A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) (NCT06305234)
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
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Colorado — Aurora, Colorado
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (11 Sites)
- University of Colorado — Aurora, Colorado
- Emory University — Atlanta, Georgia
- Northwestern University — Chicago, Illinois
- Riley Children's Hospital/ Indiana University Health — Indianapolis, Indiana
- Tulane University School of Medicine — New Orleans, Louisiana
- Boston Children's Hospital — Boston, Massachusetts
- Oregon Health & Science University — Portland, Oregon
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania
- Vanderbilt University Medical School — Nashville, Tennessee
- UT Southwestern Medical Center — Dallas, Texas
- Children's Wisconsin/Medical College of Wisconsin — Milwaukee, Wisconsin
📞 Contact the Study Coordinator
Phone: (800) 983-4587
Email: medinfo@bmrn.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Phenylketonuria (PKU)
- Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria — Phoenix, Arizona
- AAV Gene Therapy Study for Subjects with PKU — Tampa, Florida
- A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) — Aurora, Colorado
- A Long-Term Study of JNT-517 in Participants With Phenylketonuria — Gainesville, Florida
- A Study of MZE782 in Adults With PKU — Oakland, California
Explore by condition: All Phenylketonuria (PKU) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.