NCT04909346终止不适用
Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 51
- 试验地点
- 5
- 主要终点
- Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa
研究概览
简要总结
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease
详细描述
The study is primarily designed to follow a virtual model, in which Screening will take place over a telephone/video call. The study will comprise a single home health visit, either on the same day as Screening or within 30 days of enrollment. A safety follow-up may occur up to 5 days after Day 1 assessments are completed.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of OTC deficiency, GSDIa, or Wilson Disease.
- •Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
排除标准
- •Prior exposure to an AAV-based gene therapy.
- •Concurrent or previous participation in another Ultragenyx clinical study.
- •Recipient of a liver transplant.
- •Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
结局指标
主要结局
Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa
时间窗: Up to 35 days
Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease
时间窗: Up to 35 days
次要结局
未报告次要终点
研究者
研究点 (5)
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