Clinical Trial

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

Active, Not Recruiting Phase 3
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Summary
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
Protocol Amendment History 5 changes
critical Trial status changed: Recruiting → Active, Not Recruiting 2026-07-25
notable Trial sites reduced: 36 -> 26 locations 2026-07-25
notable Primary completion pushed: 2027-11-04 -> 2028-02-06 2026-07-21
minor Completion pushed: 2027-11-04 -> 2028-03-06 2026-07-21
notable Sponsor changed: Azafaros A.G. -> Azafaros B.V. 2026-06-14
Trial Details
NCT Number NCT07082543
Lead Sponsor Azafaros B.V.
Conditions Gangliosidoses, GM2, Gangliosidosis, GM1
Enrollment 75 participants
Start Date 2025-06-30
Primary Completion 2028-02-06 (estimated)
Study Completion 2028-03-06 (estimated)
Updated on ClinicalTrials.gov 2026-07-24