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.
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
Clinically significant liver dysfunction or disease
Prior treatment with gene therapy
Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
March 14, 2023
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
For more information and to contact the study team: