Skip to main content
OpenTrials
Completed

NCT Number: NCT04943393

Remote Neurocognitive and Psychological Assessment in 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.

Completed

Looking for future studies?

Notify Me

Key information

Age range

18 year–40 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Emory University, Atlanta, Georgia, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

nointervention

Other

Nointervention

Primary outcomes

  1. 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

Sponsors and collaborators

Lead sponsor

University of Missouri-Columbia

Other

Registry information

Official study title

Remote Assessment of Working Memory and Anxiety in Individuals With Early-Treated Phenylketonuria (PKU)

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Jun 29, 2021
Registry last updated
Apr 22, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.