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

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2025
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
Epistemonikos ID: 25c1a7889b0fb8766b0fcfd5839b777fc0e566e8
First added on: Jul 25, 2025