Anti-AAV8 Antibody Assessment Study of Males With Duchenne Muscular Dystrophy Aged 0 to <25 Years
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 200
- 试验地点
- 2
- 主要终点
- Prevalence of anti-AAV8 antibodies in patients with DMD
研究概览
简要总结
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
详细描述
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD). Information collected in this study may be used to identify potential participants for DMD investigational gene therapy clinical trials.
This study consists of:
- A phone/video interview or at an in-clinic visit to provide e-consent and medical history
- A single home health visit or subsequent in-clinic visit to collect blood sample for antibody testing
- A phone/video call for communication of AAV8 antibody test results
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
- 0 Years 至 25 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males at least 0 to <25 years of age
- •Diagnosis of DMD
- •Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements
排除标准
- •Prior participation in a gene therapy trial OR recipient of a gene therapy drug
- •Other inclusion/exclusion criteria apply
结局指标
主要结局
Prevalence of anti-AAV8 antibodies in patients with DMD
时间窗: 90 days
* To evaluate the prevalence of AAV8 antibodies in patients with DMD * To identify participants who may be eligible for investigational gene therapy clinical trials in males with DMD
次要结局
未报告次要终点