for people ages 2-21 (full criteria)
at La Jolla, California and other locations
study started
estimated completion



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.

Official Title

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


Cerebrotendinous Xanthomatosis (CTX) Cerebrotendinous Xanthomatosis CTX Prevalence Study CTX Idiopathic Bilateral Cataracts Cataracts Bilateral Cataracts Early-Onset Idiopathic Bilateral Cataracts Observational Study Ophthalmology Xanthomatosis, Cerebrotendinous Xanthomatosis


You can join if…

Open to people ages 2-21

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

You CAN'T join if...

  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.


  • University of California, San Diego accepting new patients
    La Jolla California 92093 United States
  • Stein Eye Institute (UCLA) accepting new patients
    Los Angeles California 90095 United States


accepting new patients
Start Date
Completion Date
Retrophin, Inc.
Study Type
Last Updated