Status:
RECRUITING
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Lead Sponsor:
Emory University
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Phenylketonurias
Eligibility:
All Genders
16+ years
Brief 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 an...
Detailed Description
Phenylketonuria (PKU) is an autosomal recessive disorder caused by more than 500 pathogenic variants in the phenylalanine hydroxylase (PAH) gene. Due to these mutations, affected individuals have redu...
Eligibility Criteria
Inclusion
- 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
- unable to provide consent
- a diagnosis of, or take medication for psychiatric, behavioral, or other inherited metabolic disorders
Key Trial Info
Start Date :
October 8 2019
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
November 1 2027
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT04404530
Start Date
October 8 2019
End Date
November 1 2027
Last Update
November 22 2024
Active Locations (2)
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1
Emory University Hospital Georgia Clinical Research Center
Atlanta, Georgia, United States, 30322
2
The Emory Clinic
Atlanta, Georgia, United States, 30322