2024-511172-33-00招募中2 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 Therapeutics Limited1 个研究点 分布在 1 个国家目标入组 1 人开始时间: 2024年4月29日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
研究概览
简要总结
To assess the long-term safety of FLT201 in participants with Gaucher disease type 1.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).
- •Participants able to give full informed consent and able to comply with all requirements of the trial.
排除标准
- •Not applicable.
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
次要结局
- 1. Incidence of AEs, SAEs, and changes from baseline in vital signs, 12-lead ECG, physical examination, and laboratory assessments.
- 2. Change from baseline in GCase activity (Dried Blood Spot [DBS]), plasma GCase activity and relative concentration, and leukocyte GCase activity over time.
- 3. Change from baseline in lyso-Gb1 concentration.
- 4. Change from baseline in hemoglobin, platelet count, spleen volume as measured by MRI, and liver volume as measured by MRI.
- 5. Change from baseline in GCase antibody levels.
- 6. Time to clearance of vector genomes in plasma and semen until three consecutive negative samples, where applicable (this may be completed in the preceding treatment trial and, if so, will not be followed-up within this trial.)
研究者
Clinical Trials Information
Scientific
Spur Therapeutics Limited
研究点 (1)
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