Phenylketonurias
Conditions
Keywords
Phenylketonuria, GMP, Sphere, Child, Adult, Phenylalanine, PKU, Glycomacropeptide
Brief summary
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.
Detailed description
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).
Interventions
DIETARY_SUPPLEMENTPKU Sphere
PKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria.
Sponsors
Oregon Health and Science University
Vitaflo International, Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Two phase study consisting of a 1 week taste introduction period followed by a 4 week period assessing acceptability and metabolic control.
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Phase 2: questionnaire data captured to evaluate change in texture perception | Phase 2, days 14 and 28. | Data captured to evaluate texture |
| Phase 2: questionnaire data captured to evaluate change in taste perception | Phase 2, days 14 and 28. | Data captured to evaluate taste |
| Phase 2: questionnaire data captured to evaluate change in smell perception | Phase 2, days 14 and 28. | Data captured to evaluate smell |
| Phase 1: taste | Days 1 - 7. | Questionnaire data captured to evaluate taste |
| Phase 1: smell | Days 1 - 7. | Questionnaire data captured to evaluate smell |
| Phase 1: texture | Days 1 - 7. | Questionnaire data captured to evaluate texture |
| Phase 1: gastrointestinal tolerance | Days 1 - 7. | Questionnaires will be completed daily during the 1-week taste test. |
| Phase 1: phenylalanine concentration | Days 1 and 7. | Phenylalanine concentrations measured via blood spot analysis. |
| Phase 1: tyrosine concentration | Days 1 and 7. | Tyrosine concentrations measured via blood spot analysis. |
| Phase 2: quantitative change in plasma amino acids at day 28 | Phase 2, day 1 and day 28. | Compare comprehensive plasma amino acid profiles at baseline and end of the trial. |
| Phase 2: gastrointestinal tolerance | Phase 2, days 1 - 28. | Questionnaires will be completed daily throughout Phase 2. |
| Phase 2: change in 3-day diet record at end of study | 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 | Phase 2, days 1 and 28. | Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated |
| Phase 2: phenylalanine concentration | Twice weekly for four weeks during phase 2 | Phenylalanine concentrations measured via blood spot analysis. |
| Phase 2: tyrosine concentration | Twice weekly for four weeks during phase 2 | Tyrosine concentrations measured via blood spot analysis. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Phase 2: questoinnaire evaluating subjective measures of satiety | Phase 2, days 1, 14 and 28. | To evaluate if PKU Sphere increases subjective measures of satiety |
| Phase 1: questoinnaire evaluating subjective measures of satiety | Phase 1, days 1 - 7. | To evaluate if PKU Sphere increases subjective measures of satiety |
Countries
United States
Outcome results
None listed