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
Parallel
Nizubaglustat
DrugAZ-3102
Placebo
DrugMatching placebo
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)
Change from baseline in functional SARA score
Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
Change from baseline in SARA score for gait/posture
Assessed on a 9-point scale with higher scores indicating inability to complete the task
Change from baseline in SARA score for speech
Assessed on a 7-point scale with higher scores indicating unintelligible speech/anarthria
Change from baseline in SARA score for kinetics
Assessed on a 5-point scale with higher scores indicating more severe impairment
Change from baseline in Vineland Adaptive Behavior Scale (VABS)
Assesses four domains of function: communication, daily living skills, socialization, and motor skills
Click on the lead sponsor to view all of their trials.