EFC18362 - A Phase 3, multicenter, multinational, randomized, open-label, active-comparator study to evaluate the efficacy and safety of venglustat compared with Cerezyme in pediatric participants 2 to 11 years of age with Gaucher disease type 3 who have reached therapeutic goals with enzyme replacement therapy

Immune system diseases

Percent change from baseline in magnetic resonance imaging (MRI) spleen volume (in multiples of normal [MN]) at Week 52

Percent change from baseline in MRI liver volume (in MN) at Week 52, Change from baseline in hemoglobin level at Week 52, Percent change from baseline in platelet count at Week 52, Changes from baseline in Vineland Adaptive Behavior Scales III Adaptive Behavior Composite (ABC) score at Week 52, Change from baseline in Vineland Adaptive Behavior Scale (VABS) III motor skill score at Week 52, Percent change from baseline in plasma glucosylceramide (GL-1), glucosylsphingosine (lyso-GL-1), and other potential biomarker levels to Week 52, Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and adverse events of special interest (AESIs), Plasma venglustat Ctrough at prespecified visits

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