PhenylketonuriasNCT02445521
Testing of Four Home Phenylalanine Monitoring Prototype Devices
An observational study of Phenylketonurias, run by Emory University at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Phenylketonurias
- How many people
- 9 participants
- Where
- 3 sitesUnited States
- Study dates
- May 2015to May 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Who may be able to join
- diagnosis of PKU or hyperphenylalaninemia (PKU group)
- any age
- in good health (PKU and control group)
Who cannot join
- pregnant
- have any medical comorbidities
- considered unfit for participation by the principal investigator
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Children's Healthcare of Atlanta (CHOA)
Atlanta, Georgia, 30322
Emory University Hospital - Atlanta Clinical and Translational Science Institute (ACTSI)
Atlanta, Georgia, 30322
Emory University Genetics Clinic
Decatur, Georgia, 30033
About this study
Official title: Testing of Four Home Phenylalanine Monitoring Prototype Devices Using Whole Blood From Normal and PKU Subjects
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.
Read the 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.
- Conditions
- Phenylketonurias
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day 1
Secondary outcomes (1)
Inter-variability of the four devices, assessed by average phenylalanine concentrations
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).
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2015 (estimated)
- Study startMay 2015
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- National PKU Alliance
- Responsible party
- Principal investigator, Rani Singh, Emory University
- Investigators
- Rani H Singh, PhD, RD, LD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02445521
- Sponsor's protocol ID
- IRB00079788
- Status last verified
- Dec 2015
- First submitted
- 13 May 2015
- Last updated
- 11 Dec 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Testing of Four Home Phenylalanine Monitoring Prototype Devices Using Whole Blood From Normal and PKU Subjects
- ClinicalTrials.gov ID
- NCT02445521
- Acronym / sponsor ID
- IRB00079788
- Conditions
- Phenylketonurias
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- National PKU Alliance
- Study start
- May 2015
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 15 May 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 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) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- diagnosis of PKU or hyperphenylalaninemia (PKU group)
- any age
- in good health (PKU and control group)
Key exclusion criteria (3 listed)
- pregnant
- have any medical comorbidities
- considered unfit for participation by the principal investigator
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Intra-variability of the four devices, assessed by phenylalanine values
Time frame: 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.
1 secondary outcome
Inter-variability of the four devices, assessed by average phenylalanine concentrations
Time frame: Day 1
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2015.
Related trials for Phenylketonurias
RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rani H Singh, PhD, RD, LD principal investigator
Emory University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonurias, run by Emory University at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Intra-variability of the four devices, assessed by phenylalanine values (time frame: Day 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Dec 2015.