Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
GM2 Gangliosidosis: later-stage trial registry
2 of 4 indexed GM2 Gangliosidosis trials are currently recruiting, with more weight in Phase 3-4.
Corpus placement: #4,115 of 28,707 conditions by registered trial count.
4 US clinical trials · 2 currently recruiting · #4,115 of 28,707 conditions by registered trial count
GM2 Gangliosidosis: 4 US clinical trials tracked, 2 recruiting.
GM2 Gangliosidosis is the subject of 4 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 0 in Phase 1-2 (earlier-stage). The most active sponsor is Terence Flotte, running 1 of these trials.
Enrollment posture brief
Registry condition gm2-gangliosidosis
OPEN 2 | RECR 50% | LATE 1 | EARLY 0 | RANK #4115 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Abdominal Aortic Aneurysms (50% recruiting · 6 trials)
GM2 Gangliosidosis lists 2 open slots (50% of 4 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 GM2 Gangliosidosis 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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GM2 Gangliosidosis is recruiting above its size-band peers.
2 of 4 indexed trials (50%) 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.
Open and recent trials
4 total, page 1 of 1
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
Azafaros A.G.
NCT07054515
A Natural History Study of the Gangliosidoses
University of Minnesota
NCT00668187
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
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 3 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on GM2 Gangliosidosis records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on GM2 Gangliosidosis 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 GM2 Gangliosidosis page
4 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 GM2 Gangliosidosis trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Terence Flotte sponsors the most GM2 Gangliosidosis trials on record, review their full trial history before evaluating a specific study. See Terence Flotte'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 GM2 Gangliosidosis condition card totals 4 registered trials; most-linked intervention AXO-AAV-GM2; top US site state Minnesota. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.