Skip to main content
TrialFinder
TrialFinder is for informational purposes only and does not provide medical advice. Always talk to your doctor about whether a trial is right for you.
RECRUITINGOBSERVATIONAL

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

Palynziq and PKU: Treatment Impacts on Diet Quality, Neurological Health, Nutritional Status, and the Metabolome

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 16 years of age or older - diagnosed with PKU through newborn screening or via diagnosis later in life - capable of providing consent for medical tests and procedures - prescription for Palynziq and be enrolled in the Palynziq Risk Evaluation and Mitigation Strategy (REMS) program - Substudy: Participants must have completed visit 2 of the main study Who Should NOT Join This Trial: - unable to provide consent - a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * 16 years of age or older * diagnosed with PKU through newborn screening or via diagnosis later in life * capable of providing consent for medical tests and procedures * prescription for Palynziq and be enrolled in the Palynziq Risk Evaluation and Mitigation Strategy (REMS) program * Substudy: Participants must have completed visit 2 of the main study Exclusion Criteria: * unable to provide consent * a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders

Treatments Being Tested

DRUG

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