Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Recruiting now
Conditions studied: Phenylketonurias
In brief
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.
Key facts
- Study ID
- NCT04404530
- Run by
- Emory University
- People needed
- 45
- Starts
- 2019-10-08
- Expected to finish
- 2028-12-01
- Last updated by the study team
- 2026-01-14
Who can join
Age: 16 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- unable to provide consent
- a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders
Where it is running
- Emory University Hospital Georgia Clinical Research Center — Atlanta, Georgia, United States (enrolling)
- The Emory Clinic — Atlanta, Georgia, United States (enrolling)
Full record on ClinicalTrials.gov
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