Cerebrotendinous Xanthomatosis (CTX)NCT02638220

Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

An observational study of Cerebrotendinous Xanthomatosis (CTX), run by Travere Therapeutics, Inc. at 44 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 2 to 21 years
Conditions the study is for: Cerebrotendinous Xanthomatosis (CTX)
How many people: 442 participants
Where: 44 sitesUnited States
Study dates: Sep 2015to 19 Feb 2021
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 to 21 years
All sexes
No healthy volunteers
Child, Adult

Study population: Early-Onset Idiopathic Bilateral Cataracts

Who may be able to join

The patient has a diagnosis of idiopathic bilateral cataracts
Between the ages of 2 to 21 years at the time of diagnosis

Who cannot join

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

Trial locations and contacts

44 sites in 1 country.

City, postcode or hospital

44 sites in 1 country

United States44 sites

Travere Investigational Site
Ann Arbor, Michigan, 48105
Travere Investigational Site
Atlanta, Georgia, 30322
Travere Investigational Site
Aurora, Colorado, 80045
Travere Investigational Site
Baltimore, Maryland, 21201
Travere Investigational Site
Birmingham, Alabama, 35294
Travere Investigational Site
Boston, Massachusetts, 02115
Travere Investigational Site
Chapel Hill, North Carolina, 27517
Travere Investigational Site
Charleston, South Carolina, 29425
Travere Investigational Site
Chicago, Illinois, 60611
Travere Investigational Site
Cincinnati, Ohio, 45229
Travere Investigational Site
Cleveland, Ohio, 44106
Travere Investigational Site
Columbus, Ohio, 43205
Travere Investigational Site
Detroit, Michigan, 48201
Travere Investigational Site
Durham, North Carolina, 27708
Travere Investigational Site
Gainesville, Florida, 32608
Travere Investigational Site
Germantown, Tennessee, 38103
Travere Investigational Site
Houston, Texas, 77025
Travere Investigational Site
Houston, Texas, 77030
Travere Investigational Site
Indianapolis, Indiana, 46202
Travere Investigational Site
Iowa City, Iowa, 52242
Travere Investigational Site
Jacksonville, Florida, 32207
Travere Investigational Site
Kansas City, Missouri, 64108
Travere Investigational Site
La Jolla, California, 92093
Travere Investigational Site
Los Angeles, California, 90027
Travere Investigational Site
Los Angeles, California, 90095
Travere Investigational Site
Louisville, Kentucky, 40202
Travere Investigational Site
Milwaukee, Wisconsin, 53226
Travere Investigational Site
Morgantown, West Virginia, 26506
Travere Investigational Site
Nashville, Tennessee, 37232
Travere Investigational Site
New Orleans, Louisiana, 70121
Travere Investigational Site
Norfolk, Virginia, 23502
Travere Investigational Site
Oklahoma City, Oklahoma, 73104
Travere Investigational Site
Palo Alto, California, 94303
Travere Investigational Site
Philadelphia, Pennsylvania, 19104
Travere Investigational Site
Philadelphia, Pennsylvania, 19107
Travere Investigational Site
Portland, Oregon, 97239-3098
Travere Investigational Site
Richmond, Virginia, 23298
Travere Investigational Site
Salt Lake City, Utah, 84113
Travere Investigational Site
Seattle, Washington, 98105
Travere Investigational Site
Spokane, Washington, 99204
Travere Investigational Site
St Louis, Missouri, 63103
Travere Investigational Site
Tacoma, Washington, 98405
Travere Investigational Site
The Bronx, New York, 10467
Travere Investigational Site
Tigard, Oregon, 97223

Other trials in United States

About this study

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

Read the detailed description

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: Cerebrotendinous Xanthomatosis (CTX)
Keywords: Cerebrotendinous Xanthomatosis, CTX Prevalence Study, CTX, Idiopathic Bilateral Cataracts, Cataracts, Bilateral Cataracts, Early-Onset Idiopathic Bilateral Cataracts, Observational Study, Ophthalmology
Medical subject headings: Xanthomatosis, Cerebrotendinous, Cataract

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants Who Had Positive Genetic Test Results
genetic test results
Measured: At genetic testing

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 201519 Feb 2021

First posted23 Dec 2015 (estimated)
Study startSep 2015
Primary completion19 Feb 2021
Study completion19 Feb 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cerebrotendinous Xanthomatosis (CTX)Studies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 442 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02638220
Sponsor's protocol ID: 018CTXX15001
Status last verified: Jun 2023
First submitted: 23 Nov 2015
Last updated: 19 Sep 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cerebrotendinous Xanthomatosis (CTX) trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed With Early-Onset Idiopathic Bilateral Cataracts
ClinicalTrials.gov ID: NCT02638220
Acronym / sponsor ID: 018CTXX15001
Conditions: Cerebrotendinous Xanthomatosis (CTX)
Keywords: Cerebrotendinous Xanthomatosis, CTX Prevalence Study, CTX, Idiopathic Bilateral Cataracts, Cataracts, Bilateral Cataracts, Early-Onset Idiopathic Bilateral Cataracts, Observational Study
Study type: Observational
Recruitment status: Completed
Enrolment: 442 participants (actual)
Lead sponsor: Travere Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: Sep 2015
Primary completion: 19 Feb 2021 (actual)
Study completion: 19 Feb 2021 (actual)
First posted: 23 Dec 2015 (estimated)
Results: Posted 19 Sep 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Observational model: Cohort
Time perspective: Prospective

Who may qualify

Ages: 2 to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult
Study population: Early-Onset Idiopathic Bilateral Cataracts

Key inclusion criteria (2 listed)

The patient has a diagnosis of idiopathic bilateral cataracts
Between the ages of 2 to 21 years at the time of diagnosis

Key exclusion criteria (6 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

Number of Participants Who Had Positive Genetic Test Results
Time frame: At genetic testing
genetic test results

Results and safety

Results posted to the registry on 19 Sep 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Participants With CTX	442	426

Primary outcome: Number of Participants Who Had Positive Genetic Test Results

Time frame: At genetic testing. Number. Unit: Participants

Measure	Participants With CTX n = 442
Value	4

Adverse events

Time frame: 4 hours. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Observational	0 of 442 (0%)	0 of 442 (0%)	0 of 442

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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Enrolling by invitationObservational study
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Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
RecruitingObservational study
The Myelin Disorders Biorepository Project
Leukodystrophy, White Matter Disease
Recruiting
''Comparative Evaluation of Change in Bite Force and Levels of Bone Turnover Markers CTX and BALP in Hyperdivergent and Hypodivergent Cases During Retention Phases-A Prospective Clinical Trial ''
Relapse, Bite Force
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Executive Director, Clinical Development Nephrology study director

Travere Therapeutics, Inc.

44 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Cerebrotendinous Xanthomatosis (CTX), run by Travere Therapeutics, Inc. at 44 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 44 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Sep 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants Who Had Positive Genetic Test Results (time frame: At genetic testing). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02638220.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.