Status: Recruiting
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
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
Participants needed: 75
Trial details
Phase: Phase 3Age: 4+Biological sex: AllType: InterventionalSponsor: Azafaros B.V.Updated: Jun 18, 2026Locations: 36
Eligibility criteria
Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant [+5]
A history of medical conditions other than GM1 or GM2 gangliosidosis that, in th... [+8]