ClinicaliQ Trial Snapshot
- 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 — Recruiting • Phase III • NCT07082543.
- Oral nizubaglustat tested against placebo over 18 months in children with late-infantile or juvenile GM1/GM2 gangliosidosis.
Verify eligibility, endpoints and current status on the original ClinicalTrials.gov registry before acting on this summary.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
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 GM1 gangliosidosis or GM2 gangliosidosis Conditions: Gangliosidoses, GM2, Gangliosidosis, GM1 Interventions: AZ-3102, Placebo Lead Sponsor: Azafaros B.V. Planned Enrollment: 75 participants