Clinical trial NCT03419819: 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
Overall status: COMPLETED
Phase: NA
Conditions: Phenylketonurias
Keywords: Phenylketonuria
GMP
Sphere
Child
Adult
Phenylalanine
PKU
Glycomacropeptide
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
Collaborators: name: Oregon Health and Science University
First posted: 2018-02-05
Enrollment: 28
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
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.
Primary purpose: TREATMENT

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).

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.

Primary outcomes:
measure: Phase 1: taste
timeFrame: Days 1 - 7.
measure: Phase 1: smell
timeFrame: Days 1 - 7.
measure: Phase 1: texture
timeFrame: Days 1 - 7.
measure: Phase 1: gastrointestinal tolerance
timeFrame: Days 1 - 7.
measure: Phase 1: phenylalanine concentration
timeFrame: Days 1 and 7.
measure: Phase 1: tyrosine concentration
timeFrame: Days 1 and 7.
measure: Phase 2: quantitative change in plasma amino acids at day 28
timeFrame: Phase 2, day 1 and day 28.
measure: Phase 2: gastrointestinal tolerance
timeFrame: Phase 2, days 1 - 28.
measure: Phase 2: change in 3-day diet record at end of study
timeFrame: Phase 2, days 1 - 3 and days 26 - 28.
measure: Phase 2: change in anthropometry at end of study
timeFrame: Phase 2, days 1 and 28.
measure: Phase 2: phenylalanine concentration
timeFrame: Twice weekly for four weeks during phase 2
measure: Phase 2: tyrosine concentration
timeFrame: Twice weekly for four weeks during phase 2
measure: Phase 2: questionnaire data captured to evaluate change in taste perception
timeFrame: Phase 2, days 14 and 28.
measure: Phase 2: questionnaire data captured to evaluate change in smell perception
timeFrame: Phase 2, days 14 and 28.
measure: Phase 2: questionnaire data captured to evaluate change in texture perception
timeFrame: Phase 2, days 14 and 28.
Secondary outcomes:
measure: Phase 1: questoinnaire evaluating subjective measures of satiety
timeFrame: Phase 1, days 1 - 7.
measure: Phase 2: questoinnaire evaluating subjective measures of satiety
timeFrame: Phase 2, days 1, 14 and 28.
Other outcomes:
Locations:
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
Study officials:
affiliation: Oregon Health and Science University
name: Sandra Van Calcar, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Oregon Health and Science University
name: Joyanna Hansen, PhD
role: PRINCIPAL_INVESTIGATOR
References: