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Long-term safety assessment of pegvaliase in patients with PKU

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

Observational BioMarin Pharmaceutical · NCT05813678

This study is testing the long-term safety of pegvaliase in people with PKU to see how well it works and if there are any side effects over time.

Quick facts

Study typeObservational
Enrollment450 (estimated)
SexAll
SponsorBioMarin Pharmaceutical Industry-sponsored
Locations26 sites (Aurora, Colorado and 25 other locations)
Trial IDNCT05813678 on ClinicalTrials.gov

What this trial studies

This is a 10-year global observational study aimed at characterizing the safety profile of pegvaliase in patients with phenylketonuria (PKU). It will monitor hypersensitivity reactions, long-term safety, and the effectiveness of additional risk minimization measures in subjects receiving pegvaliase. Eligible participants include those who are either about to start pegvaliase treatment or have already begun treatment. The study will gather data through routine clinical practice without mandated visits or procedures.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with PKU who are currently receiving or are planned to receive pegvaliase treatment.

Not a fit: Patients with disorders that impair their ability to provide informed consent or comply with the study requirements may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety and tolerability of pegvaliase for PKU patients.

How similar studies have performed: Other studies have shown success in evaluating the safety of treatments for PKU, but this long-term observational approach is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 age

Where this trial is running

Aurora, Colorado and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions PhenylketonuriaObservationalSafety StudyPegvaliasePalynziqPKUPhase 4
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.