NCT07054515

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

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: 147 (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 Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Detailed description

This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis. Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols. For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543.

Conditions

• Niemann-Pick Type C Disease
• GM1 Gangliosidosis
• GM2 Gangliosidosis

Interventions

Type	Name	Description
DRUG	AZ-3102	Oral dispersible tablets
DRUG	Placebo	A matching placebo will be administered in the same regimen as the intervention

Timeline

• Start date: 2025-06-30
• Primary completion: 2027-11-04
• Completion: 2027-11-04
• First posted: 2025-07-08
• Last updated: 2026-04-15

Locations

37 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