Evaluation of PKU Sphere

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

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

Study Overview

• Status: Completed
• Conditions: Phenylketonurias
• Intervention / Treatment: Dietary supplement: PKU Sphere

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
• Enrollment (Actual): 28
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase 1: taste	Questionnaire data captured to evaluate taste	Days 1 - 7.
Phase 1: smell	Questionnaire data captured to evaluate smell	Days 1 - 7.
Phase 1: texture	Questionnaire data captured to evaluate texture	Days 1 - 7.
Phase 1: gastrointestinal tolerance	Questionnaires will be completed daily during the 1-week taste test.	Days 1 - 7.
Phase 1: phenylalanine concentration	Phenylalanine concentrations measured via blood spot analysis.	Days 1 and 7.
Phase 1: tyrosine concentration	Tyrosine concentrations measured via blood spot analysis.	Days 1 and 7.
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, day 1 and day 28.
Phase 2: gastrointestinal tolerance	Questionnaires will be completed daily throughout Phase 2.	Phase 2, days 1 - 28.
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, days 1 - 3 and days 26 - 28.
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, days 1 and 28.
Phase 2: phenylalanine concentration	Phenylalanine concentrations measured via blood spot analysis.	Twice weekly for four weeks during phase 2
Phase 2: tyrosine concentration	Tyrosine concentrations measured via blood spot analysis.	Twice weekly for four weeks during phase 2
Phase 2: questionnaire data captured to evaluate change in taste perception	Data captured to evaluate taste	Phase 2, days 14 and 28.
Phase 2: questionnaire data captured to evaluate change in smell perception	Data captured to evaluate smell	Phase 2, days 14 and 28.
Phase 2: questionnaire data captured to evaluate change in texture perception	Data captured to evaluate texture	Phase 2, days 14 and 28.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase 1: questoinnaire evaluating subjective measures of satiety	To evaluate if PKU Sphere increases subjective measures of satiety	Phase 1, days 1 - 7.
Phase 2: questoinnaire evaluating subjective measures of satiety	To evaluate if PKU Sphere increases subjective measures of satiety	Phase 2, days 1, 14 and 28.

Collaborators and Investigators

• Sponsor: Vitaflo International, Ltd
• Collaborators: 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

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2018
• Primary Completion (Actual): May 21, 2019
• Study Completion (Actual): May 21, 2019

Study Registration Dates

• First Submitted: January 3, 2018
• First Submitted That Met QC Criteria: January 26, 2018
• First Posted (Actual): February 5, 2018

Study Record Updates

• Last Update Posted (Actual): February 16, 2024
• Last Update Submitted That Met QC Criteria: February 15, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Child; Adult; Phenylketonuria; PKU; GMP; Glycomacropeptide; Phenylalanine; Sphere
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Phenylketonurias
• Other Study ID Numbers: SCT-PKUS-2016-11-28

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No