Status:
COMPLETED
A Prospective Clinical Study of Phenylketonuria (PKU)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonurias
Eligibility:
All Genders
14+ years
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 Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 11 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 2 2022
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT04452513
Start Date
October 11 2019
End Date
June 2 2022
Last Update
March 14 2023
Active Locations (14)
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1
Children's Hosptial of Colorado
Aurora, Colorado, United States, 80045
2
University of Florida
Gainesville, Florida, United States, 32605
3
University of South Florida
Tampa, Florida, United States, 33606
4
Emory University Hospital
Atlanta, Georgia, United States, 30322