Testing of Four Home Phenylalanine Monitoring Prototype Devices

Completed

• Conditions: Phenylketonurias

• Registration Number: NCT02445521
• Lead Sponsor: Emory University

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.

Detailed Description

A real time, point-of-care and home phenylalanine monitoring system would provide patients with quick results and feedback. Home monitoring devices' phenylalanine measurements will be compared to the current gold-standard measurement of phenylalanine (plasma concentration) in four subjects with PKU and one control subject. The study will enroll subjects with different baseline phenylalanine concentrations (i.e., patients with poor to good metabolic control, assessed by comparing current phenylalanine concentrations to established treatment ranges).

For each prototype device, three separate measurements of phenylalanine per subject will be performed. Three prototype devices require whole blood, thus 1 teaspoon (approximately 4mL) of whole blood will be collected through venipuncture and blood spots will be used to conduct tests. One prototype requires capillary blood to be collected through a fingerstick, and will require three blood spots from the stick. A single plasma amino acid analysis for each participant will also be performed using leftover whole blood from the 4mL blood draw through quantitative ion exchange chromatography, reported as micromoles/L.

Study Timeline

• Study Start: 2015-05
• Primary Completion: 2015-05
• Study Completion: 2015-05
• Study First Submitted: 2015-05-13
• Study First Submitted QC: 2015-05-13
• Study First Posted: 2015-05-15
• Status Verified: 2015-12
• Last Update Submitted: 2015-12-09
• Last Update Posted: 2015-12-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

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

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Intra-variability of the four devices, assessed by phenylalanine values	Day 1	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.

Secondary Outcome Measures

Name	Time	Method
Inter-variability of the four devices, assessed by average phenylalanine concentrations	Day 1	Average phenylalanine values measured by the four tests of each device in each subject will be calculated. Mean concentrations for each device will be compared to the control (gold standard measure of plasma phenylalanine).

Trial Locations

Locations (3)

Emory University Hospital - Atlanta Clinical and Translational Science Institute (ACTSI); 🇺🇸 Atlanta, Georgia, United States

Emory University Genetics Clinic; 🇺🇸 Decatur, Georgia, United States

Children's Healthcare of Atlanta (CHOA); 🇺🇸 Atlanta, Georgia, United States