Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Alzheimer's Disease: AAV- hTERT
试验速览
- 阶段
- 1 期
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Incidence of adverse events
研究概览
简要总结
Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many neurodegenerative diseases related to aging, including Alzheimer's disease (AD).
This study will entail treating subjects with hTERT delivered via transduction using AAV. The goal is to extend the telomeres to prevent, delay, or even reverse the development of the pathology of AD. It is expected to have a direct consequence on cognitive function and quality of life in patients with neurodegenerative diseases, such as AD.
详细描述
Patients diagnosed with AD who meet with the inclusion - exclusion criteria, will be treated with a single dose of LGT delivered intravenously (IV) and intrathecally (IT).
Baseline will be performed within 8 weeks of beginning the treatment regimen. The treatment regimen will begin with IV delivery of AAV-hTERT, defined as "Day 0." Safety and efficacy analyses will be conducted at Weeks 1, 4, 13, 26, 39, and 52 post-treatment.
Study objectives
Primary: Safety and Tolerability
- Investigate the safety and tolerability of AAV-hTERT by IV and IT administration.
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
- 45 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
subjects treated with LGT
subjects will be treated with a single dose of LGT (AAV-hTERT)
干预措施: AAV-hTERT (Drug)
结局指标
主要结局
Incidence of adverse events
时间窗: 12 months
Incidence of serious adverse events and adverse events throughout the study
次要结局
- hTERT expression and telomerase activity(12 months)