Phase 3 Study of Oral Nizubaglustat AZ-3102 for Treating Late-Infantile and Juvenile Niemann-Pick Type C Disease, GM1 Gangliosidosis, and GM2 Gangliosidosis

What this Trial Is About

Researchers are conducting an 18-month Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat AZ-3102 in children aged 4 years and older diagnosed with late-infantile or juvenile forms of Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. This randomized, double-blind, placebo-controlled, multicenter trial uses a Master Protocol Research Program to study these related conditions in separate subprotocols based on disease type. Participants are randomly assigned in a 21 ratio to receive either oral nizubaglustat tablets or a matching placebo. The treatment and procedures are specific to each disease subprotocol, detailing different eligibility requirements, safety assessments, and efficacy endpoints. The study includes subprotocols for Niemann-Pick type C disease and for GM1 or GM2 gangliosidosis, each with tailored treatment plans and monitoring. Throughout the study, participants will undergo evaluations as described in their respective subprotocols, which may include clinical assessments, safety monitoring, and outcome measurements over the 18-month period. Researchers will track participant allocation to each subprotocol as the primary outcome. The trial is designed to monitor safety and treatment effects closely until the study completes in 2028, with detailed procedures provided within each disease-specific subprotocol.

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