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Evaluation of PKU Sphere

Primary Purpose

Phenylketonurias

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
PKU Sphere
Sponsored by
Vitaflo International, Ltd
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Phenylketonurias focused on measuring Phenylketonuria, GMP, Sphere, Child, Adult, Phenylalanine, PKU, Glycomacropeptide

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Oregon Health and Science University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PKU Sphere

Arm Description

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.

Outcomes

Primary Outcome Measures

Phase 1: taste
Questionnaire data captured to evaluate taste
Phase 1: smell
Questionnaire data captured to evaluate smell
Phase 1: texture
Questionnaire data captured to evaluate texture
Phase 1: gastrointestinal tolerance
Questionnaires will be completed daily during the 1-week taste test.
Phase 1: phenylalanine concentration
Phenylalanine concentrations measured via blood spot analysis.
Phase 1: tyrosine concentration
Tyrosine concentrations measured via blood spot analysis.
Phase 2: quantitative change in plasma amino acids at day 28
Compare comprehensive plasma amino acid profiles at baseline and end of the trial.
Phase 2: gastrointestinal tolerance
Questionnaires will be completed daily throughout Phase 2.
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.
Phase 2: change in anthropometry at end of study
Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated
Phase 2: phenylalanine concentration
Phenylalanine concentrations measured via blood spot analysis.
Phase 2: tyrosine concentration
Tyrosine concentrations measured via blood spot analysis.
Phase 2: questionnaire data captured to evaluate change in taste perception
Data captured to evaluate taste
Phase 2: questionnaire data captured to evaluate change in smell perception
Data captured to evaluate smell
Phase 2: questionnaire data captured to evaluate change in texture perception
Data captured to evaluate texture

Secondary Outcome Measures

Phase 1: questoinnaire evaluating subjective measures of satiety
To evaluate if PKU Sphere increases subjective measures of satiety
Phase 2: questoinnaire evaluating subjective measures of satiety
To evaluate if PKU Sphere increases subjective measures of satiety

Full Information

First Posted
January 3, 2018
Last Updated
June 4, 2019
Sponsor
Vitaflo International, Ltd
Collaborators
Oregon Health and Science University
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1. Study Identification

Unique Protocol Identification Number
NCT03419819
Brief Title
Evaluation of PKU Sphere
Official Title
Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).
Study Type
Interventional

2. Study Status

Record Verification Date
June 2019
Overall Recruitment Status
Completed
Study Start Date
May 1, 2018 (Actual)
Primary Completion Date
May 21, 2019 (Actual)
Study Completion Date
May 21, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vitaflo International, Ltd
Collaborators
Oregon Health and Science University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias
Keywords
Phenylketonuria, GMP, Sphere, Child, Adult, Phenylalanine, PKU, Glycomacropeptide

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
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)
Allocation
N/A
Enrollment
28 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PKU Sphere
Arm Type
Experimental
Arm Description
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.
Intervention Type
Dietary Supplement
Intervention Name(s)
PKU Sphere
Intervention Description
PKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria.
Primary Outcome Measure Information:
Title
Phase 1: taste
Description
Questionnaire data captured to evaluate taste
Time Frame
Days 1 - 7.
Title
Phase 1: smell
Description
Questionnaire data captured to evaluate smell
Time Frame
Days 1 - 7.
Title
Phase 1: texture
Description
Questionnaire data captured to evaluate texture
Time Frame
Days 1 - 7.
Title
Phase 1: gastrointestinal tolerance
Description
Questionnaires will be completed daily during the 1-week taste test.
Time Frame
Days 1 - 7.
Title
Phase 1: phenylalanine concentration
Description
Phenylalanine concentrations measured via blood spot analysis.
Time Frame
Days 1 and 7.
Title
Phase 1: tyrosine concentration
Description
Tyrosine concentrations measured via blood spot analysis.
Time Frame
Days 1 and 7.
Title
Phase 2: quantitative change in plasma amino acids at day 28
Description
Compare comprehensive plasma amino acid profiles at baseline and end of the trial.
Time Frame
Phase 2, day 1 and day 28.
Title
Phase 2: gastrointestinal tolerance
Description
Questionnaires will be completed daily throughout Phase 2.
Time Frame
Phase 2, days 1 - 28.
Title
Phase 2: change in 3-day diet record at end of study
Description
A 3-day diet record will be completed in the first 3 days and the last 3 days for comparison.
Time Frame
Phase 2, days 1 - 3 and days 26 - 28.
Title
Phase 2: change in anthropometry at end of study
Description
Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated
Time Frame
Phase 2, days 1 and 28.
Title
Phase 2: phenylalanine concentration
Description
Phenylalanine concentrations measured via blood spot analysis.
Time Frame
Twice weekly for four weeks during phase 2
Title
Phase 2: tyrosine concentration
Description
Tyrosine concentrations measured via blood spot analysis.
Time Frame
Twice weekly for four weeks during phase 2
Title
Phase 2: questionnaire data captured to evaluate change in taste perception
Description
Data captured to evaluate taste
Time Frame
Phase 2, days 14 and 28.
Title
Phase 2: questionnaire data captured to evaluate change in smell perception
Description
Data captured to evaluate smell
Time Frame
Phase 2, days 14 and 28.
Title
Phase 2: questionnaire data captured to evaluate change in texture perception
Description
Data captured to evaluate texture
Time Frame
Phase 2, days 14 and 28.
Secondary Outcome Measure Information:
Title
Phase 1: questoinnaire evaluating subjective measures of satiety
Description
To evaluate if PKU Sphere increases subjective measures of satiety
Time Frame
Phase 1, days 1 - 7.
Title
Phase 2: questoinnaire evaluating subjective measures of satiety
Description
To evaluate if PKU Sphere increases subjective measures of satiety
Time Frame
Phase 2, days 1, 14 and 28.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sandra Van Calcar, PhD
Organizational Affiliation
Oregon Health and Science University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Joyanna Hansen, PhD
Organizational Affiliation
Oregon Health and Science University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Evaluation of PKU Sphere

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