Condition (MeSH)
Cholesterol Ester Storage Disease
ClinicalTrials.gov lists 37 trials for Cholesterol Ester Storage Disease or a narrower condition beneath it. Alexion Pharmaceuticals, Inc. has the most among industry and government lead sponsors, with 12.
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Trials per phase (log scale)
16 of the 37 trials are tagged with Cholesterol Ester Storage Disease itself; the rest are tagged with a narrower condition beneath it.
When did Cholesterol Ester Storage Disease trials start? (18)
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 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 4
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 |
|---|---|
| 2026 (partial year) | 1 |
| 2024 | 1 |
| 2022 | 2 |
| 2021 | 3 |
| 2020 | 2 |
| 2018 | 2 |
| 2017 | 1 |
| 2016 | 2 |
| 2015 | 3 |
| 2014 | 3 |
Showing 10 of 18 earlier years.
No forecast is made for this condition: it averages 0.8 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Cholesterol Ester Storage Disease trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 1 |
| Phase 2 | 5 |
| Phase 2/3 | 2 |
| Phase 3 | 1 |
| Not applicable | 5 |
| Observational | 20 |
| Expanded access | 1 |
How many Cholesterol Ester Storage Disease trials are recruiting? (8)
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 | 16 |
| Terminated | 5 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Cholesterol Ester Storage Disease trials? (4)
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 |
|---|---|
| Alexion Pharmaceuticals, Inc. | 12 |
| Assy Nimer | 1 |
| AstraZeneca | 1 |
| CENTOGENE GmbH Rostock | 1 |
Which drugs are being tested for Cholesterol Ester Storage Disease? (3)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Sebelipase Alfa | 7 |
| CLOFARABINE | 2 |
| Melphalan | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 3 |
| Hematopoietic Stem Cell Transplantation | 2 |
| Cyclosporine 0.05% | 2 |
| Mofetil Mycophenolate | 2 |
| Human Placental Derived Stem Cell | 1 |
Showing 5 of 12 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 Cholesterol Ester Storage Disease 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 |
|---|---|---|
| Death | 60 | 2 |
| Pyrexia | 14 | 2 |
| Gastroenteritis | 13 | 3 |
| Auto recovery | 11 | 2 |
| Vomiting | 11 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Cholesterol Ester Storage Disease? (12)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Clinical Features of Lysosomal Acid Lipase Deficiency. | Journal of pediatric gastroenterology and nutrition | 2015 | Yes |
| A practical fluorometric assay method to measure lysosomal acid lipase activity in dried blood spots for the screening of cholesteryl ester storage disease and Wolman disease. | Molecular genetics and metabolism | 2014 | Yes |
| Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease. | Hepatology (Baltimore, Md.) | 2013 | Yes |
| Cholesteryl ester storage disease: review of the findings in 135 reported patients with an underdiagnosed disease. | Journal of hepatology | 2013 | Yes |
| Cholesteryl ester storage disease: protean presentations of lysosomal acid lipase deficiency. | Journal of pediatric gastroenterology and nutrition | 2013 | Yes |
Showing 5 of 12 publications.
Prevalence (6)
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 |
|---|---|---|---|---|---|
| Cholesteryl ester storage disease | Prevalence at birth | 1-9 / 100 000 | Germany | Not stated | Exact |
| Cholesteryl ester storage disease | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Cholesteryl ester storage disease | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Cholesteryl ester storage disease | Point prevalence | 1-9 / 1 000 000 | Specific population | Not stated | Exact |
| Cholesteryl ester storage disease | Point prevalence | 1-9 / 1 000 000 | Specific population | Not stated | Exact |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Cholesteryl ester storage disease | 75234 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Cholesteryl ester storage disease | 75234 | Exact | Orphadata Science knowledge files, Product 1 |
Sub-conditions in MeSH (1)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.