Research

PKU Treatment with rAvPAL-PEG – Phase II

A Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects with Phenylketonuria (PAL-002)

Protocol Description

The goal of this multi-center Phase II study is to assess the effects and side effects of rAvPAL-PEG when used to reduce 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

Subject to certain screening criteria, this study is open to participants of both genders, ages 16 to 55, who have been diagnosed with PKU.
Males: Ages 16 to 55
Females: Ages 16 to 55

Requirements
Participants receive a comprehensive physical examination, including blood tests, chest x-rays, heart monitoring and urine sampling. For the first 8-14 weeks of the study, the study drug will be given by injection weekly. In the latter weeks of the study, the drug dose may be changed. Blood testing will be done 36 to 41 times over the course of the study. Visits will be done at UPMC Montefiore Clinical Translational Research Center.
Visits: At least weekly for injections.
Duration: 22 weeks, possibly with additional testing up to 6 months after the final visit.

Status: Open for Enrollment

Sources of Support
BioMarin Pharmaceutical Inc.

Additional Resources
Study description at National Institutes of Health