Multicentre Study Project to Evaluate the New Generation of Protein Supplements With Glycomacropeptide (GMP) in Patients With Phenylketonuria (PKU) Aged 10 Years and Older Over a 16 Week Period.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Change in blood Phe
研究概览
简要总结
This study aims to measure changes in participants' blood phenylalanine (Phe) levels in participants with PKU when switching from a conventional amino acid based protein substitute to a GMP based protein substitute (PKU Sphere) over a 16 week period.
详细描述
This is a stepped wedge, open-label study of PKU Sphere for the dietary management of participants with Phenylketonuria. It will be carried out with 33 participants who will be randomised into one of three clusters. The total study length per patient is 16 weeks. Each cluster transitions from taking their standard amino acid product alone to incorporating PKU Sphere (at least 50% of their protein substitue requirement) at different timepoints: week 5, 9 or 13.
Participants will have a face-to-face clinical review at the time of recruitment (week 0) and at 16 weeks (completion of the study). They will also have an additional telephone review when they switch onto PKU Sphere.
Blood samples will be taken at baseline, and then every two weeks for the duration of the study.
In addition to these study visits and procedures, routine clinical care will continue in line with local guidelines.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Single (Participant)
盲法说明
At the point of randomisation every effort will be made to ensure that participants are unaware of the cluster they are randomised to; this is to help ensure that compliance is not affected by participants knowing when they will start the new treatment.
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of PKU made in neonatal period
- •Treated by a low-phenylalanine diet and necessity of intake of at least one protein substitute per day
- •Aged 10 years and above
- •Three blood Phe measurements taken within the preceding six months
- •Willing to replace current amino acid based protein substitute with PKU Sphere
- •Ability to take at least 50% of protein requirements as PKU Sphere
- •Ability to comply with the study protocol, in the opinion of the investigator
- •Willingly given, written, informed consent from patient or parent/guardian
- •Willingly given, written assent (if appropriate)
排除标准
- •Women who are pregnant or planning to become pregnant during the study period
- •Participants with atypical PKU (e.g. BH4 deficient)
- •Intake of sapropterin dihydrochloride (Kuvan) in the six weeks prior to recruitment in the study
- •Patients with soya, milk or fish allergies
- •Any other severe disease
研究组 & 干预措施
4 Weeks
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 4 weeks.
干预措施: PKU Sphere (Dietary Supplement)
8 Weeks
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 8 weeks.
干预措施: PKU Sphere (Dietary Supplement)
12 Weeks
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 12 weeks.
干预措施: PKU Sphere (Dietary Supplement)
结局指标
主要结局
Change in blood Phe
时间窗: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Change in blood Phe concentrations when switching from an amino acid based protein substitute to a GMP based one (PKU Sphere). A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.
次要结局
- Acceptance questionnaire(Weeks 1, 4, 8, 12 and 16)
- Gastrointestinal symptoms questionnaire(Weeks 1, 4, 8, 12 and 16)
- Change in blood tyrosine(Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.)
- Protein substitute intake(Weeks 1, 4, 8, 12 and 16)
- Dietary intake(Weeks 1 and 16.)
- Stool patterns(Weeks 1, 4, 8, 12 and 16)
- Satiety questionnaire(Weeks 1, 4, 8, 12 and 16)