A Prospective Clinical Study of Phenylketonuria (PKU)

Completed

Conditions studied: Phenylketonurias

In brief

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.

Key facts

Study ID

NCT04452513

Run by

BioMarin Pharmaceutical

People needed

32

Starts

2019-10-11

Expected to finish

2022-06-02

Last updated by the study team

2023-03-14

Who can join

Age: 14 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Children's Hosptial of Colorado — Aurora, Colorado, United States
• University of Florida — Gainesville, Florida, United States
• University of South Florida — Tampa, Florida, United States
• Emory University Hospital — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Chilren's Hospital — Chicago, Illinois, United States
• Riley Hospital for Children — Indianapolis, Indiana, United States
• University of Kentucky — Lexington, Kentucky, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Morristown Medical Center — Morristown, New Jersey, United States
• Oregon Health & Science University — Portland, Oregon, United States
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Clinic for Special Children — Strasburg, Pennsylvania, United States
• University of Texas Health Science Center at Houston — Houston, Texas, United States
• Medical College of Wisconsin — Wauwatosa, Wisconsin, United States

Full record on ClinicalTrials.gov

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