PKU Extended Treatment with rAvPAL-PEG – Phase II

Long-Term Extension of a Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects with Phenylketonuria (PAL-003)

Protocol Description

The goal of this multi-center Phase II study is to assess whether long-term use of rAvPAL-PEG is safe and can maintain reduced blood phenylalanine (Phe) concentrations in patients with Phenylketonuria (PKU). PKU is a genetic disorder characterized by an inability of the body to utilize Phe, an essential amino acid that cannot be produced by the body, but is obtained through food. Phe accumulates in individuals with PKU, causing health problems.

Study Level: Phase II

Eligibility Criteria

This study is only open to participants who completed participation in an earlier phase of this study (PAL-002).
Males: Ages 16 to 55
Females: Ages 16 to 55


Participants will need to go to the UPMC Montefiore Clinical Translational Research Center at least every week for study drug injections.
Visits: Not applicable
Duration: To be determined

Status: Open for Enrollment

Source(s) of Support

BioMarin Pharmaceutical Inc.

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For information or to enroll, please contact:
Jessica Lindenberger, RN