Treatment of ADA-SCID by Gene Therapy on Somatic Cells
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 8
- 主要终点
- Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene
研究概览
简要总结
This study investigated the safety and efficacy of different gene therapy approaches for Severe Combined Immunodeficiency (SCID) caused by the deficiency of adenosine deaminase (ADA) enzyme. This is a severe condition that can be cured by HLA-matched sibling donor bone marrow transplantation. Patients were enrolled if no HLA-identical sibling donor was available and the patient showed evidence of failure of enzyme replacement therapy or this treatment was not a long-term available option. The aim of the study was to evaluate the safety and efficacy of the procedure and to identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after retroviral vector mediated ADA gene transfer.
详细描述
This is mono-centric, non-randomized, non-controlled, open label, phase I-II trial that evaluated the safety and efficacy of ADA gene transfer into somatic cells for the treatment of ADA-SCID
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
- •Lack of HLA-identical sibling donor and
- •Evidence of failure of the enzyme replacement treatment after >6 months or
- •PEG-ADA is not available as a life long option
排除标准
- •HLA identical bone marrow sibling donor
- •HIV infection
- •Malignancy
结局指标
主要结局
Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene
次要结局
- Evaluation of extent, kinetic and duration of the engraftment of transduced cells and the potential selective advantage of ADA positive cells
- Evaluation of efficacy of the administration of autologous PBL/HSC(Clinical, immunological, hematological, microbiological, ADA activity and purine metabolism)
- To identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after gene therapy