A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
Recruiting now
Conditions studied: Phenylketonuria (PKU)
In brief
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.
Key facts
- Study ID
- NCT06305234
- Run by
- BioMarin Pharmaceutical
- People needed
- 200
- Starts
- 2024-01-20
- Expected to finish
- 2033-11-18
- Last updated by the study team
- 2026-03-24
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Subjects enrolled at US sites participating in the 165-501 study.
You may not qualify if…
- 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).
Where it is running
- University of Colorado — Aurora, Colorado, United States (enrolling)
- Emory University — Atlanta, Georgia, United States (enrolling)
- Northwestern University — Chicago, Illinois, United States (enrolling)
- Riley Children's Hospital/ Indiana University Health — Indianapolis, Indiana, United States (enrolling)
- Tulane University School of Medicine — New Orleans, Louisiana, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- Oregon Health & Science University — Portland, Oregon, United States (enrolling)
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (enrolling)
- Vanderbilt University Medical School — Nashville, Tennessee, United States (enrolling)
- UT Southwestern Medical Center — Dallas, Texas, United States (enrolling)
- Children's Wisconsin/Medical College of Wisconsin — Milwaukee, Wisconsin, United States (enrolling)
Full record on ClinicalTrials.gov
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