NCT07223944招募中3 期
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Spur Therapeutics36 个研究点 分布在 10 个国家目标入组 45 人开始时间: 2026年4月7日最近更新:
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 45
- 试验地点
- 36
- 主要终点
- To evaluate the efficacy of FLT201
研究概览
简要总结
This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.
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
- •Aged ≥18 years at time of screening.
- •Clinical diagnosis of Gaucher disease type 1
- •Stable hemoglobin concentration at baseline
- •Stable platelet count at baseline
- •Receiving ERT or SRT without interruption for at least 2 years
排除标准
- •Diagnosed or suspected Gaucher disease type 2 or type 3
- •Positive for AAVS3 neutralizing antibodies.
- •Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- •Positive pregnancy test or lactating
- •History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant.
- •History of receiving any gene therapy or cell therapy.
- •History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply
研究组 & 干预措施
FLT201
Experimental
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
干预措施: FLT201 (Genetic)
结局指标
主要结局
To evaluate the efficacy of FLT201
时间窗: 1 Year
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
次要结局
未报告次要终点
研究者
研究点 (36)
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