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 GM1 gangliosidosis or GM2 gangliosidosis
Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant
Male and female participants aged 4 years and older at the time of informed consent
Onset of neurological symptoms from 1 to 10 years
Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
A history of medical conditions other than GM1 or GM2 gangliosidosis 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
AZ-3102
DrugNizubaglustat
Placebo
DrugPlacebo
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
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