Clinical trial NCT03939052: Protein Requirements in Adults With Phenylketonuria (PKU)
Official title: Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Phenylketonuria
Keywords: Phenylketonuria
PKU
Phenylalanine metabolism
Protein requirements
Indicator Amino acid Oxidation
Stable isotopes
GMP
Glycomacropeptide
Lead sponsor: University of British Columbia
Sponsor class: OTHER
Collaborators: name: Vitaflo International, Ltd
First posted: 2019-05-06
Enrollment: 6
Sex: ALL
Minimum age (years): 19
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: Phenylketonuria (PKU) is an inherited inborn error of phenylalanine (PHE) metabolism caused by decreased activity of phenylalanine hydroxylase (PAH) enzyme. Therefore, PHE accumulates in plasma leading to mental problems. Treatment is a phenylalanine-restricted diet with sufficient protein. However, the optimum protein requirements are still unknown and compliance with diet is not satisfactory in PKU adults. A Previously established technique called indicator amino acid oxidation (IAAO) will be used to determine protein requirements from amino acid based formula vs. glycomacropeptide (GMP) in adults with PKU (≥ 19y). This study will help treat adults with enough protein ensuring maintenance of health.


Eligibility criteria: Inclusion Criteria:

-Adults more than 19 years of age who are diagnosed with PKU and clinically stable with no acute illness

Exclusion Criteria:

* Adults with PKU under age 19 year
* Adults diagnosed with PKU but are currently ill with a fever or cold

Primary outcomes:
measure: 13 Co2 production
timeFrame: 8 hours (1 study day). 3 samples will collected as a baseline prior to isotope protocol and 6 samples after 2 hours and 30 minutes of starting the tracer protocol. Data will be reported an average of 2 years.
measure: Lysine flux
timeFrame: 8 hours (1 study day). 1 sample will collected as a baseline prior to isotope protocol and 2 samples after 2 hours and 30 minutes of starting the tracer protocol. Data will be reported an average of 2 years.
Secondary outcomes:
measure: Phenylalanine concentrations
timeFrame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
measure: Tyrosine concentrations
timeFrame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
measure: 16 other amino acids
timeFrame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
Other outcomes:
Locations:
city: Vancouver
country: Canada
facility: BC Children's Hospital Research Institute, University of British Columbia
state: British Columbia
Study officials:
affiliation: BC Children's Hospital Research Institute, University of British Columbia
name: Rajavel Elango, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of British Columbia
name: Sandra Sirrs, MD
role: STUDY_CHAIR
affiliation: University of British Columbia
name: Sylvia Stockler, MD
role: STUDY_CHAIR
References: