A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.
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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
PKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria.
Oregon Health and Science University
Portland, Oregon, United States
Phase 1: taste
Questionnaire data captured to evaluate taste
Time frame: Days 1 - 7.
Phase 1: smell
Questionnaire data captured to evaluate smell
Time frame: Days 1 - 7.
Phase 1: texture
Questionnaire data captured to evaluate texture
Time frame: Days 1 - 7.
Phase 1: gastrointestinal tolerance
Questionnaires will be completed daily during the 1-week taste test.
Time frame: Days 1 - 7.
Phase 1: phenylalanine concentration
Phenylalanine concentrations measured via blood spot analysis.
Time frame: Days 1 and 7.
Phase 1: tyrosine concentration
Tyrosine concentrations measured via blood spot analysis.
Time frame: Days 1 and 7.
Phase 2: quantitative change in plasma amino acids at day 28
Compare comprehensive plasma amino acid profiles at baseline and end of the trial.
Time frame: Phase 2, day 1 and day 28.
Phase 2: gastrointestinal tolerance
Questionnaires will be completed daily throughout Phase 2.
Time frame: Phase 2, days 1 - 28.
Phase 2: change in 3-day diet record at end of study
A 3-day diet record will be completed in the first 3 days and the last 3 days for comparison.
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Time frame: Phase 2, days 1 - 3 and days 26 - 28.
Phase 2: change in anthropometry at end of study
Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated
Time frame: Phase 2, days 1 and 28.
Phase 2: phenylalanine concentration
Phenylalanine concentrations measured via blood spot analysis.
Time frame: Twice weekly for four weeks during phase 2
Phase 2: tyrosine concentration
Tyrosine concentrations measured via blood spot analysis.
Time frame: Twice weekly for four weeks during phase 2
Phase 2: questionnaire data captured to evaluate change in taste perception
Data captured to evaluate taste
Time frame: Phase 2, days 14 and 28.
Phase 2: questionnaire data captured to evaluate change in smell perception
Data captured to evaluate smell
Time frame: Phase 2, days 14 and 28.
Phase 2: questionnaire data captured to evaluate change in texture perception
Data captured to evaluate texture
Time frame: Phase 2, days 14 and 28.
Phase 1: questoinnaire evaluating subjective measures of satiety
To evaluate if PKU Sphere increases subjective measures of satiety
Time frame: Phase 1, days 1 - 7.
Phase 2: questoinnaire evaluating subjective measures of satiety
To evaluate if PKU Sphere increases subjective measures of satiety
Time frame: Phase 2, days 1, 14 and 28.