Long-term evaluation of immune response in PKU patients receiving Palynziq
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
Run by BioMarin Pharmaceutical · NCT06305234 · Data updated 2026-11-12
This study is testing how well Palynziq works over 10 years for people with PKU by looking at their immune responses and overall health.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 200 (estimated) |
| Sponsor | BioMarin Pharmaceutical Industry-sponsored |
| Locations | 11 sites (Aurora, Colorado and 10 other locations) |
| Status | Recruiting |
| Started | January 20, 2024 |
| Main results expected | November 18, 2033 |
| Study ends | November 18, 2033 |
| Registry record updated | March 24, 2026 |
| Trial ID | NCT06305234 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals with phenylketonuria who are either starting or currently receiving pegvaliase treatment.
✕Probably not a fit
Patients who are not receiving pegvaliase or those unable to provide informed consent may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a 10-year observational study that evaluates the immunologic and inflammatory responses in patients with phenylketonuria (PKU) who are receiving Palynziq (pegvaliase) treatment. The study involves multiple centers across the United States and includes both new and existing users of pegvaliase. Participants will provide blood samples for various laboratory tests at regular intervals to monitor their health and treatment response. The data collected will help understand the long-term effects of Palynziq on patients with PKU.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and efficacy of Palynziq treatment for PKU patients.
How similar studies have gone: While this study builds on existing knowledge of pegvaliase, it focuses on long-term outcomes, which may provide new insights into its use.
Who can join: the full rules
Show full inclusion / exclusion criteria
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).
Where it's running
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University of Colorado
Aurora, Colorado, United States Recruiting -
Emory University
Atlanta, Georgia, United States Recruiting -
Northwestern University
Chicago, Illinois, United States Recruiting -
Riley Children's Hospital/ Indiana University Health
Indianapolis, Indiana, United States Recruiting -
Tulane University School of Medicine
New Orleans, Louisiana, United States Recruiting -
Boston Children's Hospital
Boston, Massachusetts, United States Recruiting
Show 5 more locations
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Oregon Health & Science University
Portland, Oregon, United States Recruiting -
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States Recruiting -
Vanderbilt University Medical School
Nashville, Tennessee, United States Recruiting -
UT Southwestern Medical Center
Dallas, Texas, United States Recruiting -
Children's Wisconsin/Medical College of Wisconsin
Milwaukee, Wisconsin, United States Recruiting
Study contacts
- Study coordinator: 165-503 Program Director
- Email: medinfo@bmrn.com
- Phone: 1-800-983-4587
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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This study tests whether checking phenylalanine levels weekly instead of monthly helps adults with classic PKU keep their levels under better control.
-
MZE782 in adults with phenylketonuria (PKU) testing safety and efficacy
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-
PL54 injection safety in adults with PKU
This study tests whether PL54 is safe and tolerable when given once or in multiple weekly shots to adults with phenylketonuria.
-
Multiomics (genome plus metabolome) approach for adults with phenylketonuria
This project will test whether combining whole-genome sequencing from saliva and non-targeted blood metabolomics can find genetic and metabolic patterns linked to symptoms in adults who were treated for phenylketonuria from birth.
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Investigating brain aging in adults with phenylketonuria
This study looks at how living with phenylketonuria (PKU) affects brain aging and thinking skills in adults who have followed a special diet since they were babies.
-
Long-term JNT-517 treatment for people with phenylketonuria
This trial tests the long-term safety of JNT-517 in children and adults with PKU, including people who previously took JNT-517 and those who are new to the drug.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.