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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

Active, not recruiting

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

January 31, 2022

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: