A 4-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamic, and exploratory efficacy of venglustat in combination with Cerezyme in adult patients with Gaucher disease Type 3 with venglustat monotherapy extension

Gaucher disease type 3, Gaucher disease type 1

Number of participants with Treatment Emergent Adverse Events (TEAEs), Assessment of pharmacodynamic (PD) parameter: Lyso-glucosylceramide (lyso-GL1) and glucosylceramide (GL-1) in cerebrospinal fluid (CSF)

Assessment of pharmacodynamic (PD) parameter: Lyso-glucosylceramide (lyso-GL1) and glucosylceramide (GL-1) in plasma, Assessment of plasma pharmacokinetic parameter: Cmax (Part 2), Assessment of plasma pharmacokinetic parameter: Tmax (Part 2), Assessment of plasma pharmacokinetic parameter: AUC 0-24h (Part2), Assessment of plasma pharmacokinetic parameter: Ctrough, Assessment of CSF pharmacokinetic parameter: Cmax, Assessment of spleen volume, Assessment of spleen volume (Part4), Assessment of liver volume, Assessment of liver volume (Part 4), Assessment of hemoglobin level, Assessment of hemoglobin level (Part 4), Assessment of platelet level, Assessment of platelet level (Part 4), Assessment of Ataxia, Assessment of Ataxia (Part 4)

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Germany