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A Prospective Clinical Study of Phenylketonuria (PKU)

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04452513
Collaborator
(none)
90
Enrollment
14
Locations
44.6
Anticipated Duration (Months)
6.4
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    90 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    A Prospective Clinical Study of Phenylketonuria (PKU)
    Actual Study Start Date :
    Oct 11, 2019
    Anticipated Primary Completion Date :
    Jul 1, 2023
    Anticipated Study Completion Date :
    Jul 1, 2023

    Outcome Measures

    Primary Outcome Measures

    1. Change in Plasma Phe Levels [Baseline, week 92]

    Secondary Outcome Measures

    1. Change in dietary protein intake from medical and intact food [Baseline, week 92]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    14 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    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
    5 Ann & Robert H. Lurie Chilren's Hospital Chicago Illinois United States 60611
    6 Riley Hospital for Children Indianapolis Indiana United States 46202
    7 University of Kentucky Lexington Kentucky United States 40536-0293
    8 Boston Children's Hospital Boston Massachusetts United States 02115
    9 Morristown Medical Center Morristown New Jersey United States 07960
    10 Oregon Health & Science University Portland Oregon United States 97239
    11 UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania United States 15224
    12 Clinic for Special Children Strasburg Pennsylvania United States 17579
    13 University of Texas Health Science Center at Houston Houston Texas United States 77030
    14 Medical College of Wisconsin Wauwatosa Wisconsin United States 53226

    Sponsors and Collaborators

    • BioMarin Pharmaceutical

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    BioMarin Pharmaceutical
    ClinicalTrials.gov Identifier:
    NCT04452513
    Other Study ID Numbers:
    • 307-902
    First Posted:
    Jun 30, 2020
    Last Update Posted:
    Jan 31, 2022
    Last Verified:
    Jan 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by BioMarin Pharmaceutical
    PKU
    Phenylketonuria
    PAH Deficiency
    Additional relevant MeSH terms:
    Phenylketonurias

    Study Results

    No Results Posted as of Jan 31, 2022