PhenylketonuriaNCT01965691
Protein Requirements in Children With Phenylketonuria (PKU)
A study testing Protein intake in people with Phenylketonuria, run by University of British Columbia at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- Protein intake
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteCanada
- Study dates
- Oct 2013to Dec 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Children (5-18y) who are diagnosed with Phenylketonuria (PKU), and clinically stable with no acute illness
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Canada1 site
Child and Family Research Institute
Vancouver, British Columbia, V5Z4H4
About this study
Official title: 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.
- Conditions
- Phenylketonuria
- Being tested
- Protein intake
- Keywords
- Phenylketonuria, PKU, Phenylalanine metabolism, Protein requirements, Indicator Amino acid Oxidation, Stable isotopes
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Protein intakeDietary supplement
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.
| Study group | Role | What they receive |
|---|---|---|
| Protein intake | Experimental | Protein intake- Dietary supplement Receives: Dietary Supplement: Protein intake |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 8 hours (1 study day)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2013 (estimated)
- Study startOct 2013
- Primary completionAug 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- More trials run by University of British ColumbiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of British Columbia
- Sponsor type
- University, hospital or other
- Collaborators
- Rare Disease Foundation, Vancouver, Canada, Saudi Arabian Cultural Bureau, Ottawa
- Responsible party
- Principal investigator, Rajavel Elango, PhD, University of British Columbia
- Investigators
- Rajavel Elango, PhD, Principal investigator, Child and Family Research Institute, University of British ColumbiaSylvia Stockler-Ipsiroglu, MD, Study chair, University of British ColumbiaKeiko Ueda, MPH, RD, Study chair, Provincial Health Services Authority British Columbia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01965691
- Sponsor's protocol ID
- H13-00220
- Status last verified
- Sep 2020
- First submitted
- 16 Oct 2013
- Last updated
- 24 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 28053173
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Application of Stable Isotopes to Determine Protein Requirements in Children With Phenylketonuria (PKU)
- ClinicalTrials.gov ID
- NCT01965691
- Acronym / sponsor ID
- H13-00220
- Conditions
- Phenylketonuria
- Keywords
- Phenylketonuria, PKU, Phenylalanine metabolism, Protein requirements, Indicator Amino acid Oxidation, Stable isotopes
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- University of British ColumbiaUniversity, hospital or other
- Collaborators
- Rare Disease Foundation, Vancouver, Canada, Saudi Arabian Cultural Bureau, Ottawa
- Study start
- Oct 2013
- Primary completion
- Aug 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 18 Oct 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Protein intake | Experimental | Dietary Supplement: Protein intakeProtein intake- Dietary supplement |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Children (5-18y) who are diagnosed with Phenylketonuria (PKU), and clinically stable with no acute illness
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Protein intakeOral 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. | Dietary supplement | Protein intake |
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2020.
- Derived from this study: PubMed 28053173Turki 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rajavel Elango, PhD principal investigator
Child and Family Research Institute, University of British Columbia
Sylvia Stockler-Ipsiroglu, MD study chair
University of British Columbia
Keiko Ueda, MPH, RD study chair
Provincial Health Services Authority British Columbia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Protein intake in people with Phenylketonuria, run by University of British Columbia at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
13 Co2 production (time frame: 8 hours (1 study day)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01965691, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2020.