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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
University of Missouri-Columbia
Study ID
NCT05356377
Status
Completed

Conditions

  • Phenylketonurias

Eligibility Criteria

Sex
ALL
Age
18 Years - 55 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
May 25, 2022
Status verified
Dec 2025
Primary completion
Jul 1, 2025
Completion
Jul 1, 2025

Study Design

Enrollment
6 participants (actual)

Arms

  • Arm: PKU Palynziq Group
    Individuals with PKU who have previously completed baseline (pre-Palnyziq) neurocognitive and neuroimaging evaluations and have since demonstrated a significant and prolonged Phe response to Palynziq (as reflected by at least 3 consecutive months of Phe levels below 360 μmol/L).

Primary Outcome Measure

Neurologic Integrity [ Time Frame: Single timepoint for 1 hour ]

Locations (2)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115-
University of MissouriColumbiaMissouri65211-

Find similar trials in Boston, MA

By research site
Boston Children's Hospital· Boston, MAUniversity of Missouri· Columbia, MO

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