ClinicaliQ Trial Snapshot
- 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 — Recruiting • Phase III • NCT07054515.
- Oral nizubaglustat tested against placebo for safety and effectiveness in children with rare inherited brain storage diseases.
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What This Trial Is Studying
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 Conditions: Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis Interventions: AZ-3102, Placebo Lead Sponsor: Azafaros A.G. Planned Enrollment: 147 participants