10-Year study to track immune risks of PKU drug

NCT ID NCT06305234

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• 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