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Nutrition Status of Adults Treated With Pegvaliase

Sponsor
Boston Children's Hospital (Other)
Overall Status
Completed
CT.gov ID
NCT03792451
Collaborator
BioMarin Pharmaceutical (Industry)
18
Enrollment
1
Location
23.5
Actual Duration (Months)
0.8
Patients Per Site Per Month

Study Details

Study Description

Brief 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. 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.

Condition or Disease Intervention/Treatment Phase

Study Design

Study Type:
Observational
Actual Enrollment :
18 participants
Observational Model:
Cohort
Time Perspective:
Cross-Sectional
Official Title:
Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase
Actual Study Start Date :
Jan 17, 2019
Actual Primary Completion Date :
Dec 31, 2020
Actual Study Completion Date :
Dec 31, 2020

Outcome Measures

Primary Outcome Measures

  1. Mean concentrations of serum marker(s) of protein status [through study completion, an average of 1 year]

  2. Mean concentrations of serum marker(s) of micronutrient status [through study completion, an average of 1 year]

  3. Mean concentrations of serum marker(s) of essential fatty acid status [through study completion, an average of 1 year]

  4. Mean concentrations of serum marker(s) of cardiovascular status [through study completion, an average of 1 year]

  5. Mean protein intake (g/day) as reported on a three-day food record [through study completion, an average of 1 year]

  6. Mean micronutrient intake (%DRI) as reported on a three-day food record [through study completion, an average of 1 year]

Secondary Outcome Measures

  1. Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III [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.

  2. Correlation between serum micronutrient concentrations and average micronutrient intake [through study completion, an average of 1 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Boston Children's Hospital Boston Massachusetts United States 02115

Sponsors and Collaborators

  • Boston Children's Hospital
  • BioMarin Pharmaceutical

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Krista Viau, Clinical Nutrition Specialist II, Boston Children's Hospital
ClinicalTrials.gov Identifier:
NCT03792451
Other Study ID Numbers:
  • P00029611
First Posted:
Jan 3, 2019
Last Update Posted:
Mar 4, 2021
Last Verified:
Mar 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
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
Additional relevant MeSH terms:
Phenylketonurias

Study Results

No Results Posted as of Mar 4, 2021