18-month Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Oral Nizubaglustat (AZ-3102) Safety and Efficacy in Late-infantile and Juvenile Niemann-Pick Type C Disease

What this Trial Is About

Researchers are evaluating the safety and effectiveness of an oral drug called nizubaglustat (AZ-3102) for children and young people with late-infantile and juvenile forms of Niemann-Pick type C disease. This is an 18-month, double-blind, randomized, placebo-controlled Phase 3 study conducted at multiple centers. The main goal is to see if nizubaglustat improves ataxic symptoms compared to a placebo, while also assessing other neurological and functional effects, drug behavior in the body, and safety. Participants will take oral dispersible tablets once daily, receiving either nizubaglustat or a matching placebo. The study includes thorough monitoring over 18 months, with evaluations of drug levels in the blood at the start, steady state, and end of treatment. The study is designed to carefully compare changes in neurological function and disease severity between groups receiving the active drug and placebo. Throughout the study, participants will undergo regular assessments including the Scale for the Assessment and Rating of Ataxia (SARA), behavior and swallowing scales, clinical severity scales, and seizure tracking, among others. Researchers will track changes in these measures from baseline at months 6, 12, and 18. Safety and tolerability are closely monitored, and participants' progress is evaluated over the full 18-month period to understand the drug's effects and any side effects.

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 (NPC)