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

Description

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.

Conditions

Phenylketonuria (PKU)

Study Overview

Study Details

Study overview

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.

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

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

Condition
Phenylketonuria (PKU)
Intervention / Treatment

-

Contacts and Locations

Aurora

University of Colorado, Aurora, Colorado, United States, 80045

Chicago

Northwestern University, Chicago, Illinois, United States, 60611

New Orleans

Tulane University School of Medicine, New Orleans, Louisiana, United States, 70112

Portland

Oregon Health & Science University, Portland, Oregon, United States, 97239

Pittsburgh

University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States, 15213

Dallas

UT Southwestern Medical Center, Dallas, Texas, United States, 75390

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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).

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

BioMarin Pharmaceutical,

Medical Director, MD, STUDY_DIRECTOR, BioMarin Pharmaceutical

Study Record Dates

2033-11-18