Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)

Retrospective Cohort Study to Investigate the Safety and Efficacy of Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05499026

Enrollment

Registered

2022-08-12

Start date

2014-12-09

Completion date

2015-06-03

Last updated

2022-08-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cerebrotendinous Xanthomatoses

Brief summary

Retrospective study of CTX patients to investigate the safety and clinical efficacy of Chenodeoxycholic Acid

Interventions

DRUGChenodeoxycholic Acid

Bile acid

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

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

Design outcomes

Primary

Measure	Time frame
Serum Cholestanol Levels	34 years

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026