An Exploratory Study to Evaluate the Acceptability of PKU Explore, a Food for Special Medical Purposes, for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 3 Years of Age With Regard to Product-acceptability, Tolerance, Phe Levels and Growth
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 2
- 主要终点
- Gastrointestinal tolerance daily diary as reported by the patient
研究概览
简要总结
PKU explore France is an exploratory study to evaluate the acceptability of PKU explore, a food for special medical purposes, for use in the dietary management of phenylketonuria in infants from 6 months to 3 years of age, assessing participant adherence, GI tolerance, phe levels, growth and product palatability.
详细描述
The study product, PKU explore, is a concentrated, spoonable, protein substitute food for special medical purposes, used in the dietary management of PKU. The recommended amount will be determined by a dietitian or clinician and the product used is unflavoured.
PKU explore France is an exploratory study to evaluate the gastrointestinal tolerance, palatability, and participant adherence over a four-week period in patients with PKU that follow an appropriate restricted therapeutic diet. This will be followed by a maximum 24-month follow-up period monitoring growth as per routine standard of care.
Up to 10 participants will be recruited at a single centre in France.
For 28 consecutive days, up to 10 participants, aged 6 months to 3 years inclusive, take their usual restricted therapeutic diet, either replacing their usual second-stage, concentrated protein substitute with PKU explore or commencing PKU explore and gradually reducing their Phe-free formula for infants.
Their parent/guardian complete a daily questionnaire on adherence and tolerance, and a final questionnaire on usage and acceptability.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 3 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid protein substitute.
- •Aged between 6 months and 3 years (inclusive at screening).
- •Already taking part of their protein substitute in a spoonable form OR is at the stage in their PKU management when a second stage spoonable protein substitute is recommended to commence.
- •Well-controlled PKU, evidenced by the latest three routine blood spots being within the acceptable range, in the investigator's opinion.
- •Able to comply with the study protocol and take the study product, according to the opinion of the investigator.
- •Willingly given, written, informed consent from parents/guardian.
排除标准
- •Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and Phe-free L-amino acid supplements.
- •Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.
- •Known milk or fish allergy/intolerance.
- •Patients who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to the screening visit.
- •Existing significant GI issues which may affect compliance with the study protocol, according to the opinion of the investigator.
- •Any medical conditions precluding the study intervention, which in the opinion of the investigator may impact on metabolic control during the study period.
- •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).
- •Where applicable, patients NOT covered by Health Insurance System and/or not in compliance with the recommendations of National Law in force.
结局指标
主要结局
Gastrointestinal tolerance daily diary as reported by the patient
时间窗: Days 1 - 28
Qualitative assessments from subject questionnaires to allow evaluation of any self-reported gastrointestinal symptoms during the study period.
Adherence to the recommended amount of study product
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), measured in grams consumed per day.
Product palatability rated on a Likert scale by the patient after 28 days
时间窗: Day 28
Patient assessment of study product's palatability using a Likert scale. At the end of the 28-day acceptability phase, participants/parents/guardians will be required to complete a Product Acceptability Questionnaire to record perceptions about: the appearance, smell, taste, aftertaste, texture, packaging/presentation of the product, also the ease of preparation and administration. These will be on a 5-point Likert scale as Loved it, Liked it, Neither liked nor disliked it, Didn't like it and Really didn't like it.
Change in Phe levels
时间窗: Previous three routine results prior to Visit 1, Visit 1 (Day 0), Week 1, Week 2, Week 3, Week 4 and Visit 2 (Day 28)
Analysis of results from routine dried blood spots. Change in Phe level at diagnosis and at different timepoints measured as µmol/l.
次要结局
- Evaluation of growth for a minimum of two years follow up period(Visit 1 (day 0), Visit 2 (Day 28) and Every 4 months for a max. 24 Months' Followup)
- Product adherence for a minimum of two years follow up period by measuring the quantity of product intake(days 1 - 28)
- Product adherence for a minimum of two years follow up period by measuring the Phe levels(Visit 1 (Day 0), Week 1, Week 2, Week 3, Week 4, Visit 2 (Day 28) and every 4 months for a max. 24 Months' Follow-up period)