Phenylketonuria (PKU)PALaceNCT05813678
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
An observational study of Phenylketonuria (PKU), run by BioMarin Pharmaceutical at 26 sites in 3 countries.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Phenylketonuria (PKU)
- What is being tested?
- Pegvaliase
- How many people
- 450 participants
- Where
- 26 sitesin 3 countries
- Study dates
- 6 Jun 2022to 1 Nov 2033
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.
Who may be able to join
- 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.
Who cannot join
- 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- Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)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
Trial locations and contacts
26 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
26 sites in 3 countries
United States12 sites, 12 recruiting
- Recruiting
Emory University
Atlanta, Georgia, 30322
- Recruiting
University of Colorado
Aurora, Colorado, 80045
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02215
- Recruiting
Northwestern University
Chicago, Illinois, 60611
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Indiana University School of Medicine
Indianapolis, Indiana, 46202
- Recruiting
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
- Recruiting
Vanderbilt University Medical School
Nashville, Tennessee, 37235
- Recruiting
Tulane University Medical Center
New Orleans, Louisiana, 70118
- Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
- Recruiting
Oregon Health and Science University
Portland, Oregon, 97239
- Recruiting
University of Utah
Salt Lake City, Utah, 84112
Italy8 sites, 8 recruiting
- Recruiting
Policlinico Sant'orsola Malpighi
Bologna
- Recruiting
University Hospital Careggi
Florence
- Recruiting
Ospedale San Paolo
Milan
- Recruiting
Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
Naples
- Recruiting
Azienda Ospedaliera di Padova
Padova
- Recruiting
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo
- Recruiting
Policlinico Gemelli
Rome
- Recruiting
Sapienza University of Rome Hospital
Rome
Germany6 sites, 6 recruiting
- Recruiting
Hospital Carl-Thiem-Klinikum Cottbus
Cottbus
- Recruiting
Universitaetsklinikum Hamburg Eppendorf
Hamburg
- Recruiting
Universitaetslinikum Leipzig AoeR
Leipzig
- Recruiting
Johannes Gutenberg University of Mainz
Mainz
- Recruiting
Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
Münster
- Recruiting
Universität Ulm
Ulm
About this study
Official title: 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.
Read the 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.
- Conditions
- Phenylketonuria (PKU)
- Being tested
- Pegvaliase
- Keywords
- Observational, Safety Study, Pegvaliase, Palynziq, PKU, Phenylketonuria, Phase 4
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegvaliaseDrug
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).
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
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
Measured: A maximum of 10 years treatment duration.
Secondary outcomes (1)
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.
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
Measured: A maximum of 10 years treatment follow up duration.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2023
- Study start6 Jun 2022
- Primary completion1 Nov 2033 (estimated)
- Study completion1 Nov 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Phenylketonuria (PKU)Studies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 450 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05813678
- Sponsor's protocol ID
- 165-501
- Other IDs
- EUPAS34992
- Acronym
- PALace
- Status last verified
- Mar 2026
- First submitted
- 3 Apr 2023
- Last updated
- 25 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria
- ClinicalTrials.gov ID
- NCT05813678
- Acronym / sponsor ID
- PALace / 165-501
- Conditions
- Phenylketonuria (PKU)
- Keywords
- Observational, Safety Study, Pegvaliase, Palynziq, PKU, Phenylketonuria, Phase 4
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 450 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 6 Jun 2022 (actual)
- Primary completion
- 1 Nov 2033 (estimated)
- Study completion
- 1 Nov 2033 (estimated)
- First posted
- 14 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegvaliaseThis is an uncontrolled, single-cohort, Phase 4 observational study not falling under the definition of an Applicable Clinical Trial (ACT) (i.e., non-interventional). | Drug | – |
Primary outcome
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
1 secondary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director, MD study director
BioMarin Pharmaceutical
26 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonuria (PKU), run by BioMarin Pharmaceutical at 26 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Italy, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact 165-501 Program Director (1-800-983-4587, medinfo@bmrn.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2026.