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

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

NCT Number	NCT07054515
Lead Sponsor	Azafaros B.V.
Conditions	Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Enrollment	147 participants
Start Date	2025-06-30
Primary Completion	2028-05-14 (estimated)
Study Completion	2028-06-14 (estimated)
Updated on ClinicalTrials.gov	2026-08-14