Clinical Research Directory

Inclusion Criteria: * 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 * Female participants of childbearing potential who are sexually active and willing to follow contraceptive guidance * Male participants with a female partner of childbearing potential who are willing to follow contraceptive guidance Exclusion Criteria: * 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 * Transfusion dependence * Prior splenectomy * The presence of severe renal impairment at Screening * Prior use of an investigational drug within 3 months before Screening * Prior participation in a clinical study involving gene therapy or stem cell transplantation * Current treatment, or treatment in the last 12 months before Screening, with enzyme replacement therapy (ERT) and/or substrate reduction therapy (SRT) * A positive serum pregnancy test (for women of childbearing potential) * Electrocardiogram (ECG) with an average Fridericia-corrected QT interval (QTcF) of \>450 msec for males and \>470 msec for females at Screening * Known allergies to azasugars or any study drug excipient