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
PHASE3
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phas...
Status
Active Not Recruiting
Location
UCSF Children's Hospital and Research Center at Oakland
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
PHASE3
Primary Objectives:
Primary population (adult participants with late-onset...
Status
Terminated
Location
Ataxia Center and HD Center of Excellence, 300 UCLA Med Plaza, Suite B200 - Investigational site number 8400004
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
PHASE3
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phas...
Status
Recruiting
Location
UCSF Children's Hospital and Research Center at Oakland