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)
1 other identifier
observational
28
0 countries
N/A
Brief Summary
Retrospective study of CTX patients to investigate the safety and clinical efficacy of Chenodeoxycholic Acid
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Dec 2014
Shorter than P25 for all trials
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 9, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 3, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
June 3, 2015
CompletedFirst Submitted
Initial submission to the registry
August 10, 2022
CompletedFirst Posted
Study publicly available on registry
August 12, 2022
CompletedAugust 12, 2022
August 1, 2022
6 months
August 10, 2022
August 10, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Serum Cholestanol Levels
34 years
Interventions
Bile acid
Eligibility Criteria
CTX patients
You may qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 10, 2022
First Posted
August 12, 2022
Study Start
December 9, 2014
Primary Completion
June 3, 2015
Study Completion
June 3, 2015
Last Updated
August 12, 2022
Record last verified: 2022-08
Data Sharing
- IPD Sharing
- Will not share