Evaluating the Adherence, Tolerance, Acceptability and Safety of New GMP-based Powdered Protein Substitutes in PKU and TYR: a Case Study Series
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Gastro-intetsinal tolerance
研究概览
简要总结
Four new GMP-based protein substitutes have been developed to support the dietary management of PKU and TYR. These products are powdered protein substitutes, low in phenylalanine and low in phenylalanine and tyrosine respectively, with a mix of glycomacropeptide (GMP), essential and non-essential amino acids, carbohydrates, fibres, fats (including DHA) and micronutrients. The proteins in these new protein substitutes are based on GMP, a peptide isolated from milk during cheese manufacturing and the only known naturally derived protein source low in phenylalanine and tyrosine. The GMP is supplemented with other amino acids which are naturally low or not present in GMP, as well as carbohydrates, fats, fibre and micronutrients. Studies to date have illustrated that PKU patients who replace their regular phenylalanine-free amino acid-based formula with GMP-based foods may have better diet compliance and prefer the taste whilst maintaining metabolic control.
This series of case-studies aims to evaluate the gastrointestinal tolerance, acceptability, compliance, and safety of these four GMP based protein substitutes in both adults and children over 3 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. A series of case studies is undertaken due to the rarity of these conditions, the diversity of patient types and the difficulty in recruiting these patients to trials.
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
- 3 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 3 years of age
- •Patients diagnosed with any phenotype of PKU characterised by hyperphenylalaninaemia or patients diagnosed with tyrosinaemia
- •Have been compliant in taking at least one protein substitute, providing at least 15g protein equivalents, for at least 1 month prior to trial commencement
- •Have a prescribed daily phenylalanine or phenylalanine and tyrosine allowance for PKU or TYR respectively
- •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 one sachet of GMP 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
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). Information about appearance of stools will also be collected using the Bristol Stool Chart© at the same time points.
次要结局
- Safety/ Adverse Events(Throughout the case study series (day 1 to day 29).)
- Compliance(Measured at baseline (day 1) and end of intervention (day 29))
- Acceptability(Measured at baseline (day 1) and end of intervention (day 29))
- Blood phenylalanine, blood tyrosine and other amino acid levels(Day 1 (baseline) and day 29 (end of intervention))
- Anthropometry(Day 1 (baseline) and day 29 (end of intervention))
- Patient history(Day 1 (baseline) and day 29 (end of intervention))