Nutrition Status of Adults Treated With Pegvaliase

Completed

• Conditions: Phenylketonurias

• Registration Number: NCT03792451
• Lead Sponsor: Boston Children's Hospital

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-12-31
• Study First Submitted QC: 2019-01-02
• Study First Posted: 2019-01-03
• Study Start: 2019-01-17
• Primary Completion: 2020-12-31
• Study Completion: 2020-12-31
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-02
• Last Update Posted: 2021-03-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Mean concentrations of serum marker(s) of protein status	through study completion, an average of 1 year
Mean concentrations of serum marker(s) of micronutrient status	through study completion, an average of 1 year
Mean concentrations of serum marker(s) of essential fatty acid status	through study completion, an average of 1 year
Mean concentrations of serum marker(s) of cardiovascular status	through study completion, an average of 1 year
Mean protein intake (g/day) as reported on a three-day food record	through study completion, an average of 1 year
Mean micronutrient intake (%DRI) as reported on a three-day food record	through study completion, an average of 1 year

Secondary Outcome Measures

Name	Time	Method
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.
Correlation between serum micronutrient concentrations and average micronutrient intake	through study completion, an average of 1 year

Trial Locations

Locations (1)

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States