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

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Trial Status Enrolling By Invitation

Trial Identifier

NCT05356377

Condition

Phenylketonuria, Phenylketonurias

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

18 Years to 55 Years

Healthy Volunteers

No

Drug/Treatment:

Pegvaliase-Pqpz

Study Type:

Observational

Number of Participants:

13

Study Started:

2022-05-25

Study Updated:

2024-07-26

Trial Locations

  • Boston Children's Hospital

    Boston, Massachusetts, United States

  • University of Missouri

    Columbia, Missouri, United States

Inclusion Criteria

  • Age 18-55 years
  • Participants identified by newborn screening with PKU as evidenced by a blood Phe level ≥ 360μmol/L; received treatment within the first 30 days of life
  • Previously received a neuropsychological and neuroimaging evaluation (as part of previously described MU or BCH studies) prior to enrollment in the present study (while Palynziq-naïve).
  • Capable of providing informed consent
  • Presently being treated with Palynziq and has demonstrated a prolonged Phe response to Palynziq (as reflected by at least 3 consecutive months of Phe levels below 360 μmol/L)

Exclusion Criteria

  • History of major neurologic disorder unrelated to PKU
  • Contraindications for safe MRI participation such as (a) pregnancy or plans to become pregnant during period of study enrollment; or (b) metallic objects inside the body (e.g., surgical staples left in the body following surgery, middle ear prosthesis, metal foreign objects lodged inside the eye, heart pacemakers).
  • Current participation in an interventional clinical trial (not Palynziq-related)

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