PhenylketonuriaNCT03939052
Protein Requirements in Adults With Phenylketonuria (PKU)
A study testing Free amino acids 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 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. Read more about this study
- Who is it for?
- Ages 19 to 50 years
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- Free amino acids intake
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteCanada
- Study dates
- 19 Jul 2019to 13 Jan 2023
- 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 19 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- -Adults more than 19 years of age who are diagnosed with PKU and clinically stable with no acute illness
Who cannot join
- Adults with PKU under age 19 year
- Adults diagnosed with PKU but are currently ill with a fever or cold
Trial locations and contacts
1 site in 1 country.
Canada1 site
BC Children's Hospital Research Institute, University of British Columbia
Vancouver, British Columbia, V5Z4H4
About this study
Official title: Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product
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.
- Conditions
- Phenylketonuria
- Being tested
- Free amino acids intake
- Keywords
- Phenylketonuria, PKU, Phenylalanine metabolism, Protein requirements, Indicator Amino acid Oxidation, Stable isotopes, GMP, Glycomacropeptide
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Free amino acids intakeDietary supplement
Also called glycomacropeptide (GMP) 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).
| Study group | Role | What they receive |
|---|---|---|
| Protein intake | Experimental | Free amino acids vs. Glycomacropeptide (GMP) Receives: Dietary Supplement: Free amino acids intake |
What is being measured
The primary outcome is the main question the study is designed to answer.
13 Co2 production
Breath samples will be collected during the study to measure the rate of oxidation of tracer in the expired breath.
Measured: 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.
Lysine flux
Urine samples will be collected during the study to measure the flux enrichment in urine.
Measured: 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.
Secondary outcomes (3)
Phenylalanine concentrations
Phenylalanine concentrations will be measured in blood.
Measured: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
Tyrosine concentrations
Tyrosine concentrations will be measured in blood.
Measured: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
16 other amino acids
16 other amino acids will be measured in blood.
Measured: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2019
- Study start19 Jul 2019
- Primary completion1 Dec 2021
- Study completion13 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of British Columbia
- Sponsor type
- University, hospital or other
- Collaborators
- Vitaflo International, Ltd
- Responsible party
- Principal investigator, Rajavel Elango, PhD, University of British Columbia
- Investigators
- Rajavel Elango, PhD, Principal investigator, BC Children's Hospital Research Institute, University of British ColumbiaSandra Sirrs, MD, Study chair, University of British ColumbiaSylvia Stockler, MD, Study chair, University of British Columbia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03939052
- Sponsor's protocol ID
- H18-03464
- Status last verified
- Jul 2024
- First submitted
- 1 May 2019
- Last updated
- 25 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product
- ClinicalTrials.gov ID
- NCT03939052
- Acronym / sponsor ID
- H18-03464
- Conditions
- Phenylketonuria
- Keywords
- Phenylketonuria, PKU, Phenylalanine metabolism, Protein requirements, Indicator Amino acid Oxidation, Stable isotopes, GMP, Glycomacropeptide
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- University of British ColumbiaUniversity, hospital or other
- Collaborators
- Vitaflo International, Ltd
- Study start
- 19 Jul 2019 (actual)
- Primary completion
- 1 Dec 2021 (actual)
- Study completion
- 13 Jan 2023 (actual)
- First posted
- 6 May 2019 (actual)
- 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 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.
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: Free amino acids intakeFree amino acids vs. Glycomacropeptide (GMP) |
Who may qualify
- Ages
- 19 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- -Adults more than 19 years of age who are diagnosed with PKU and clinically stable with no acute illness
Key exclusion criteria (2 listed)
- Adults with PKU under age 19 year
- Adults diagnosed with PKU but are currently ill with a fever or cold
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 |
|---|---|---|
| Free amino acids intakeAlso: glycomacropeptide (GMP) intakeOral 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). | Dietary supplement | Protein intake |
Primary outcome
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.
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.
3 secondary outcomes
Phenylalanine 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
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
Time frame: One sample (at 6th meal) after starting of the tracer protocol. Data will be reported an average of 2 years.
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 25 Jul 2024.
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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
BC Children's Hospital Research Institute, University of British Columbia
Sandra Sirrs, MD study chair
University of British Columbia
Sylvia Stockler, MD study chair
University of 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 Free amino acids 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 25 Jul 2024. 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). 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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Jul 2024.