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Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).\n* Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile\u002F juvenile onset. Detailed inclusion criteria are presented in the GM1\u002FGM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).\n\nExclusion Criteria:\n\n* Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC\n* Detailed exclusion criteria are presented in the GM1\u002FGM2 gangliosidosis-specific subprotocol AZA-001-301-GMx","ALL","4 Years",{"count":594,"type":595},147,"ESTIMATED","INTERVENTIONAL",[598],"PHASE3","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",[601,602,603],"Niemann-Pick Type C Disease","GM1 Gangliosidosis","GM2 Gangliosidosis",[605],"Nizubaglustat","2026-08-13",{"date":608,"type":609},"2026-08-14","ACTUAL",{"date":611,"type":609},"2025-06-30",{"date":613,"type":595},"2028-06-14",{"name":5,"class":6},38]