Condition (MeSH)
Xanthomatosis, Cerebrotendinous
ClinicalTrials.gov lists 23 trials for Xanthomatosis, Cerebrotendinous or a narrower condition beneath it. TRPHARM has the most among industry and government lead sponsors, with 3.
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Trials per phase
23 of the 23 trials are tagged with Xanthomatosis, Cerebrotendinous itself; the rest are tagged with a narrower condition beneath it.
When did Xanthomatosis, Cerebrotendinous trials start? (15)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 2
- Phase 3
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2024 | 2 |
| 2022 | 1 |
| 2021 | 3 |
| 2020 | 1 |
| 2019 | 2 |
| 2018 | 5 |
| 2017 | 1 |
| 2016 | 1 |
| 2015 | 1 |
| 2014 | 1 |
Showing 10 of 15 earlier years.
No forecast is made for this condition: it averages 0.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 23 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Xanthomatosis, Cerebrotendinous trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 1 |
| Phase 3 | 3 |
| Not applicable | 6 |
| Observational | 11 |
How many Xanthomatosis, Cerebrotendinous trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Completed | 8 |
| Terminated | 2 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Xanthomatosis, Cerebrotendinous trials? (7)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| TRPHARM | 3 |
| Humanis Saglık Anonim Sirketi | 1 |
| Leadiant Biosciences Ltd. | 1 |
| Leadiant Biosciences, Inc. | 1 |
| Mirum Pharmaceuticals, Inc. | 1 |
Showing 5 of 7 sponsors.
Interventions as named in the registry (15)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Chenodeoxycholic Acid | 5 |
| Rescue Medication CDCA 250 mg TID | 1 |
| Blinded CDCA 250 mg TID | 1 |
| Blood tests | 1 |
| acid Zoledronic | 1 |
Showing 5 of 15 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Xanthomatosis, Cerebrotendinous trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Angina pectoris | 2 | 1 |
| Cerebrovascular accident | 2 | 1 |
| Cystocele | 2 | 1 |
| Haematoma | 2 | 1 |
| Hypokalaemia | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Xanthomatosis, Cerebrotendinous? (7)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Epidemiology, diagnosis, and treatment of cerebrotendinous xanthomatosis (CTX). | Journal of inherited metabolic disease | 2017 | Yes |
| A suspicion index for early diagnosis and treatment of cerebrotendinous xanthomatosis. | Journal of inherited metabolic disease | 2014 | Yes |
| Cerebrotendinous xanthomatosis: a comprehensive review of pathogenesis, clinical manifestations, diagnosis, and management. | Orphanet journal of rare diseases | 2014 | Yes |
| Juvenile cataract morphology in 3 siblings not yet diagnosed with cerebrotendinous xanthomatosis. | Ophthalmology | 2013 | Yes |
| Neurological outcome in cerebrotendinous xanthomatosis treated with chenodeoxycholic acid: early versus late diagnosis. | Clinical neuropharmacology | 2013 | Yes |
Showing 5 of 7 publications.
Prevalence (12)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Cerebrotendinous xanthomatosis | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Cerebrotendinous xanthomatosis | Point prevalence | 1-9 / 100 000 | United States | US | Unspecified |
| Cerebrotendinous xanthomatosis | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Cerebrotendinous xanthomatosis | Point prevalence | 1-9 / 100 000 | Specific population | Not stated | Exact |
| Cerebrotendinous xanthomatosis | Point prevalence | <1 / 1 000 000 | Spain | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Cerebrotendinous xanthomatosis | 909 | Exact | Orphadata Science knowledge files, Product 1 |
| Cerebrotendinous xanthomatosis | 909 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.