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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

12 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-03-08

Study Completion Date

2023-10-20

Brief Summary

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

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Detailed Description

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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

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Phenylketonurias

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of phenylketonuria
* Aged 18-65 years
* Following a protein-restricted diet with or without medical food within the last 30 days
* Routine natural protein intake from food is less than the RDA for protein based on weight (0.8 g/kg/day).
* If not consuming animal proteins, recommended protein intake is 115% RDA due to lower bioavailability of plant proteins.
* Within ±90 days of starting treatment with pegvaliase at time of study enrollment

Exclusion Criteria

* Unable to consent to study
* Under age 18 or over age 65 years
* Routine natural protein intake is greater than the RDA for protein (0.8 g/kg/d) within the last 30 days.
* Females who are currently pregnant or lactating

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No