ClinicalTrials.gov record NCT05813678
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
A 10-year, prospective observational study that collects safety information on pegvaliase (Palynziq) in people with PKU. It follows people who are starting pegvaliase within about 30 days or who are already receiving it, using routine clinic visits and medical records. No experimental treatments or extra procedures are required by the study.
What it is studying. The study aims to characterize the long-term safety and tolerability of pegvaliase, including hypersensitivity reactions and other protocol-defined safety events, and to assess the effectiveness of additional risk minimization measures in the EU.
What the researchers hope to learn. To quantify incidence rates of defined safety events (for example acute systemic hypersensitivity, anaphylaxis, serum sickness, severe injection-site reactions, hypophenylalaninemia) in real-world pegvaliase users, to evaluate other serious or severe adverse reactions and treatment discontinuations, and to assess effectiveness of EU additional risk minimization measures.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Pegvaliase
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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 description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participation is observational: investigators will collect medical history and routine-care information about PKU management, pegvaliase use, and concomitant medications. There are no protocol-mandated visits or extra procedures; participants are followed per routine clinical practice (typically monthly during induction/titration and about every 6 months during maintenance) for up to 10 years. Investigators will record and report protocol-defined safety events (for example hypersensitivity reactions, anaphylaxis, serum sickness) and an Independent Adjudication Committee will periodically review these events. No experimental treatments or study-only assessments are planned.
How the study is structured. Prospective, single-group observational study (no experimental treatment assigned). Investigators provide medical history, treatment details, and reports of specific safety events from routine care; there are no protocol-mandated visits or extra procedures. An Independent Adjudication Committee will periodically review defined safety events.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Germany, Italy, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on phenylketonuria
Important dates
- Study start
- Jun 6, 2022
- Primary completion
- Nov 1, 2033 (estimated)
- Study completion
- Nov 1, 2033 (estimated)
- Target enrollment
- 450 participants (estimated)
- Last updated on registry
- Mar 25, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- BioMarin Pharmaceutical
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- 165-501 Program Director 📞 1-800-983-4587 ✉ medinfo@bmrn.com
- Medical Director, MD 📞 1-800-983-4587 ✉ medinfo@bmrn.com
Participating sites (26)
- University of ColoradoRecruiting
Aurora, Colorado, 80045 · United States
- Emory UniversityRecruiting
Atlanta, Georgia, 30322 · United States
- Northwestern UniversityRecruiting
Chicago, Illinois, 60611 · United States
- Indiana University School of MedicineRecruiting
Indianapolis, Indiana, 46202 · United States
- Tulane University Medical CenterRecruiting
New Orleans, Louisiana, 70118 · United States
- Boston Children's HospitalRecruiting
Boston, Massachusetts, 02215 · United States
- Oregon Health and Science UniversityRecruiting
Portland, Oregon, 97239 · United States
- University of Pittsburgh Medical CenterRecruiting
Pittsburgh, Pennsylvania, 15224 · United States
- Vanderbilt University Medical SchoolRecruiting
Nashville, Tennessee, 37235 · United States
- University of Texas Southwestern Medical CenterRecruiting
Dallas, Texas, 75390 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- standard of care
- Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
- legally authorized representative
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- interventional study
- A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
- investigational product
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- concomitant medication
- Other medicines a participant takes during the study. Some must be reported; some are restricted.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
← Back to clinical trials