evaluate the safety and efficacy of universal CD19/BCMA CAR T-cells in Relapsed or Refractory Neurological Autoimmune Diseases.
Study intervention consists of a single infusion of universal CAR T-cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.
Interim analysis will be performed when participants finish the visit 90 days after CAR T-cell infusion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years.
- •Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
- •Subjects with relapsed or refractory neurological autoimmune diseases, Including neuromyelitis optica spectrum disorders(NMOSD), myasthenia gravis(MG), multiple sclerosis(MS)，Autoimmune encephalitis(AE) and chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP).
- •Female subjects of childbearing potential and male subjects with partners of childbearing potential must use medically approved contraception or abstinence during the study treatment period and for at least 6 months after the end of the study treatment; Female subjects of childbearing potential must have a negative Human chorionic gonadotropin (HCG) test within 7 days before study enrollment and not be lactating.
- •Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
排除标准
- •Subjects with a history of severe drug allergies or allergic tendencies.
- •History of malignancy within five years.
- •Subjects with insufficient cardiac function.
- •Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA >the upper limit of detection; subjects positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; individuals positive for human immunodeficiency virus (HIV) antibody; individuals positive for syphilis testing.
- •Pregnant women or women planning to conceive.
研究组 & 干预措施
UCAR T-cell group
Universal allogeneic CD19/BCMA CAR T-cells
干预措施: UCAR T-cell (Drug)
结局指标
主要结局
The number and severity of dose-limiting toxicity (DLT) events
时间窗: Within 28 Days After UCAR T-cell Infusion
DLT will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, and the ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells.
The total number, incidence, and severity of AEs
时间窗: Up to 90 days After UCAR T-cell Infusion
次要结局
- NMOSD、MS: Expanded Disability Status Scale (EDSS) score(Up to 24 Months After UCAR T-cell Infusion)
- MG: Quantitative Myasthenia Gravis Score (QMG)(Up to 24 Months After UCAR T-cell Infusion)
- NMOSD、MS: Modified Rankin Scale(Up to 24 Months After UCAR T-cell Infusion)
- CIDP: Inflammatory Neuropathy Cause and Treatment (INCAT) Score(Up to 24 Months After UCAR T-cell Infusion)
- MG: Myasthenia Gravis Activities if Daily Living (MG-ADL) Score(Up to 24 Months After UCAR T-cell Infusion)
- AE: Change in CASE(Up to 24 Months After UCAR T-cell Infusion)
研究者
Jialing Wu
Principal Investigator
Tianjin Huanhu Hospital