A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Part of paid clinical trials in Aurora, Colorado.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT06305234
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
    Must be enrolled in the 165-501 PALace study. Subjects currently receiving or who plan to receive pegvaliase treatment within 30 days after the date of enrollment in the 165-501 study, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation

Study Details

This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US 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 in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.

Key Dates

Start date
Jan 20, 2024
Status verified
Mar 2026
Primary completion
Nov 18, 2033
Completion
Nov 18, 2033

Study Design

Enrollment
200 participants (estimated)

Primary Outcome Measure

To evaluate immunologic and inflammatory responses associated with occurrences of [ Time Frame: A maximum of 10 years treatment duration. ]

Central Contacts

Locations (11)

FacilityCityStateZIPSite coordinators
University of ColoradoAuroraColorado80045-
Emory UniversityAtlantaGeorgia30322-
Northwestern UniversityChicagoIllinois60611-
Riley Children's Hospital/ Indiana University HealthIndianapolisIndiana46202-
Tulane University School of MedicineNew OrleansLouisiana70112-
Boston Children's HospitalBostonMassachusetts02215-
Oregon Health & Science UniversityPortlandOregon97239-
University of Pittsburgh Medical CenterPittsburghPennsylvania15213-
Vanderbilt University Medical SchoolNashvilleTennessee37235-
UT Southwestern Medical CenterDallasTexas75390-
Children's Wisconsin/Medical College of WisconsinMilwaukeeWisconsin53226-

Find similar trials in Aurora, CO

By research site
University of Colorado· Aurora, COEmory University· Atlanta, GANorthwestern University· Chicago, ILRiley Children's Hospital/ Indiana University Health· Indianapolis, INTulane University School of Medicine· New Orleans, LABoston Children's Hospital· Boston, MA

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