A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Condition(s) studied

Investigational drug(s) / intervention(s)

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

Study summary

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.

Eligibility

Primary outcome measure(s)

• To evaluate immunologic and inflammatory responses associated with occurrences of — A maximum of 10 years treatment duration.
  * Acute systemic hypersensitivity reaction * Anaphylaxis * Angioedema * Serum sickness * Severe hypersensitivity reaction * Severe or Persistent arthralgia * Severe injection site reaction

Trial sites (11)