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Long-term Safety Study of Palynziq for PKU

This study, called PALace, is looking at the long-term safety of a medicine called pegvaliase (Palynziq) for people with Phenylketonuria (PKU). PKU is a rare genetic condition where the body can't break down a substance called phenylalanine. The study wants to understand any side effects, including allergic reactions, over a long period, up to 10 years. You can join if you have PKU and are either starting pegvaliase treatment within 30 days or are already taking it. The main goal is to track and understand any safety events that happen while people are using pegvaliase in their daily lives.

Study design
This is an observational study involving up to 450 participants. It is not a clinical trial where a new treatment is being tested, but rather observes people already using pegvaliase.
What's involved
Your doctor will provide information about your medical history, treatments, and any safety concerns after you start pegvaliase. There are no required study visits or procedures.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 10 years of treatment duration to monitor safety events.

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NCT05813678

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

Recruiting
Not specifiedAll AgesObservational
BioMarin Pharmaceutical
~450 participants
Updated 2026-03-25 on ClinicalTrials.gov
What's tested:Pegvaliase

At a glance

Recruiting sites
26 of 26 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
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.
Measured over A maximum of 10 years treatment duration.

Conditions

Phenylketonuria (PKU)

Explore related trials

Phenylketonuria

Where it's being run

26 sites across 14 states
Italy8
Germany6
Colorado1
Georgia1
Illinois1
Indiana1
Louisiana1
Massachusetts1

Study leadership

  • Medical Director, MD · STUDY_DIRECTOR · BioMarin Pharmaceutical

Who to contact

165-501 Program Director
[email protected]
1-800-983-4587
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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • 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.A maximum of 10 years treatment duration.

    Analyses on incident-users are considered primary. The primary analysis is the incidence rate of: Acute systemic hypersensitivity reaction Anaphylaxis Angioedema Serum sickness Severe hypersensitivity reaction Severe or Persistent (≥ 6 months) or arthralgia Severe injection site reaction Hypophenylalaninemia