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
Single group
AZ-3102
DrugDaily oral intake of AZ-3102 orodispersible tablets
Change in hemoglobin levels from Baseline
Change in platelet count from Baseline
Change in total spleen volume from Baseline
Assessed using magnetic resonance imaging (MRI)
Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline
Scale ranges from 1 to 7, where a higher score equals a worse outcome.
Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline
Scale ranges from 1 to 7, where a higher score equals a worse outcome.
Clinical Global Impression of Change (CGI-C) score
Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.
Participant/Caregiver Global Impression of Change (PGI-C) score
Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.
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