A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Summary
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 PD of daily oral dosing of venglustat and 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 * To assess the acceptability and palatability of the venglustat tablet
Timeline
- Start
- 2020-06-29
- Primary completion
- 2024-12-26
- Completion
- 2024-12-26
Outcome
Mixed primary results
Stopped: “Discontinued based on the absence of positive trends on clinical endpoints.”
registry MEAN PAP: PP: Placebo: -11.33 (Standard Error 4.23) percent change NCT04221451 ↗
registry MEAN PAP: PP: Venglustat: -47.57 (Standard Error 3.04) percent change NCT04221451 ↗
registry analysis (superiority test); PAP: PP: Placebo vs PAP: PP: Venglustat; p <0.0001; Mean difference -36.24 NCT04221451 ↗
registry analysis (superiority test); PAP: PP: Placebo vs PAP: PP: Venglustat; p = 0.7435; Mean difference 1.54 NCT04221451 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Venglustat | Small molecule | — | Oral |