NCT07054515: 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

Sponsor: Azafaros B.V. · Phase: PHASE3 · Area: CARDIOLOGY_METABOLIC · Target Enrollment: 147 pts