Evaluation of PKU Sphere

Phenylketonurias Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03419819
• Other study ID #: SCT-PKUS-2016-11-28
• Status: Completed
• Phase: N/A
• Start date: May 1, 2018
• Est. completion date: May 21, 2019

Study information

• Verified date: February 2024
• Source: Vitaflo International, Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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 information / eligibility

• Status: Completed
• Enrollment: 28
• Est. completion date: May 21, 2019
• Est. primary completion date: May 21, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

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

Locations

Country	Name	City	State
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (2)

Lead Sponsor	Collaborator
Vitaflo International, Ltd	Oregon Health and Science University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Phase 1: taste	Questionnaire data captured to evaluate taste	Days 1 - 7.
Primary	Phase 1: smell	Questionnaire data captured to evaluate smell	Days 1 - 7.
Primary	Phase 1: texture	Questionnaire data captured to evaluate texture	Days 1 - 7.
Primary	Phase 1: gastrointestinal tolerance	Questionnaires will be completed daily during the 1-week taste test.	Days 1 - 7.
Primary	Phase 1: phenylalanine concentration	Phenylalanine concentrations measured via blood spot analysis.	Days 1 and 7.
Primary	Phase 1: tyrosine concentration	Tyrosine concentrations measured via blood spot analysis.	Days 1 and 7.
Primary	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.
Primary	Phase 2: gastrointestinal tolerance	Questionnaires will be completed daily throughout Phase 2.	Phase 2, days 1 - 28.
Primary	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.
Primary	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.
Primary	Phase 2: phenylalanine concentration	Phenylalanine concentrations measured via blood spot analysis.	Twice weekly for four weeks during phase 2
Primary	Phase 2: tyrosine concentration	Tyrosine concentrations measured via blood spot analysis.	Twice weekly for four weeks during phase 2
Primary	Phase 2: questionnaire data captured to evaluate change in taste perception	Data captured to evaluate taste	Phase 2, days 14 and 28.
Primary	Phase 2: questionnaire data captured to evaluate change in smell perception	Data captured to evaluate smell	Phase 2, days 14 and 28.
Primary	Phase 2: questionnaire data captured to evaluate change in texture perception	Data captured to evaluate texture	Phase 2, days 14 and 28.
Secondary	Phase 1: questoinnaire evaluating subjective measures of satiety	To evaluate if PKU Sphere increases subjective measures of satiety	Phase 1, days 1 - 7.
Secondary	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.