GM1 Gangliosidosis clinical trials at UCSF
4 in progress, 3 open to eligible people
GM1 gangliosidosis is a genetic disorder that causes worsening movement and learning problems. UCSF is conducting 18‑month studies that test an oral medication in late‑infantile and juvenile patients. The studies are randomized, double‑blind, and use a placebo control.
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
Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
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 Niemann-Pick type C disease
Oakland, California and other locations
Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, 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 Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
Oakland, California and other locations
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
Sorry, not yet accepting patients
This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.
Our lead scientists for GM1 Gangliosidosis research studies include Caroline Hastings, MD.
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