Current Environment:

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:

A Prospective Clinical Study of Phenylketonuria (PKU) NCT04452513