Condition (MeSH)
Gangliosidoses
ClinicalTrials.gov lists 49 trials for Gangliosidoses or a narrower condition beneath it. Azafaros B.V. has the most among industry and government lead sponsors, with 5.
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Trials per phase (log scale)
11 of the 49 trials are tagged with Gangliosidoses itself; the rest are tagged with a narrower condition beneath it.
When did Gangliosidoses trials start? (23)
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. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 6
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) | 3 |
| 2025 | 2 |
| 2024 | 2 |
| 2023 | 1 |
| 2022 | 1 |
| 2021 | 5 |
| 2020 | 6 |
| 2019 | 5 |
| 2018 | 1 |
| 2017 | 1 |
Showing 10 of 23 earlier years.
No forecast is made for this condition: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 6 trials still running at the 2026-10-09 snapshot; registered median of 43 months across 29 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Gangliosidoses trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 5 |
| Phase 1/2 | 6 |
| Phase 2 | 6 |
| Phase 2/3 | 1 |
| Phase 3 | 7 |
| Phase 4 | 1 |
| Not applicable | 3 |
| Observational | 20 |
How many Gangliosidoses trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 3 |
| Recruiting | 9 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 4 |
| Completed | 18 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Gangliosidoses trials? (5)
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 |
|---|---|
| Azafaros B.V. | 5 |
| LYSOGENE | 2 |
| National Human Genome Research Institute (NHGRI) | 2 |
| Aldagen | 1 |
| CENTOGENE GmbH Rostock | 1 |
Which drugs are being tested for Gangliosidoses? (5)
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 |
|---|---|
| AZ-3102 | 4 |
| Miglustat | 4 |
| CLOFARABINE | 2 |
| Melphalan | 2 |
| Pyrimethamine | 2 |
Interventions as named in the registry (10)
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 |
| Lumbar puncture | 1 |
Showing 5 of 10 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 Gangliosidoses 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 | 54 | 1 |
| Auto recovery | 9 | 1 |
| Disease Progression | 4 | 1 |
| Graft Failure | 4 | 1 |
| Pneumonia | 3 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Gangliosidoses? (5)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Quantitative longitudinal natural history of 8 gangliosidoses-conceptual framework and baseline data of the German 8-in-1 disease registry. A cross-sectional analysis. | Genetics in medicine : official journal of the American College of Medical Genetics | 2022 | Yes |
| Infantile gangliosidoses: Mapping a timeline of clinical changes. | Molecular genetics and metabolism | 2017 | Yes |
| Biomarkers of central nervous system inflammation in infantile and juvenile gangliosidoses. | Molecular genetics and metabolism | 2015 | Yes |
| Generalized gangliosidosis type II (juvenile GM1 gangliosidosis). A pathological, histochemical and ultrastructural study. | Zeitschrift fur Kinderheilkunde | 1975 | Yes |
| Enzymatic abnormalities in diseases of sphingolipid metabolism. | Clinical chemistry | 1967 | No |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Gangliosidosis | 309144 | Exact | Orphadata Science knowledge files, Product 1 |
| Gangliosidosis | 309144 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Sub-conditions in MeSH (2)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence.