Evaluation of PKU Sphere
- Conditions
- Phenylketonurias
- Interventions
- Dietary Supplement: PKU Sphere
- Registration Number
- NCT03419819
- Lead Sponsor
- Vitaflo International, Ltd
- 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).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 28
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description PKU Sphere PKU Sphere Phase 1: 1 week To evaluate the acceptability of PKU Sphere during a short-term (1 week) period. Individuals with PKU will aim to consume a minimum of 30% of the medical food component of the diet as PKU Sphere. The amount will be assessed and advised on an individual basis. Phase 2: 4 weeks To evaluate longer-term acceptability and metabolic control in individuals with PKU consuming an agreed target of 50 - 100% of their medical food component of the diet as PKU Sphere for 4 weeks. Some individuals, particularly young children between the ages of 3 - 6 years, may require a 1 - 3 week build up period to reach target volume which will be assessed on an individual basis.
- Primary Outcome Measures
Name Time Method Phase 2: questionnaire data captured to evaluate change in smell perception Phase 2, days 14 and 28. Data captured to evaluate smell
Phase 1: tyrosine concentration Days 1 and 7. Tyrosine concentrations measured via blood spot analysis.
Phase 1: taste Days 1 - 7. Questionnaire data captured to evaluate taste
Phase 1: gastrointestinal tolerance Days 1 - 7. Questionnaires will be completed daily during the 1-week taste test.
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 2: questionnaire data captured to evaluate change in taste perception Phase 2, days 14 and 28. Data captured to evaluate taste
Phase 2: gastrointestinal tolerance Phase 2, days 1 - 28. Questionnaires will be completed daily throughout Phase 2.
Phase 1: phenylalanine concentration Days 1 and 7. Phenylalanine 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: 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.
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: questionnaire data captured to evaluate change in texture perception Phase 2, days 14 and 28. Data captured to evaluate texture
- Secondary Outcome Measures
Name Time Method Phase 1: questoinnaire evaluating subjective measures of satiety Phase 1, days 1 - 7. To evaluate if PKU Sphere increases subjective measures of satiety
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
Trial Locations
- Locations (1)
Oregon Health and Science University
🇺🇸Portland, Oregon, United States