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Venglustat in late-onset GM2 gangliosidosis
NCT04221451Phase 3Terminated
- Sponsor
- Sanofi
- Start
- Jun 2020
- Primary completion
- Dec 2024
- Modality
- Small Molecules
- Countries
- 13
01
Overview
Phase 3, randomised, double-blind trial of venglustat in Tay-Sachs disease (TSD), Sandhoff disease, GM1 gangliosidosis (GM1) and 3 more (3 arms), sponsored by Sanofi. Primary endpoints: Percent Change From Baseline in CSF GM2 Biomarker at 104 weeks, Annualized Rate of Change From Baseline in the 9-Hole Peg Test at 104 weeks and 1 more. Started Jun 2020; status: terminated.
02
Drugs
venglustat
03
Conditions
Tay-Sachs disease (TSD)Sandhoff diseaseGM1 gangliosidosis (GM1)Saposin C Deficiency (SCD)Sialidosis Type 1
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