Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
Completed
- Conditions
- Cerebrotendinous Xanthomatoses
- Registration Number
- NCT05499026
- Lead Sponsor
- Leadiant Biosciences Ltd.
- Brief Summary
Retrospective study of CTX patients to investigate the safety and clinical efficacy of Chenodeoxycholic Acid
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 28
Inclusion Criteria
Diagnosis of CTX Received treatment with CDCA Age between 2 and 75 years Having at least one cholestanol level and/or urinary bile alcohol level no more than 3 months prior to treatment with CDCA and one cholestanol level and/or urinary bile alcohol level post-treatment within 2 years from the beginning of therapy with CDCA
Exclusion Criteria
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Serum Cholestanol Levels 34 years
- Secondary Outcome Measures
Name Time Method