N/A
N=442
Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Cerebrotendinous Xanthomatosis (CTX)
Bottom Line
View on ClinicalTrials.gov: NCT02638220 ↗Enrolled (actual)
442
Serious AEs
0.0%
Results posted
Sep 2024
Primary outcome: Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- —
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Travere Therapeutics, Inc.
- Primary completion
- Feb 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Had Positive Genetic Test Results |
4 | — |
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.
Eligibility Criteria
Inclusion Criteria
- The patient has a diagnosis of idiopathic bilateral cataracts
- Between the ages of 2 to 21 years at the time of diagnosis
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT02638220). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.