Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

ClinicalTrials.gov ID: NCT01212744

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Official title

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria

Brief summary

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

The purpose of this study is to evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU.

Study identification

NCT ID: NCT01212744
Recruitment status: Completed
Study type: Interventional
Phase: Phase 2
Lead sponsor: BioMarin Pharmaceutical; Industry
Enrollment: 16 participants

Conditions and interventions

Conditions

Phenylketonuria

Interventions

rAvPAL-PEG Drug

Drug

Eligibility (public fields only)

Age range: 16 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: Mar 2011 (Type not available)
Primary completion: Apr 2015 (Actual)
Completion: Apr 2015 (Actual)
Last update posted: Feb 26, 2019

2011 – 2015

United States locations

U.S. sites: 9
U.S. states: 8
U.S. cities: 9

Facility	City	State	ZIP	Site status
The Children's Hospital	Aurora	Colorado	80045	—
University of Florida	Gainesville	Florida	32610	—
Weisskopf Child Evaluation Center / University of Louisville	Louisville	Kentucky	40202	—
University of Missouri	Columbia	Missouri	65212	—
Washington University Center for Applied Research Sciences	St Louis	Missouri	63110	—
Albany Medical Center	Albany	New York	12208	—
Nationwide Children's Hospital	Columbus	Ohio	43205	—
University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania	15213	—
University of Wisconsin-Madison	Madison	Wisconsin	53705	—

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 NCT01212744, 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 26, 2019 · Synced Nov 15, 2026