Recruiting

Palynziq

Sponsor:

BioMarin Pharmaceutical

Code:

NCT06305234

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, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US 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 in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.

Conditions

Phenylketonuria (PKU)

Study ID

NCT06305234

Start date

Jan 20, 2024

Status verified date

Mar, 2026

Completion date

Nov 18, 2033

Anticipated

Primary completion date

Nov 18, 2033

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Subjects enrolled at US sites participating in the 165-501 study.

Exclusion Criteria:

  • Legal incapacity or limited legal capacity without legal guardian representation.
  • Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling).

Study Design

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Interventions

Pegvaliase

Must be enrolled in the 165-501 PALace study. Subjects currently receiving or who plan to receive pegvaliase treatment within 30 days after the date of enrollment in the 165-501 study, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation

Primary outcome measure

  • To evaluate immunologic and inflammatory responses associated with occurrences of [ Time Frame: A maximum of 10 years treatment duration. ]

Central Contacts and Locations

Central contacts

165-503 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

Riley Children's Hospital/ Indiana University Health

Recruiting

Indianapolis, Indiana, United States, 46202

Tulane University School of Medicine

Recruiting

New Orleans, Louisiana, United States, 70112

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02215

Oregon Health & Science University

Recruiting

Portland, Oregon, United States, 97239

University of Pittsburgh Medical Center

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

Vanderbilt University Medical School

Recruiting

Nashville, Tennessee, United States, 37235

UT Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

Children's Wisconsin/Medical College of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

Mar 24, 2026

Last verified

Mar, 2026

Keywords

  • PKU
  • Phenylketonuria
  • Palynziq
  • pegvaliase
  • observational
  • safety study
  • immunogenicity assessment
  • inflammatory assessment

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 2026-03-24.