A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years 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
- 22
- 试验地点
- 6
- 主要终点
- Product compliance daily diary
研究概览
简要总结
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.
详细描述
This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU.
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 Explore based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free protein substitute with PKU Explore for one (1) month. The sponsor will supply PKU Explore free of charge.
Parents/guardians will be asked to record information about the following:
Daily PKU Explore intake Stools Vomiting and Spit-up Feed / Fluid Intake Compliance Phenylalanine Levels (from their routine blood spot testing)
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
- 6 Months 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •i. A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid supplements.
- •ii. Aged between 6 months and 5 years.
- •iii. Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence.
- •iv. Willingly given, written, informed consent from parent/guardian.
- •v. Willingly given, written assent (if appropriate).
排除标准
- •i. Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment with a low protein diet and Phe-free L-amino acid supplements.
- •ii. Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.
- •iii. Participation in any other clinical trial/acceptability study.
- •iv. Any serious medical precluding the study intervention.
- •v. Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).
结局指标
主要结局
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.
GI tolerance daily diary assessing change
时间窗: Days 1-7 and 22-28
Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.
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.
Weekly phenylalanine control
时间窗: Days 1-28
Collection of quantitative data regarding phenylalanine control using routine biochemical testing
次要结局
未报告次要终点