🏛️ Verified NIH Record • Updated 2026-06-23
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (NCT05270837)
A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria- Open-Label Randomized Two-Arm (Active vs Diet-Only Control)
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: BioMarin Pharmaceutical
🩺 Plain-English Summary (8th-Grade Level)
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
Who can join: Review the eligibility criteria below.
Location: Phoenix Children's Hospital — Phoenix, Arizona
Age range: 12 Years to 17 Years
🧪 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 (16 Sites)
- Phoenix Children's Hospital — Phoenix, Arizona
- Arkansas Children's Hospital — Little Rock, Arkansas
- Children's Hospital of Colorado — Aurora, Colorado
- University of South Florida — Tampa, Florida
- Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- IU Health University Hospital — Indianapolis, Indiana
- University of Kentucky College of Medicine — Lexington, Kentucky
- Boston Children's Hospital — Boston, Massachusetts
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania
- McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) — Houston, Texas
- University of Utah Medical Center — Salt Lake City, Utah
- University of Virginia School of Medicine — Charlottesville, Virginia
- Children's Hospital of Wisconsin — Milwaukee, Wisconsin
- Charité - Universitätsmedizin Berlin — Berlin,
- Universitätsklinikum Hamburg-Eppendorf — Hamburg,
- Universitat Mainz — Mainz,
💰 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)
- 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
- A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) — Aurora, Colorado
Explore by condition: All Phenylketonuria (PKU) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.