Condition
GM2 Gangliosidosis clinical trials and research
As of November 12, 2026, Find a Trial lists 3 GM2 Gangliosidosis clinical trials that are recruiting, at sites in 17 countries (most in United States (3), Argentina (1), Australia (1)). By phase: Phase 3 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 17Countries
Newest GM2 Gangliosidosis clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick C, GM1, and GM2 gangliosidoses | Phase 3 | Oakland, California and 38 other locations | Recruiting | Jul 8, 2025 |
| Collecting data and samples from patients with leukodystrophy | — | Los Angeles, California and 22 other locations | Recruiting | Feb 9, 2017 |
| Understanding the progression of gangliosidosis diseases | — | Minneapolis, Minnesota | Recruiting | Apr 29, 2008 |
All recruiting trials (3)
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Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick C, GM1, and GM2 gangliosidoses
This 18‑month study will test whether daily oral nizubaglustat (AZ‑3102) helps people age 4 and up with late‑infantile or juvenile Niemann‑Pick C, GM1, or GM2 gangliosidosis compared with placebo.
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Collecting data and samples from patients with leukodystrophy
This study is collecting health information and samples from people with leukodystrophy to help find new causes and better treatments for the condition.
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Understanding the progression of gangliosidosis diseases
This study looks at how gangliosidosis diseases, like Tay-Sachs and Sandhoff, progress over time in order to help doctors and families understand the conditions better and prepare for future treatments.
Related topics
Quick answers
How many GM2 Gangliosidosis clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 17 countries. The most sites are in United States (3 trials), Argentina (1 trial), Australia (1 trial), Brazil (1 trial), Canada (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.