Phase I Study of Chemokine and Cytokine Gene Modified Autologous Neuroblastoma Cells for Treatment of Relapsed/Refractory Neuroblastoma Using an Adenoviral Vector (CYCHE)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- To determine the safety of up to four subcutaneous (SC) injections of autologous neuroblastoma cells which have been genetically modified by adenoviral vectors to secrete lymphotactin (Lptn) and Interleukin-2 (IL-2).
研究概览
简要总结
This research study is designed to determine the safety and dosage of special cells that may make a patients own immune system fight the cancer. To do this we will put two special genes into cancer cells taken from the patients body. The genes we put in make the cancer cells produce lymphotactin, a natural substance that attracts immune system cells to the cancer, and IL-2 a natural substance that may help the immune system kill cancer cells. Some of these cells will then be put back in the patient's body. Studies of cancers in animals and in cancer cells that are grown in laboratories suggest that substances like lymphotactin and IL-2 help the body kill cancer cells. A treatment similar to this has been used in ten children previously and similar treatments are being used in adults with other cancers.
The purpose of this study is to learn the side effects and safe 'dosage' of these special cells.
详细描述
Neuroblastoma cells taken from the patient will be separated in the laboratory and two specially produced human viruses (adenovirus) that carry the lymphotactin and the IL-2 gene were put into the cells. These lymphotactin and IL-2 genes are meant to help the immune system fight the cancer.
The modified cancer cells will be injected under the patient's skin. There will be four shots. The second and subsequent shots will have ten times as many cells producing lymphotactin as the first. We do not know the best amount of special cells to use, so different patients will get different numbers of cells.
Before the second shot, and then again about 2 weeks later, we will remove some of the modified cells from the patient's body and study them. We will do this by removing a section of skin (referred to as a skin biopsy) at the place where the cells were injected. This test will help us to see whether or not the modified cells are killing cancer cells.
To study how the immunity is working in the patient's system, we will take blood samples prior to each injection, two to four days after each injection, and may repeat this 5-7 days after each injection if your doctor thinks that is necessary.
After patients have received the first set of 4 injections a complete evaluation is performed to see how this research treatment is working. If these evaluations look okay and if more injections are available, patients will have the option to receive a second set of 4 injections. If patients receive the second set of 4 injections, they will again have blood samples taken after each injection.
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
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
To determine the safety of up to four subcutaneous (SC) injections of autologous neuroblastoma cells which have been genetically modified by adenoviral vectors to secrete lymphotactin (Lptn) and Interleukin-2 (IL-2).
时间窗: 2 months
To determine the safety of up to eight (total) of these injections in patients who have received the first four injections without unacceptable toxicity and have evidence of stable disease or better after receiving these injections.
时间窗: 6 months
次要结局
- To determine whether MHC restricted or unrestricted antitumor immune responses are induced by SC injection of modified autologous neuroblasts and the cell doses required to produce these effects.(3 weeks)
- To obtain preliminary data on the antitumor effects of this treatment regimen(8 weeks)
研究者
Malcolm Brenner
Professor, Director Center for Cell and Gene Therapy
Baylor College of Medicine