Nutrition Status of Adults Treated With Pegvaliase

March 2, 2021 updated by: Krista Viau, Boston Children's Hospital

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

18

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

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital

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

16 years to 63 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

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.

Description

Inclusion Criteria:

  1. Diagnosis of phenylketonuria
  2. Aged 18-65 years
  3. Currently treated with pegvaliase
  4. 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
  5. Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)

Exclusion Criteria:

  1. Unable to consent to study
  2. Under age 18 years or over age 65
  3. Routine food intake provides less than the RDA for protein (0.8 g/kg/d)
  4. Consuming any PKU medical foods

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

  • Observational Models: Cohort
  • Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Mean concentrations of serum marker(s) of protein status
Time Frame: through study completion, an average of 1 year
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
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
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
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
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
through study completion, an average of 1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III
Time Frame: through study completion, an average of 1 year

"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.
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
through study completion, an average of 1 year

Collaborators and Investigators

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

Sponsor

Boston Children's Hospital

Collaborators

BioMarin Pharmaceutical

Investigators

  • Principal Investigator: Krista Viau, PhD, RD, Boston Children's Hospital

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 (Actual)

January 17, 2019

Primary Completion (Actual)

December 31, 2020

Study Completion (Actual)

December 31, 2020

Study Registration Dates

First Submitted

December 31, 2018

First Submitted That Met QC Criteria

January 2, 2019

First Posted (Actual)

January 3, 2019

Study Record Updates

Last Update Posted (Actual)

March 4, 2021

Last Update Submitted That Met QC Criteria

March 2, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P00029611

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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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