NCT03858101已完成不适用
An Exploratory Matched Case-control Study to Measure Blood Nutrient Levels of Adult PKU Patients on a Protein Substitute.
Nutricia Research4 个研究点 分布在 3 个国家目标入组 71 人开始时间: 2019年4月15日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 71
- 试验地点
- 4
- 主要终点
- Blood nutritional status
研究概览
简要总结
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). PKU patients have specific dietary needs and must follow a restrictive diet in the aim of preventing toxic levels of the amino acid phenylalanine (Phe) accumulation.
详细描述
The aim of this explorative cross-sectional study is to gain quantitative insights on blood nutrient levels of adult PKU patients on a protein substitute.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Both PKU and Non-PKU comparison subjects:
- •Age ≥18 years
- •Willing and able to provide signed informed consent PKU specific inclusion criteria
- •PKU patients identified by newborn screening and started low Phe diet before 1 month age
- •Usage of at least one Phe-free protein substitute (i.e. an amino acid mixture including micronutrients) on a daily basis for at least 26 consecutive weeks up to Visit 1
- •Non-PKU comparison subjects specific inclusion criteria:
- •Same age- (±3 years) and sex as an included PKU subject
排除标准
- •Both PKU and Non-PKU comparison subjects:
- •For women: Currently pregnant or lactating
- •Current psychiatric disorders
- •Current Substance Use Disorders (as described in DSM V)
- •Current use of psychotropic and/or inotropic medication
- •Omega-3, antioxidant, (multi)vitamin and/or (multi)mineral supplement use within six weeks prior to entry in the study
- •Severe hepatic, thyroid or renal dysfunction
- •No acute illnesses like flu, diarrhea, or vomiting (subjects should be symptom free for a week prior to V1)
- •Participation in any other clinical intervention studies involving test products concomitantly or within six weeks prior to entry into the study
- •Other family members taking part in this study
- •PKU subject specific exclusion criteria:
- •Use of BH4, or drugs that may interfere with main outcomes
- •Non-PKU comparison subjects specific exclusion criteria:
- •Any condition or special diet (e.g. vegan or vegetarian diet) that effects the metabolism and/or normal dietary pattern/intake
- •A first or second degree relative with inborn errors of metabolism
- •Living together with someone with inborn errors of metabolism (e.g. partner, spouse or roommate)
结局指标
主要结局
Blood nutritional status
时间窗: day 1
Micro-and macronutrients and amino acid levels \[in a.o. mg/L\]
次要结局
- Nutrient intake(day 1 - day 7)
- Subjective cognitive well-being(day 1 - day 7)
- Blood nutritional status(day 1)
研究者
研究点 (4)
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