Gene Therapy for X Linked Severe Combined Immunodeficiency
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- 1-year survival rate 1-year survival rate
研究概览
简要总结
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.
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 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •X-SCID patients diagnosed by IL2RG single gene mutation
- •No HLA（human leukocyte antigen） matching donor
- •Hematopoietic stem cell transplantation failed and the time from transplantation was more than 18 months
- •Severe and persistent refractory infections
- •Life expectancy of > : 4 months
- •HIV PCR in peripheral blood was negative
- •the children and their families signed informed consent and were willing to enter the clinical trial and complete follow-up
排除标准
- •The patient has diagnosed with hematological malignant diseases
- •Received chemotherapy within 3 months
- •HIV infection or HBV（hepatitis B virus） infection
- •The patient or his first-degree relative has developed a malignant tumor within the age of 18 or has been diagnosed with malignant tumor prone genes
- •Although the patient with X-SCID was diagnosed as IL2RG single gene mutation , the clinical phenotype was not severe, so they could continue to wait for the donor search;
- •Patients whose family members have no intention to continue the follow-up treatment in any link
结局指标
主要结局
1-year survival rate 1-year survival rate
时间窗: one year after gene therapy of last recruited patient
1-year survival rate of 10 recruited patients
5-year survival rate
时间窗: five years after gene therapy of last recruited patient
5-year survival rate of 10 recruited patients
3-year survival rate
时间窗: three years after gene therapy of last recruited patient
3-year survival rate of 10 recruited patients
次要结局
- Number of patients who recovers from previous infection（virus and bacteria）(through study completion, an average of 2 year)
- Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood mononuclear cells(through study completion, an average of 1 year)
- Growth velocity after gene therapy,weight in kilograms, height in meters(through study completion, an average of 2 year)
- Absolute numbers of peripheral-blood immune-cell subsets(through study completion, an average of 1 year)
- Vector marking (vector copy number per cell) in blood and bone marrow cells(through study completion, an average of 1 year)
- Serum immunoglobulins levels(through study completion, an average of 2 year)
- Number of patients without intravenous immune globulin supplementation(through study completion, an average of 2 year)
- Number of patients who has a response to vaccines(through study completion, an average of 2 year)
研究者
mingfeng hu
Resident physician
Children's Hospital of Chongqing Medical University