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.
Conditions
Phenylketonurias
Recruitment Status
COMPLETED
Eligibility Criteria
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
Gender
ALL
Min Age
14 Years
Max Age
N/A
Download Date
2023-03-14
Principal Investigator
N/A
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: