Phenylketonuria (PKU)NCT06305234
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
An observational study of Phenylketonuria (PKU), run by BioMarin Pharmaceutical at 11 sites in United States.
Trial summary
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). 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
- 200 participants
- Where
- 11 sitesUnited States
- Study dates
- 20 Jan 2024to 18 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: Subjects enrolled at US sites participating in the 165-501 study.
Who may be able to join
- Subjects enrolled at US sites participating in the 165-501 study.
Who cannot join
- 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).
Trial locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
United States11 sites, 11 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
UT Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Riley Children's Hospital/ Indiana University Health
Indianapolis, Indiana, 46202
- Recruiting
Children's Wisconsin/Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
- Recruiting
Vanderbilt University Medical School
Nashville, Tennessee, 37235
- Recruiting
Tulane University School of Medicine
New Orleans, Louisiana, 70112
- Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: 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
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.
Read the detailed description
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.
- Conditions
- Phenylketonuria (PKU)
- Being tested
- Pegvaliase
- Keywords
- PKU, Phenylketonuria, Palynziq, pegvaliase, observational, safety study, immunogenicity assessment, inflammatory assessment
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegvaliaseDrug
Must be enrolled in the 165-501 PALace study. Subjects currently receiving or who plan to receive pegvaliase treatment within 30 days after the date of enrollment in the 165-501 study, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate immunologic and inflammatory responses associated with occurrences of
- Acute systemic hypersensitivity reaction
- Anaphylaxis
- Angioedema
- Serum sickness
- Severe hypersensitivity reaction
- Severe or Persistent arthralgia
- Severe injection site reaction
Measured: A maximum of 10 years treatment duration.
Secondary outcomes (2)
Evaluation of immunologic and/or inflammatory responses over time
To evaluate immunologic and inflammatory responses (immunologic testing, inflammatory markers) associated with occurrences of end-organ function (eg, kidney, liver) related, immune-mediated adverse drug reactions (ADRs)*
Measured: A maximum of 10 years treatment duration.
Evaluation of immunologic responses over time
To evaluate the potential association between immunologic responses and blood Phe levels
- A list of end-organ function related, immune-mediated ADRs is included as an appendix to the statistical analysis plan (SAP).
Measured: A maximum of 10 years treatment duration.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2024
- Study start20 Jan 2024
- Primary completion18 Nov 2033 (estimated)
- Study completion18 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
- 200 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
- NCT06305234
- Sponsor's protocol ID
- 165-503
- Status last verified
- Mar 2026
- First submitted
- 30 Jan 2024
- Last updated
- 24 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06305234
- Acronym / sponsor ID
- 165-503
- Conditions
- Phenylketonuria (PKU)
- Keywords
- PKU, Phenylketonuria, Palynziq, pegvaliase, observational, safety study, immunogenicity assessment, inflammatory assessment
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 20 Jan 2024 (actual)
- Primary completion
- 18 Nov 2033 (estimated)
- Study completion
- 18 Nov 2033 (estimated)
- First posted
- 12 Mar 2024 (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, 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.
Detailed description
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.
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
- Subjects enrolled at US sites participating in the 165-501 study.
Key inclusion criteria (1 listed)
- Subjects enrolled at US sites participating in the 165-501 study.
Key exclusion criteria (2 listed)
- 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).
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 |
|---|---|---|
| PegvaliaseMust be enrolled in the 165-501 PALace study. Subjects currently receiving or who plan to receive pegvaliase treatment within 30 days after the date of enrollment in the 165-501 study, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation | Drug | – |
Primary outcome
To evaluate immunologic and inflammatory responses associated with occurrences of
Time frame: A maximum of 10 years treatment duration.
* Acute systemic hypersensitivity reaction * Anaphylaxis * Angioedema * Serum sickness * Severe hypersensitivity reaction * Severe or Persistent arthralgia * Severe injection site reaction
2 secondary outcomes
Evaluation of immunologic and/or inflammatory responses over time
Time frame: A maximum of 10 years treatment duration.
Evaluation of immunologic responses over time
Time frame: A maximum of 10 years treatment 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 24 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
11 sites in 1 country: 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 11 sites in United States.
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 1 inclusion and 2 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 24 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 11 sites in United States. 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 165-503 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 evaluate immunologic and inflammatory responses associated with occurrences of (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 24 Mar 2026; AzTrials last refreshed its copy on 6 Nov 2026.