- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04943393
Remote Neurocognitive and Psychological Assessment in PKU
April 21, 2022 updated by: Shawn Christ, University of Missouri-Columbia
Remote Assessment of Working Memory and Anxiety in Individuals With Early-Treated Phenylketonuria (PKU)
PKU is a rare autosomal recessive condition associated with disruption in dopamine synthesis.
Although early diagnosis and treatment prevent the severe impairments associated with untreated PKU, individuals with early-treated PKU (ETPKU) nonetheless experience significant sequelae, including impaired working memory and increased risk for anxiety problems.
Past research with other clinical populations (e.g., generalized anxiety disorder) suggests that the two phenomena may be linked, with increased anxiety contributing to poorer WM performance.
Currently we propose to initiate an exciting new line of research examining the potential relationship between anxiety and WM in ETPKU.
The proposed study will also apply remote neuropsychological performance-based assessment to the study of ETPKU and thus overcoming the geographical limitations/challenges associated with recruitment of participants with this rare disease.
Study Overview
Study Type
Observational
Enrollment (Actual)
84
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Georgia
-
Atlanta, Georgia, United States, 30322
- Emory University
-
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Missouri
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Columbia, Missouri, United States, 65211
- University of Missouri
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 40 years (Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Individuals age 18-40 years old with and without PKU
Description
Inclusion Criteria:
- Participants with PKU will be included if they were diagnosed as newborns and immediately started on treatment.
Exclusion Criteria:
- Individuals with a history of neurologic compromise or major medical disorder unrelated to PKU (e.g., closed head injury, diabetes, multiple sclerosis) will be excluded. In addition, individuals with PKU will be excluded if they are on a medication called Peg-Valiant/ PEG-PAL/ Pegvaliase/Palynziq.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Cross-Sectional
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Individuals with PKU
Adults with early-treated PKU
|
Nointervention
|
Individuals without PKU
Adults without PKU who are otherwise healthy
|
Nointervention
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Working Memory performance
Time Frame: 2.5-3 hrs total over approx 1-3 weeks
|
Composite score based on multiple working memory-related CANTAB tests
|
2.5-3 hrs total over approx 1-3 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Shawn Christ, PhD, University of Missouri-Columbia
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 24, 2020
Primary Completion (Actual)
October 2, 2021
Study Completion (Actual)
October 2, 2021
Study Registration Dates
First Submitted
June 2, 2021
First Submitted That Met QC Criteria
June 27, 2021
First Posted (Actual)
June 29, 2021
Study Record Updates
Last Update Posted (Actual)
April 22, 2022
Last Update Submitted That Met QC Criteria
April 21, 2022
Last Verified
April 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2034504
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
IPD Plan Description
To be determined
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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