Protein Requirements in Children With Phenylketonuria (PKU)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by University of British Columbia
Sponsor:
Collaborators:
Rare Disease Foundation
Royal Embassy of Saudi Arabia-Cultural Bureau in Ottawa
Information provided by (Responsible Party):
University of British Columbia
ClinicalTrials.gov Identifier:
NCT01965691
First received: October 16, 2013
Last updated: May 26, 2014
Last verified: May 2014
  Purpose

Phenylketonuria (PKU) is an inherited inborn error of an amino acid phenylalanine (PHE) metabolism affecting 1:15,000 births. It is caused by a decreased activity of an enzyme in the liver called phenylalanine hydroxylase (PAH) which is important to convert PHE into tyrosine, another amino acid. Consequently, PHE accumulates in the blood leading to mental and developmental delays. Nutritional management is the primary choice of treatment that includes providing sufficient protein in the diet and at the same time restricting PHE. However the amount of protein to be given is unknown. A new technique called Indicator Amino Acid Oxidation (IAAO) will be used to determine the protein requirements in children with PKU (5-18y). The study will help treat and manage these children with sufficient protein to ensure proper growth and development. Current dietary recommendations range from 35-65 g/day and is based on factorial calculations.

The investigators hypothesize that the protein requirement in children with PKU will be higher than the current mathematically calculated recommended intake of 35-65 g/day for the 5-18y children.


Condition Intervention
Phenylketonuria
Dietary Supplement: Protein intake

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Single Blind (Subject)
Primary Purpose: Supportive Care
Official Title: Application of Stable Isotopes to Determine Protein Requirements in Children With Phenylketonuria (PKU)

Resource links provided by NLM:


Further study details as provided by University of British Columbia:

Primary Outcome Measures:
  • 13 Co2 production [ Time Frame: 8 hours (1 study day) ] [ Designated as safety issue: No ]
    Urine and breath samples will be collected during the study to measure the rate of oxidation of tracer in the expired breath and flux enrichment in urine.


Estimated Enrollment: 6
Study Start Date: October 2013
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Protein intake
Protein intake- Dietary supplement
Dietary Supplement: Protein 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 flavored liquid, protein free cookies and corn oil
  • 4 isotopically labeled experimental meals.

  Eligibility

Ages Eligible for Study:   5 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Children (5-18y) who are diagnosed with Phenylketonuria (PKU), and clinically stable with no acute illness

Exclusion Criteria:

  • Children less than 5y of age and who are diagnosed with PKU, as it may be difficult to take breath samples and perform indirect calorimetry in very young children.
  • Children diagnosed with PKU, but are currently ill, with a fever, cold, vomiting or diarrhea.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01965691

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

Locations
Canada, British Columbia
Child and Family Research Institute Recruiting
Vancouver, British Columbia, Canada, V5Z4H4
Contact: Rajavel Elango, Ph.D    604-875-2000 ext 4911    relango@cfri.ubc.ca   
Sponsors and Collaborators
University of British Columbia
Rare Disease Foundation
Royal Embassy of Saudi Arabia-Cultural Bureau in Ottawa
Investigators
Principal Investigator: Rajavel Elango, PhD Child and Family Research Institute, University of British Columbia
Study Chair: Sylvia Stockler-Ipsiroglu, MD University of British Columbia
Study Chair: Keiko Ueda, MPH, RD Provincial Health Services Authority
  More Information

No publications provided

Responsible Party: University of British Columbia
ClinicalTrials.gov Identifier: NCT01965691     History of Changes
Other Study ID Numbers: H13-00220
Study First Received: October 16, 2013
Last Updated: May 26, 2014
Health Authority: Canada: Health Canada

Keywords provided by University of British Columbia:
Phenylketonuria
PKU
Phenylalanine metabolism
Protein requirements
Indicator Amino acid Oxidation
Stable isotopes

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

ClinicalTrials.gov processed this record on August 20, 2014