Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed With Early-Onset Idiopathic Bilateral Cataracts
1 other identifier
observational
442
1 country
44
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2015
Longer than P75 for all trials
44 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2015
CompletedFirst Submitted
Initial submission to the registry
November 23, 2015
CompletedFirst Posted
Study publicly available on registry
December 23, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 19, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
February 19, 2021
CompletedResults Posted
Study results publicly available
September 19, 2024
CompletedSeptember 19, 2024
June 1, 2023
5.5 years
November 23, 2015
June 8, 2023
April 30, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants Who Had Positive Genetic Test Results
genetic test results
At genetic testing
Eligibility Criteria
Early-Onset Idiopathic Bilateral Cataracts
You may qualify if:
- The patient has a diagnosis of idiopathic bilateral cataracts
- Between the ages of 2 to 21 years at the time of diagnosis
You may not qualify if:
- The patient has a diagnosis of cataracts with known etiology other than CTX
- The patient has a diagnosis of CTX
- The patient has cataracts caused by cataractogenic treatments
- The patient has taken or is currently taking cholic acid or chenodeoxycholic acid
- The patient has participated in an interventional clinical trial in the past 30 days
- The patient and/or their parent/legal guardian, in the opinion of the Investigator, is unable to adhere to the requirements of the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (44)
Travere Investigational Site
Birmingham, Alabama, 35294, United States
Travere Investigational Site
La Jolla, California, 92093, United States
Travere Investigational Site
Los Angeles, California, 90027, United States
Travere Investigational Site
Los Angeles, California, 90095, United States
Travere Investigational Site
Palo Alto, California, 94303, United States
Travere Investigational Site
Aurora, Colorado, 80045, United States
Travere Investigational Site
Gainesville, Florida, 32608, United States
Travere Investigational Site
Jacksonville, Florida, 32207, United States
Travere Investigational Site
Atlanta, Georgia, 30322, United States
Travere Investigational Site
Chicago, Illinois, 60611, United States
Travere Investigational Site
Indianapolis, Indiana, 46202, United States
Travere Investigational Site
Iowa City, Iowa, 52242, United States
Travere Investigational Site
Louisville, Kentucky, 40202, United States
Travere Investigational Site
New Orleans, Louisiana, 70121, United States
Travere Investigational Site
Baltimore, Maryland, 21201, United States
Travere Investigational Site
Boston, Massachusetts, 02115, United States
Travere Investigational Site
Ann Arbor, Michigan, 48105, United States
Travere Investigational Site
Detroit, Michigan, 48201, United States
Travere Investigational Site
Kansas City, Missouri, 64108, United States
Travere Investigational Site
St Louis, Missouri, 63103, United States
Travere Investigational Site
The Bronx, New York, 10467, United States
Travere Investigational Site
Chapel Hill, North Carolina, 27517, United States
Travere Investigational Site
Durham, North Carolina, 27708, United States
Travere Investigational Site
Cincinnati, Ohio, 45229, United States
Travere Investigational Site
Cleveland, Ohio, 44106, United States
Travere Investigational Site
Columbus, Ohio, 43205, United States
Travere Investigational Site
Oklahoma City, Oklahoma, 73104, United States
Travere Investigational Site
Portland, Oregon, 97239-3098, United States
Travere Investigational Site
Tigard, Oregon, 97223, United States
Travere Investigational Site
Philadelphia, Pennsylvania, 19104, United States
Travere Investigational Site
Philadelphia, Pennsylvania, 19107, United States
Travere Investigational Site
Charleston, South Carolina, 29425, United States
Travere Investigational Site
Germantown, Tennessee, 38103, United States
Travere Investigational Site
Nashville, Tennessee, 37232, United States
Travere Investigational Site
Houston, Texas, 77025, United States
Travere Investigational Site
Houston, Texas, 77030, United States
Travere Investigational Site
Salt Lake City, Utah, 84113, United States
Travere Investigational Site
Norfolk, Virginia, 23502, United States
Travere Investigational Site
Richmond, Virginia, 23298, United States
Travere Investigational Site
Seattle, Washington, 98105, United States
Travere Investigational Site
Spokane, Washington, 99204, United States
Travere Investigational Site
Tacoma, Washington, 98405, United States
Travere Investigational Site
Morgantown, West Virginia, 26506, United States
Travere Investigational Site
Milwaukee, Wisconsin, 53226, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Limitations and Caveats
This was an observational study; no treatment was administered and therefore no efficacy assessment was conducted. The results presented are used to evaluate the prevalence of CTX in a patient population diagnosed with early-onset idiopathic bilateral cataracts. Only SAEs that were related to a research procedure (eg, blood sample collection) were to be recorded.
Results Point of Contact
- Title
- Cheryl Wong Po Foo/Director, Clinical Development
- Organization
- Travere Therapeutics, Inc.
Study Officials
- STUDY DIRECTOR
Executive Director, Clinical Development Nephrology
Travere Therapeutics, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 23, 2015
First Posted
December 23, 2015
Study Start
September 1, 2015
Primary Completion
February 19, 2021
Study Completion
February 19, 2021
Last Updated
September 19, 2024
Results First Posted
September 19, 2024
Record last verified: 2023-06