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GM1 Gangliosidosis: early-phase trial registry
2 of 7 indexed GM1 Gangliosidosis 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 · 2 currently recruiting · #2,497 of 28,707 conditions by registered trial count
GM1 Gangliosidosis: 7 US clinical trials tracked, 2 recruiting.
GM1 Gangliosidosis is the subject of 7 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is LYSOGENE, running 1 of these trials.
Enrollment posture brief
Registry condition gm1-gangliosidosis
OPEN 2 | RECR 29% | LATE 1 | EARLY 2 | RANK #2497 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Acromegaly (29% recruiting · 14 trials)
GM1 Gangliosidosis lists 2 open slots (29% 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 GM1 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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GM1 Gangliosidosis is recruiting below its size-band peers.
2 of 7 indexed trials (28.6%) 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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Sponsorship of GM1 Gangliosidosis trials is spread across many organizations.
LYSOGENE accounts for 1 of 7 trials (14.3%). 7 distinct sponsors appear on at least one study for this condition label.
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Typical GM1 Gangliosidosis 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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GM1 Gangliosidosis splits between interventional and observational designs.
3 interventional and 4 observational studies make up this index (42.9% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
7 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
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
Gemma Biotherapeutics
NCT04713475
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
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
University of Pennsylvania
NCT04041102
Interviews and Video Capture in Patients With GM1 Gangliosidosis
LYSOGENE
NCT04310163
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 1 |
| Phase 3 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on GM1 Gangliosidosis records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on GM1 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 GM1 Gangliosidosis page
7 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 GM1 Gangliosidosis trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- LYSOGENE sponsors the most GM1 Gangliosidosis trials on record, review their full trial history before evaluating a specific study. See LYSOGENE'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 GM1 Gangliosidosis 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.