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A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Sponsor: BioMarin Pharmaceutical
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.
Official title: A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
450
Start Date
2022-06-06
Completion Date
2033-11-01
Last Updated
2026-03-25
Healthy Volunteers
No
Conditions
Interventions
Pegvaliase
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).
Locations (26)
University of Colorado
Aurora, Colorado, United States
Emory University
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Tulane University Medical Center
New Orleans, Louisiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Oregon Health and Science University
Portland, Oregon, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical School
Nashville, Tennessee, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 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
Padova, Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, Italy
Policlinico Gemelli
Rome, Italy
Sapienza University of Rome Hospital
Rome, Italy