A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
PALace
A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria
2 other identifiers
observational
450
3 countries
26
Brief Summary
This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects 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 (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2022
Longer than P75 for all trials
26 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
June 6, 2022
CompletedFirst Submitted
Initial submission to the registry
April 3, 2023
CompletedFirst Posted
Study publicly available on registry
April 14, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2033
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 1, 2033
March 25, 2026
March 1, 2026
11.4 years
April 3, 2023
March 23, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real¬ world setting.
Analyses on incident-users are considered primary. The primary analysis is the incidence rate of: Acute systemic hypersensitivity reaction Anaphylaxis Angioedema Serum sickness Severe hypersensitivity reaction Severe or Persistent (≥ 6 months) or arthralgia Severe injection site reaction Hypophenylalaninemia
A maximum of 10 years treatment duration.
Secondary Outcomes (1)
To quantify and characterize the risk of protocol-defined safety events in subjects receiving pegvaliase for the treatment of PKU in a real-world setting.
A maximum of 10 years treatment follow up duration.
Interventions
This is an uncontrolled, single-cohort, Phase 4 observational study not falling under the definition of an Applicable Clinical Trial (ACT) (i.e., non-interventional).
Eligibility Criteria
All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.
You may qualify if:
- Documented diagnosis of PKU per local standard of care
- Currently receiving or planned to receive pegvaliase treatment within 30 days after the date of enrollment, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation.
- Subject (or legally authorized representative) is willing and able to provide written informed consent after the nature of the study has been explained and prior to any data collection.
You may not qualify if:
- Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or comply with any aspect of the study.
- Currently participating in an interventional study of any investigational product, device, or procedure
- Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)
- German subjects \<16 years if age
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (26)
University of Colorado
Aurora, Colorado, 80045, United States
Emory University
Atlanta, Georgia, 30322, United States
Northwestern University
Chicago, Illinois, 60611, United States
Indiana University School of Medicine
Indianapolis, Indiana, 46202, United States
Tulane University Medical Center
New Orleans, Louisiana, 70118, United States
Boston Children's Hospital
Boston, Massachusetts, 02215, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
Vanderbilt University Medical School
Nashville, Tennessee, 37235, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
University of Utah
Salt Lake City, Utah, 84112, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Hospital Carl-Thiem-Klinikum Cottbus
Cottbus, Germany
Universitaetsklinikum Hamburg Eppendorf
Hamburg, Germany
Universitaetslinikum Leipzig AoeR
Leipzig, Germany
Johannes Gutenberg University of Mainz
Mainz, Germany
Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
Münster, Germany
Universität Ulm
Ulm, Germany
Policlinico Sant'orsola Malpighi
Bologna, Italy
University Hospital Careggi
Florence, Italy
Ospedale San Paolo
Milan, Italy
Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
Naples, Italy
Azienda Ospedaliera di Padova
Padua, Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, Italy
Policlinico Gemelli
Rome, Italy
Sapienza University of Rome Hospital
Rome, Italy
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
April 3, 2023
First Posted
April 14, 2023
Study Start
June 6, 2022
Primary Completion (Estimated)
November 1, 2033
Study Completion (Estimated)
November 1, 2033
Last Updated
March 25, 2026
Record last verified: 2026-03