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Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

NCT05356377 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 6

Last updated 2025-12-29

No results posted yet for this study

Summary

The goal of the proposed study is to elucidate neurologic and neuropsychological improvements associated with Palynziq-related reduction in plasma Phe levels in individuals with PKU. To this end, investigators will utilize state-of-the-art neuropsychological and multi-modal neuroimaging methods to examine the effects of large Phe level reduction (levels \<360 μmol/L for at least 3 consecutive months) on GM and WM brain structures, brain concentrations of Phe, functionality of brain networks, and associated cognitive functioning in a sample of individuals with PKU who are being treated with Palyzniq.

Conditions

  • Phenylketonurias

Interventions

DRUG

Pegvaliase-Pqpz

Participants will have been prescribed pegvaliase-pqpz (Palynziq) by their primary metabolic care providers as part of their standard care. \[Note that prescription and standard care are independent of the present study.\]

Sponsors & Collaborators

Principal Investigators

  • Shawn Christ, PhD · University of Missouri-Columbia

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-05-25
Primary Completion
2025-07-01
Completion
2025-07-01

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05356377 on ClinicalTrials.gov