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

Description

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.

Conditions

Phenylketonuria (PKU)

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Phenylketonuria (PKU)
Intervention / Treatment

-

Contacts and Locations

Aurora

University of Colorado, Aurora, Colorado, United States, 80045

Chicago

Northwestern University, Chicago, Illinois, United States, 60611

Indianapolis

Indiana University School of Medicine, Indianapolis, Indiana, United States, 46202

New Orleans

Tulane University Medical Center, New Orleans, Louisiana, United States, 70118

Portland

Oregon Health and Science University, Portland, Oregon, United States, 97239

Pittsburgh

University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States, 15224

Dallas

University of Texas Southwestern Medical Center, Dallas, Texas, United States, 75390

Milwaukee

Medical College of Wisconsin, Milwaukee, Wisconsin, United States, 53226

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

BioMarin Pharmaceutical,

Medical Director, MD, STUDY_DIRECTOR, BioMarin Pharmaceutical

Study Record Dates

2033-11-01