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Active, not recruiting Phase 3 Interventional

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

ClinicalTrials.gov ID: NCT05270837

Public ClinicalTrials.gov record NCT05270837. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Jun 26, 2026, 4:59 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design

Study identification

NCT ID
NCT05270837
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 3
Lead sponsor
BioMarin Pharmaceutical
Industry
Enrollment
55 participants

Conditions and interventions

Interventions

  • Pegvaliase Drug
  • Diet Only Other

Drug · Other

Eligibility (public fields only)

Age range
12 Years to 17 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jun 16, 2022
Primary completion
Jan 13, 2025
Completion
Sep 30, 2027
Last update posted
Sep 11, 2025

2022 – 2027

United States locations

U.S. sites
13
U.S. states
13
U.S. cities
13
Facility City State ZIP Site status
Phoenix Children's Hospital Phoenix Arizona 85016
Arkansas Children's Hospital Little Rock Arkansas 72202
Children's Hospital of Colorado Aurora Colorado 80045
University of South Florida Tampa Florida 33606
Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois 60611
IU Health University Hospital Indianapolis Indiana 46202
University of Kentucky College of Medicine Lexington Kentucky 40506
Boston Children's Hospital Boston Massachusetts 02115
University of Pittsburgh Medical Center Pittsburgh Pennsylvania 15224
McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) Houston Texas 77030
University of Utah Medical Center Salt Lake City Utah 84132
University of Virginia School of Medicine Charlottesville Virginia 22903
Children's Hospital of Wisconsin Milwaukee Wisconsin 53226

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05270837, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Sep 11, 2025 · Synced Jun 26, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05270837 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →