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RECRUITINGOBSERVATIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - Subjects enrolled at US sites participating in the 165-501 study. Who Should NOT Join This Trial: - Legal incapacity or limited legal capacity without legal guardian representation. - Subject is unable or unwilling to provide willing to sign a consent form for the additional interventional burden of the study (blood sampling). Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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).

Treatments Being Tested

DRUG

Pegvaliase

Must be enrolled in the 165-501 PALace study. Subjects currently receiving or who plan to receive pegvaliase treatment within 30 days after the date of enrollment in the 165-501 study, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation

Locations (11)

University of Colorado
Aurora, Colorado, United States
Emory University
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
Riley Children's Hospital/ Indiana University Health
Indianapolis, Indiana, United States
Tulane University School of Medicine
New Orleans, Louisiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical School
Nashville, Tennessee, United States
UT Southwestern Medical Center
Dallas, Texas, United States
Children's Wisconsin/Medical College of Wisconsin
Milwaukee, Wisconsin, United States