Protein Requirements in Adults With Phenylketonuria (PKU)

• ClinicalTrials.gov Identifier: NCT03939052
• Recruitment Status: Unknown
• First Posted: May 6, 2019
• Last Update Posted: August 22, 2019
• Sponsor: University of British Columbia
• Collaborator: Vitaflo International, Ltd
• Information provided by (Responsible Party): Rajavel Elango, PhD, University of British Columbia

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Phenylketonuria	Dietary Supplement: Free amino acids intake	Not Applicable

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 6 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Supportive Care
• Official Title: Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product
• Actual Study Start Date: July 19, 2019
• Estimated Primary Completion Date: May 2020
• Estimated Study Completion Date: May 2020

Arms and Interventions

Arm

Intervention/treatment

Experimental: Protein intake; Free amino acids vs. Glycomacropeptide (GMP)

Dietary Supplement: Free amino acids intake; Oral consumption of eight hourly experimental meals- -4 tracer free experimental meals containing a mixture of free amino acids and calories from protein free flavoured liquid, protein free cookies and corn oil -4 isotopically labeled experimental meals. The same protocol will be repeated with glycomacropeptide (GMP).; Other Name: glycomacropeptide (GMP) intake

Outcome Measures

Primary Outcome Measures :

1. 13 Co2 production [ Time Frame: 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. ]
   Breath samples will be collected during the study to measure the rate of oxidation of tracer in the expired breath.
2. Lysine flux [ Time Frame: 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. ]
   Urine samples will be collected during the study to measure the flux enrichment in urine.

Secondary Outcome Measures :

1. Phenylalanine concentrations [ Time Frame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years. ]
   Phenylalanine concentrations will be measured in blood.
2. Tyrosine concentrations [ Time Frame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years. ]
   Tyrosine concentrations will be measured in blood.
3. 16 other amino acids [ Time Frame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years. ]
   16 other amino acids will be measured in blood.

Eligibility Criteria

• Ages Eligible for Study: 19 Years to 50 Years (Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Contacts

Contact: Rajavel Elango, PhD; 604-875-2000 ext 4911; relango@bcchr.ubc.ca

Locations

Canada, British Columbia

BC Children's Hospital Research Institute, University of British Columbia; Recruiting

Vancouver, British Columbia, Canada, V5Z4H4

Contact: Rajavel Elango, Ph.D

Sponsors and Collaborators

University of British Columbia

Vitaflo International, Ltd

Investigators

• Principal Investigator: Rajavel Elango, PhD; BC Children's Hospital Research Institute, University of British Columbia
• Study Chair: Sandra Sirrs, MD; University of British Columbia
• Study Chair: Sylvia Stockler, MD; University of British Columbia

More Information

• Responsible Party: Rajavel Elango, PhD, Associate Professor, University of British Columbia
• ClinicalTrials.gov Identifier: NCT03939052
• Other Study ID Numbers: H18-03464
• First Posted: May 6, 2019
• Last Update Posted: August 22, 2019
• Last Verified: August 2019

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Rajavel Elango, PhD, University of British Columbia:

Phenylketonuria; PKU; Phenylalanine metabolism; Protein requirements; Indicator Amino acid Oxidation; Stable isotopes; GMP; Glycomacropeptide

Additional relevant MeSH terms:

Phenylketonurias; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases