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

Part of paid clinical trials in Aurora, Colorado.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT05813678
Status
Recruiting
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Conditions

  • Phenylketonuria (PKU)

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Pegvaliase — DRUG
    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).

Study Details

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 Dates

Start date
Jun 6, 2022
Status verified
Mar 2026
Primary completion
Nov 1, 2033
Completion
Nov 1, 2033

Study Design

Enrollment
450 participants (estimated)

Primary Outcome Measure

To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real¬ world setting. [ Time Frame: A maximum of 10 years treatment duration. ]

Central Contacts

Locations (12)

FacilityCityStateZIPSite coordinators
University of ColoradoAuroraColorado80045-
Emory UniversityAtlantaGeorgia30322-
Northwestern UniversityChicagoIllinois60611-
Indiana University School of MedicineIndianapolisIndiana46202-
Tulane University Medical CenterNew OrleansLouisiana70118-
Boston Children's HospitalBostonMassachusetts02215-
Oregon Health and Science UniversityPortlandOregon97239-
University of Pittsburgh Medical CenterPittsburghPennsylvania15224-
Vanderbilt University Medical SchoolNashvilleTennessee37235-
University of Texas Southwestern Medical CenterDallasTexas75390-
University of UtahSalt Lake CityUtah84112-
Medical College of WisconsinMilwaukeeWisconsin53226-

Find similar trials in Aurora, CO

By research site
University of Colorado· Aurora, COEmory University· Atlanta, GANorthwestern University· Chicago, ILIndiana University School of Medicine· Indianapolis, INTulane University Medical Center· New Orleans, LABoston Children's Hospital· Boston, MA

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