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GM1 Gangliosidosis
Open-data reference.
7 US clinical trials · 2 currently recruiting
Active & Recent Trials
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 |
|---|---|
| Early Phase 1 | 1 |
| Phase 2 | 1 |
| Phase 3 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the GM1 Gangliosidosis Trial Landscape
ClinicalTrials.gov lists 7 US studies indexed under GM1 Gangliosidosis, and 2 of those are currently open to recruitment — roughly 29% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for GM1 Gangliosidosis shows 1 late-stage studies (Phase 3 and Phase 4 combined) alongside 2 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for GM1 Gangliosidosis is led by LYSOGENE with 1 indexed trial, alongside 6 other organizations in the top contributor list. The list on this page surfaces up to 7 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for GM1 Gangliosidosis?
PlainTrial tracks 7 US clinical trials for GM1 Gangliosidosis, of which 2 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for GM1 Gangliosidosis?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.