RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-01-20
Study Completion:2033-11-18
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06305234

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Subjects enrolled at US sites participating in the 165-501 study.
  1. * Legal incapacity or limited legal capacity without legal guardian representation.
  2. * Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling).

Contacts and Locations

Study Contact

165-503 Program Director
CONTACT
1-800-983-4587
medinfo@bmrn.com
Medical Director, MD
CONTACT
1-800-983-4587
medinfo@bmrn.com

Principal Investigator

Medical Director, MD
STUDY_DIRECTOR
BioMarin Pharmaceutical

Study Locations (Sites)

University of Colorado
Aurora, Colorado, 80045
United States
Northwestern University
Chicago, Illinois, 60611
United States
Tulane University School of Medicine
New Orleans, Louisiana, 70112
United States
Oregon Health & Science University
Portland, Oregon, 97239
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
United States
UT Southwestern Medical Center
Dallas, Texas, 75390
United States

Collaborators and Investigators

Sponsor: BioMarin Pharmaceutical

  • Medical Director, MD, STUDY_DIRECTOR, BioMarin Pharmaceutical

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-01-20
Study Completion Date2033-11-18

Study Record Updates

Study Start Date2024-01-20
Study Completion Date2033-11-18

Terms related to this study

Keywords Provided by Researchers

  • PKU
  • Phenylketonuria
  • Palynziq
  • pegvaliase
  • observational
  • safety study
  • immunogenicity assessment
  • inflammatory assessment

Additional Relevant MeSH Terms

  • Phenylketonuria (PKU)