Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I (GSD-DIET)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 3
- 主要终点
- Change in lactate during the dietary intervention compared to baseline under the usual diet
研究概览
简要总结
The present project will specifically assess metabolic effects of dietary interventions with controlled intake of fructose and fructose/galactose in GSDI, with the aim to provide evidence whether relaxed dietary restrictions of fructose and galactose may be justified in treatment recommendations at least for adults, which would considerably enlarge food choice in everyday life of the patients with an expected positive impact on the quality of life of patients with this rare disorder.
详细描述
To assess relaxed restriction of fructose and fructose/galactose intake on secondary metabolic alterations in GSDI, (i) by looking at the traditional parameters for assessing metabolic control in clinical chemistry (lactate, triglycerides, uric acid), and (ii) by using a broad analytical approach relying on targeted metabolomics/lipidomics.
It is hypothesized that relaxed restrictions on the intake of fructose and/or galactose as part of the diet in everyday life may lead to an increase in blood lactate levels (=primary outcome), triglycerides, and uric acid to a certain degree compared to baseline. However, this increase is expected to remain within a range that is not clinically relevant for adult patients, especially when fructose/galactose intake is not excessive and stays within the usual daily allowances for healthy individuals, as planned in this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically and/or enzymatically confirmed diagnosis of GSDI (GSDIa or GSDIb)
- •Male or female ≥ 18y
- •Restriction of fructose intake in usual dietary treatment
- •Written informed consent
排除标准
- •Non-compliance with routine dietary treatment
- •Pregnancy or lactation
- •Liver transplant
- •Recurrent hospitalisations due to metabolic decompensation within the last 12 months
- •Severe chronic kidney disease with glomerular filtration rate (GFR) < 30 ml/min
- •For GSDIb: Severe, uncontrolled symptomatic inflammatory bowel disease
研究组 & 干预措施
Diet with additional fructose and galactose intake
干预措施: Fructose and galactose (Dietary Supplement)
Diet with additional fructose intake
干预措施: Fructose (Dietary Supplement)
结局指标
主要结局
Change in lactate during the dietary intervention compared to baseline under the usual diet
时间窗: 4 Weeks
Lactate belongs to the parameters of secondary metabolic disturbance traditionally used to estimate metabolic control in GSDI in routine clinical practice (lactate, triglycerides, uric acid). Redundant measurements of lactate are performed in blood as well as in collected urine.
次要结局
- Change in plasma uric acid during the dietary intervention compared to baseline (as parameters traditionally measured together with lactate to estimate overall metabolic control)(4 Weeks)
- Plasma metabolite changes in targeted metabolomics during the dietary intervention compared to baseline measurements.(4 Weeks)
- Change in plasma triglycerides during the dietary intervention compared to baseline (as parameters traditionally measured together with lactate to estimate overall metabolic control)(4 Weeks)
- Plasma metabolite changes in targeted lipidomics during the dietary intervention compared to baseline measurements.(4 Weeks)