Phase 3 Study to Test Safety and Effectiveness of Oral Nizubaglustat in Children and Teens with Late-infantile and Juvenile Niemann-Pick Type C Disease

What this Trial Is About

Researchers are conducting an 18-month Phase 3 study to evaluate the safety and effectiveness of oral nizubaglustat AZ-3102 in children and adolescents with late-infantile and juvenile forms of Niemann-Pick type C disease. This randomized, double-blind, placebo-controlled trial aims to show whether nizubaglustat improves ataxic symptoms compared with placebo. The study also assesses other neurological and behavioral effects, pharmacokinetics, pharmacodynamics, and safety of the treatment. Participants receive either daily oral dispersible tablets of nizubaglustat or matching placebo. The study follows a parallel design with random assignment to treatment groups. The treatment period lasts for 18 months, during which participants take the assigned medication once daily. The study monitors treatment effects and safety throughout this period. During the trial, participants undergo assessments including the Scale for the Assessment and Rating of Ataxia SARA, Vineland Adaptive Behavior Scale, Penetration-Aspiration Scale, 9-Hole Peg Test, and other neurological and functional tests at baseline and at months 6, 12, and 18. Blood samples are collected to study drug levels and biomarkers. Researchers also track seizure frequency and adverse events. The primary outcome measures focus on changes in total and functional SARA scores from baseline to month 18, with safety and tolerability evaluated throughout participation.

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)