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Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Sponsor: Emory University
Summary
Phenylketonuria (PKU) is an inherited metabolic disorder that impairs the metabolism of the essential amino acid phenylalanine (Phe). Without stringent dietary control, Phe accumulates in the blood and brain of PKU patients, leading to severe cognitive deficits. Achieving metabolic control, defined as blood Phe levels within the range of 120-360 μmol/L, has been a significant challenge for PKU patients using traditional diet therapy. The new FDA approved pharmacologic treatment, Palynziq, offers a new approach that could significantly reduce the burden of PKU by improving blood Phe levels and allowing for a less restrictive diet. As little is known about the global metabolic and physiologic effects of Palynziq, the present study aims to capture changes in diet quality, neurological health, nutritional status, the nutritional metabolome, and patient perceptions of mental and social health with sustained Palynziq therapy.
Official title: Palynziq and PKU: Treatment Impacts on Diet Quality, Neurological Health, Nutritional Status, and the Metabolome
Key Details
Gender
All
Age Range
16 Years - Any
Study Type
OBSERVATIONAL
Enrollment
45
Start Date
2019-10-08
Completion Date
2028-12
Last Updated
2026-01-14
Healthy Volunteers
No
Conditions
Interventions
Palynziq
Participants will take Palynziq as prescribed by their genetics doctor. In accordance with the Palynziq Risk Evaluation and Mitigation Strategy (REMS) protocol, patients will have their first injection of Palynziq at the Emory Genetics Clinic under the supervision of a physician. After initiating therapy, patients will continue to mail in blood spot filter papers and 3-day dietary records to their clinic providers as standard components of clinical care.
Locations (2)
Emory University Hospital Georgia Clinical Research Center
Atlanta, Georgia, United States
The Emory Clinic
Atlanta, Georgia, United States