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Protein Requirements in Children With Phenylketonuria (PKU)

Primary Purpose

Phenylketonuria

Status

Completed

Phase

Not Applicable

Locations

Canada

Study Type

Interventional

Intervention

Protein intake

About this trial

This is an interventional supportive care trial for Phenylketonuria focused on measuring Phenylketonuria, PKU, Phenylalanine metabolism, Protein requirements, Indicator Amino acid Oxidation, Stable isotopes

Eligibility Criteria

5 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Child and Family Research Institute

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Protein intake

Arm Description

Protein intake- Dietary supplement

Outcomes

Primary Outcome Measures

13 Co2 production

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT01965691

First Posted

October 16, 2013

Last Updated

September 22, 2020

Sponsor

University of British Columbia

Collaborators

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

1. Study Identification

Unique Protocol Identification Number

NCT01965691

Brief Title

Protein Requirements in Children With Phenylketonuria (PKU)

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2020

Overall Recruitment Status

Completed

Study Start Date

October 2013 (undefined)

Primary Completion Date

August 2015 (Actual)

Study Completion Date

December 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University of British Columbia

Collaborators

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

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Phenylketonuria

Keywords

Phenylketonuria, PKU, Phenylalanine metabolism, Protein requirements, Indicator Amino acid Oxidation, Stable isotopes

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

4 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Protein intake

Arm Type

Experimental

Arm Description

Protein intake- Dietary supplement

Intervention Type

Dietary Supplement

Intervention Name(s)

Protein intake

Intervention Description

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.

Primary Outcome Measure Information:

Title

13 Co2 production

Description

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.

Time Frame

8 hours (1 study day)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Rajavel Elango, PhD

Organizational Affiliation

Child and Family Research Institute, University of British Columbia

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Sylvia Stockler-Ipsiroglu, MD

Organizational Affiliation

University of British Columbia

Official's Role

Study Chair

First Name & Middle Initial & Last Name & Degree

Keiko Ueda, MPH, RD

Organizational Affiliation

Provincial Health Services Authority

Official's Role

Study Chair

Facility Information:

Facility Name

Child and Family Research Institute

City

Vancouver

State/Province

British Columbia

ZIP/Postal Code

V5Z4H4

Country

Canada

12. IPD Sharing Statement

Citations:

PubMed Identifier

28053173

Citation

Turki A, Ueda K, Cheng B, Giezen A, Salvarinova R, Stockler-Ipsiroglu S, Elango R. The Indicator Amino Acid Oxidation Method with the Use of l-[1-13C]Leucine Suggests a Higher than Currently Recommended Protein Requirement in Children with Phenylketonuria. J Nutr. 2017 Feb;147(2):211-217. doi: 10.3945/jn.116.240218. Epub 2017 Jan 4.

Results Reference

derived

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Protein Requirements in Children With Phenylketonuria (PKU)

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