A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 61
- 试验地点
- 36
- 主要终点
- Neutralizing Antibodies Titer to SPK-3006 capsid
研究概览
简要总结
The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Screening
- 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 and authorization to use protected health information in accordance with national and local privacy regulations
- •Male or females ≥18 years of age
- •Currently on ERT using regular recombinant human GAA infusions for at least 18 months prior to screening
- •Documented history of clinically moderate late-onset Pompe disease.
排除标准
- •History of HIV infection
- •Requires any invasive ventilation (other than BiPAP at night) or noninvasive ventilation while awake and upright
- •Previously received SPK-3006
- •Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks (vaccination studies are accepted)
- •Any concurrent clinically significant condition that would not allow the potential participant to complete the Day 1 examinations, or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study
- •Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol.
结局指标
主要结局
Neutralizing Antibodies Titer to SPK-3006 capsid
时间窗: 1 day
The neutralizing antibodies titer to SPK-3006 is measured once prospectively at one site visit.
Occurrence of Neutralizing Antibodies to SPK-3006 capsid
时间窗: 1 day
The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid.
次要结局
- Anti-GAA binding antibodies Titer(1 day)
- GAA activity level(1 day)
- Occurrence of Anti-GAA binding antibodies across participants(1 day)
- Neutralizing antibodies to circulating GAA Titer(1 day)
- GAA antigen level(1 day)
- Occurrence of Neutralizing antibodies to circulating GAA(1 day)