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

March 23, 2026 updated by: BioMarin Pharmaceutical

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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 aRMMs (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. Once the subject has been enrolled, the investigator (ie, subject's primary physician) will be asked to provide information about the subject's medical history, treatment(s) received to manage their blood Phe levels, treatment with pegvaliase, and concomitant medication use. The investigator will also be asked to provide evaluation of the following safety concerns: Acute systemic hypersensitivity reaction, Anaphylaxis, Angioedema, Serum sickness, Severe hypersensitivity reaction, Severe or Persistent arthralgia, Severe injection site reaction, Hypophenylalaninemia and other protocol-defined safety events, following the initiation of pegvaliase therapy. There are no protocol-mandated visits or procedures associated with this study. Enrolled subjects should be followed per routine clinical practice at the institution based upon their diagnosis, with clinical outcomes assessed at regular intervals (typically every month during the induction and titration phases of pegvaliase treatment and every 6 months once the maintenance phase is reached). No experimental treatment or assessments are planned as part of this observational study. An Independent Adjudication Committee (IAC) will periodically review protocol-defined safety events, including Acute systemic hypersensitivity reaction and Serum sickness.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
450

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Medical Director, MD
  • Phone Number: 1-800-983-4587
  • Email: medinfo@bmrn.com

Study Contact Backup

  • Name: 165-501 Program Director
  • Phone Number: 1-800-983-4587
  • Email: medinfo@bmrn.com

Study Locations

  • Germany
      • Cottbus, Germany
        • Recruiting
        • Hospital Carl-Thiem-Klinikum Cottbus
      • Hamburg, Germany
        • Recruiting
        • Universitaetsklinikum Hamburg Eppendorf
      • Leipzig, Germany
        • Recruiting
        • Universitaetslinikum Leipzig AoeR
      • Mainz, Germany
        • Recruiting
        • Johannes Gutenberg University of Mainz
      • Münster, Germany
        • Recruiting
        • Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
      • Ulm, Germany
        • Recruiting
        • Universität Ulm
  • Italy
      • Bologna, Italy
        • Recruiting
        • Policlinico Sant'Orsola Malpighi
      • Florence, Italy
        • Recruiting
        • University Hospital Careggi
      • Milan, Italy
        • Recruiting
        • Ospedale San Paolo
      • Naples, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
      • Padua, Italy
        • Recruiting
        • Azienda Ospedaliera di Padova
      • Palermo, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
      • Rome, Italy
        • Recruiting
        • Policlinico Gemelli
      • Rome, Italy
        • Recruiting
        • Sapienza University of Rome Hospital
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Indiana University School of Medicine
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Recruiting
        • Tulane University Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Boston Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • University of Pittsburgh Medical Center
    • Tennessee
      • Nashville, Tennessee, United States, 37235
        • Recruiting
        • Vanderbilt University Medical School
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • University of Texas Southwestern Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Recruiting
        • University of Utah
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.

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
A maximum of 10 years treatment duration.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
To quantify and characterize the risk of protocol-defined safety events in subjects receiving pegvaliase for the treatment of PKU in a real-world setting.
Time Frame: A maximum of 10 years treatment follow up duration.

Secondary analyses under safety events will include:

The incidence rate of:

  • End-organ damage associated with immune-complex formation, or PEG accumulation.
  • SAEs• Severe ADRs
  • ADRs leading to treatment interruption or discontinuation and/or study discontinuation
A maximum of 10 years treatment follow up duration.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, MD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 6, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 1, 2033

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2033

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 3, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 3, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 14, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 23, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 165-501
  • EUPAS34992 (Registry Identifier: European Network of Centres for Pharmacoepidemiology and Pharmacovigilance)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings