NCT07082543

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

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: Azafaros A.G. · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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

Detailed description

Please see NCT #07054515 for information on the AZA-001-301 Master Protocol PRIMARY OBJECTIVE The primary objective of this study is to demonstrate superior efficacy on ataxic manifestations with oral nizubaglustat dosing compared with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis SECONDARY OBJECTIVES I. To assess additional efficacy in ataxic and non-ataxic manifestations comparing nizubaglustat dosing with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis II. To assess the pharmacokinetic (PK) properties of nizubaglustat after administration of the first dose (Visit 1) and at steady state after multiple once daily doses III. To assess the pharmacodynamic (PD) effects of nizubaglustat IV. To assess the safety and tolerability of daily oral nizubaglustat dosing compared with placebo, when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis

Conditions

• Gangliosidoses, GM2
• Gangliosidosis, GM1

Interventions

Type	Name	Description
DRUG	AZ-3102	Nizubaglustat
DRUG	Placebo	Placebo

Timeline

• Start date: 2025-06-30
• Primary completion: 2027-11-04
• Completion: 2027-11-04
• First posted: 2025-07-24
• Last updated: 2026-04-15

Locations

36 sites across 16 countries: United States, Argentina, Australia, Brazil, Canada, France, Germany, India, Italy, Mexico, Portugal, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study