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A Prospective Clinical Study of Phenylketonuria (PKU)

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Trial Status Completed

Trial Identifier

NCT04452513

Condition

Phenylketonuria, Phenylketonurias

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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

Birth Sex

All

Age

From 14 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

32

Study Started:

2019-10-11

Study Updated:

2023-03-14

Trial Locations

  • 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

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 stud

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