Nutrition Status of Adults Treated With Pegvaliase

NCT ID: NCT03792451

Last Updated: 2021-03-04

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

18 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-01-17

Study Completion Date

2020-12-31

Brief Summary

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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

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Conditions

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Phenylketonurias

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Interventions

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Pegvaliase

Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.

Intervention Type DRUG

Other Intervention Names

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Palynziq

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioMarin Pharmaceutical

INDUSTRY

Sponsor Role collaborator

Boston Children's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Krista Viau

Clinical Nutrition Specialist II

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Krista Viau, PhD, RD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

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Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Countries

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United States

Other Identifiers

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P00029611

Identifier Type: -

Identifier Source: org_study_id

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