NCT03792451

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2019

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 31, 2018

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 3, 2019

Completed
14 days until next milestone

Study Start

First participant enrolled

January 17, 2019

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2020

Completed
Last Updated

March 4, 2021

Status Verified

March 1, 2021

Enrollment Period

2 years

First QC Date

December 31, 2018

Last Update Submit

March 2, 2021

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (6)

  • 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 Outcomes (2)

  • Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III

    through study completion, an average of 1 year

  • Correlation between serum micronutrient concentrations and average micronutrient intake

    through study completion, an average of 1 year

Interventions

PegvaliaseDRUG

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

Also known as: Palynziq

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

This cross-sectional study will recruit adults with PKU who have been treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. This may include individuals who participated in the phase III clinical trials for pegvaliase.

You may qualify if:

  • Diagnosis of phenylketonuria
  • Aged 18-65 years
  • Currently treated with pegvaliase
  • 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
  • Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)

You may not qualify if:

  • Unable to consent to study
  • Under age 18 years or over age 65
  • Routine food intake provides less than the RDA for protein (0.8 g/kg/d)
  • Consuming any PKU medical foods

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Phenylketonurias

Interventions

pegvaliase

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Krista Viau, PhD, RD

    Boston Children's Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Clinical Nutrition Specialist II

Study Record Dates

First Submitted

December 31, 2018

First Posted

January 3, 2019

Study Start

January 17, 2019

Primary Completion

December 31, 2020

Study Completion

December 31, 2020

Last Updated

March 4, 2021

Record last verified: 2021-03

Data Sharing

IPD Sharing
Will not share

Locations

US(1)