Clinical trials
GM2 Gangliosidosis clinical trials
Below are recruiting gm2 gangliosidosis clinical trials, each written for real people, not researchers. We’re tracking 4 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07054515RecruitingPhase 3
Study of oral nizubaglustat for rare genetic disorders
This trial tests a new oral medicine, nizubaglustat (AZ-3102), for children and adults (4+) with late-infantile or juvenile Niemann-Pick type C, GM1, or GM2 gangliosidosis. It aims to see if the drug is safe and helps slow disease progression.
Oakland, CaliforniaAges 4 years+ - NCT07399704RecruitingPhase 2
Testing a new drug for two rare brain diseases
This trial is for people with Niemann-Pick type C or GM2 gangliosidosis who are already on or have been in a related study. It tests a new drug called Nizubaglustat to see if it is safe and works better than current treatments.
Água Verde, CuritibaAges 12 years+ - NCT00668187Recruiting
Study of the natural history of gangliosidosis
This study follows people with gangliosidosis to better understand how the condition changes over time. You may be asked to complete thinking/behavior tests and, if you have late-onset disease, get a head MRI (a painless scan) to help researchers track changes in the brain.
Minneapolis, MinnesotaAges Any age - NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Los Angeles, CaliforniaAges Any age
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GM2 Gangliosidosis trials by city
Studies with a site in or near these metro areas.
GM2 Gangliosidosis trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for gm2 gangliosidosis?
- Yes. Clin2 currently lists 4 recruiting gm2 gangliosidosis studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a gm2 gangliosidosis trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a gm2 gangliosidosis trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.