NCT04574830已完成不适用
Clinical Survey Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 4
- 主要终点
- Urine hexose tetrasaccharide (Hex4): mean and variance
研究概览
简要总结
The primary objective of this study is to evaluate potential biomarkers of GSD III.
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
- 5 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed historical diagnosis of GSD III based on pathogenic mutations in the AGL gene on both alleles or GDE deficiency based on biopsy of liver, muscle, or fibroblasts
- •Willing to comply with all study procedures
- •Willing and able to provide written informed consent. If a minor, willing and able to provide written assent and have a legally authorized representative willing and able to provide written informed consent
排除标准
- •Presence or history of any condition that, in the view of the Investigator, places the subject at high risk of poor study compliance, interferes with study participation, or interferes with the subject's ability to safely or reliably complete the study assessments
- •Use of any IP within 30 days prior to informed consent/assent or at the time of enrollment
结局指标
主要结局
Urine hexose tetrasaccharide (Hex4): mean and variance
时间窗: Up to Day 35
次要结局
未报告次要终点
研究者
研究点 (4)
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