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

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Trial Status Recruiting

Trial Identifier

NCT05813678

Condition

Phenylketonuria, Phenylketonuria (PKU)

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Drug/Treatment:

Pegvaliase

Study Type:

Observational

Number of Participants:

450

Study Started:

2022-06-06

Study Updated:

2024-05-13

Trial Locations

  • University of Colorado

    Aurora, Colorado, 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

  • Oregon Health and Science University

    Portland, Oregon, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, United States

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, United States

  • Hospital Carl-Thiem-Klinikum Cottbus

    Cottbus, , Germany

  • Universitaetsklinikum Hamburg Eppendorf

    Hamburg, , Germany

  • Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster

    Münster, , Germany

  • Universität Ulm

    Ulm, , Germany

  • Policlinico Sant'orsola Malpighi

    Bologna, , Italy

  • University Hospital Careggi

    Firenze, , Italy

  • Ospedale San Paolo

    Milan, , Italy

  • Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria

    Naples, , Italy

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 ag

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