EUCTR2018-004473-27-ES进行中（未招募）1 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 Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
ltragenyx Pharmaceutical Inc.0 个研究点目标入组 12 人开始时间: 2021年6月14日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Received DTX401 in Study 401GSDIA01.
- •2. Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed.
- •3. Willing and able to comply with all scheduled study visits, procedures, and requirements.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 11
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
- •2. Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.
研究者
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