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A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
A Multi-Center, Prospective, Longitudinal, Study Evaluating Immunologic, Inflammatory, and Laboratory Parameters Associated With Long-Term Palynziq® (Pegvaliase) Treatment in Subjects With Phenylketonuria (PKU) in the United States
Who can join
All ages · All sexes
Full eligibility criteria
Inclusion Criteria: * Subjects enrolled at US sites participating in the 165-501 study. Exclusion Criteria: * Legal incapacity or limited legal capacity without legal guardian representation. * Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling).
About the study
This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.
What is being tested
- Pegvaliase (drug)
Sponsor: BioMarin Pharmaceutical · Participants: 200 · Started: Jan 20, 2024
Contact the study team
- 165-503 Program Director · Phone: 1-800-983-4587
- Medical Director, MD · Phone: 1-800-983-4587
Official record on ClinicalTrials.gov — NCT06305234
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.