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A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway

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Trial Profile

A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway

Status: Discontinued
Phase of Trial: Phase III

Latest Information Update: 06 Jan 2025

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At a glance

  • Drugs Venglustat (Primary)
  • Indications GM1 gangliosidosis; GM2 gangliosidoses; Sandhoff disease; Tay-Sachs disease
  • Focus Biomarker; Pharmacodynamics; Registrational; Therapeutic Use
  • Acronyms AMETHIST
  • Sponsors Sanofi Genzyme

Most Recent Events

  • 16 May 2024 This trial has been discontinued in Germany According to European Clinical Trials Database record.
  • 09 May 2024 This trial has been completed in France (Global end date: 25 Apr 2024).
  • 25 Apr 2024 Status changed from active, no longer recruiting to discontinued, according to a Sanofi media release.

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