ClinicalTrials.gov record NCT06305234
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
A Multi-Center, Prospective, Longitudinal, Study Evaluating Immunologic, Inflammatory, and Laboratory Parameters Associated With Long-Term Palynziq® (Pegvaliase) Treatment in Subjects With Phenylketonuria (PKU) in the United States
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.
This is a 10-year, prospective, single‑arm observational study that follows people in a related study (165-501) who are receiving or will receive pegvaliase (Palynziq). It collects blood samples over time to measure immune, inflammatory, and laboratory markers. Data from this study are combined with the parent study to report long-term immune and safety-related laboratory findings.
What it is studying. The study is testing how immunologic and inflammatory markers change during long-term pegvaliase (Palynziq) treatment and whether those markers are associated with hypersensitivity or immune-related adverse events and with blood phenylalanine (Phe) levels. Examples of markers measured include blood Phe, antibodies to pegvaliase (PAL IgG, PEG IgG, PEG IgM, anti-pegvaliase IgE) and inflammatory markers (C3/C4, hsCRP).
What the researchers hope to learn. To describe and track immunologic and inflammatory laboratory changes over long-term pegvaliase treatment; to evaluate associations between those markers and specific hypersensitivity or immune-mediated adverse events and with blood Phe levels; and to compile combined long-term safety and laboratory data for reporting.
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, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US 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 in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.
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
- Subjects enrolled at US sites participating in the 165-501 study.
Exclusion criteria
- Legal incapacity or limited legal capacity without legal guardian representation.
- Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling).
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, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US 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 in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503. Subjects participating in the 165-501 study who consent to participate in this study (165-503) will be requested to provide pre-dose blood samples for Phe, immunologic and inflammatory marker testing approximately every 3 months for the first 3 years of participation, then every 6 months for the remainder of the study. Subjects will be requested to provide additional pre-dose blood samples at the time of Study 165-501 -protocol defined safety events. Additional pre-dose samples will be drawn as per standard of care as detailed in protocol 165-501. The blood Phe, immunologic (PAL IgG, PEG IgG, PEG IgM, anti-pegvaliase IgE), and inflammatory (C3/C4, hsCRP) markers, will be sent to a central laboratory for processing. Data collected in the 165-501 study will be combined with the data collected in this study to decrease burden on sites for data entry and to avoid duplication of ADR reporting (see Criteria for Evaluation). The combined data will be reported in the Clinical Study Report for this study. Subjects can withdraw from Study 165-503 and remain on Study 165-501, but they cannot withdraw from Study 165-501 and remain on Study 165-503 as safety events and other data-points are collected in Study 165-501.
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.
Participants will give pre-dose blood samples for blood phenylalanine (Phe) and immunologic and inflammatory markers about every 3 months for the first 3 years, then every 6 months thereafter, for up to 10 years. Additional pre-dose blood draws may be requested after protocol-defined safety events or per the parent Study 165-501 standard of care. Blood tests include Phe and immunologic markers (PAL IgG, PEG IgG, PEG IgM, anti-pegvaliase IgE) and inflammatory markers (C3/C4, hsCRP); samples are sent to a central laboratory. If you withdraw from this study you may remain in Study 165-501, but you cannot remain in this study if you withdraw from Study 165-501.
How the study is structured. A prospective, observational cohort (single‑arm) study lasting up to 10 years. Participants who are part of the related Study 165-501 and are receiving or planning to receive pegvaliase provide pre-dose blood samples roughly every 3 months for the first 3 years, then every 6 months thereafter. Samples are sent to a central lab for analysis. Data collected here are combined with Study 165-501 to avoid duplicate reporting.
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 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
- Jan 20, 2024
- Primary completion
- Nov 18, 2033 (estimated)
- Study completion
- Nov 18, 2033 (estimated)
- Target enrollment
- 200 participants (estimated)
- Last updated on registry
- Mar 24, 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.
- 165-503 Program Director 📞 1-800-983-4587 ✉ medinfo@bmrn.com
- Medical Director, MD 📞 1-800-983-4587 ✉ medinfo@bmrn.com
Participating sites (11)
- University of ColoradoRecruiting
Aurora, Colorado, 80045 · United States
- Emory UniversityRecruiting
Atlanta, Georgia, 30322 · United States
- Northwestern UniversityRecruiting
Chicago, Illinois, 60611 · United States
- Riley Children's Hospital/ Indiana University HealthRecruiting
Indianapolis, Indiana, 46202 · United States
- Tulane University School of MedicineRecruiting
New Orleans, Louisiana, 70112 · United States
- Boston Children's HospitalRecruiting
Boston, Massachusetts, 02215 · United States
- Oregon Health & Science UniversityRecruiting
Portland, Oregon, 97239 · United States
- University of Pittsburgh Medical CenterRecruiting
Pittsburgh, Pennsylvania, 15213 · United States
- Vanderbilt University Medical SchoolRecruiting
Nashville, Tennessee, 37235 · United States
- UT Southwestern Medical CenterRecruiting
Dallas, Texas, 75390 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- single arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- 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
- 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.
- 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.
- 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.
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.
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