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Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

Sponsor
Travere Therapeutics, Inc. (Industry)
Overall Status
Unknown status
CT.gov ID
NCT02638220
Collaborator
(none)
500
Enrollment
45
Locations
39
Duration (Months)
11.1
Patients Per Site
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is an observational, multicenter study to determine the prevalence of Cerebrotendinous Xanthomatosis (CTX) in patient populations diagnosed with early-onset idiopathic bilateral cataracts. Patients who are potentially eligible for study participation will be identified through a chart review of patients who were seen at each study site prior to that site's initiation, or by entering care at the site while the site is participating in the trial.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    500 participants
    Observational Model:
    Cohort
    Time Perspective:
    Other
    Official Title:
    An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed With Early-Onset Idiopathic Bilateral Cataracts
    Study Start Date :
    Sep 1, 2015
    Anticipated Primary Completion Date :
    Dec 1, 2018
    Anticipated Study Completion Date :
    Dec 1, 2018

    Outcome Measures

    Primary Outcome Measures

    1. To calculate the prevalence of Cerebrotendinous Xanthomatosis (CTX) in a patient population diagnosed up to age 21 with early-onset idiopathic bilateral cataracts [8 weeks]

      (by blood draw where plasma cholenstanol levels and/or urine bile alcohol results will be used to determine whether genetic testing is indicated. Genetic Testing, if indicated, will be used to determine the prevalence of CTX)

    Secondary Outcome Measures

    1. To assess other manifestations of CTX within patients presenting with idiopathic bilateral cataracts. [8 weeks]

      Based on a patient reported, CTX disease-specific medical history for the patients diagnosed with CTX, the manifestations and symptoms related to the disease will be presented by body system.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 21 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. The patient has a diagnosis of idiopathic bilateral cataracts.

    2. Between the ages of 2 to 21 years at the time of diagnosis

    Exclusion Criteria:

    1. The patient has a diagnosis of cataracts with known etiology other than CTX.

    2. The patient has a diagnosis of CTX.

    3. The patient has cataracts caused by cataractogenic treatments.

    4. The patient has taken or is currently taking cholic acid or chenodeoxycholic acid.

    5. The patient has participated in an interventional clinical trial in the past 30 days.

    6. The patient and/or their parent/legal guardian, in the opinion of the Investigator, is unable to adhere to the requirements of the study.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama at Birmingham Birmingham Alabama United States 35294
    2 University of California, San Diego La Jolla California United States 92093
    3 Children's Hospital of Los Angeles Los Angeles California United States 90027
    4 Stein Eye Institute (UCLA) Los Angeles California United States 90095
    5 Stanford University Palo Alto California United States 94303
    6 Children's Hospital Colorado Aurora Colorado United States 80045
    7 University of Florida - Shands Hosptial Gainesville Florida United States 32608
    8 Nemours Children's Specialty Care Jacksonville Florida United States 32207
    9 Emory Univerisity Atlanta Georgia United States 30322
    10 Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    11 Riley Hospital for Children Indianapolis Indiana United States 46202
    12 University of Iowa Iowa City Iowa United States 52242
    13 KY Lions Eye Center Louisville Kentucky United States 40202
    14 Ochsner Clinic Foundation New Orleans Louisiana United States 70121
    15 University of Maryland Baltimore Baltimore Maryland United States 21201
    16 Boston Children's Hospital Boston Massachusetts United States 02115
    17 University of Michigan Kellogg Eye Center Ann Arbor Michigan United States 48105
    18 Children's Hospital of Michigan Detroit Michigan United States 48201
    19 Children's Mercy Hospitals and Clinics Kansas City Missouri United States 64108
    20 Saint Louis University Eye Institute Saint Louis Missouri United States 63103
    21 Montefiore Bronx New York United States 10467
    22 University of North Carolina Kittner Eye Center Chapel Hill North Carolina United States 27517
    23 Duke University Durham North Carolina United States 27708
    24 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    25 Cole Eye Institute, Cleveland Clinic Foundation Cleveland Ohio United States 44106
    26 Rainbow Babies and Children's Hospital Cleveland Ohio United States 44106
    27 Nationwide Children's Hospital Columbus Ohio United States 43205
    28 Dean McGee Eye Institute-Oklahoma Health Center Oklahoma City Oklahoma United States 73104
    29 Orgegon Health & Science University Portland Oregon United States 97239-3098
    30 Child Eye Care Associates Tigard Oregon United States 97223
    31 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    32 Wills Eye Hospital Philadelphia Pennsylvania United States 19107
    33 Storm Eye Institute Charleston South Carolina United States 29425
    34 University of Tennessee Health Science Center, Hamilton Eye Institute Germantown Tennessee United States 38103
    35 Vanderbilt Medical Medical Center Nashville Tennessee United States 37232
    36 Houston Eye Associates Houston Texas United States 77025
    37 Baylor College of Medicine Houston Texas United States 77030
    38 University of Utah Salt Lake City Utah United States 84113
    39 Children's Hospital of the King's Daughter Norfolk Virginia United States 23502
    40 Virginia Commonwealth University Richmond Virginia United States 23298
    41 Seattle Children's Hospital Seattle Washington United States 98105
    42 Spokane Eye Associates Spokane Washington United States 99204
    43 Multicare Institute for Research and Innovation Tacoma Washington United States 98405
    44 West Virginia University Morgantown West Virginia United States 26506
    45 Children's Hospital of Wisocnsin Milwaukee Wisconsin United States 53226

    Sponsors and Collaborators

    • Travere Therapeutics, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Travere Therapeutics, Inc.
    ClinicalTrials.gov Identifier:
    NCT02638220
    Other Study ID Numbers:
    • 018CTXX15001
    First Posted:
    Dec 23, 2015
    Last Update Posted:
    Nov 9, 2018
    Last Verified:
    Nov 1, 2018
    Keywords provided by Travere Therapeutics, Inc.
    Cerebrotendinous Xanthomatosis
    CTX Prevalence Study
    CTX
    Idiopathic Bilateral Cataracts
    Cataracts
    Bilateral Cataracts
    Early-Onset Idiopathic Bilateral Cataracts
    Observational Study
    Ophthalmology
    Additional relevant MeSH terms:
    Xanthomatosis, Cerebrotendinous
    Xanthomatosis

    Study Results

    No Results Posted as of Nov 9, 2018