A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 6
- 主要终点
- Daily phenylalanine control
研究概览
简要总结
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.
详细描述
This is an assessment of ten (10) infants who require a protein restricted diet that is low in Phe. Infants who routinely use a Phe free infant formula as part of their dietary therapy will be recruited for a 28-day assessment of PKU Start, to evaluate tolerance and acceptability.
The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK.
The participant's dietitian will advise on an appropriate amount of PKU Start based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free infant formula with PKU Start for one (1) month. The sponsor will supply PKU Start free of charge.
Prior to starting PKU Start, parents/carers will be asked to record information about the infant's usual GI tolerance and feeding pattern for a period of three (3) days, to allow for comparison between their existing formula and PKU Start.
They will be asked to record information about the following:
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
- 4 Weeks 至 2 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
- •Taking a minimum of one (1) feed of a Phe-free infant formula
- •A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents
排除标准
- •Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
- •Diagnosis of a congenital condition
结局指标
主要结局
Daily phenylalanine control
时间窗: Days 1-28
Collection of quantitative data regarding phenylalanine control using routine biochemical testing
GI tolerance daily diary
时间窗: Days 1-28
Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product.
Product compliance daily diary
时间窗: Days 1-28
Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.
Ease of use questionnaire
时间窗: Day 29
Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
次要结局
未报告次要终点