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RECRUITINGOBSERVATIONAL

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

A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 willing to sign a consent form after the nature of the study has been explained and prior to any data collection. Who Should NOT Join This Trial: - Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written willing to sign a consent form 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of any investigational product, device, or procedure - Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date) - German subjects \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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * 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. Exclusion Criteria: * 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

Treatments Being Tested

DRUG

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 (20)

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