10-Year study to track immune risks of PKU drug

NCT ID NCT06305234

Recruiting now Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This 10-year study will follow 200 people with PKU who are taking or about to start Pegvaliase (Palynziq). Researchers will monitor immune reactions, inflammation, and lab results to better understand long-term safety. The goal is to improve management of side effects like allergic reactions and joint pain.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Pegvaliase (Palynziq)
What this could lead to: If successful, this study could help doctors better manage long-term Pegvaliase treatment for PKU, reducing risks like allergic reactions and joint pain.
What could go wrong: This is an observational study, not a treatment trial. It won't test if Pegvaliase works, only monitors side effects over 10 years. Results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHENYLKETONURIA (PKU)

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••
Contact

Email: •••••@•••••

Locations

Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02215, United States
Children's Wisconsin/Medical College of Wisconsin
RECRUITING

Milwaukee, Wisconsin, 53226, United States
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Northwestern University
RECRUITING

Chicago, Illinois, 60611, United States
Oregon Health & Science University
RECRUITING

Portland, Oregon, 97239, United States
Riley Children's Hospital/ Indiana University Health
RECRUITING

Indianapolis, Indiana, 46202, United States
Tulane University School of Medicine
RECRUITING

New Orleans, Louisiana, 70112, United States
UT Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
University of Colorado
RECRUITING

Aurora, Colorado, 80045, United States
University of Pittsburgh Medical Center
RECRUITING

Pittsburgh, Pennsylvania, 15213, United States
Vanderbilt University Medical School
RECRUITING

Nashville, Tennessee, 37235, United States

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