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10-Year study tracks safety of PKU drug palynziq

NCT ID NCT05813678

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Study facts

What this study's own registry entry says, in plain language.

Participants

About 450 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2022

Expected to finish

Nov 2033

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Documented diagnosis of PKU per local standard of care * Currently receiving or planned to receive pegvaliase treatment within 30 days after the date of enrollment, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation. * Subject (or legally authorized representative) is willing and able to provide written informed consent after the nature of the study has been explained and prior to any data collection. Exclusion Criteria: * Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or comply with any aspect of the study. * Currently participating in an interventional study of any investigational product, device, or procedure * Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date) * German subjects \<16 years if age

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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)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    26 sites in 3 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    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

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