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Evaluation of PKU Sphere

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ClinicalTrials.gov Identifier: NCT03419819
Recruitment Status : Recruiting
First Posted : February 2, 2018
Last Update Posted : May 3, 2018
Sponsor:
Collaborator:
Oregon Health and Science University
Information provided by (Responsible Party):
Vitaflo International, Ltd

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

Condition or disease Intervention/treatment Phase
Phenylketonurias Dietary Supplement: PKU Sphere Not Applicable

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).


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
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.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).
Actual Study Start Date : May 1, 2018
Estimated Primary Completion Date : May 1, 2020
Estimated Study Completion Date : November 1, 2020


Arm Intervention/treatment
Experimental: 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.

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




Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03419819


Contacts
Contact: Elissaveta Johnson 1 571 216 0601 elissaveta.johnson@vitaflousa.com
Contact: Rachel Coode 1 571 481 8833 rachel.coode@vitaflousa.com

Locations
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Sandra Van Calcar, PhD    503-494-5500    vancalca@ohsu.edu   
Contact: Joyanna Hansen, PhD    1 503 494 4263    hansejo@ohsu.edu   
Principal Investigator: Sandra Van Calcar, PhD         
Principal Investigator: Joyanna Hansen, PhD         
Sponsors and Collaborators
Vitaflo International, Ltd
Oregon Health and Science University
Investigators
Principal Investigator: Sandra Van Calcar, PhD Oregon Health and Science University
Principal Investigator: Joyanna Hansen, PhD Oregon Health and Science University

Responsible Party: Vitaflo International, Ltd
ClinicalTrials.gov Identifier: NCT03419819     History of Changes
Other Study ID Numbers: SCT-PKUS-2016-11-28
First Posted: February 2, 2018    Key Record Dates
Last Update Posted: May 3, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Vitaflo International, Ltd:
Phenylketonuria
GMP
Sphere
Child
Adult
Phenylalanine
PKU
Glycomacropeptide

Additional relevant MeSH terms:
Phenylketonurias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Caseins
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action