Condition (MeSH)
Gangliosidoses, GM2
ClinicalTrials.gov lists 36 trials for Gangliosidoses, GM2 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
25 of the 36 trials are tagged with Gangliosidoses, GM2 itself; the rest are tagged with a narrower condition beneath it.
When did Gangliosidoses, GM2 trials start? (22)
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
- Phase 4
- 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) | 2 |
| 2025 | 2 |
| 2024 | 1 |
| 2023 | 1 |
| 2022 | 1 |
| 2021 | 2 |
| 2020 | 2 |
| 2019 | 4 |
| 2017 | 1 |
| 2016 | 2 |
Showing 10 of 22 earlier years.
No forecast is made for this condition: it averages 1.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 4 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 Gangliosidoses, GM2 trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 4 |
| Phase 1/2 | 3 |
| Phase 2 | 6 |
| Phase 2/3 | 1 |
| Phase 3 | 6 |
| Phase 4 | 1 |
| Not applicable | 3 |
| Observational | 12 |
How many Gangliosidoses, GM2 trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 7 |
| Active, not recruiting | 3 |
| Completed | 16 |
| Terminated | 6 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Gangliosidoses, GM2 trials? (6)
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 |
| Aldagen | 1 |
| Exsar Corporation | 1 |
| Genzyme, a Sanofi Company | 1 |
| Idorsia Pharmaceuticals Ltd. | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Gangliosidoses, GM2? (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 |
| Hematopoetic Stem Cell Transplantation | 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, GM2 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, GM2? (19)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial. | Nature medicine | 2025 | Yes |
| Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses. | Neurology | 2023 | Yes |
| Gene expression changes in Tay-Sachs disease begin early in fetal brain development. | Journal of inherited metabolic disease | 2023 | Yes |
| 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 |
| A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia. | Trials | 2021 | Yes |
Showing 5 of 19 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 |
|---|---|---|---|---|---|
| GM2 gangliosidosis | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Exact |
| GM2 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Exact |
| GM2 gangliosidosis | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Unspecified |
| GM2 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Unspecified |
| GM2 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Sweden | 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 |
|---|---|---|---|
| GM2 gangliosidosis | 309152 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| GM2 gangliosidosis | 309152 | Exact | Orphadata Science knowledge files, Product 1 |
Sub-conditions in MeSH (3)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.