Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Tay-Sachs Disease: early-phase trial registry
3 of 7 indexed Tay-Sachs Disease trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #2,497 of 28,707 conditions by registered trial count.
7 US clinical trials · 3 currently recruiting · #2,497 of 28,707 conditions by registered trial count
Tay-Sachs Disease: 7 US clinical trials tracked, 3 recruiting.
Tay-Sachs Disease is the subject of 7 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is University of Minnesota, running 2 of these trials.
Enrollment posture brief
Registry condition tay-sachs-disease
OPEN 3 | RECR 43% | LATE 0 | EARLY 2 | RANK #2497 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Adrenoleukodystrophy (43% recruiting · 7 trials)
Tay-Sachs Disease lists 3 open slots (43% of 7 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Tay-Sachs Disease on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Tay-Sachs Disease is recruiting below its size-band peers.
3 of 7 indexed trials (42.9%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Tay-Sachs Disease research.
University of Minnesota accounts for 2 of 7 trials (28.6%). 6 distinct sponsors appear on at least one study for this condition label.
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Typical Tay-Sachs Disease enrolment sits around 52 participants.
Median target enrolment is 52 among the 7 trials that report a genuine target (sentinel values excluded). 4 of those (57.1%) plan for fewer than 100 participants.
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Tay-Sachs Disease research leans observational.
2 interventional and 5 observational studies make up this index (28.6% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
7 total, page 1 of 1
Longitudinal Study of Neurodegenerative Disorders
University of Pittsburgh
NCT03333200
A Natural History Study of the Gangliosidoses
University of Minnesota
NCT00668187
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Joanne Kurtzberg, MD
NCT02254863
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
Terence Flotte
NCT06614569
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Idorsia Pharmaceuticals
NCT04470713
Stem Cell Transplant for Inborn Errors of Metabolism
Masonic Cancer Center, University of Minnesota
NCT00176904
Gene Therapy for Tay-Sachs Disease
University of Minnesota
NCT01869270
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Tay-Sachs Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Tay-Sachs Disease trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Tay-Sachs Disease page
7 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 3 Tay-Sachs Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- University of Minnesota sponsors the most Tay-Sachs Disease trials on record, review their full trial history before evaluating a specific study. See University of Minnesota's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Tay-Sachs Disease condition card totals 7 registered trials; most-linked intervention Busulfan, Cyclophosphamide, Antithymocyte Globulin; top US site state Minnesota. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.