Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

November 7, 2018 updated by: Travere Therapeutics, Inc.

An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed With Early-Onset Idiopathic Bilateral Cataracts

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.

Study Overview

Status

Unknown

Conditions

Study Type

Observational

Enrollment (Anticipated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
    • California
      • La Jolla, California, United States, 92093
        • Recruiting
        • University of California, San Diego
        • Contact:
        • Contact:
      • Los Angeles, California, United States, 90027
      • Los Angeles, California, United States, 90095
      • Palo Alto, California, United States, 94303
    • Colorado
    • Florida
      • Gainesville, Florida, United States, 32608
        • Recruiting
        • University of Florida - Shands Hosptial
        • Contact:
        • Contact:
      • Jacksonville, Florida, United States, 32207
    • Georgia
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Hospital for Children
        • Contact:
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Kentucky
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
    • Maryland
    • Massachusetts
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • Recruiting
        • University of Michigan Kellogg Eye Center
        • Contact:
        • Contact:
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Children's Hospital of Michigan
        • Contact:
        • Contact:
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Hospitals and Clinics
        • Contact:
        • Contact:
      • Saint Louis, Missouri, United States, 63103
    • New York
      • Bronx, New York, United States, 10467
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
      • Durham, North Carolina, United States, 27708
    • Ohio
      • Cincinnati, Ohio, United States, 45229
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Cole Eye Institute, Cleveland Clinic Foundation
        • Contact:
        • Contact:
      • Cleveland, Ohio, United States, 44106
      • Columbus, Ohio, United States, 43205
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
    • Oregon
      • Portland, Oregon, United States, 97239-3098
        • Recruiting
        • Orgegon Health & Science University
        • Contact:
        • Contact:
      • Tigard, Oregon, United States, 97223
        • Completed
        • Child Eye Care Associates
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
        • Contact:
      • Philadelphia, Pennsylvania, United States, 19107
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Storm Eye Institute
        • Contact:
        • Contact:
    • Tennessee
      • Germantown, Tennessee, United States, 38103
        • Recruiting
        • University of Tennessee Health Science Center, Hamilton Eye Institute
        • Contact:
        • Contact:
      • Nashville, Tennessee, United States, 37232
    • Texas
    • Utah
    • Virginia
      • Norfolk, Virginia, United States, 23502
        • Recruiting
        • Children's Hospital of The King's Daughter
        • Contact:
        • Contact:
      • Richmond, Virginia, United States, 23298
    • Washington
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Children's Hospital of Wisocnsin
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Early-Onset Idiopathic Bilateral Cataracts

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To calculate the prevalence of Cerebrotendinous Xanthomatosis (CTX) in a patient population diagnosed up to age 21 with early-onset idiopathic bilateral cataracts
Time Frame: 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)
8 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To assess other manifestations of CTX within patients presenting with idiopathic bilateral cataracts.
Time Frame: 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.
8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Travere Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2015

Primary Completion (Anticipated)

December 1, 2018

Study Completion (Anticipated)

December 1, 2018

Study Registration Dates

First Submitted

November 23, 2015

First Submitted That Met QC Criteria

December 18, 2015

First Posted (Estimate)

December 23, 2015

Study Record Updates

Last Update Posted (Actual)

November 9, 2018

Last Update Submitted That Met QC Criteria

November 7, 2018

Last Verified

November 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 018CTXX15001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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