NCT04093349进行中（未招募）1 期
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 4
- 试验地点
- 58
- 主要终点
- Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 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
- •Provide written informed consent;
- •Males and Females ≥18 years of age with late-onset Pompe disease;
- •Received ERT for at least the previous 24 months
- •Have clinically moderate, late-onset Pompe disease characteristics;
- •Agree to use reliable contraception.
排除标准
- •Active hepatitis B and/or C;
- •Significant underlying liver disease;
- •Human immunodeficiency virus (HIV) infection;
- •Prior hypersensitivity to rhGAA;
- •Pre-existing anti-AAV neutralizing antibody titers;
- •High titer antibody responses to rhGAA;
- •Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
- •Received any prior vector or gene transfer agent;
- •Active malignancy (except non-melanoma skin cancer);
- •History of liver cancer;
- •Pregnant or nursing women;
- •Any evidence of active infection at the time of SPK-3006 infusion.
结局指标
主要结局
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
时间窗: Up to 5 years
Adverse events.
Occurrence of immune response against GAA transgene
时间窗: Up to 5 years
Occurrence of immune response against AAV capsid
时间窗: Up to 5 years
次要结局
未报告次要终点
研究者
研究点 (58)
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