NCT02445521

Brief Summary

The objective of this study is to test four home phenylalanine monitoring prototype devices, selected out of a pool of candidates by the National PKU Alliance Scientific Committee.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
9

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started May 2015

Geographic Reach
1 country

3 active sites

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

Study Start

First participant enrolled

May 1, 2015

Completed
Same day until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2015

Completed
12 days until next milestone

First Submitted

Initial submission to the registry

May 13, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 15, 2015

Completed
Last Updated

December 11, 2015

Status Verified

December 1, 2015

Enrollment Period

Same day

First QC Date

May 13, 2015

Last Update Submit

December 9, 2015

Conditions

Outcome Measures

Primary Outcomes (1)

  • Intra-variability of the four devices, assessed by phenylalanine values

    Phenylalanine values for each of the three runs on each device for the same individual will be compared. Each PKU subject will be assessed separately to assess the reliability of each device since the subjects will have a range of phenylalanine concentrations.

    Day 1

Secondary Outcomes (1)

  • Inter-variability of the four devices, assessed by average phenylalanine concentrations

    Day 1

Study Arms (5)

PKU (low phe-level)

A subject diagnosed with phenylketonuria (PKU) with low phenylalanine levels, defined as 1-5 mg/dL

PKU (mid phe-level)

A subject diagnosed with phenylketonuria (PKU) with middle phenylalanine levels, defined as 6-10 mg/dL

PKU (high mid phe-level)

A subject diagnosed with phenylketonuria (PKU) with high middle phenylalanine levels, defined as 11-15 mg/dL

PKU (high phe-level)

A subject diagnosed with phenylketonuria (PKU) with high phenylalanine levels, defined as 16-20+ mg/dL

Control

A normal subject without phenylketonuria (PKU)

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Control will be recruited by distributing an email to all staff on the Emory Genetics Clinic listserv to solicit a volunteer to provide 4ml of whole blood only. Respondents who are within two years of age and the same gender of at least one of the PKU subjects will be considered eligible. Remaining subjects will be recruited through Emory Genetics Clinic and possibly through the Emory Metabolic Nutrition Program's Annual Metabolic Camp.

You may qualify if:

  • diagnosis of PKU or hyperphenylalaninemia (PKU group)
  • any age
  • in good health (PKU and control group)

You may not qualify if:

  • pregnant
  • have any medical comorbidities
  • considered unfit for participation by the principal investigator

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Children's Healthcare of Atlanta (CHOA)

Atlanta, Georgia, 30322, United States

Location

Emory University Hospital - Atlanta Clinical and Translational Science Institute (ACTSI)

Atlanta, Georgia, 30322, United States

Location

Emory University Genetics Clinic

Decatur, Georgia, 30033, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Whole blood and plasma

MeSH Terms

Conditions

Phenylketonurias

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

  • Rani H Singh, PhD, RD, LD

    Emory University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

May 13, 2015

First Posted

May 15, 2015

Study Start

May 1, 2015

Primary Completion

May 1, 2015

Study Completion

May 1, 2015

Last Updated

December 11, 2015

Record last verified: 2015-12

Locations