CTIS2023-504004-29-00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) - 401GSDIA02
ltragenyx Pharmaceutical Inc.0 个研究点目标入组 12 人开始时间: 2023年12月15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
- 年龄范围
- 18 至 65+（—）
- 性别
- All
入选标准
- •Individuals eligible to participate in this study must meet all of the following criteria: 1. Received DTX401 in Study 401GSDIA01, 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, Willing and able to comply with all scheduled study visits, procedures, and requirements
排除标准
- •Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study: 1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study, 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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