Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life: Acceptability and Efficacy of a New Protein Substitute
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 46
- 试验地点
- 3
- 主要终点
- Intake of energy and nutritients: Weaning
研究概览
简要总结
In this study the feeding patterns and practices at the introduction of a second stage phe-free protein substitute will be assessed in young children with PKU. This will be compared with the normal weaning process in healthy non-PKU age-matched children. In addition, tolerability and acceptability of the study product will be evaluated in a smaller group of subjects who have already been transferred onto a second stage protein substitute. The study is performed in 3 centers in the United Kingdom.
详细描述
Part 1:
For subjects with PKU, the study product will be incorporated in the diet as the second stage protein substitute until the subjects are 2 years of age.
The healthy non-PKU age-matched subjects will not have the study product incorporated in their diet in order to be able to compare the weaning experience and nutritional intake of PKU subjects with non-PKU subjects. The healthy subjects will also be part of the study until the subjects are 2 years of age.
Part 2:
All PKU subjects will take the study product for 7 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of PKU identified on screening during the newborn period and requiring a phenylalanine free protein substitute
- •Subjects who are about to commence weaning and require a concentrated phe-free protein substitute to meet their total protein requirements and are aged between 4 and 10 months (Part 1)
- •Subjects aged under 5 years who have already been fully transferred onto a concentrated phe-free protein substitute and are willing to try the study product for 7 days (Part 2)
- •Written informed consent obtained from subject or parents / caregiver
- •May or may not have commenced weaning solids (i.e. using normal foods)
排除标准
- •Presence of serious concurrent illness
- •Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- •Any child who has not commenced a second stage protein substitute and is over the age of 10 months
- •Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study
- •Any children having taken antibiotics over the previous 2 weeks leading up to the study
研究组 & 干预措施
Part 1
Part 1 of the study will recruit subjects who are about to be introduced to a concentrated phe-free protein substitute (i.e. second stage protein substitute) for the first time.
干预措施: B2970 Metabolic Texture Modified Protein Substitute (Other)
Part 2
Part 2 of the study will recruit subjects who have already been introduced to a concentrated phe-free protein substitute (i.e. have already transferred from phe-free infant formula onto a second stage protein substitute). This group will be included to evaluate the acceptability of the study product in patients who have already moved onto a second stage protein substitute.
干预措施: B2970 Metabolic Texture Modified Protein Substitute (Other)
结局指标
主要结局
Intake of energy and nutritients: Weaning
时间窗: up to 2 years
Intakes of energy and nutrients from the weaning diet: descriptive information, intakes \[g/day\]
Intake of energy and nutritients: Study product
时间窗: up to 2 years
Intakes of energy and nutrients from the study product \[diet diary; #g product/day\]
Feeding practices and difficulties by questionnaire; position
时间窗: up to 2 years
Feeding practices entered by parents in diaries: the way the child has been fed will be assessed : categories \[sitting position e.g on lap, in chair etc\]
Feeding patterns- product intake
时间窗: up to 2 years
Actual intake of product and prescribed amount of product \[# sachets\] will be recorded as documented in diaries.
Feeding patterns - intake other than study product
时间窗: up to 2 years
Intakes of standard formulas / breast-milk and normal foods \[mL\] or \[g/day\] and \[frequency\] of breastfeeding, as documented in diaries.
Feeding practices and difficulties by questionnaire; self feeding
时间窗: up to 2 years
Feeding practices entered by parents in diaries; the way the child has been fed will be assessed : \[self-feeding; parent feeding\]; \[type of self feeding: bottle, finger; spoon, fork\]
Occurence of Adverse Events [ Safety and tolerability]
时间窗: up to 2 years
Occurence of Adverse Events
Gastro-Intestinal symptoms
时间窗: up to 2 years
Occurence of GI symptoms \[yes/no\] for: * Mouth Ulcers * Tummy upset * Harder poos * Constipation * Softer poos * Vomiting * Colic * Other (free entry)
Product Acceptability yes/no
时间窗: up to 2 years
Acceptability questions \[yes/no\] for: * Refuses more than takes * Refuses as much as takes * Takes more than refuses * Spits out product * Puts spoon away * Holds in mouth but won't swallow * Closes mouth on feeding * Cries at beginning of feed * Cries at end of feed * Deliberately spills feed * Turns head away * Other (free entry)
Blood Phenylalanine control: Phe levels
时间窗: up to 2 years
Phe levels \[umol/L\]
Blood Tyrosine levels
时间窗: up to 2 years
Tyrosine levels \[umol/L\]
Anthropometry: weight
时间窗: up to 2 years
Weight \[g\]
Anthropometry: length
时间窗: up to 2 years
Length \[cm\]
次要结局
- Anxiety levels of parents - Beck's Anxiety inventory(up to 2 years)
- Weaning per group (with our without protein substitute) - descriptive(up to 2 years)
- Coping mechanisms of parents by questionnaire(up to 2 years)
- Parent experience of Feeding via questionnaire(up to 2 years)