Long-term evaluation of immune response in PKU patients receiving Palynziq
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 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 |
|---|---|
| Enrollment | 200 (estimated) |
| Sex | All |
| Sponsor | BioMarin Pharmaceutical Industry-sponsored |
| Locations | 11 sites (Aurora, Colorado and 10 other locations) |
| Trial ID | NCT06305234 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are individuals with phenylketonuria who are either starting or currently receiving pegvaliase treatment.
Not a fit: Patients who are not receiving pegvaliase or those unable to provide informed consent may not benefit from this study.
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 performed: While this study builds on existing knowledge of pegvaliase, it focuses on long-term outcomes, which may provide new insights into its use.
Eligibility criteria
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 this trial is running
Aurora, Colorado and 10 other locations
- 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)
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.