10-Year study tracks safety of PKU drug palynziq

NCT ID NCT05813678

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This 10-year observational study is following 450 people with PKU who are taking or starting pegvaliase (Palynziq) to monitor long-term safety, especially allergic reactions. Researchers are tracking serious side effects like anaphylaxis, severe joint pain, and low phenylalanine levels. The goal is to better understand real-world risks and how well safety measures work.

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 will provide a clearer picture of the long-term safety of pegvaliase for managing PKU, helping doctors and patients make informed treatment decisions.

What could go wrong

This is an observational study, not a controlled trial, so it cannot prove cause and effect. It focuses on safety, not new benefits, and results may take years to emerge.

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.

PHENYLKETONURIA (PKU)

Contacts and locations

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Study contacts

Contact

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

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

Locations

Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
RECRUITING

Naples, Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
RECRUITING

Palermo, Italy
Azienda Ospedaliera di Padova
RECRUITING

Padova, Italy
Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02215, United States
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Hospital Carl-Thiem-Klinikum Cottbus
RECRUITING

Cottbus, Germany
Indiana University School of Medicine
RECRUITING

Indianapolis, Indiana, 46202, United States
Johannes Gutenberg University of Mainz
RECRUITING

Mainz, Germany
Medical College of Wisconsin
RECRUITING

Milwaukee, Wisconsin, 53226, United States
Northwestern University
RECRUITING

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

Portland, Oregon, 97239, United States
Ospedale San Paolo
RECRUITING

Milan, Italy
Policlinico Gemelli
RECRUITING

Rome, Italy
Policlinico Sant'orsola Malpighi
RECRUITING

Bologna, Italy
Sapienza University of Rome Hospital
RECRUITING

Rome, Italy
Tulane University Medical Center
RECRUITING

New Orleans, Louisiana, 70118, United States
Universitaetsklinikum Hamburg Eppendorf
RECRUITING

Hamburg, Germany
Universitaetslinikum Leipzig AoeR
RECRUITING

Leipzig, Germany
University Hospital Careggi
RECRUITING

Florence, Italy
University of Colorado
RECRUITING

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

Pittsburgh, Pennsylvania, 15224, United States
University of Texas Southwestern Medical Center
RECRUITING

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

Salt Lake City, Utah, 84112, United States
Universität Ulm
RECRUITING

Ulm, Germany
Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
RECRUITING

Münster, Germany
Vanderbilt University Medical School
RECRUITING

Nashville, Tennessee, 37235, United States