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

NCT05813678 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 450

Last updated 2026-03-25

No results posted yet for this study

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.

Conditions

  • Phenylketonuria (PKU)

Interventions

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).

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-06
Primary Completion
2033-11-01
Completion
2033-11-01
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Italy

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05813678 on ClinicalTrials.gov