NCT01279720已完成1 期
Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency
Great Ormond Street Hospital for Children NHS Foundation Trust2 个研究点 分布在 1 个国家目标入组 8 人开始时间: 2003年10月最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 2
- 主要终点
- Immunological reconstitution
研究概览
简要总结
Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.
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
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who lack a human leukocyte antigen (HLA)-genotypically identical bone marrow donor OR phenotypically matched family or unrelated donor AND who show incomplete immune reconstitution on Polyethylene glycol-modified adenosine deaminase (PEG-ADA) enzyme replacement therapy (defined by absolute CD4+ count <300 cell/mm3 and who remain on immunoglobulin replacement therapy)
- •Diagnosis of ADA-SCID (Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA)confirmed by DNA sequencing OR by confirmed absence of <3% of ADA enzymatic activity in peripheral blood or (for neonates) in umbilical cord blood erythrocytes and/or leukocytes or in cultured fetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of PEG-ADA replacement therapy
- •Parental/guardian/patient signed informed consent
排除标准
- 未提供
结局指标
主要结局
Immunological reconstitution
时间窗: 5 years
Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study
次要结局
- Incidence of adverse reactions(5 years)
- Normalisation of nutritional status, growth, and development(5 years)
- Molecular characterisation of gene transfer(5 years)
研究者
研究点 (2)
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