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Tay-Sachs Disease Sandhoff Disease clinical trials at UC Health

1 research study open to eligible people

Showing trials for
  • A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

    open to eligible people ages 2 years and up

    Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess PD response (plasma and CSF GL-1 biomarker and disease specific biomarkers) of venglustat when administered once daily over a 104-week period Secondary Objectives: Primary population: - To assess the effect of venglustat on selected performance test and scale over a 104-week period - To determine the safety and tolerability of venglustat when administered orally once daily over a 104-week period - To assess the pharmacokinetics (PK) of venglustat in plasma and cerebrospinal fluid (CSF) Secondary population: - To assess the effect of venglustat on selected performance tests and scale over a 104-week period - To determine the safety and tolerability of venglustat when administered once daily over a 104-week period - To assess the PK of venglustat in plasma and CSF

    at UCLA

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