A Prospective, Observational Study Evaluating Seroprevalence and Rate of Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes and Exploratory Vectors in Subjects With Hemophilia A in the United States
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 186
- 试验地点
- 1
- 主要终点
- Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A
研究概览
简要总结
To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A
详细描述
This is a single-center, decentralized, patient-centered, prospective, observational study utilizing biospecimen samples collected from hemophilia A subjects across the United States to evaluate and characterize seroprevalence and the rate of seroconversion of antibodies against AAV serotypes and exploratory vectors, and to investigate the associated factors that may influence the vector titers.
Relevant medical findings will also be collected from the subject, as well as symptoms related to hemophilia A. The collection of medical history may include major illnesses, diagnoses, and surgeries.
研究设计
- 研究类型
- Observational
- 观察模型
- Ecologic Or Community
- 时间视角
- Prospective
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
- •Subjects diagnosed with Hemophilia A
- •Subjects aged 18years or over at time of entry
排除标准
- •Currently participating in an interventional study of any investigational product, device or procedure.
- •Subjects who have been previously treated with AAV vector gene therapy
结局指标
主要结局
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A
时间窗: Baseline
To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline
Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A
时间窗: Change from baseline through Week 12
次要结局
- To describe and characterize AAV vector titer values in subjects with Hemophilia A(Change from baseline through Week 24)