NCT03636438进行中（未招募）不适用
A Long-Term Follow-up Study to Evaluate Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 11
- 试验地点
- 15
- 主要终点
- Number of Participants with Adverse Events and Serious Adverse Events
研究概览
简要总结
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
详细描述
Study 301OTC02 is a long-term follow-up study to evaluate the safety and efficacy of adeno-associated virus (AAV) serotype 8 (AAV8)-mediated gene transfer of human OTC in adults with late-onset OTC deficiency. Only subjects who complete Study 301OTC01 (NCT02991144) are eligible to participate in Study 301OTC02.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 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
- •Completed the Week 52 visit in Study 301OTC
- •Willing and able to provide written informed consent.
- •Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.
排除标准
- •Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study.
- •Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.
结局指标
主要结局
Number of Participants with Adverse Events and Serious Adverse Events
时间窗: Up to 416 weeks
次要结局
- Change from Baseline Over Time in 24-Hour Area Under the Curve for Plasma Ammonia(Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration)
- Change from Baseline Over Time in the Ureagenesis Rate(Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration)
研究者
研究点 (15)
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