EUCTR2019-003595-38-GB进行中（未招募）1 期
FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease - FORTIS
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 8
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Subjects must meet all of the following inclusion criteria to be eligible for enrollment:
- •1. Subject is aged = 18 years (ambulatory or nonambulatory).
- •2. Subject has a documented clinical diagnosis of Pompe disease by genetic testing.
- •3. Subject has received ERT with rhGAA for the previous = 2 years.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 6
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Subject is currently participating in an interventional study or has received gene or cell therapy
- •2. Subject tests positive for AAV8 antibodies
研究者
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