Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 6
- 主要终点
- Nutritional Status (objective measure)
研究概览
简要总结
This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. Fifty eligible adults (≥ 16 years) with proven phenylketonuria or hyperphenylalaninemia will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.
详细描述
This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. In particular, this randomised controlled trial aims to evaluate the efficacy (changes relating to nutritional status and metabolic control) of this new protein substitute, while also capturing data pertaining to tolerance, compliance, safety and acceptability. Designed for poorly compliant adult patients with proven phenylketonuria or hyperphenylalaninemia, this new protein substitute is composed of an adapted mixture of other essential and non-essential amino acids, carbohydrates, vitamins and selected minerals and trace elements and enriched with docosahexaenoic acid (DHA). Fifty eligible adults (≥ 16 years) will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female
- •Over 16 years of age
- •Diagnosed with proven PKU or hyperphenylalaninemia with an increased phenylalanine-tolerance/intake
- •Currently taking a maximum of 1 protein substitute per day (equal to 20g protein equivalent)
- •Have a minimum blood phenylalanine level of ≥ 600 umol/L (for PKU patients)
- •Have relaxed (if not stopped) their dietary and protein substitute regimen for at least 1 month prior to trial commencement
- •Have Written informed consent from patient
排除标准
- •Pregnant or lactating
- •Requiring nutritional support (including enteral and parenteral nutrition)
- •Major hepatic or renal dysfunction
- •Participation in other studies within 1 month prior to entry of this study
- •Allergy to any of the study product ingredients, including milk protein or soya
- •Investigator concern around willingness/ability of patient to comply with protocol requirements
结局指标
主要结局
Nutritional Status (objective measure)
时间窗: 2 days
Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)
次要结局
- Nutritional Status (Objective measure)(2 days)
- Daily compliance with prescribed protein substitute as assessed by standarised questionnaire(31 days)
- Nutritional Status (subjective measure)(6 days)
- Gastrointestinal tolerance as assessed by standarised questionnaire(12 days)
- Metabolic Control(2 days)
- Anthropometry(2 days)
- Acceptability as assessed by standarised questionnaire(2 days)
- Subjective Mood(3 days)