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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
NameTimeMethod
Serum Cholestanol Levels34 years
Secondary Outcome Measures
NameTimeMethod
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