Clinical Trials Directory

Trials / Completed

CompletedNCT03856203

Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Longitudinal Assessment of Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Status
Completed
Phase
Study type
Observational
Enrollment
12 (actual)
Sponsor
Boston Children's Hospital · Academic / Other
Sex
All
Age
18 Years – 65 Years
Healthy volunteers
Not accepted

Summary

Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).

Detailed description

The investigators will compare intra-subject change in adults with PKU who follow a protein-restricted diet (protein intake less than the RDA for protein based on weight (0.8 g/kg/day) with or without a PKU medical food) within 90 days of starting pegvaliase (baseline) and 9 and 15 months later. The investigators will collect laboratory measures of nutrition status, anthropometrics, Dual-Energy X-Ray Absorptiometry (DXA) to measure body composition and bone mineral density (BMD), and indirect calorimetry to measure resting energy expenditure (REE). In addition, three-day food records will be obtained to assess protein intake and overall diet quality.

Conditions

Timeline

Start date
2019-03-08
Primary completion
2022-12-31
Completion
2023-10-20
First posted
2019-02-27
Last updated
2023-11-18

Locations

1 site across 1 country: United States

Source: ClinicalTrials.gov record NCT03856203. Inclusion in this directory is not an endorsement.