Clinical Research Directory

Inclusion criteria: * ≥ 12 years of age * Clinical diagnosis of GD3 with at least 1 CNS manifestation * Body weight ≥15 kg * On a stable ERT dose for at least 6 months * Clinically stable with respect to hematologic manifestations * Clinically stable with respect to spleen and liver volume * If the patient has a history of seizures, they must be well controlled without use of medications that are strong/moderate inducers or strong/moderate inhibitors of CYP3A * Documented negative pregnancy test * Contraceptive use consistent with local regulations. Exclusion criteria: * Inability to safely swallow (or chew and swallow) a venglustat tablet * Use of any investigational drugs within the last 30 days or 5 half-lives or gene therapy at any time * Current use of any unapproved therapy for GD * Pregnant or breastfeeding females * Drug contraindications * History of major organ transplant (eg, bone marrow or liver) * History of drug and/or alcohol abuse within the last year * Patients with severe depression and/or a history of a major affective disorder in the past year * Patients with suicidal ideation or behavior prior to study. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.