Clinical trials
GM1 Gangliosidosis clinical trials
Below are recruiting gm1 gangliosidosis clinical trials, each written for real people, not researchers. We’re tracking 6 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+ - NCT03952637RecruitingPhase 1/Phase 2
Gene therapy for GM1 disease with IV delivery
This early-stage study tests an IV gene therapy that delivers a working copy of an enzyme gene to help the body make beta-galactosidase in GM1 gangliosidosis. It may help slow or improve disease symptoms and looks closely at safety and how well the treatment works.
Bethesda, MarylandAges 6 months–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 - NCT06539169Recruiting
Following people with rare diseases over time
This study follows people with rare diseases over time to learn more about how these conditions progress and how they are treated. Joining may help researchers understand your disease better and find better ways to care for others.
Los Altos, CaliforniaAges Any age - NCT05368038Enrolling by invitation
Newborn screening program for babies up to 4 weeks old
This trial tests a flexible newborn screening process that looks for certain conditions early in life. It may help by catching problems sooner, when treatment can be started earlier.
Brooklyn, New YorkAges Up to 4 weeks - NCT03333200Recruiting
Study of genetic brain diseases over time
This trial follows people with genetic neurodegenerative (brain-wasting) disorders over time to better understand how the condition changes. The goal is to learn patterns that could help future treatments or care plans.
Pittsburgh, PennsylvaniaAges Any age
Hear when a new GM1 Gangliosidosis trial opens
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Common questions
- Are there clinical trials for gm1 gangliosidosis?
- Yes. Clin2 currently lists 6 recruiting gm1 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 gm1 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 gm1 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.