Calendario›SNY›venglustat GZ402671 · AMETHIST
venglustat GZ402671 · AMETHIST
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Ensayo de Sanofi en Tay-Sachs Disease · Sandhoff Disease (del registro, en inglés).
- Fase
- Fase 3
- Estado
- Interrumpido
- Participantes
- 75
- Centros
- 23
- Fin del objetivo primario
- dic 2024
Qué significa cada fase y cada estado.
Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en jun 2020.
Este ensayo se detuvo. Motivo declarado por el promotor (del registro, en inglés): «Discontinued based on the absence of positive trends on clinical endpoints.».
Qué mide
PAP: PP: Percent Change From Baseline in CSF GM2 Biomarker to Week 104 (Baseline (Day 1) and Week 104) (del registro, en inglés)
Cómo lo describe el promotor
En el documentoEn inglés, del registroPrimary 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
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Ficha completa en ClinicalTrials.gov (NCT04221451), actualizada en ene 2026.