Condition (MeSH)
Mucolipidoses
ClinicalTrials.gov lists 15 trials for Mucolipidoses or a narrower condition beneath it.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
15 of the 15 trials are tagged with Mucolipidoses itself; the rest are tagged with a narrower condition beneath it.
When did Mucolipidoses trials start? (13)
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 2
- Phase 3
- Not applicable
- Observational
Top of scale: 2
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 |
| 2023 | 1 |
| 2022 | 1 |
| 2020 | 1 |
| 2018 | 1 |
| 2012 | 1 |
| 2010 | 1 |
| 2009 | 2 |
| 2008 | 1 |
| 2006 | 1 |
Showing 10 of 13 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: 1 trial 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 Mucolipidoses trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 2 |
| Phase 3 | 1 |
| Not applicable | 1 |
| Observational | 10 |
How many Mucolipidoses 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 | 5 |
| Terminated | 2 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucolipidoses 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 |
|---|---|
| Aldagen | 1 |
| CENTOGENE GmbH Rostock | 1 |
| National Human Genome Research Institute (NHGRI) | 1 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 1 |
Which drugs are being tested for Mucolipidoses? (2)
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 |
|---|---|
| CLOFARABINE | 2 |
| Melphalan | 2 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Hematopoietic Stem Cell Transplantation | 2 |
| Alemtuzumab s.c. | 2 |
| Cyclosporine 0.05% | 2 |
| Mofetil Mycophenolate | 2 |
| Busulfan, Cyclophosphamide, ATG | 1 |
Showing 5 of 11 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 Mucolipidoses trials report? (4)
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 |
|---|---|---|
| Disease Progression | 4 | 1 |
| Graft Failure | 4 | 1 |
| Death Due to Disease Progression | 2 | 1 |
| Infection with Grade 3 or 4 Neutrophils | 1 | 1 |
Which publications cover Mucolipidoses? (15)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| A sialidosis type I cohort and a quantitative approach to multimodal ophthalmic imaging of the macular cherry-red spot. | The British journal of ophthalmology | 2021 | Yes |
| Unique molecular signature in mucolipidosis type IV microglia. | Journal of neuroinflammation | 2019 | Yes |
| Loss of syntaxin 3 causes variant microvillus inclusion disease. | Gastroenterology | 2014 | Yes |
| Myosin Vb uncoupling from RAB8A and RAB11A elicits microvillus inclusion disease. | The Journal of clinical investigation | 2014 | Yes |
| An overview and online registry of microvillus inclusion disease patients and their MYO5B mutations. | Human mutation | 2013 | Yes |
Showing 5 of 15 publications.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mucolipidosis | 79212 | Exact | Orphadata Science knowledge files, Product 1 |
| Mucolipidosis | 79212 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and sub-conditions in mesh.