Gangliosidoses, GM2 clinical trials at UCSF
1 research study open to eligible people
Gangliosidoses, GM2, are inherited disorders that cause nerve cell damage from stored ganglioside fats. UCSF is running an 18-month, double-blind, randomized, placebo-controlled study of an oral drug in late-infantile and juvenile GM2. UCSF is monitoring safety and clinical changes over time.
Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
open to eligible people ages 4 years and up
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis
Oakland, California and other locations
Our lead scientists for Gangliosidoses, GM2 research studies include Caroline Hastings, MD.
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