Protein Requirements in Children With Phenylketonuria (PKU)

September 22, 2020 updated by: Rajavel Elango, PhD, University of British Columbia

Application of Stable Isotopes to Determine Protein Requirements in Children With Phenylketonuria (PKU)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z4H4
        • Child and Family Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Supportive Care
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
13 Co2 production
Time Frame: 8 hours (1 study day)
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.
8 hours (1 study day)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of British Columbia

Collaborators

Rare Disease Foundation, Vancouver, Canada
Saudi Arabian Cultural Bureau, 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2013

Primary Completion (Actual)

August 1, 2015

Study Completion (Actual)

December 1, 2015

Study Registration Dates

First Submitted

October 16, 2013

First Submitted That Met QC Criteria

October 17, 2013

First Posted (Estimate)

October 18, 2013

Study Record Updates

Last Update Posted (Actual)

September 24, 2020

Last Update Submitted That Met QC Criteria

September 22, 2020

Last Verified

September 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • H13-00220

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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