A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Recruiting now

Conditions studied: Phenylketonuria (PKU)

In brief

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.

Key facts

Study ID

NCT05813678

Run by

BioMarin Pharmaceutical

People needed

450

Starts

2022-06-06

Expected to finish

2033-11-01

Last updated by the study team

2026-03-25

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

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• Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)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

Where it is running

• Universitaetsklinikum Hamburg Eppendorf — Hamburg, Germany (enrolling)
• Universitaetslinikum Leipzig AoeR — Leipzig, Germany (enrolling)
• Johannes Gutenberg University of Mainz — Mainz, Germany (enrolling)
• Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster — Münster, Germany (enrolling)
• Universität Ulm — Ulm, Germany (enrolling)
• Policlinico Sant'orsola Malpighi — Bologna, Italy (enrolling)
• University Hospital Careggi — Florence, Italy (enrolling)
• Ospedale San Paolo — Milan, Italy (enrolling)
• Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria — Naples, Italy (enrolling)
• Azienda Ospedaliera di Padova — Padova, Italy (enrolling)
• Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone — Palermo, Italy (enrolling)
• Policlinico Gemelli — Rome, Italy (enrolling)
• Sapienza University of Rome Hospital — Rome, Italy (enrolling)
• University of Colorado — Aurora, Colorado, United States (enrolling)
• Emory University — Atlanta, Georgia, United States (enrolling)
• Northwestern University — Chicago, Illinois, United States (enrolling)
• Indiana University School of Medicine — Indianapolis, Indiana, United States (enrolling)
• Tulane University Medical Center — New Orleans, Louisiana, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Oregon Health and Science University — Portland, Oregon, United States (enrolling)
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (enrolling)
• Vanderbilt University Medical School — Nashville, Tennessee, United States (enrolling)
• University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• University of Utah — Salt Lake City, Utah, United States (enrolling)
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States (enrolling)

Full record on ClinicalTrials.gov

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