Long-term Follow-up Study to Evaluate the Safety and Efficacy in Patients Who Have Ever Received Lentiviral-based Gene-edited Immune Cell Therapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 49
- 试验地点
- 5
- 主要终点
- To assess delayed adverse events which are suspected related to previous gene-edited immune cell therapy
研究概览
简要总结
According to health authorities guidances (FDA 2006, EMA(European Medicines Agency) 2009) for gene therapy clinical trials, observing subjects for delayed adverse events for 15 years is recommended. This purpose of this long-term follow-up study is to evaluate the safety and efficacy in patients who have ever received lentiviral-based gene-edited immune cells which are manufactured by Pell Bio-Med Technology Co. Ltd.
详细描述
After completion or early withdraw from the other treatment protocol, patients should be enrolled into this long-term follow-up study. If patients do not enter this study right after leaving the treatment protocol, they may have the option to enter this long-term follow-up study at any time within 15 years after the last lentiviral-based gene-edited immune cell infusion.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have ever received Pell's lentiviral-based gene-edited immune cell as monotherapy or as combination therapy in clinical trials.
- •The last lentiviral-based gene-edited immune cell infusion within 15 years.
- •Patient/patient's parent/legal guardian is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
排除标准
- •There are no specific exclusion criteria for this study.
结局指标
主要结局
To assess delayed adverse events which are suspected related to previous gene-edited immune cell therapy
时间窗: 15 years
• Proportion of patients with any events of the following items which are suspected related to previous gene-edited immune cell therapy. 1. New malignancies 2. New incidence or exacerbation of a pre-existing neurologic disorder 3. New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder 4. New incidence of a hematologic disorder, including hypogammaglobulinemia 5. New incidence of infection (potentially product-related) 6. Other than the above adverse events, which are suspected related to gene-edited immune cell therapy judged by the investigator
次要结局
- Monitor for Replication Competent of Lentivirus (RCL)(15 years)
- Monitor the persistence of gene-edited immune cells in peripheral blood(By qPCR)(15 years)
- Monitor the persistence of gene-edited immune cells in peripheral blood(By Flowcytometry)(5 years)
- To assess the long-term efficacy of gene-edited immune cells(15 years)