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.
Subjects enrolled at US sites participating in the 165-501 study.
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).
Subjects enrolled at US sites participating in the 165-501 study.
Cohort
Prospective
Pegvaliase
DrugMust 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
To evaluate immunologic and inflammatory responses associated with occurrences of
* Acute systemic hypersensitivity reaction * Anaphylaxis * Angioedema * Serum sickness * Severe hypersensitivity reaction * Severe or Persistent arthralgia * Severe injection site reaction
Evaluation of immunologic and/or inflammatory responses over time
To evaluate immunologic and inflammatory responses (immunologic testing, inflammatory markers) associated with occurrences of end-organ function (eg, kidney, liver) related, immune-mediated adverse drug reactions (ADRs)\*
Evaluation of immunologic responses over time
To evaluate the potential association between immunologic responses and blood Phe levels \* A list of end-organ function related, immune-mediated ADRs is included as an appendix to the statistical analysis plan (SAP).
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