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.

• Protocol code: AZA-001-301
• EudraCT code: No aplica
• Research group: Pediatric Neurology
• Service: General Pediatrics and Specialties
• Principal investigator:  Del Toro Riera, Mireia
• Pathology: Malalties del sistema nerviós
• Phase: Fase III