BMN 307-902 - A Prospective Clinical Study of Phenylketonuria (PKU)

Clinicaltrials.gov ID# NCT04452513  

Brief Summary: This is a study for adults and children ≥ 14 years old who have Phenylketonuria (PKU) with uncontrolled plasma Phe levels. No clinical intervention or study drug is provided by BioMarin in this study.

ELIGIBILITY: 

Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency

Ability and willingness to maintain dietary protein intake consistent with baseline intake

Willingness and capable per investigator opinion to comply with study procedures and requirements

Plasma Phe Levels > 600umol/L​

Recruitment status/timeline: Active study status

Primary Investigator: Stephanie Sacharow, MD

Contact information for investigator or study team: Kimberly Parkin – kimberly.parkin@childrens.harvard.edu