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

Phase 3 Recruiting 75 enrolled Azafaros B.V.

RandomizedParallel-groupQuadruple-blindTreatment

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

Timeline

Start: 2025-06-30
Primary completion: 2028-02-06
Completion: 2028-03-06

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Nizubaglustat	Unknown	—	Oral

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

Summary

Timeline

Drugs

Indications