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Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

NCT ID: NCT02638220

Last Updated: 2024-09-19

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

442 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-09-30

Study Completion Date

2021-02-19

Brief Summary

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Iiopathic bilateral cataracts occur in many children with Cerebrotendinous Xanthomatosis (CTX) so the opportunity to use contact with health care providers in connection with pediatric cataract evaluation and treatment is a promising avenue for disease detection and prevention.

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Detailed Description

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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.

Conditions

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Cerebrotendinous Xanthomatosis (CTX)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Travere Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Executive Director, Clinical Development Nephrology

Role: STUDY_DIRECTOR

Travere Therapeutics, Inc.

Locations

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Travere Investigational Site

Birmingham, Alabama, United States

Site Status

Travere Investigational Site

La Jolla, California, United States

Site Status

Travere Investigational Site

Los Angeles, California, United States

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Travere Investigational Site

Los Angeles, California, United States

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Travere Investigational Site

Palo Alto, California, United States

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Travere Investigational Site

Aurora, Colorado, United States

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Travere Investigational Site

Gainesville, Florida, United States

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Travere Investigational Site

Jacksonville, Florida, United States

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Travere Investigational Site

Atlanta, Georgia, United States

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Travere Investigational Site

Chicago, Illinois, United States

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Travere Investigational Site

Indianapolis, Indiana, United States

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Iowa City, Iowa, United States

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Travere Investigational Site

Louisville, Kentucky, United States

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Travere Investigational Site

New Orleans, Louisiana, United States

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Baltimore, Maryland, United States

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Travere Investigational Site

Boston, Massachusetts, United States

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Travere Investigational Site

Ann Arbor, Michigan, United States

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Detroit, Michigan, United States

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Kansas City, Missouri, United States

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St Louis, Missouri, United States

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The Bronx, New York, United States

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Travere Investigational Site

Chapel Hill, North Carolina, United States

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Travere Investigational Site

Durham, North Carolina, United States

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Travere Investigational Site

Cincinnati, Ohio, United States

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Cleveland, Ohio, United States

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Columbus, Ohio, United States

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Oklahoma City, Oklahoma, United States

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Travere Investigational Site

Portland, Oregon, United States

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Travere Investigational Site

Tigard, Oregon, United States

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Travere Investigational Site

Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Travere Investigational Site

Charleston, South Carolina, United States

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Travere Investigational Site

Germantown, Tennessee, United States

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Travere Investigational Site

Nashville, Tennessee, United States

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Travere Investigational Site

Houston, Texas, United States

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Travere Investigational Site

Houston, Texas, United States

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Travere Investigational Site

Salt Lake City, Utah, United States

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Travere Investigational Site

Norfolk, Virginia, United States

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Travere Investigational Site

Richmond, Virginia, United States

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Travere Investigational Site

Seattle, Washington, United States

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Travere Investigational Site

Spokane, Washington, United States

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Travere Investigational Site

Tacoma, Washington, United States

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Travere Investigational Site

Morgantown, West Virginia, United States

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Travere Investigational Site

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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018CTXX15001

Identifier Type: -

Identifier Source: org_study_id

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