Actively Recruiting
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Led by Emory University · Updated on 2026-01-14
45
Participants Needed
2
Research Sites
477 weeks
Total Duration
On this page
Sponsors
E
Emory University
Lead Sponsor
B
BioMarin Pharmaceutical
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 16 years of age or older
- Diagnosed with PKU through newborn screening or later diagnosis
- Able to provide consent for medical tests and procedures
- Have a prescription for Palynziq and enrolled in the Palynziq REMS program
- For sub-study: Completed visit 2 of the main study
You will not qualify if you...
- Unable to provide consent
- Diagnosed with or taking medication for psychiatric, behavioral, or other inherited metabolic disorders
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Emory University Hospital Georgia Clinical Research Center
Atlanta, Georgia, United States, 30322
Actively Recruiting
2
The Emory Clinic
Atlanta, Georgia, United States, 30322
Actively Recruiting
Research Team
R
Rani Singh, PhD, RDN, LD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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