Remote Neurocognitive and Psychological Assessment in PKU

Phenylketonurias Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04943393
• Other study ID #: 2034504
• Status: Completed
• Start date: November 24, 2020
• Est. completion date: October 2, 2021

Study information

• Verified date: April 2022
• Source: University of Missouri-Columbia
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 84
• Est. completion date: October 2, 2021
• Est. primary completion date: October 2, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 40 Years

Eligibility

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.

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Other:
• nointervention
Nointervention

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia
United States	University of Missouri	Columbia	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
University of Missouri-Columbia

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Working Memory performance	Composite score based on multiple working memory-related CANTAB tests	2.5-3 hrs total over approx 1-3 weeks