Condition (MeSH)
Tay-Sachs Disease
ClinicalTrials.gov lists 24 trials for Tay-Sachs Disease 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
24 of the 24 trials are tagged with Tay-Sachs Disease itself; the rest are tagged with a narrower condition beneath it.
When did Tay-Sachs Disease trials start? (16)
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: 3
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 |
|---|---|
| 2024 | 1 |
| 2022 | 1 |
| 2021 | 2 |
| 2020 | 2 |
| 2019 | 3 |
| 2015 | 1 |
| 2014 | 2 |
| 2012 | 2 |
| 2011 | 1 |
| 2010 | 2 |
Showing 10 of 16 earlier years.
No forecast is made for this condition: it averages 0.4 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 Tay-Sachs Disease trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 3 |
| Phase 2 | 3 |
| Phase 2/3 | 1 |
| Phase 3 | 3 |
| Phase 4 | 1 |
| Not applicable | 2 |
| Observational | 8 |
How many Tay-Sachs Disease trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Active, not recruiting | 2 |
| Completed | 11 |
| Terminated | 6 |
| Withdrawn | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Tay-Sachs Disease 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 |
|---|---|
| Aldagen | 1 |
| Azafaros B.V. | 1 |
| Exsar Corporation | 1 |
| Genzyme, a Sanofi Company | 1 |
| Idorsia Pharmaceuticals Ltd. | 1 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Tay-Sachs Disease? (4)
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 |
|---|---|
| Miglustat | 3 |
| CLOFARABINE | 2 |
| Melphalan | 2 |
| Pyrimethamine | 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 |
| Hematopoetic Stem Cell Transplantation | 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 Tay-Sachs 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 | 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 Tay-Sachs Disease? (18)
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 |
| 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 |
| Genetics and Therapies for GM2 Gangliosidosis. | Current gene therapy | 2018 | Yes |
| Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment. | Genetics in medicine : official journal of the American College of Medical Genetics | 2009 | Yes |
Showing 5 of 18 publications.
Prevalence (42)
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 |
|---|---|---|---|---|---|
| Tay-Sachs disease | Prevalence at birth | 1-9 / 100 000 | Portugal | Not stated | Unspecified |
| Tay-Sachs disease | Prevalence at birth | 1-9 / 1 000 000 | Canada | Not stated | Exact |
| Tay-Sachs disease | Prevalence at birth | 1-9 / 1 000 000 | Netherlands | Not stated | Exact |
| Tay-Sachs disease | Prevalence at birth | 1-9 / 1 000 000 | Australia | Not stated | Exact |
| Tay-Sachs disease | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Unspecified |
Showing 5 of 42 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Tay-Sachs disease | 845 | Exact | Orphadata Science knowledge files, Product 1 |
| Tay-Sachs disease | 845 | 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.