Long-term safety assessment of pegvaliase in patients with PKU

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a 10-year global observational study aimed at characterizing the safety profile of pegvaliase in patients with phenylketonuria (PKU). It will monitor hypersensitivity reactions, long-term safety, and the effectiveness of additional risk minimization measures in subjects receiving pegvaliase. Eligible participants include those who are either about to start pegvaliase treatment or have already begun treatment. The study will gather data through routine clinical practice without mandated visits or procedures.

Why it matters

Who can join: the full rules

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* Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)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

Where it's running

• University of Colorado
  Aurora, Colorado, United States Recruiting
• Emory University
  Atlanta, Georgia, United States Recruiting
• Northwestern University
  Chicago, Illinois, United States Recruiting
• Indiana University School of Medicine
  Indianapolis, Indiana, United States Recruiting
• Tulane University Medical Center
  New Orleans, Louisiana, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting

Study contacts

• Study coordinator: 165-501 Program Director
• Email: medinfo@bmrn.com
• Phone: 1-800-983-4587

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• How often to check phenylalanine in adults with classic PKU

  This study tests whether checking phenylalanine levels weekly instead of monthly helps adults with classic PKU keep their levels under better control.

  Recruiting Jagiellonian University · Krakow · NCT07825883

• MZE782 in adults with phenylketonuria (PKU) testing safety and efficacy

  This study tests whether MZE782 works safely for adults with PKU compared with a placebo.

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• PL54 injection safety in adults with PKU

  This study tests whether PL54 is safe and tolerable when given once or in multiple weekly shots to adults with phenylketonuria.

  Recruiting Phase 1 Chongqing Peg-Bio Biopharm Co., Ltd. · Hefei, Anhui · NCT07672756

• Multiomics (genome plus metabolome) approach for adults with phenylketonuria

  This project will test whether combining whole-genome sequencing from saliva and non-targeted blood metabolomics can find genetic and metabolic patterns linked to symptoms in adults who were treated for phenylketonuria from birth.

  Recruiting University Hospital, Tours · Angers and 14 other locations · NCT07484945

• Investigating brain aging in adults with phenylketonuria

  This study looks at how living with phenylketonuria (PKU) affects brain aging and thinking skills in adults who have followed a special diet since they were babies.

  Recruiting Insel Gruppe AG, University Hospital Bern · Bern · NCT06969209

• Long-term JNT-517 treatment for people with phenylketonuria

  This trial tests the long-term safety of JNT-517 in children and adults with PKU, including people who previously took JNT-517 and those who are new to the drug.

  Recruiting at 12 sites Phase 3 Otsuka Pharmaceutical Development & Commercialization, Inc. · Gainesville, Florida and 11 other locations · NCT06628128

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.