Actively Recruiting

All Genders
NCT06305234

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

Led by BioMarin Pharmaceutical · Updated on 2026-03-24

200

Participants Needed

11

Research Sites

512 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Subjects enrolled at US sites participating in the 165-501 study.
Not Eligible

You will not qualify if you...

  • 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).

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 11 locations

1

University of Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

2

Emory University

Atlanta, Georgia, United States, 30322

Actively Recruiting

3

Northwestern University

Chicago, Illinois, United States, 60611

Actively Recruiting

4

Riley Children's Hospital/ Indiana University Health

Indianapolis, Indiana, United States, 46202

Actively Recruiting

5

Tulane University School of Medicine

New Orleans, Louisiana, United States, 70112

Actively Recruiting

6

Boston Children's Hospital

Boston, Massachusetts, United States, 02215

Actively Recruiting

7

Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

8

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States, 15213

Actively Recruiting

9

Vanderbilt University Medical School

Nashville, Tennessee, United States, 37235

Actively Recruiting

10

UT Southwestern Medical Center

Dallas, Texas, United States, 75390

Actively Recruiting

11

Children's Wisconsin/Medical College of Wisconsin

Milwaukee, Wisconsin, United States, 53226

Actively Recruiting

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Research Team

1

165-503 Program Director

CONTACT

M

Medical Director, MD

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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