Cerebrotendinous XanthomatosesNCT05499026
Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
An observational study of Cerebrotendinous Xanthomatoses, run by Leadiant Biosciences Ltd..
Trial summary
Retrospective study of CTX patients to investigate the safety and clinical efficacy of Chenodeoxycholic Acid Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cerebrotendinous Xanthomatoses
- What is being tested?
- Chenodeoxycholic Acid
- How many people
- 28 participants
- Where
- No sites listed yet
- Study dates
- 9 Dec 2014to 3 Jun 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Study population: CTX patients
Who may be able to join
- 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
Who cannot join
- -
About this study
Official title: Retrospective Cohort Study to Investigate the Safety and Efficacy of 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
- Conditions
- Cerebrotendinous Xanthomatoses
- Being tested
- Chenodeoxycholic Acid
- Medical subject headings
- Xanthomatosis, Cerebrotendinous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Chenodeoxycholic AcidDrug
Bile acid
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum Cholestanol Levels
Measured: 34 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2022
- Study start9 Dec 2014
- Primary completion3 Jun 2015
- Study completion3 Jun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebrotendinous XanthomatosesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Leadiant Biosciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05499026
- Sponsor's protocol ID
- CDCA-STUK-15-001
- Status last verified
- Aug 2022
- First submitted
- 10 Aug 2022
- Last updated
- 12 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Cohort Study to Investigate the Safety and Efficacy of Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
- ClinicalTrials.gov ID
- NCT05499026
- Acronym / sponsor ID
- CDCA-STUK-15-001
- Conditions
- Cerebrotendinous Xanthomatoses
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Leadiant Biosciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 9 Dec 2014 (actual)
- Primary completion
- 3 Jun 2015 (actual)
- Study completion
- 3 Jun 2015 (actual)
- First posted
- 12 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Retrospective study of CTX patients to investigate the safety and clinical efficacy of Chenodeoxycholic Acid
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- CTX patients
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Chenodeoxycholic AcidBile acid | Drug | – |
Primary outcome
Serum Cholestanol Levels
Time frame: 34 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebrotendinous Xanthomatoses, run by Leadiant Biosciences Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serum Cholestanol Levels (time frame: 34 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2022.