GM1 Gangliosidosis clinical trials
8 active trials name this condition. Describe your situation to see which ones look like a fit.
By location
- A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
- 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
- A Natural History Study of the Gangliosidoses
- Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
- FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
- Longitudinal Study of Neurodegenerative Disorders
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Guides for people searching for a trial
- Why people get turned down — and what to do nextWhat eligibility criteria are for, what keeps people out, and where to go when the first door closes.
- How your biomarkers decide which trials you qualify forEGFR, HER2, BRAF, PD-L1 and the rest — what they are and why they increasingly gate access.
- How to search for a trial without losing your mindWhere to look, what to gather first, and how to read what you find.
- What Phase 1, 2 and 3 actually mean for youWhat each phase is testing, and how to weigh an early-phase trial against a later one.
- 10 questions to ask before you say yesTreatment, time, cost, safety and your rights. Worth bringing to a screening visit.
- You matched with a trial. What happens now?Phone screens, the screening visit, informed consent, and day one.
Showing active trials that list “GM1 Gangliosidosis” as a condition. Eligibility shown on each trial is an estimate — the trial's study team makes the final decision.