A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 242
- 试验地点
- 23
- 主要终点
- Prevalence of neutralizing antibodies (NAb) - Year 3 Visit
研究概览
简要总结
Assess the seroprevalence of neutralizing antibodies (NAb) to AAV in adults with severe hemophilia A (coagulation factor VIII [FVIII] <1%) or moderately severe to severe hemophilia B (coagulation factor IX [FIX] ≤2%).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participant is male between 18 and 75 years old at the time of screening.
- •Established severe hemophilia A (plasma Factor VIII (FVIII) activity <1%) or B (plasma Factor IX (FIX) activity ≤2%).
- •Provision of signed informed consent form (ICF).
- •Participant is willing and able to comply with the requirements of the protocol.
排除标准
- •Bleeding disorder(s) other than hemophilia A or B.
- •Personal laboratory evidence of having developed inhibitors to FVIII or FIX protein at any time (≥0.6 Bethesda Units [BU] on any single test).
- •Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy.
- •Received systemic antiviral and/or interferon therapy within 30 days of blood draw, with the exception of treatment for human immunodeficiency virus (HIV).
- •Currently receiving ribavirin and/or interferon based therapy for active hepatitis C virus (HCV).
- •Received immunoglobulin therapy or plasma infusion within 120 days of the blood draw.
- •Has a known immune deficiency other than HIV.
- •Has lymphocyte or plasma cell malignancies.
- •Participant is a family member or employee of the investigator.
结局指标
主要结局
Prevalence of neutralizing antibodies (NAb) - Year 3 Visit
时间窗: Year 3 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Prevalence of neutralizing antibodies (NAb) - Baseline visit
时间窗: Baseline visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Prevalence of neutralizing antibodies (NAb) - Year 1 Visit
时间窗: Year 1 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Prevalence of neutralizing antibodies (NAb) - Year 2 Visit
时间窗: Year 2 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
次要结局
- Prevalence of binding antibodies to AAV, including AAV8 and AAV2(Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit)
- Prevalence of NAb to AAV including AAV2 and AAV8(Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit)
- T-cell mediated immune response to AAV8(Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit)