A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
Have a confirmed diagnosis of GD3 disease
Have at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening
Be aged 4-30 years old at the time of informed consent
Have the following symptoms of GD3 identified at Screening: GD-related anemia, thrombocytopenia, splenomegaly
Any condition at Screening or Baseline that, in the opinion of the Principal Investigator, could interfere with study assessments
History of medical conditions other than GD3 that, in the opinion of the Principal Investigator, could confound scientific rigor or interpretation of results
Body weight <10 kg at Screening
Presence of a neurologic disease other than GD3