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Completed Phase 3 Interventional Results available

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

ClinicalTrials.gov ID: NCT03694353

Public ClinicalTrials.gov record NCT03694353. 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 Sep 2, 2026, 4:14 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

An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570). The study is designed to evaluate the long-term safety and efficacy of pegvaliase administered at doses \> 40 mg/day to 60 mg/day.

Study identification

NCT ID
NCT03694353
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
BioMarin Pharmaceutical
Industry
Enrollment
37 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
18 Years to 70 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
Sep 12, 2018
Primary completion
Jan 12, 2021
Completion
Jan 12, 2021
Last update posted
Feb 16, 2022

2018 – 2021

United States locations

U.S. sites
17
U.S. states
14
U.S. cities
17
Facility City State ZIP Site status
University of Florida Gainesville Florida 32610
University of South Florida Tampa Florida 33606
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois 60611
Indiana CTSI Clinical Research Center Indianapolis Indiana 46202
University of Kentucky Medical Center Lexington Kentucky 40536
Wayne State University, Center for Molecular Medicine and Genetics, Pediatrics and Pathology Detroit Michigan 48201
University of Missouri Health Care Columbia Missouri 65201
Washington University School of Medicine St Louis Missouri 63110
University of Nebraska Medical Center Omaha Nebraska 68198
Morristown Medical Center Morristown New Jersey 07960
University of Oklahoma Health Science Center Oklahoma City Oklahoma 73104
Oregon Health and Science University Portland Oregon 97239
St. Christopher's Hospital for Children Philadelphia Pennsylvania 19134
UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania 15224
Vanderbilt University Medical Center Nashville Tennessee 37232
University of Texas Health Science Center at Houston Houston Texas 77030
Medical College of Milwaukee 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.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03694353, 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 Feb 16, 2022 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03694353 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →