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
Signed informed consent
Confirmed diagnosis of NPC disease
Patient is unable or unwilling to take miglustat, or is, in the opinion of the investigator, unsatisfactorily treated with miglustat
Male and female participants aged 4 years and older at the time of informed consent
A history of medical conditions other than NPC disease that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results
Body weight of <10 kg
The presence of another neurologic disease
The presence of moderate or severe hepatic impairment