ISRCTN57061190已完成未知
Phase I/II open-label trial to determine the safety and tolerability of intracranial gene therapy in GM2 gangliosidosis using recombinant adeno-associated viral vectors
适应症
试验速览
- 阶段
- 未知
- 状态
- 已完成
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Male and female infants and young subjects aged 3 months to 18 years
- •2. GM2 gangliosidosis confirmed by biochemical analysis and molecular analysis of cognate HEXA or HEXB genes in the presymptomatic phase with normal neuromotor development, physical examination and cerebral MR imaging
排除标准
- •1. GM2 activator deficiency
- •2. Developmental regression or other features of symptomatic GM2 gangliosidosis
- •3. Clinical or radiological abnormalities of the central nervous system
研究者
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