PKU Treatment with BMN 165 (rAvPAL-PEG) – Phase III

Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self Administered by Adults With Phenylketonuria

Protocol Description

This research study is to learn if a drug called BMN 165 (also known as rAvPAL-PEG) can help people with Phenylketonuria (PKU). Specifically, researchers are determining whether BMN 165 can reduce concentrations of phenylalanine (Phe) in the blood of patients with phenylketonuria (PKU) when injected by the patient, or someone who helps with their care. 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 III

Eligibility Criteria

Subject to exclusion criteria, the study is accepting adult patients of both genders who have a confirmed diagnosis of PKU and who participated in a prior study where they were given BMN 165.
Men and Women: Ages 18 to 70


Through the screening process, participants will be given physical examinations, which will include blood and urine sampling, and electrocardiogram. Participants or caregivers will be taught how to administer an injection. For most of the duration of the study, which lasts about 4-1/2 years, participants will need daily injections of the study drug. There are four parts to the study. For certain intervals, by random selection some participants will take a placebo. At other intervals, additional study medications will be consumed along with the daily injections. Clinic visits for additional examinations, blood testing and monitoring range from two weeks apart to eight weeks apart, depending on the study phase.
Visits: About 42
Duration: About 4-1/2 years

Status: Enrolling by Invitation

Source(s) of Support

BioMarin Pharmaceutical

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Jessica Lindenberger, RN