Condition (MeSH)
Gangliosidosis, GM1
ClinicalTrials.gov lists 21 trials for Gangliosidosis, GM1 or a narrower condition beneath it. Azafaros B.V. has the most among industry and government lead sponsors, with 3.
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Trials per phase
21 of the 21 trials are tagged with Gangliosidosis, GM1 itself; the rest are tagged with a narrower condition beneath it.
When did Gangliosidosis, GM1 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. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- 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 |
|---|---|
| 2025 | 2 |
| 2024 | 1 |
| 2022 | 1 |
| 2021 | 3 |
| 2020 | 5 |
| 2019 | 1 |
| 2015 | 1 |
| 2012 | 1 |
| 2010 | 1 |
| 2008 | 1 |
Showing 10 of 13 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: 3 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 Gangliosidosis, GM1 trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 2 |
| Phase 2 | 1 |
| Phase 2/3 | 1 |
| Phase 3 | 4 |
| Phase 4 | 1 |
| Not applicable | 1 |
| Observational | 10 |
How many Gangliosidosis, GM1 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 | 4 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 2 |
| Completed | 7 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Gangliosidosis, GM1 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 |
|---|---|
| Azafaros B.V. | 3 |
| LYSOGENE | 2 |
| Aldagen | 1 |
| Gemma Biotherapeutics | 1 |
| Idorsia Pharmaceuticals Ltd. | 1 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Gangliosidosis, GM1? (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 |
|---|---|
| AZ-3102 | 2 |
| CLOFARABINE | 2 |
| Melphalan | 2 |
Interventions as named in the registry (12)
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 |
| Stem Cell Transplant | 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 Gangliosidosis, GM1 trials report? (7)
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 |
| Death Due to Disease Progression | 2 | 1 |
Showing 5 of 7 adverse events.
Which publications cover Gangliosidosis, GM1? (14)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| GM1 gangliosidosis type II: Results of a 10-year prospective study. | Genetics in medicine : official journal of the American College of Medical Genetics | 2024 | Yes |
| Anesthesia outcomes in lysosomal disorders: CLN3 and GM1 gangliosidosis. | American journal of medical genetics. Part A | 2023 | Yes |
| AAV9-coGLB1 Improves Lysosomal Storage and Rescues Central Nervous System Inflammation in a Mutant Mouse Model of GM1 Gangliosidosis. | Current gene therapy | 2022 | Yes |
| Distinct progression patterns of brain disease in infantile and juvenile gangliosidoses: Volumetric quantitative MRI study. | Molecular genetics and metabolism | 2018 | Yes |
| Infantile gangliosidoses: Mapping a timeline of clinical changes. | Molecular genetics and metabolism | 2017 | Yes |
Showing 5 of 14 publications.
Prevalence (15)
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 |
|---|---|---|---|---|---|
| GM1 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Unspecified |
| GM1 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| GM1 gangliosidosis | Prevalence at birth | 1-9 / 100 000 | Brazil | Not stated | Exact |
| GM1 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Exact |
| GM1 gangliosidosis | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
Showing 5 of 15 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| GM1 gangliosidosis | 354 | Exact | Orphadata Science knowledge files, Product 1 |
| GM1 gangliosidosis | 354 | 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.