Actively Recruiting
A Multi-Center Observational Study Evaluating Long-Term Safety of Pegvaliase (Palynziq) Injections for Phenylketonuria
Led by BioMarin Pharmaceutical · Updated on 2026-03-25
450
Participants Needed
26
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a 10-year global, multi-center observational study to better understand the safety profile of pegvaliase, a treatment for Phenylketonuria (PKU). The study focuses on monitoring hypersensitivity reactions, long-term safety, tolerability, and the effectiveness of risk minimization measures used in the European Union. Both new users starting pegvaliase within 30 days of enrollment and those who have already been receiving pegvaliase can participate. Participants in this study will not receive any experimental treatments or undergo special procedures as part of the trial. Instead, their usual pegvaliase treatment and clinical care will continue as normal. Information about their medical history, pegvaliase use, other medications, and safety concerns such as allergic reactions and injection site issues will be collected by their primary physicians. Safety events will be reviewed periodically by an independent committee. Participants are followed according to routine clinical practice, with clinical outcomes assessed regularly, typically monthly during initial treatment phases and every six months during maintenance. The study measures risks of specific safety events over up to 10 years of pegvaliase treatment in a real-world setting. There are no mandatory study visits, and all data collection is based on regular medical care visits.
CONDITIONS
Brief Title
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented diagnosis of PKU according to local standard of care
- Currently receiving or planned to receive pegvaliase treatment within 30 days of enrollment, including those who completed clinical development studies
- Willing and able to provide written informed consent before any data collection
You will not qualify if you...
- Any disorder that may affect ability to provide informed consent or comply with study procedures
- Currently participating in an interventional study involving any investigational product, device, or procedure
- Previously enrolled in this study (including those withdrawn who want to rejoin)
- German subjects under 16 years of age
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - Up to 10 years
Participants who are receiving pegvaliase treatment for PKU are observed for safety and tolerability under routine clinical practice over a period of up to 10 years.
Monthly visits during induction and titration phases, then visits every 6 months during maintenance phase
Trial Site Locations
Total: 26 locations
1
University of Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
2
Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Northwestern University
Chicago, Illinois, United States, 60611
Actively Recruiting
4
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
Actively Recruiting
5
Tulane University Medical Center
New Orleans, Louisiana, United States, 70118
Actively Recruiting
6
Boston Children's Hospital
Boston, Massachusetts, United States, 02215
Actively Recruiting
7
Oregon Health and Science University
Portland, Oregon, United States, 97239
Actively Recruiting
8
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
9
Vanderbilt University Medical School
Nashville, Tennessee, United States, 37235
Actively Recruiting
10
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
11
University of Utah
Salt Lake City, Utah, United States, 84112
Actively Recruiting
12
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
13
Hospital Carl-Thiem-Klinikum Cottbus
Cottbus, Germany
Actively Recruiting
14
Universitaetsklinikum Hamburg Eppendorf
Hamburg, Germany
Actively Recruiting
15
Universitaetslinikum Leipzig AoeR
Leipzig, Germany
Actively Recruiting
16
Johannes Gutenberg University of Mainz
Mainz, Germany
Actively Recruiting
17
Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
Münster, Germany
Actively Recruiting
18
Universität Ulm
Ulm, Germany
Actively Recruiting
19
Policlinico Sant'orsola Malpighi
Bologna, Italy
Actively Recruiting
20
University Hospital Careggi
Florence, Italy
Actively Recruiting
21
Ospedale San Paolo
Milan, Italy
Actively Recruiting
22
Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
Naples, Italy
Actively Recruiting
23
Azienda Ospedaliera di Padova
Padova, Italy
Actively Recruiting
24
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, Italy
Actively Recruiting
25
Policlinico Gemelli
Rome, Italy
Actively Recruiting
26
Sapienza University of Rome Hospital
Rome, Italy
Actively Recruiting
Research Team
1
165-501 Program Director
M
Medical Director, MD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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