NCT06545136进行中（未招募）1 期
A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects With Gaucher Disease (GALILEO-2)
Spur Therapeutics5 个研究点 分布在 4 个国家目标入组 75 人开始时间: 2024年5月13日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 75
- 试验地点
- 5
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
研究概览
简要总结
This is a multicenter, long-term, follow-up trial of participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants will be followed for 5 years post-treatment.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).
- •2. Participants able to give full informed consent and able to comply with all requirements of the trial.
排除标准
- 未提供
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
时间窗: Week 38 to Month 60
Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded per CTCAE version 5.0 (or later)
次要结局
未报告次要终点
研究者
研究点 (5)
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