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Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02638220
Recruitment Status : Recruiting
First Posted : December 23, 2015
Last Update Posted : November 9, 2018
Sponsor:
Information provided by (Responsible Party):
Retrophin, Inc.

Tracking Information
First Submitted Date November 23, 2015
First Posted Date December 23, 2015
Last Update Posted Date November 9, 2018
Study Start Date September 2015
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 18, 2015)
To calculate the prevalence of Cerebrotendinous Xanthomatosis (CTX) in a patient population diagnosed up to age 21 with early-onset idiopathic bilateral cataracts [ Time Frame: 8 weeks ]
(by blood draw where plasma cholenstanol levels and/or urine bile alcohol results will be used to determine whether genetic testing is indicated. Genetic Testing, if indicated, will be used to determine the prevalence of CTX)
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: December 18, 2015)
To assess other manifestations of CTX within patients presenting with idiopathic bilateral cataracts. [ Time Frame: 8 weeks ]
Based on a patient reported, CTX disease-specific medical history for the patients diagnosed with CTX, the manifestations and symptoms related to the disease will be presented by body system.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Official Title An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed With Early-Onset Idiopathic Bilateral Cataracts
Brief Summary 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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population Early-Onset Idiopathic Bilateral Cataracts
Condition Cerebrotendinous Xanthomatosis (CTX)
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: December 18, 2015)
500
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2018
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages 2 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Retrophin Medical Information 1-877-659-5518 medinfo@retrophin.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02638220
Other Study ID Numbers 018CTXX15001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Retrophin, Inc.
Study Sponsor Retrophin, Inc.
Collaborators Not Provided
Investigators Not Provided
PRS Account Retrophin, Inc.
Verification Date November 2018