Ignite Creation Date:
2025-12-24 @ 7:36 PM
Ignite Modification Date:
2026-01-02 @ 9:59 AM
Study NCT ID:
NCT03856203
Status:
COMPLETED
Last Update Posted:
2023-11-18
First Post:
2019-02-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Sponsor:
Boston Children's Hospital
Organization:
Study Overview
Official Title:
Longitudinal Assessment of Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Status:
COMPLETED
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: