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

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

Completed

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Key information

Conditions

Cerebrotendinous Xanthomatoses Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Xanthomatosis Xanthomatosis, Cerebrotendinous

Sex eligibility

All sexes

Study type

Observational

Who can participate

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Chenodeoxycholic Acid

Drug

Bile acid

Primary outcomes

Serum Cholestanol Levels

Time frame: 34 years

Sponsors and collaborators

Lead sponsor

Leadiant Biosciences Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start: 2014
Primary completion: 2015
Study completion: 2015
First posted: Aug 12, 2022
Registry last updated: Aug 12, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed