Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 28
- 试验地点
- 2
- 主要终点
- Phase 1: tyrosine concentration
研究概览
简要总结
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.
详细描述
The rationale for this study is to assess acceptability, adherence and metabolic control in individuals with PKU consuming PKU Sphere, a GMP-based medical food.
The sponsor developed PKU Sphere in response to growing interest in GMP medical foods for the purpose of improving adherence and quality of life by offering an alternative to amino acid based medical foods. PKU Sphere is a powdered, low phenylalanine medical food containing a balanced mix of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP); docosahexaenoic acid (DHA). PKU Sphere has been designed for use in the dietary management of Phenylketonuria. It is available in two flavors, Red Berry and Vanilla, packaged in individual serving sachets of 35g and containing 20g protein equivalent (PE).
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
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of PKU, identified by newborn screening (NBS) and treated since birth.
- •Ages 3 years and above (this includes pregnant women who will be assessed on an individual basis by the Co-Principal Investigators).
- •Currently or previously consuming a low phenylalanine diet supplemented with a medical food designed for the dietary management of PKU.
- •English as primary language.
- •Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/caregiver for participants < 18 years.
- •Willingly given, written assent (if appropriate) for those < 18 years.
排除标准
- •Currently consuming 100% of the medical food component of the diet as PKU sphere.
- •An inability, in the opinion of the investigator, to comply with the requirements of the protocol.
- •Any other type of inherited metabolic disease.
结局指标
主要结局
Phase 1: tyrosine concentration
时间窗: Days 1 and 7.
Tyrosine concentrations measured via blood spot analysis.
Phase 1: taste
时间窗: Days 1 - 7.
Questionnaire data captured to evaluate taste
Phase 1: gastrointestinal tolerance
时间窗: Days 1 - 7.
Questionnaires will be completed daily during the 1-week taste test.
Phase 1: smell
时间窗: Days 1 - 7.
Questionnaire data captured to evaluate smell
Phase 1: texture
时间窗: Days 1 - 7.
Questionnaire data captured to evaluate texture
Phase 2: questionnaire data captured to evaluate change in taste perception
时间窗: Phase 2, days 14 and 28.
Data captured to evaluate taste
Phase 2: questionnaire data captured to evaluate change in smell perception
时间窗: Phase 2, days 14 and 28.
Data captured to evaluate smell
Phase 2: gastrointestinal tolerance
时间窗: Phase 2, days 1 - 28.
Questionnaires will be completed daily throughout Phase 2.
Phase 1: phenylalanine concentration
时间窗: Days 1 and 7.
Phenylalanine concentrations measured via blood spot analysis.
Phase 2: quantitative change in plasma amino acids at day 28
时间窗: Phase 2, day 1 and day 28.
Compare comprehensive plasma amino acid profiles at baseline and end of the trial.
Phase 2: phenylalanine concentration
时间窗: Twice weekly for four weeks during phase 2
Phenylalanine concentrations measured via blood spot analysis.
Phase 2: tyrosine concentration
时间窗: Twice weekly for four weeks during phase 2
Tyrosine concentrations measured via blood spot analysis.
Phase 2: change in 3-day diet record at end of study
时间窗: Phase 2, days 1 - 3 and days 26 - 28.
A 3-day diet record will be completed in the first 3 days and the last 3 days for comparison.
Phase 2: change in anthropometry at end of study
时间窗: Phase 2, days 1 and 28.
Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated
Phase 2: questionnaire data captured to evaluate change in texture perception
时间窗: Phase 2, days 14 and 28.
Data captured to evaluate texture
次要结局
- Phase 1: questoinnaire evaluating subjective measures of satiety(Phase 1, days 1 - 7.)
- Phase 2: questoinnaire evaluating subjective measures of satiety(Phase 2, days 1, 14 and 28.)