A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
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
1 other identifier
observational
200
1 country
11
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jan 2024
Longer than P75 for all trials
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 20, 2024
CompletedFirst Submitted
Initial submission to the registry
January 30, 2024
CompletedFirst Posted
Study publicly available on registry
March 12, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 18, 2033
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 18, 2033
March 24, 2026
March 1, 2026
9.8 years
January 30, 2024
March 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
A maximum of 10 years treatment duration.
Secondary Outcomes (2)
Evaluation of immunologic and/or inflammatory responses over time
A maximum of 10 years treatment duration.
Evaluation of immunologic responses over time
A maximum of 10 years treatment duration.
Interventions
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
Eligibility Criteria
Subjects enrolled at US sites participating in the 165-501 study.
You may qualify if:
- Subjects enrolled at US sites participating in the 165-501 study.
You may not qualify if:
- 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).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
University of Colorado
Aurora, Colorado, 80045, United States
Emory University
Atlanta, Georgia, 30322, United States
Northwestern University
Chicago, Illinois, 60611, United States
Riley Children's Hospital/ Indiana University Health
Indianapolis, Indiana, 46202, United States
Tulane University School of Medicine
New Orleans, Louisiana, 70112, United States
Boston Children's Hospital
Boston, Massachusetts, 02215, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
Vanderbilt University Medical School
Nashville, Tennessee, 37235, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
Children's Wisconsin/Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Biospecimen
A small blood sample may be kept to be used only in a health authority request.
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director, MD
BioMarin Pharmaceutical
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 30, 2024
First Posted
March 12, 2024
Study Start
January 20, 2024
Primary Completion (Estimated)
November 18, 2033
Study Completion (Estimated)
November 18, 2033
Last Updated
March 24, 2026
Record last verified: 2026-03