2022-501146-30-00招募中2 期
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 Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 4
- 试验地点
- 3
- 主要终点
- The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.
研究概览
简要总结
To determine the long-term safety of DTX301 following a single IV dose in adults with late-onset OTC deficiency.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Overall Study
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Completed the Week 52 visit in Study 301OTC
- •Note that the Day 0 visit of Study 301OTC02 may coincide with the Week 52 visit of 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
The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.
The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.
次要结局
- The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 208 weeks following IV administration of DTX301
- The change from baseline (the baseline of each subject before DTX301 administration in Study 301OTC01) in the rate of ureagenesis (as measured by the generation of [13 C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 416 weeks following IV administration of DTX301 The rate of ureagenesis was assessed prior to Amendment 3; ureagenesis data collected will be analyzed as a secondary efficacy endpoint.
研究者
Medical information
Scientific
Ultragenyx Pharmaceutical Inc.
研究点 (3)
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