A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
试验速览
- 阶段
- 早期 1 期
- 发起方
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Dose-limiting toxicity (DLT)
研究概览
简要总结
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
详细描述
POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis and other diseases may only show local pathological damage or systemic lesions. If they are not diagnosed and treated in time or poorly controlled, they will progress as the course of the disease progresses. Risk of disability or even death.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Diagnosed with POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, and the curative effect of conventional hormones, radiotherapy and chemotherapy, protease inhibitors is not good and (or) no effective treatment means.
- •2. After glucocorticoids, cyclophosphamide or methotrexate treatments there are still relapsed and refractory diseases, or clearly show intolerance/toxicity to these drugs.
- •3. Estimated survival time> 12 weeks;
- •Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up;
- •Patients or their legal guardians volunteer to participate in the study and sign the informed consent.
排除标准
- •Subjects with any of the following exclusion criteria were not eligible for this trial:
- •History of craniocerebral trauma, conscious disturbance, epilepsy,cerebrovascular ischemia, and cerebrovascular, hemorrhagic diseases;
- •Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
- •Pregnant (or lactating) women;
- •Patients with severe active infections (excluding simple urinary tract infection and bacterial pharyngitis);
- •Active infection of hepatitis B virus or hepatitis C virus;
- •Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
- •Those who have used any gene therapy products before.
- •The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- •Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0mg/dl;
- •Those who suffer from other uncontrolled diseases are not suitable to join the study;
- •HIV infection;
- •Any situation that the researchers believe may increase the risk of patients or interfere with the test results.
结局指标
主要结局
Dose-limiting toxicity (DLT)
时间窗: Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of treatment-emergent adverse events (TEAEs)
时间窗: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Incidence of treatment-emergent adverse events \[Safety and Tolerability\]
次要结局
- Titer of auto-antibody Titer of auto-antibody titer of auto-antibody(Up to 2 years after CD19/BCMA targeted CAR T-cells infusion)
- Overall response rate (ORR)(Up to 2 years after CD19/BCMA targeted CAR T-cells infusion)
- Duration of remission, DOR(2 years post CD19/BCMA CAR-T cells infusion)
- Best overall response, BOR(At ≤3 month)
- Overall survival (OS)(From CD19/BCMA CAR-T infusion to death，up to 2 years)
研究者
He Huang
The President of The First Affiliated Hospital, College of Medicine, Zhejiang University
Zhejiang University