PhenylketonuriasNCT03792451
Nutrition Status of Adults Treated With Pegvaliase
An observational study of Phenylketonurias, run by Boston Children's Hospital at 1 site in United States.
Trial summary
Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- Pegvaliase
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Jan 2019to 31 Dec 2020
- Phase
- Observational
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: This cross-sectional study will recruit adults with PKU who have been treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. This may include individuals who participated in the phase III clinical trials for pegvaliase.
Who may be able to join
- Diagnosis of phenylketonuria
- Aged 18-65 years
- Currently treated with pegvaliase
- Have either 1) received treatment with pegvaliase for one or more years or 2) followed an unrestricted diet for 6 or more months while on pegvaliase
- Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)
Who cannot join
- Unable to consent to study
- Under age 18 years or over age 65
- Routine food intake provides less than the RDA for protein (0.8 g/kg/d)
- Consuming any PKU medical foods
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
Official title: Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase
Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. We will evaluate laboratory markers of nutritional status, including markers of protein and micronutrient nutriture. In addition, a three-day food record and food frequency questionnaire will be obtained to assess protein intake and overall diet quality.
- Conditions
- Phenylketonurias
- Being tested
- Pegvaliase
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegvaliaseDrug
Also called Palynziq
Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean concentrations of serum marker(s) of protein status
Measured: through study completion, an average of 1 year
Mean concentrations of serum marker(s) of micronutrient status
Measured: through study completion, an average of 1 year
Mean concentrations of serum marker(s) of essential fatty acid status
Measured: through study completion, an average of 1 year
Mean concentrations of serum marker(s) of cardiovascular status
Measured: through study completion, an average of 1 year
Mean protein intake (g/day) as reported on a three-day food record
Measured: through study completion, an average of 1 year
Mean micronutrient intake (%DRI) as reported on a three-day food record
Measured: through study completion, an average of 1 year
Secondary outcomes (2)
Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III
"The Healthy Eating Index-2015 (HEI-2015) is… used to assess how well a set of foods aligns with the 2015-2020 Dietary Guidelines for Americans. The HEI-2015 includes 13 components that can be summed to a maximum total score of 100 points. The components capture… adequacy components and… moderation components. For the adequacy components, higher scores reflect higher intakes that meet or exceed the standards. For the moderation components, higher scores reflect lower intakes because lower intakes are more desirable. A higher total score indicates a diet that aligns better with the Dietary Guidelines." - https://www.cnpp.usda.gov/sites/default/files/healthy_eating_index/HEI2015-ScoresForAmericans.pdf
Adequacy components include total fruits, whole fruits, total vegetables, greens and beans, whole grains, dairy, total protein foods, seafood and plant proteins, fatty acids. Moderation components include refined grains, sodium, added sugars, and saturated fats.
Measured: through study completion, an average of 1 year
Correlation between serum micronutrient concentrations and average micronutrient intake
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jan 2019
- Study start17 Jan 2019
- Primary completion31 Dec 2020
- Study completion31 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Responsible party
- Principal investigator, Krista Viau, Boston Children's Hospital
- Investigators
- Krista Viau, PhD, RD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03792451
- Sponsor's protocol ID
- P00029611
- Status last verified
- Mar 2021
- First submitted
- 31 Dec 2018
- Last updated
- 4 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase
- ClinicalTrials.gov ID
- NCT03792451
- Acronym / sponsor ID
- P00029611
- Conditions
- Phenylketonurias
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Study start
- 17 Jan 2019 (actual)
- Primary completion
- 31 Dec 2020 (actual)
- Study completion
- 31 Dec 2020 (actual)
- First posted
- 3 Jan 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.
Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. We will evaluate laboratory markers of nutritional status, including markers of protein and micronutrient nutriture. In addition, a three-day food record and food frequency questionnaire will be obtained to assess protein intake and overall diet quality.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This cross-sectional study will recruit adults with PKU who have been treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. This may include individuals who participated in the phase III clinical trials for pegvaliase.
Key inclusion criteria (5 listed)
- Diagnosis of phenylketonuria
- Aged 18-65 years
- Currently treated with pegvaliase
- Have either 1) received treatment with pegvaliase for one or more years or 2) followed an unrestricted diet for 6 or more months while on pegvaliase
- Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)
Key exclusion criteria (4 listed)
- Unable to consent to study
- Under age 18 years or over age 65
- Routine food intake provides less than the RDA for protein (0.8 g/kg/d)
- Consuming any PKU medical foods
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 |
|---|---|---|
| PegvaliaseAlso: PalynziqTreated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase. | Drug | – |
Primary outcome
Mean concentrations of serum marker(s) of protein status
Time frame: through study completion, an average of 1 year
Mean concentrations of serum marker(s) of micronutrient status
Time frame: through study completion, an average of 1 year
Mean concentrations of serum marker(s) of essential fatty acid status
Time frame: through study completion, an average of 1 year
Mean concentrations of serum marker(s) of cardiovascular status
Time frame: through study completion, an average of 1 year
Mean protein intake (g/day) as reported on a three-day food record
Time frame: through study completion, an average of 1 year
Mean micronutrient intake (%DRI) as reported on a three-day food record
Time frame: through study completion, an average of 1 year
2 secondary outcomes
Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III
Time frame: through study completion, an average of 1 year
Correlation between serum micronutrient concentrations and average micronutrient intake
Time frame: through study completion, an average of 1 year
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 4 Mar 2021.
- Individual participant dataSharing planned: No
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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.
Krista Viau, PhD, RD principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonurias, run by Boston Children's Hospital at 1 site in United States.
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 5 inclusion and 4 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 4 Mar 2021. 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 United States. 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?
Mean concentrations of serum marker(s) of protein status (time frame: through study completion, an average of 1 year). The study lists 6 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 4 Mar 2021.