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 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
Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
PHASE2
This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks st...