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Clinical Trials in Italy / NCT05813678
Recruiting Observational

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT05813678 · tracked via the Priya Life Science Italy tracker
Sponsor
BioMarin Pharmaceutical
Phase
Observational
Started
2022-06-06
Last updated
2026-03-25

Condition(s) studied

Phenylketonuria (PKU)

Investigational drug(s) / intervention(s)

Pegvaliase →

Pegvaliase: This is an uncontrolled, single-cohort, Phase 4 observational study not falling under the definition of an Applicable Clinical Trial (ACT) (i.e., non-interventional).

Study summary

This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study.

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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of any investigational product, device, or procedure * Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date) * German subjects \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

Primary outcome measure(s)

  • To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real¬ world setting. — A maximum of 10 years treatment duration.
    Analyses on incident-users are considered primary. The primary analysis is the incidence rate of: Acute systemic hypersensitivity reaction Anaphylaxis Angioedema Serum sickness Severe hypersensitivity reaction Severe or Persistent (≥ 6 months) or arthralgia Severe injection site reaction Hypophenylalaninemia

Trial sites (26)

FacilityCityRegionStatus
University of Colorado Aurora Colorado Recruiting
Emory University Atlanta Georgia Recruiting
Northwestern University Chicago Illinois Recruiting
Indiana University School of Medicine Indianapolis Indiana Recruiting
Tulane University Medical Center New Orleans Louisiana Recruiting
Boston Children's Hospital Boston Massachusetts Recruiting
Oregon Health and Science University Portland Oregon Recruiting
University of Pittsburgh Medical Center Pittsburgh Pennsylvania Recruiting
Vanderbilt University Medical School Nashville Tennessee Recruiting
University of Texas Southwestern Medical Center Dallas Texas Recruiting
University of Utah Salt Lake City Utah Recruiting
Medical College of Wisconsin Milwaukee Wisconsin Recruiting
Hospital Carl-Thiem-Klinikum Cottbus Cottbus Germany Recruiting
Universitaetsklinikum Hamburg Eppendorf Hamburg Germany Recruiting
Universitaetslinikum Leipzig AoeR Leipzig Germany Recruiting
Johannes Gutenberg University of Mainz Mainz Germany Recruiting
Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster Münster Germany Recruiting
Universität Ulm Ulm Germany Recruiting
Policlinico Sant'orsola Malpighi Bologna Italy Recruiting
University Hospital Careggi Florence Italy Recruiting
Ospedale San Paolo Milan Italy Recruiting
Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria Naples Italy Recruiting
Azienda Ospedaliera di Padova Padova Italy Recruiting
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone Palermo Italy Recruiting
Policlinico Gemelli Rome Italy Recruiting
Sapienza University of Rome Hospital Rome Italy Recruiting

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Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia Enrolling by invitation
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature Recruiting
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05813678 on ClinicalTrials.gov ↗ ← All trials in Italy