Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects with PKU (BioMarin Pharmaceutical Inc. Protocol PAL-003)
ID Number 09-2049Principal Investigator(s)
Melissa P. Wasserstein
Department(s) or Division(s)
Genetics and Genomic Sciences
The purpose of the study is to find out what good effects and side effects the study drug rAvPAL-PEG has on people with Phenylketonuria (PKU). It is also being done to see if the drug has any effect on Phenylalanine (Phe) levels for people with PKU. PKU is a condition that people are born with and it cannot be cured. Phe is a substance needed in the body that can only be obtained from the foods we eat. People with PKU cannot get rid of Phe in their body. Large amounts of Phe can cause problems, such as mental retardation and problems with attention and impulse control. This trial is a Phase 2 study which means there will be still be more studies completed before the FDA will consider this drug for the market.
Recruiting Patients: No