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Decade-Long safety watch launched for rare disease treatment

NCT ID NCT05813678

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

Summary

This 10-year observational study aims to monitor the long-term safety of Palynziq (pegvaliase) injections in people with phenylketonuria (PKU). Researchers will follow 450 participants who are already taking or planning to start the medication to track potential side effects like allergic reactions and injection site issues. The study collects real-world safety data without requiring any experimental treatments or extra procedures beyond normal care.

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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Contacts and locations

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

    Padua, 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

Conditions

Explore the condition pages connected to this study.

PHENYLKETONURIA (PKU)