Evaluating the Adherence, Tolerance, Acceptability and Safety of a New Amino Acid Based, Microtablet Protein Substitute in PKU: a Case Study Series
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Gastro-intetsinal tolerance
研究概览
简要总结
Phenylalanine (Phe) free protein substitutes are typically presented in ready to drink liquid or powder format and are made up with water to a set volume. Despite recent advancements related to the taste, scent and texture of commercially available protein substitutes, a proportion of PKU patients choose to consume tablet-based protein substitutes. Protein substitutes in tablet format may help overcome taste, scent and texture concerns associated with powdered and liquid preparations. Indeed, previous research has shown that protein substitute tablets are an effective strategy to manage PKU in children, teenagers and adults. Nonetheless, the daily burden and discipline required often impedes compliance, especially alongside the restrictive low-protein diet. Achieving compliance with multivitamin supplements when given alongside protein substitutes therefore becomes especially challenging. This is particularly true for adolescent, adult and maternal populations.
PKU Microtablets have recently been developed. PKU Microtablets are a new phenylalanine-free tablet protein substitute combined with vitamins, minerals and trace elements. As patient acceptability is fundamental to the successful use of any protein substitute, investigation is needed to assess the compliance, tolerance and acceptability of PKU Microtablets as part of a low phenylalanine dietary regimen in PKU patients.
This series of case-studies aims to evaluate the gastrointestinal tolerance, acceptability, compliance, and safety of this protein substitute in both adults and children from 4 years. These case studies will last 29 days in total, including a 1-day baseline period followed by a 28-day intervention period. The case studies will be conducted across multiple specialist metabolic centres in the UK.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female
- •Over 4 years of age
- •Patients diagnosed with any phenotype of PKU characterised by hyperphenylalaninaemia
- •Have been compliant in taking at least one protein substitute, providing at least 20g protein equivalent, for at least 1 month prior to trial commencement
- •Have a prescribed daily phenylalanine allowance for PKU
- •Written or electronic informed consent from patient, and/or from parent/caregiver if applicable
- •Participants who are anticipated to be able to take at least 20g protein equivalent of the case study product per day
排除标准
- •Pregnant or lactating
- •Requiring 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
- •Investigator concern around willingness/ability of patient or parent/caregiver to comply with protocol requirement
- •Treatment with Sapropterin dihydrochloride for less than 6 months
结局指标
主要结局
Gastro-intetsinal tolerance
时间窗: Measured at baseline (day 1) and end of intervention (day 29)
GI tolerance will be recorded by the Dietitian at baseline on Day 1 and at the end of the case study period (Day 29). GI tolerance will be recorded using a standardised questionnaire to capture perceived severity (none, mild, moderate or severe) of common gastrointestinal symptoms (diarrhoea, constipation, bloating, abdominal discomfort, vomiting and nausea).
次要结局
- Acceptability(Measured at baseline (day 1) and end of intervention (day 29))
- Compliance(Measured at baseline (day 1) and end of intervention (day 29))
- Blood Phe and Tyrosine(Day 2 (baseline) and day 29 (end of intervention))
- Anthropometry - Height(Day 1 (baseline) and day 29 (end of intervention))
- Anthropometry - Weight(Day 1 (baseline) and day 29 (end of intervention))
- Patient History(Day 1 (baseline) and day 29 (end of intervention))
- Safety/ Adverse events(Throughout the case study series (day 1 to day 29).)