Oral Supplementation of Gangliosides - A Potential Treatment for GM3 Synthase Deficiency
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 20
- 主要终点
- Change in scores of standardized developmental assessments (Vineland & Batelle)
研究概览
简要总结
The purpose of this pilot project is to see if a supplemental form of dietary gangliosides can serve as a potential treatment for the rare metabolic condition called ganglioside GM3 synthase deficiency.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of GM3 synthase deficiency
排除标准
- 未提供
研究组 & 干预措施
Supplementation of gangliosides
Supplementation of dairy-derived concentrated gangliosides
干预措施: Supplementation of dairy-derived concentrated gangliosides. (Dietary Supplement)
结局指标
主要结局
Change in scores of standardized developmental assessments (Vineland & Batelle)
时间窗: Quarterly measures - change from baseline over 24 months
Body Weight compared to normal pediatric growth curves
时间窗: Quarterly measurements from baseline over 24 months
Head circumference compared to normal pediatric growth curves.
时间窗: Quarterly measures from baseline over 24 months
Body length compared to normal pediatric growth curves.
时间窗: Quarterly measures- change from baseline over 24 months.
次要结局
- Concentration of ganglioside GM3 in blood plasma(Six times per year over 24 months)