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Completed

NCT Number: NCT03419819

Evaluation of PKU Sphere

A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.

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Key information

Conditions

Phenylketonurias Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Oregon Health and Science University

Portland, Oregon, 97239, United States

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

PKU sphere

Dietary Supplement

PKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria.

Primary outcomes

  1. Phase 1: taste

    Time frame: Days 1 - 7.

    Questionnaire data captured to evaluate taste

  2. Phase 1: smell

    Time frame: Days 1 - 7.

    Questionnaire data captured to evaluate smell

  3. Phase 1: texture

    Time frame: Days 1 - 7.

    Questionnaire data captured to evaluate texture

  4. Phase 1: gastrointestinal tolerance

    Time frame: Days 1 - 7.

    Questionnaires will be completed daily during the 1-week taste test.

  5. Phase 1: phenylalanine concentration

    Time frame: Days 1 and 7.

    Phenylalanine concentrations measured via blood spot analysis.

  6. Phase 1: tyrosine concentration

    Time frame: Days 1 and 7.

    Tyrosine concentrations measured via blood spot analysis.

  7. Phase 2: quantitative change in plasma amino acids at day 28

    Time frame: Phase 2, day 1 and day 28.

    Compare comprehensive plasma amino acid profiles at baseline and end of the trial.

  8. Phase 2: gastrointestinal tolerance

    Time frame: Phase 2, days 1 - 28.

    Questionnaires will be completed daily throughout Phase 2.

  9. Phase 2: change in 3-day diet record at end of study

    Time frame: 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.

  10. Phase 2: change in anthropometry at end of study

    Time frame: Phase 2, days 1 and 28.

    Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated

  11. Phase 2: phenylalanine concentration

    Time frame: Twice weekly for four weeks during phase 2

    Phenylalanine concentrations measured via blood spot analysis.

  12. Phase 2: tyrosine concentration

    Time frame: Twice weekly for four weeks during phase 2

    Tyrosine concentrations measured via blood spot analysis.

  13. Phase 2: questionnaire data captured to evaluate change in taste perception

    Time frame: Phase 2, days 14 and 28.

    Data captured to evaluate taste

  14. Phase 2: questionnaire data captured to evaluate change in smell perception

    Time frame: Phase 2, days 14 and 28.

    Data captured to evaluate smell

  15. Phase 2: questionnaire data captured to evaluate change in texture perception

    Time frame: Phase 2, days 14 and 28.

    Data captured to evaluate texture

Secondary outcomes

  1. Phase 1: questoinnaire evaluating subjective measures of satiety

    Time frame: Phase 1, days 1 - 7.

    To evaluate if PKU Sphere increases subjective measures of satiety

  2. Phase 2: questoinnaire evaluating subjective measures of satiety

    Time frame: Phase 2, days 1, 14 and 28.

    To evaluate if PKU Sphere increases subjective measures of satiety

Sponsors and collaborators

Lead sponsor

Vitaflo International, Ltd

Industry

Collaborators

  • Oregon Health and Science University

Registry information

Official study title

Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Feb 5, 2018
Registry last updated
Feb 16, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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