Recruiting

Palynziq

Sponsor:

BioMarin Pharmaceutical

Code:

NCT05813678

Conditions

Phenylketonuria (PKU)

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Pegvaliase

Study Details

Brief 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.

Conditions

Phenylketonuria (PKU)

Study ID

NCT05813678

Start date

Jun 6, 2022

Status verified date

Mar, 2026

Completion date

Nov 1, 2033

Anticipated

Primary completion date

Nov 1, 2033

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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  • Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)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

Study Design

Enrollment

450 participants

Anticipated

Interventions and Outcome Measures

Interventions

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).

Primary outcome measure

  • 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. [ Time Frame: A maximum of 10 years treatment duration. ]

Central Contacts and Locations

Central contacts

165-501 Program Director

1-800-983-4587medinfo@bmrn.com

Medical Director, MD

1-800-983-4587medinfo@bmrn.com

Locations

University of Colorado

Recruiting

Aurora, Colorado, United States, 80045

Emory University

Recruiting

Atlanta, Georgia, United States, 30322

Northwestern University

Recruiting

Chicago, Illinois, United States, 60611

Indiana University School of Medicine

Recruiting

Indianapolis, Indiana, United States, 46202

Tulane University Medical Center

Recruiting

New Orleans, Louisiana, United States, 70118

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02215

Oregon Health and Science University

Recruiting

Portland, Oregon, United States, 97239

University of Pittsburgh Medical Center

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Vanderbilt University Medical School

Recruiting

Nashville, Tennessee, United States, 37235

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84112

Medical College of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

Mar 25, 2026

Last verified

Mar, 2026

Keywords

  • Observational
  • Safety Study
  • Pegvaliase
  • Palynziq
  • PKU
  • Phenylketonuria
  • Phase 4

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by BioMarin Pharmaceutical on 2026-03-25.