An AllTrials project

NCT04221451: An ongoing trial by Genzyme, a Sanofi Company

This trial is ongoing. It must report results 1 week, 3 days from now.

Full data

Full entry on ClinicalTrials.gov NCT04221451
Title 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
Results Status Ongoing
ACT or pACT? This is what FDAAA officially calls an "Applicable Clinical Trial"
Start date June 29, 2020
Completion date Dec. 26, 2024
Required reporting date Dec. 26, 2025, midnight
Actual reporting date None
Date last checked at ClinicalTrials.gov Dec. 12, 2025
Days late None